Penn Dermatology

Penn Dermatology Clinical Trials

Penn Dermatology's Clinical Studies Unit Conducts Trials for Common to Rare Dermatologic Diseases

Penn Dermatology's Clinical Studies Unit is one of the first of its kind in the nation that specializes in dermatology. The Clinical Studies Unit receives patient referrals from Penn dermatologists and clinical faculty as well as community-based dermatologists and physicians. It also receives inquiries and referrals from all over the world. Its medical director is Joel M. Gelfand, MD, MSCE. Dr. Gelfand oversees a team of highly skilled, experienced and certified research coordinators, nurses and investigators in a manner that enhances the efficiency of clinical research and optimizes the safety of the trial participants.

The Clinical Studies Unit and the Department of Dermatology offer a variety of interventional clinical trials related to both common and rare diseases ranging from acne, atopic eczema, hidradenitis, skin cancer and psoriasis to cutaneous t-cell lymphoma. Penn Dermatology conducts cutting-edge research and clinical trials on an ongoing basis and is one of the top funded research departments in the nation in National Institutes of Health (NIH) funding. In addition, the Clinical Studies Unit receives funding from the Food and Drug Administration, foundations, and pharmaceutical companies in order to conduct trials designed to advance the care of patients with skin disease.

The Penn Dermatology Clinical Studies Unit is located at the Hospital of the University of Pennsylvania. To refer a patient, or to learn how to participate in clinical trials, contact 215-662-SKIN (7546).

Clinical Studies Unit trials that are currently in progress and offer ongoing enrollment include:

Vascular Inflammation in Psoriasis Extension Trial (The VIP-E Study)
Principal Investigator Joel M. Gelfand, MD, MSCE
Start Date May 2013
Sponsor Abbvie and National Heart, Lung, and Blood Institute (NHLBI)
Coordinator Rosemary Attor, MS, CRC
Number of Patients 2
Enrolling Yes
Purpose The purpose of this study is to determine if extended treatment of psoriasis with adalimumab over a period of up to 52 weeks is associated with durable improvements in cardiometabolic disease.
Description of the Study VIP-E is a one-arm, open-label, 40-52 week extension study to continue or cross over subjects of the VIP Study to active drug (adalimumab) to determine if there is sustained improvement in vascular inflammation, lipid metabolism, and inflammatory markers. VIP-E extends VIP study procedures for 40-52 weeks including questionnaires, physical exams, blood and urine samples, lab tests, one additional FDG-PET/CT scan, and adalimumab injections following FDA-approved psoriasis treatment regimen.
Inclusion Criteria
  • Males and females 18 years of age and older.
  • Completed the 12 week VIP Study.
  • Willing and able to avoid prolonged exposure of skin affected by psoriasis to natural or sunlight or tanning beds during the course of the study.
  • Willing and able to avoid topical or systemic prescription treatments for psoriasis besides adalimumab during the course of the study.
  • Women are eligible to participate in the study if they meet one of the following criteria:
    • Willing to undergo regular pregnancy testing and agree to use one method of contraception throughout the study (for women of childbearing age).
    • Are postmenopausal (for at least one year), sterile, or hysterectomized.
    • Have undergone tubal ligation and agree to regular pregnancy testing.
    • Agree to be sexually abstinent, defined as total abstinence from sexual intercourse, as a form of contraception.
  • Subject is judged to be in good general health as determined by the Principal Investigator based upon the results of medical history, laboratory profile, and physical examination.
  • Able and willing to give written informed consent and to comply with requirements of this study protocol.
Exclusion Criteria
  • Previous adverse event following exposure to a TNF-alpha antagonist that led to discontinuation of the TNF inihibitor and contraindicates future treatment.
  • Previous lack of response to a TNF-alpha antagonist led to discontinuation.
  • Diagnosis of erythrodermic psoriasis, generalized pustular psoriasis, or medication-induced or medication-exacerbated psoriasis.
  • Diagnosis of other active skin diseases or skin infections (bacterial, fungal, or viral) that may interfere with evaluation of psoriasis.
  • Taking or requires oral or injectable corticosteroids during the study. Inhaled corticosteroids for stable medical conditions are allowed.
  • Poorly controlled medical condition, such as unstable ischemic heart disease, congestive heart failure, recent cerebrovascular accidents, psychiatric disease requiring frequent hospitalization, and any other condition, which, in the opinion of the Investigator, would put the subject at risk by participation in the study.
  • History of diabetes mellitus, type 1 or type 2 (patients with type 2 diabetes may be enrolled if the duration of diabetes is <10 years and HbA1c is <7.0%)
  • Uncontrolled hypertension, with measured systolic blood pressure >180 mmHg or diastolic blood pressure >90 mmHg
  • History of demyelinating diseases or lupus.
  • Subject has infection or risk factors for severe infections, for example:
    • Known history of HIV, hepatitis B or C, or other severe, recurrent, or persistent infections;
    • Excessive immunosuppression or other factors associated with it, including human immunodeficiency virus infection;
    • Active tuberculosis (TB) disease;
    • Evidence of latent TB infection demonstrated by Purified Protein Derivative (PPD) ≥ 5 mm of induration or positive Quantiferon-GOLD results as determined within 6 months of the baseline visit for VIP-E; except if prophylactic treatment for TB, as recommended by local guidelines, is initiated prior to administration of study drug or if there is documentation that the subject has received prophylactic treatment for TB previously.
    • Any other significant infection requiring hospitalization or intravenous (IV) antibiotics in the month prior to Baseline;
    • Infection requiring treatment with oral or parenteral antibiotics within 14 days prior to Baseline;
    • Require a live vaccination during study participation including up to 30 days after the last dose of study drug.
  • History of hematological or solid malignancy within the past five years other than successfully treated basal cell carcinoma, non-metastatic cutaneous squamous cell carcinoma or cervical carcinoma in situ.
  • Female participant who is pregnant or breast-feeding or considering becoming pregnant during the study.
  • Clinic laboratory analyses showing any of the following abnormal results:
    • Hemoglobin (Hgb) < 10 g/dL in females or <12 g/dL in males;
    • White blood cell (WBC) count <2.5 x 109/L
      • Participants can be included if WBC count is <2.5 x x 109/L and absolute neutrophil count (ANC) is >1000 cells / mm3.
    • WBC count > 15 x 109/L;
    • Platelet count < 100 x 109/L;
    • Serum aspartate transaminase (AST) or alanine transaminase (ALT) >2.5 upper limits of normal (ULN);
    • Serum total bilirubin ≥2 mg/dL (≥26 µmol/L)
  • Recent history of substance abuse or psychiatric illness that could preclude compliance with the protocol.
  • If taking cholesterol-lowering medication (e.g. statin), dose and form of medication must be stable for 90 days prior to baseline and remain stable throughout the duration of the study.
Vascular Inflammation in Psoriasis Study (The VIP study): A Trial to Determine the Effect of Psoriasis Treatment on Cardiometabolic Disease
Principal Investigator Joel M. Gelfand, MD, MSCE
Start Date February 2012
Sponsor National Heart, Lung, and Blood Institute (NHLBI)
(Study product provided by Abbvie)
Coordinator Rosemary Attor, MS, CRC
Number of Patients 9
Enrolling Yes
Purpose The purpose of this study is to assess the effect of adalimumab (Humira), when compared to NB-UVB (narrow-band ultraviolet B) phototherapy or placebo (an inactive substance that may resemble an active substance but has no medical value) injection. The study will compare the effects of each on systemic inflammation and cardiovascular disease risk factors in subjects diagnosed with moderate to severe psoriasis
Description of the Study The study is a three-arm, randomized, double-blind, placebo-controlled, 12-week clinical trial to investigate the efficacy of adalimumab and narrow band UVB phototherapy in reducing vascular inflammation and cardiometabolic risk biomarkers in patients with moderate to severe psoriasis.
Inclusion Criteria
  • Males and Females 18 years of age and older.
  • Diagnosis of psoriasis for at least 6 months.
  • Stable plaque psoriasis for at least 2 months before Screening and at Baseline (Week 0) as determined by subject interview of his/her medical history.
  • Moderate to severe psoriasis determined by the study doctor.
  • PASI score of ≥ 12 at the Baseline (Week 0) visit.
  • Candidate for systemic therapy or phototherapy and has active psoriasis despite prior treatment with topical agents.
  • Women are eligible to participate in the study if they meet one of the following criteria:
    • Willing to undergo monthly pregnancy testing during the study and agree to use one of the following methods of contraception throughout the study:
      • Oral contraceptives;
      • Transdermal contraceptives
      • Injectable or implantable methods
      • Intrauterine devices
      • Barrier methods (for example but not limited to a diaphragm with spermicide, condom with spermicide); or
      • Vasectomized partner
      • Subjects using oral or parental forms of contraceptives must have been practicing birth control for at least three months prior to study drug administration.
    • Are postmenopausal (for at least one year), sterile, or hysterectomized;
    • Have undergone tubal ligation and are willing to undergo monthly pregnancy testing during the duration of the study.
    • Agree to be sexually abstinent, defined as total abstinence from sexual intercourse, as a form of contraception.
  • Judged to be in good general health as determined by the Principal Investigator based upon the results of medical history, laboratory profile, physical examination, and 12-lead electrocardiogram (ECG) performed at screening.
  • Able and willing to give written informed consent and to comply with requirements of this study protocol.
Exclusion Criteria
  • Previous adverse event following exposure to a TNF-alpha antagonist and/or UV phototherapy that led to discontinuation of either of these therapies and contraindicates future treatment.
  • Previous lack of response to a TNF-alpha antagonist and/or UV phototherapy that led to discontinuation of either of these therapies.
  • Diagnosis of erythrodermic psoriasis, generalized or localized pustular psoriasis, medication-induced or medication-exacerbated psoriasis, or new onset guttate psoriasis.
  • Diagnosis of other active skin diseases or skin infections (bacterial, fungal, or viral) that may interfere with evaluation of psoriasis.
  • Cannot avoid UVB phototherapy for at least 14 days prior to the Baseline (Week 0) visit.
  • Cannot avoid psoralen-UVA phototherapy for at least 30 days prior to the Baseline (Week 0) visit and during the study.
  • Cannot discontinue systemic therapies for the treatment of psoriasis, or systemic therapies known to improve psoriasis, during the study:
    • Systemic (investigational or marketed) therapies must be discontinued at least 30 days prior to the Baseline (Week 0) visit except for biologics.
      • All biologics, except ustekinumab, must be discontinued for at least 90 days prior to Baseline (Week 0).
      • The IL-12/IL-23 antagonist ustekinumab (half-life of 45.6 ± 80.2 days) must be discontinued for at least 180 days prior to Baseline (Week 0).
    • Investigational agents must be discontinued at least 30 days or 5 half-lives (whichever is longer) prior to the Baseline (Week 0) visit.
  • Taking or requires oral or injectable corticosteroids during the study. Inhaled corticosteroids for stable medical conditions are allowed.
  • Poorly controlled medical condition, such as unstable ischemic heart disease, congestive heart failure, recent cerebrovascular accidents, psychiatric disease requiring frequent hospitalization, and any other condition, which, in the opinion of the Investigator, would put the subject at risk by participation in the study.
  • History of diabetes mellitus, type 1 or type 2- note that patients with type 2 diabetes may be enrolled if the duration of diabetes is <10 years and HbA1c is <7.0%)
  • Uncontrolled hypertension, with measured systolic blood pressure >180 mmHg or diastolic blood pressure >90 mmHg
  • History of demyelinating diseases or lupus.
  • Infection or risk factors for severe infections, for example:
    • Positive serology or known history of HIV, hepatitis B or C, or other severe, recurrent, or persistent infections;
    • Excessive immunosuppression or other factors associated with it, including human immunodeficiency virus infection;
    • Active tuberculosis (TB) disease;
    • Evidence of latent TB infection demonstrated by Purified Protein Derivative (PPD) ≥ 5 mm of induration or positive Quantiferon-GOLD results; except if prophylactic treatment for TB, as recommended by local guidelines, is initiated prior to administration of study drug or if there is documentation that the subject has received prophylactic treatment for TB previously.
    • Any other significant infection requiring hospitalization or intravenous (IV) antibiotics in the month prior to Baseline;
    • Infection requiring treatment with oral or parenteral antibiotics within 14 days prior to Baseline;
    • Received vaccination with Bacille Calmette-Guerin (BCG) within 365 days prior to Screening;
    • Received vaccination with a live viral agent 30 days prior to Screening or will require a live vaccination during study participation including up to 30 days after the last dose of study drug.
  • History of hematological or solid malignancy within the past five years other than successfully treated basal cell carcinoma, non-metastatic cutaneous squamous cell carcinoma or cervical carcinoma in situ.
  • Female subject who is pregnant or breast-feeding or considering becoming pregnant during the study.
  • Screening clinical laboratory analyses showing any of the following abnormal results:
    • Hemoglobin (Hgb) < 10 g/dL in females or <12 g/dL in males;
    • White blood cell (WBC) count <2.5 x 109/L
      • Subject can be included if WBC count is <2.5 x x 109/L and absolute neutrophil count (ANC) is >1000 cells / mm3.
    • WBC count > 15 x 109/L;
    • Platelet count < 100 x 109/L;
    • Serum aspartate transaminase (AST) or alanine transaminase (ALT) >2.5 upper limits of normal (ULN);
    • Serum total bilirubin ≥2 mg/dL (≥26 µmol/L); or
  • Recent history of substance abuse or psychiatric illness that could preclude compliance with the protocol.
  • History of any substance abuse within 365 days of screening visit
  • Alcohol use >14 drinks per week at the screening visit or within 30 days of the screening period
  • If taking cholesterol-lowering medication (e.g. statin), dose and form of medication must be stable for 90 days prior to week 0 and remain stable throughout the duration of the study.
  • History of photosensitivity of medical condition that may be exacerbated by UV exposures such as lupus or dermatomyositis
Phase I/IIa: Dose-Ranging Safety and Efficacy Study of Topical Resiquimod for the Treatment of Early-Stage Cutaneous T-Cell Lymphoma
Principal Investigator Alain Rook, MD
Start Date April 2012
Sponsor Alain Rook, MD
Coordinator Marie Buchanan, RN
Number of Patients 16
Enrolling Yes
Purpose The purpose of this study is to explore the safety and preliminary efficacy of two concentrations of resiquimod gel (0.06% and 0.2%) applied to lesions of early stage (lA, lB, llA) CTCL
Description of the Study

Treatment groups will be:

  1. Resiquimod 0.06% will be applied in dosing frequencies that are periodically adjusted according to tolerability. Subjects will begin dosing at 3 times per week (3x/wk), and will be evaluated at the clinic every two weeks. The dosing frequency (1, 2, 3, 5, or 7x/wk) may be adjusted in a stepwise manner after each two week interval based on the physician assessment of tolerability (maintained, increased, decreased with or without a dosing interruption [rest period]). Resiquimod will be applied for 8 weeks (COT1) followed by a 4 week no-treatment period. If the subject has not required permanent discontinuation from treatment, the subject will repeat a second course of treatment of 8 weeks (COT2) followed by a 4 week no-treatment period. Subjects will apply up to 500 mg of study drug per day based upon the total surface area that is treated (~250 mg of product / 50 cm2 of lesion surface area).
  2. Resiquimod 0.2% applied as described for Treatment Group 1. However, initial applications will be once weekly due to the increased potency of this concentration with the dosing frequency adjusted upward as tolerated every two weeks.
Inclusion Criteria
  1. Males or female ≥18 years of age at the time of study enrollment
  2. Have a clinical diagnosis of cutaneous T cell lymphoma CTCL, including documentation of a skin biopsy within the prior 3 years with histological findings consistent with CTCL (atypical epidermotrophic or folliculocentric T-cells).
  3. Have Stage IA, IB or IIA: T1 or T2 (patches or plaques) with measurable lesions.
  4. Previous treatment with at least one standard therapy used to treat Stage IA, IB or IIA CTCL including but not limited to oral corticosteroids, high-potency topical corticosteroids, topical mechlorethamine, topical bexarotene, PUVA, UVB, total body electron beam radiation, biological response or oral methotrexate.
  5. Have measurable skin disease with at least 1 to 4 eligible baseline target lesions with a total area >25 cm2 but <100 cm2. Eligible lesions must be below the neck and may not involve the genitalia, intertriginous areas, internally, or to frankly ulcerated or infected skin.
  6. Generally healthy other than for CTCL, or with other stable diseases/conditions that are adequately controlled.
  7. Willing and able to provide written informed consent.
  8. Willing and able to adhere to the protocol requirements, including but not limited to study drug dosing, study drug visits, medication and treatment restrictions, and laboratory tests.
  9. Willing and able to discontinue concomitant medications or treatments for CTCL during the study.
  10. If a female of child bearing potential, willing to use adequate contraception (defined as double-method contraception, e.g. oral contraceptive usage by subject and condom by partner). Non-child bearing potential is defined as being at least 2 years post-menopausal or being surgically sterile.
  11. Willing to abstain from therapeutic sunbathing, tanning beds, etc. for the duration of the study.
Exclusion Criteria
  1. Have a known allergy to resiquimod or any of the excipients in the study drug.
  2. Stage IIB or greater CTCL.
  3. Require immediate treatment for progressive CTCL.
  4. Are unable to discontinue current treatment for CTCL due to risk of progression.
  5. Within 8 weeks of treatment initiation (Day 0), have received treatment with:
    • Imiquimod
    • Total body electron beam radiation
    • Investigational drugs or treatments
    • PUVA
  6. Within 4 weeks of treatment initiation (Day 0), have received treatment with:
    • Local radiation therapy
    • UVB therapy
    • Any topical chemotherapy
    • Photopheresis
    • Systemic retinoids, corticosteroids, immune response modifiers (other than imiquimod), interferon inducers, chemotherapeutic agents, biologic agents including interferon
    • Topical corticosteroids or retinoids
  7. Within 2 weeks of treatment initiation (Day 0), have received at or adjacent to the target treatment lesions.
    • Any surgical procedures other than biopsies related to CTCL diagnosis or follow-up
    • Any topical treatment other than bland moisturizers (creams, lotions, emollients, etc).
  8. Have other concurrent cutaneous conditions in the treatment area or immediately adjacent to the treatment area that would be exacerbated by resiquimod or interfere with assessments.
  9. Have a grade 2 or greater laboratory abnormalities (CTCAE v4) at baseline for any of the following:
    • Hemoglobin
    • White blood cell count
    • Platelet count
    • Alanine transferase
    • Aspartate transferase
    • Creatinine
  10. Have a known history of or a positive serologic test for infection with human immunodeficiency virus or human T lymphotrophic virus.
  11. Are pregnant or nursing, or intending to become pregnant within the duration of the study.
  12. Have any clinically significant medical conditions that are unstable, progressive, or inadequately controlled in the opinion of the investigator, that would pose a potential risk for the subject, result in poor compliance with the study requirements, or require treatment with an excluded medication or treatment during the study.
  13. Have an active chemical or alcohol dependency as assessed by the investigator.
  14. Have systemic collagen vascular disorder, systemic autoimmune disease, an organ transplant or diagnosis of cancer within 5 years other than CTCL (not including basal cell carcinoma, non-invasive squamous cell cancer of the skin, malignant melanoma in situ, or cervical carcinoma in situ).
Mental Health Outcomes of Patients Undergoing Minimally-Invasive Cosmetic Surgery
Principal Investigator Joseph Sobanko
Co-Investigator: Ivona Percec, MD, PhD
Start Date
Sponsor Center for Human Appearance
Coordinator Joseph Sobanko, MD
Number of Patients Up to 140 subjects (70 cosmetic subjects and 70 non-cosmetic control subjects)
Enrolling Yes
Purpose

This study intends to evaluate the demographic and psychosocial aspects of patients seeking minimally invasive cosmetic procedures in the outpatient setting. This study will also examine the body image, self-esteem, and quality of life of subjects seeking such procedures, in addition to the change in these psychosocial parameters after treatment. Finally, the relationship between patient desire for facial rejuvenation and objective rating of attractiveness and apparent age will be assessed.

Description of the Study
  • Survey research (the main focus of the research is administration of a survey to research subjects)
  • Surveys and photos will be collected at time 0 and in follow up between 2-6 weeks and at 12 weeks.
  • Subjects will complete questionnaires (demographics, RSE, DAS59, BIQLI) at day 0/pre-procedure, and at 2-6 and 12 weeks post-procedure
  • Subjects will be evaluated by the Glogau Aging Scale at day 0, 2-6 weeks, and 12 weeks post-procedure.
  • Photographs taken at weeks 0, 6, and 12 for all subjects.
  • The visit at 12 weeks allows the investigator to determine if additional treatments are necessary particularly for botulinum toxin as its duration wears off between 12-16 weeks.
Inclusion Criteria
  1. Patients between the ages of 25 and 70 seeking minimally-invasive facial rejuvenation cosmetic procedures: botulinum toxin injection or soft tissue filler injection
  2. Patients between the ages of 25 and 70 seeking non-cosmetic procedures (control group)
Exclusion Criteria
  1. Age < 25 years or >70 years
  2. Inability to provide informed consent
  3. History of any prior facial cosmetic surgery or minimally-invasive procedure as this may obscure the objective physical outcomes of the procedures.
  4. History of body dysmorphic disorder as this may complicate the ability to measure changes in body image and self-esteem after surgery and would limit the ability to generalize our findings.
  5. Significant craniofacial abnormalities or facial scarring (i.e. acne scarring, traumatic scarring) as this population may have a unique intrinsic motivation for seeking aesthetic improvement of their appearance.
Ablative Fractional Laser Resurfacing of Surgical Scars 1 Week after Mohs Surgery
Principal Investigator Joseph F. Sobanko, MD
Co-principal investigator Christopher J. Miller and J. Tzu
Start Date
Sponsor Investigator-initiated study
Coordinator
Number of Patients Up to 24 subjects
Enrolling Yes
Purpose The purpose of this study is to assess the efficacy of the 10,600-nm CO2 fractional laser on postsurgical scars.
Description of the Study A prospective randomized, split-scar study will be conducted on 24 subjects between the ages of 20-90. Subjects will have one half of their scar treated with a single-session of the 10,600-nm CO2 fractional laser. The remaining scar half will be designated as a control. Scars will be re-evaluated clinically and histologically 12 weeks later.
Inclusion Criteria
  1. Patients between the ages of 20 and 90 years with a post-surgical scar on the face.
  2. Patients whose scar is linear and visibly symmetric, measuring at least 4 cm.
  3. Able and willing to follow study procedures.
  4. Able to understand and sign a written informed consent.
Exclusion Criteria
  1. Patients with prior laser or resurfacing procedures to the scar
  2. Patients with propensity for keloid scarring
  3. Patients with recent use of oral retinoids
  4. Pregnancy
  5. Immunosuppression
  6. Patients with Fitzpatrick skin type V-VI
An Open-Label, Multi-Center, Randomized Study of Anti-CCR4 Monoclonal Antibody KW-0761 (mogamulizumab) Versus Vorinostat in Subjects with Previously Treated Cutaneous T-Cell Lymphoma
Principal Investigator Ellen Kim, MD
Start Date July 2013
Sponsor Kyowa Hakko Kirin Pharma, Inc.
Coordinator Kathleen Kirchner, MS
Number of Patients 10
Enrolling Yes
Purpose

The purpose of this study is to compare the treatment of KW-0761(mogamulizumab) versus vorinostat (Zolinza) in subjects with relapsed or refractory CTCL. Comparisons of progression free survival, overall response rates, and improvements in skin condition and quality of life will be conducted.

Description of the Study

This is an open-label, multi-center, randomized, phase 3 study with a 1:1 randomization of study drug, KW-0761 versus the comparator, vorinostat in subjects diagnosed with Stage IB, II-A, II-B, III and IV mycosis fungoides (MF) or Sézary Syndrome (SS) who have progressed following at least one prior course of systemic therapy. Treatment will be administered on an outpatient basis. The dose of KW-0761 will be 1.0 mg/kg. The dose of vorinostat will be the recommended dose of 400 mg (once daily with food). Each treatment cycle is 28 days. Subjects will receive KW-0761 as an IV infusion over at least 1 hour on four times throughout the first cycle and twice during each subsequent cycle. Vorinostat will be administered orally daily. Subjects may remain in the treatment phase up until progressive disease, drug intolerance or unacceptable toxicity, or until any of the other criteria for study removal are met. Subjects who have received at least two full treatment cycles and demonstrate progression of disease on treatment with vorinostat at the 8 week assessment of the second cycle may be eligible to cross over to treatment with KW-0761.

Inclusion Criteria
  1. Voluntarily signed and dated Institutional Review Board / Ethics Committee approved informed consent form in accordance with regulatory and institutional guidelines. Written informed consent must be obtained prior to performing any study-related procedure.
  2. Males and female subjects > 18 years of age at the time of enrollment.
  3. Histologically confirmed diagnosis of MF or SS within 3 months of the Pre-treatment Visit.
    • For SS (defined as meeting T4 plus B2 criteria), where the biopsy of erythrodermic skin may only reveal suggestive but not diagnostic histopathologic features, the diagnosis may be based on either a node biopsy or fulfillment of B2 criteria including a clone in the blood that matches that of the skin.
  4. Stage IB, II-A, II-B, III and IV.
  5. Subjects who have progressed following at least one prior course of systemic therapy (e.g., interferon, denileukin diftitox, bexarotene, photopheresis, anti-neoplastic chemotherapy, etc.).
  6. Eastern Cooperative Oncology Group (ECOG) performance status score of < 1 at study entry.
  7. The subject has resolution of all clinically significant toxic effects of prior cancer therapy to Grade < 1 by the National Cancer Institute Common Terminology Criteria for Adverse Events, version 4.0 (NCI-CTCAE, v.4.0) excluding the specifications required in 8, 9 and 10 below.
  8. Adequate hematological function:
    • absolute neutrophil count (ANC) > 1,500 cells/μL (> 1,500/mm3)
    • platelets > 100,000 cells/μL; (> 100,000/mm3)
    • in subjects with known bone marrow involvement, ANC must be > 1,000 cells/μL (>1,000/mm3) and platelets > 75,000 cells/μL. (> 75,000/mm3)
  9. Adequate hepatic function:
    • bilirubin < 1.5 times the specific institutional upper limit of normal (ULN), except for subjects
    • with Gilbert's syndrome
    • aspartate transaminase (AST) and alanine transaminase (ALT) each < 2.5 x ULN or < 5.0 x ULN in the presence of known hepatic malignancy
  10. Adequate renal function:
    • serum creatinine < 1.5 x ULN or
    • calculated creatinine clearance > 50 mL/min using the Cockcroft-Gault formula
  11. Subjects previously treated with anti-CD4 antibody or alemtuzumab are eligible provided their CD4+ cell counts are > 200/mm3.
  12. Subjects with MF and a known history of non-complicated staphylococcus infection/colonization are eligible provided they continue to receive stable doses of prophylactic antibiotics.
  13. Women of childbearing potential (WOCBP) must have a negative pregnancy test within 7 days of receiving study medication.
  14. WOCBP must agree to use effective contraception, defined as oral contraceptives, double barrier method (condom plus spermicide or diaphragm plus spermicide) or practice true abstinence from sexual intercourse (periodic abstinence, e.g., calendar, ovulation, symptothermal, post-ovulation methods and withdrawal are not acceptable methods of contraception) during the study and for 3 months after the last dose. WOCBP includes any female who has experienced menarche and who has not undergone successful surgical sterilization or is not postmenopausal (defined as amenorrhea > 12 consecutive months).
  15. Male subjects and their female partners of child bearing potential must be willing to use an appropriate method of contraception defined as oral contraceptives, double barrier method (condom plus spermicide or diaphragm plus spermicide) or practice true abstinence from sexual intercourse (periodic abstinence, e.g., calendar, ovulation, symptothermal, post-ovulation methods and withdrawal are not acceptable methods of contraception) during the study and for 3 months after the last dose.
Exclusion Criteria
  1. Large cell transformation of SS as well as transformed MF.
  2. Have had a malignancy in the past two years. However, subjects with non-melanoma skin cancers, melanoma in situ, localized cancer of the prostate with current prostate-specific antigen of < 0.1 μg/mL, treated thyroid cancer or cervical carcinoma in situ or ductal/lobular carcinoma in situ of the breast within the past two years may enroll as long as there is no current evidence of disease.
  3. (< 1 ng/mL), treated thyroid cancer or cervical carcinoma in situ or ductal/lobular carcinoma in situ of the breast with in the past two years may enroll as long as there is no current evidence of disease.
  4. Clinical evidence of central nervous system (CNS) metastasis.

Medical or pharmaceutical industry professionals interested in sponsoring or learning more about Penn Dermatology's clinical trials can contact joel.gelfand@uphs.upenn.edu or 215-662-2737.

Autoimmune Skin Disease Study Unit

The autoimmune skin disease studies are directed by Victoria Werth, MD. Dr. Werth oversees a team of highly skilled and experienced and certified research coordinators and nurses.

Autoimmune Skin Disease Clinical trials that are currently in progress and offer ongoing enrollment include:

A Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of QGE031 in the Treatment of Patients with Bullous Pemphigoid with Disease Refractory to Oral Steroid Treatment
Principal Investigator Victoria Werth, MD
Start Date August 2012
Sponsor Novartis Pharmaceuticals
Coordinator Nicole Jochym, BS, BA
Number of Patients 4
Enrolling Yes
Purpose Study of efficacy, safety and blood-related changes of QGE031 in bullous pemphigoid (BP) patients not responding adequately to oral steroid treatment.
Description of the Study

This is a multicenter, randomized, placebo controlled study designed to assess proof of concept for the treatment of BP with QGE031. The study will examine the efficacy of QGE031 relative to placebo at 12 weeks in patients with BP by reducing disease activity as determined by the Clinical Global Assessment of Change (CGA-C) responder rate.

Study Design: This study is divided into 2 distinct parts.

In Part 1, patients with IgE levels up to 5000 IU/mL inclusive at Screening will begin a 12 week treatment period with QGE031 or placebo in a 2:1 ratio. When predefined efficacy criteria are met, patients will begin a steroid taper, as necessary, to the lowest dose needed to address disease symptoms in combination with study treatment. Conversely, if patients show a lack of efficacy at predefined decision points they will be withdrawn from treatment. After the treatment period patients will enter a follow-up period lasting for up to 12 months where disease relapse activity will be monitored.

Part 2 of the study will be open label and will evaluate 2 lower dose levels and regimens of QGE031 over 12 weeks of treatment in successive cohorts.

Inclusion Criteria
  • Active disease is defined as =3 % body surface area with any combination of urticarial plaques, erosions or blisters, as assessed by the investigator.
  • Patients must be on a stable dose of prednisone of =10mg per day (or equivalent oral steroid dose) but no greater than 1 mg/kg/day at baseline.
  • Patients with a total IgE up to 5000 IU/mL inclusive at screening.
Exclusion Criteria
  • Use of rituximab within the preceding 1 year of baseline. .
  • Use of prednisone or other systemic steroids (excluding inhaled) for conditions other than bullous pemphigoid within the 1 month prior to baseline. .
  • Use of topical steroids within 2 weeks prior to baseline. .
  • Recent previous treatment with phototherapy, biological therapy, immunosupressive agents such as azathioprine, dapsone, or methotrexate.
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of R333 6% Ointment Administered Topically to Discoid Lupus Erythematosus (DLE) and Systemic Lupus Erythematosus (SLE) Patients with Active Cutaneous Discoid Lesions
Principal Investigator Victoria Werth, MD
Start Date July 2012
Sponsor Rigel Pharmaceuticals
Coordinator Nicole Jochym, BS, BA
Number of Patients 4
Enrolling Yes
Purpose

To assess the preliminary efficacy, safety, tolerability and pharmacokinetics of R333 6% ointment administered topically for 28 days to active discoid lesions as determined by change from baseline in erythema and scaling.

Description of the Study

This is a Phase II, multi-center, randomized, double-blind, placebo-controlled study to evaluate the preliminary efficacy, safety, tolerability, and pharmacokinetics (PK) of topical R333 ointment in patients with discoid lupus (DLE) and systemic lupus (SLE) with active discoid lesions. Patients will be randomized into one of two groups (either R333 or placebo), resulting in a randomization ratio of 2:1 active to placebo. Study drug will be applied topically twice a day for 28 days, using an amount to cover completely the selected active discoid lesions. The concentration per patient will not exceed a total R333 dose of 480 mg/day (8 g of R333 6%) and a body surface area (BSA) of = 5%. Follow-up visits will occur 7 and 14 days after the final dosing.

Inclusion Criteria
  • At least 2 active discoid lesions secondary to SLE or DLE prior to study entry, each with a minimum Erythema Rating Score of = 2. At least 1 of the active discoid lesions must have been present (by history) for = 3 weeks prior to screening (Visit 1).
  • Patients who are taking oral prednisone must be receiving stable daily doses for = 3 weeks prior to randomization and must remain on the same dose throughout the study. Prednisone as high as 10 mg/day (or equivalent) is allowed.
  • Patients who are taking azathioprine, hydroxychloroquine, chloroquine, quinicrine, methotrexate, and/ or oral glucocorticoids, must be receiving a stable daily dose = 4 weeks prior to randomization and must remain on the same dose throughout the study.
Exclusion Criteria
Prevalence and Clinical Severity of Cutaneous Lupus Erythematosus (CLE Database)
Principal Investigator Victoria Werth, MD
Start Date 2006
Sponsor Investigator initiated
Coordinator Ioannis Koutroulis, MD
Number of Patients 400
Enrolling Enrollment of subjects began in 2006 and continues on a regular basis in the dermatology clinic at the Penn Perelman Medical Center. At present there are over 300 subjects enrolled in the CLE database and enrollment is ongoing.
Purpose The CLE database, which is conducted at Penn and at its sub-site, the University of Texas Southwestern Medical Center in Dallas (UTSW), is designed to assess disease severity and treatment responsiveness in various subtypes of CLE. The database includes a blood and tissue bank CLE Tissue Bank, which was started at Penn in 2009 as a foundation for future genetic, biomarker and pathophysiologic studies. The CLE database is a seminal source of patient data that has led to numerous other investigations about the CLE population.
Description of the Study The CLE database for lupus is a prospective, questionnaire-based study that is designed to assess disease severity and treatment responsiveness in various subtypes of CLE. Begun at Penn in 2006, the CLE database is expected to continue enrolling patients until the year 2019. Potential subjects are recruited from among the dermatologic clinic patients seen by the principal investigators at Penn and at the sub-site, University of Texas Southwestern Medical Center in Dallas (UTSW). Subjects are interviewed at their regularly scheduled clinic appointments and receive skin evaluations using the CLASI (cutaneous lupus area and severity index), which was developed by the principal investigator, Dr. Werth. The lesional CLASI sub-study, which is included in the CLE database, evaluates a cohort of subjects with cutaneous lupus in terms of individual lesion response to treatment.
Inclusion Criteria
Exclusion Criteria
Evaluation of Clinical Responsiveness Using the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)—The CDASI Study
Principal Investigator Victoria Werth, MD
Start Date 2008
Sponsor Investigator-initiated study
Coordinator Ioannis Koutroulis, MD
Number of Patients No established limit
Enrolling Subjects have been steadily enrolled in the CDASI study since its inception in 2008. Over 100 participants are currently enrolled in the study.
Purpose The Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) is a validated, reliable research tool that objectively evaluates skin severity in patients with dermatomyositis (DM). The CDASI study utilizes this tool to evaluate prospectively whether CDASI scores correlate reliably with results of successful therapy over time. Patients with dermatomyositis from the investigator's dermatology clinic who are being treated at their regularly scheduled appointments are given questionnaires and surveys to complete along with having their skin evaluated using the CDASI tool.
Description of the Study This is a study of DM subjects who are followed during regularly scheduled clinic visits and are asked to provide data leading to evaluation of clinical responsiveness of their skin disease to the CDASI and other evaluative instruments.
Inclusion Criteria
Exclusion Criteria

Medical or pharmaceutical industry professionals interested in sponsoring or learning more about Penn Dermatology's autoimmune skin disease clinical trials can contact werth@mail.med.upenn.edu or 215-898-0168.