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 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 July 2012
Sponsor National Heart, Lung, and Blood Institute (NHLBI)
(Study product provided by Abbott)
Coordinator Suzette VanderBeek and Rosemary Attor, CRC
Number of Patients 5
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.
  • Clinical diagnosis of psoriasis for at least 6 months as determined by subject interview of his/her medical history and confirmation of diagnosis through physical examination by Investigator.
  • 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 defined by ≥ 10 percent Body Surface Area (BSA) involvement at the Baseline (Week 0) visit.
  • PASI score of ≥ 12 at the Baseline (Week 0) visit.
  • Subject is a 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:
    • Women of childbearing potential must undergo monthly pregnancy testing during the study and agree to use two of the following methods of contraception throughout the 6-month 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.
    • Women who are postmenopausal (for at least one year), sterile, or hysterectomized;
    • Women who have undergone tubal ligation will be required to undergo monthly pregnancy testing during the duration of the study and agree to use a second form of contraception which includes:
      • 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
    • Sexual abstinence, defined as total abstinence from sexual intercourse, is considered an adequate form of contraception. (Agreement to comply with sexual abstinence must be recorded in the source document.)
  • Subject is 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.
  • Subject is 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.
  • Subject has 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;
    • Subject has received vaccination with Bacille Calmette-Guerin (BCG) within 365 days prior to Screening;
    • Subject has 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.
  • Subject has 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 subject is on 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 Up to 96 patients will be enrolled across 6 different study sites including the University of Pennsylvania
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
  • A screening period prior to study treatment initiation will allow for study eligibility assessment and washout of current psoriasis therapies (at least 14 days for topical treatments or UVB phototherapy; 30 days for psoralen-ultraviolet A phototherapy or non-biologic systemic therapy, 90 days for all biologics other than ustekinumab, and 180 days for ustekinumab).
  • After the washout period is completed, patients will be randomized to one of three treatment groups and receive either 1) adalimumab; 2) NB-UVB phototherapy; or 3) placebo injections. Study staff will administer subcutaneous adalimumab or placebo injections according to its FDA-approved schedule for subjects who are randomized into one of the two groups receiving injections. Study subjects randomized to the NB-UVB phototherapy group will be seen 3 times per week for NB-UVB, for up to 36 treatments of each lasting approximately 5-10 minutes as part of routine care.
  • During the screening period, baseline, week 4, 8, and 12, the patient will be evaluated by investigators blinded to treatment allocation status. Psoriasis severity will be assessed using the Psoriasis Area and Severity Index (PASI) and the Physician Global Assessment (PGA), both widely accepted measurement tools for psoriasis. Patient-reported outcomes, e.g. the EuroQol EQ-5D, the Dermatology Life Quality Index (DLQI), the MEDFICTS dietary assessment instrument, and the International Physical Activity Questionnaire (IPAQ) will be assessed at baseline and weeks 4, 8 and 12.
  • FDG-PET/CT will be conducted at baseline and week 12 for assessing the primary study endpoints.
  • Laboratory tests will be conducted at screening, baseline and weeks 4 and 12 for screening safety labs and/or CV biomarker assessment.
Inclusion Criteria
  1. Males and females 18 years of age and older.
  2. Clinical diagnosis of psoriasis for at least 6 months as determined by subject interview of his/her medical history and confirmation of diagnosis through physical examination by Investigator.
  3. 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.
  4. Moderate to severe psoriasis defined by ≥ 10 percent Body Surface Area (BSA) involvement at the Baseline (Week 0) visit.
  5. PASI score of ≥ 12 at the Baseline (Week 0) visit.
  6. Subject is a candidate for systemic therapy or phototherapy and has active psoriasis despite prior treatment with topical agents.
  7. Women are eligible to participate in the study if they meet one of the following criteria:
    1. Women of childbearing potential must undergo monthly pregnancy testing during the study and agree to use two of the following methods of contraception throughout the 6-month study:
      1. Oral contraceptives;
      2. Transdermal contraceptives
      3. Injectable or implantable methods
      4. Intrauterine devices
      5. Barrier methods (for example but not limited to a diaphragm with spermicide, condom with spermicide); or
      6. 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.

    2. Women who are postmenopausal (for at least one year), sterile, or hysterectomized;
    3. Women who have undergone tubal ligation will be required to undergo monthly pregnancy testing during the duration of the study and agree to use a second form of contraception which includes:
      1. Oral contraceptives;
      2. Transdermal contraceptives
      3. Injectable or implantable methods
      4. Intrauterine devices
      5. Barrier methods (for example but not limited to a diaphragm with spermicide, condom with spermicide); or
      6. Vasectomized partner
    4. Sexual abstinence, defined as total abstinence from sexual intercourse, is considered an adequate form of contraception. (Agreement to comply with sexual abstinence must be recorded in the source document.)
  8. Subject is 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.
  9. Able and willing to give written informed consent and to comply with requirements of this study protocol.
Exclusion Criteria

Subjects meeting any of the following criteria will be excluded from the study:

  1. 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.
  2. Previous lack of response to a TNF-alpha antagonist and/or UV phototherapy that led to discontinuation of either of these therapies.
  3. Diagnosis of erythrodermic psoriasis, generalized or localized pustular psoriasis, medication-induced or medication-exacerbated psoriasis, or new onset guttate psoriasis.
  4. Diagnosis of other active skin diseases or skin infections (bacterial, fungal, or viral) that may interfere with evaluation of psoriasis.
  5. Cannot avoid UVB phototherapy for at least 14 days prior to the Baseline (Week 0) visit.
  6. Cannot avoid psoralen-UVA phototherapy for at least 30 days prior to the Baseline (Week 0) visit and during the study.
  7. 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.
  8. Subject is taking or requires oral or injectable corticosteroids during the study.  Inhaled corticosteroids for stable medical conditions are allowed. 
  9. 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.
  10. 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%)
  11. Uncontrolled hypertension, with measured systolic blood pressure >180 mmHg or diastolic blood pressure >90 mmHg
  12. History of demyelinating diseases or lupus.
  13. Subject has 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;
    • Subject has received vaccination with Bacille Calmette-Guerin (BCG) within 365 days prior to Screening;
    • Subject has 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.
  14. Subject has 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.
  15. Female subject who is pregnant or breast-feeding or considering becoming pregnant during the study.
  16. 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
  17. Recent history of substance abuse or psychiatric illness that could preclude compliance with the protocol.
  18. History of any substance abuse within 365 days of screening visit
  19. Alcohol use >14 drinks per week at the screening visit or within 30 days of the screening period
  20. If subject is on 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.
  21. History of photosensitivity of medical condition that may be exacerbated by UV exposures such as lupus or dermatomyositis
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

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.