Dystrophic Epidermolysis Bullosa Clinical Trial
— CELEBOfficial title:
A Randomized,Single-Blind, Placebo-controlled, Self-matched Pairing, Independent Evaluated Study to Evaluate the Efficacy and Safety of RGN-137 Topical Gel in Subjects With Junctional and Dystrophic Epidermolysis Bullosa (CELEB)
Verified date | August 2022 |
Source | Lenus Therapeutics, LLC |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The objective of this study is to compare the efficacy and safety of RGN-137 topical gel with that of placebo gel for treatment of junctional epidermolysis bullosa (JEB) or dystrophic epidermolysis bullosa (DEB).
Status | Terminated |
Enrollment | 4 |
Est. completion date | November 23, 2021 |
Est. primary completion date | September 1, 2021 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 4 Years and older |
Eligibility | Inclusion Criteria: - Male or female and at least 4 years old with a diagnosis of DEB or JEB - Patients and their parents or guardians must be willing and able to provide written informed consent/assent - Presence of 1 pair of stable index wounds within the specified size range at study 1. Index wounds must have a surface area between 5 cm2 and 50 cm2, inclusive at the screening visit 2. The index wounds selected as pairing must be relatively matched in terms of size and location - Women of childbearing potential must have a negative pregnancy test prior to randomization - Sexually active subjects must agree to use medically accepted methods of contraception during the study Exclusion Criteria: - Have any clinical evidence of local infection of the index lesion - Use of prior or concomitant medication (Any investigational drug within 30 days, Immunotherapy or cytotoxic chemotherapy within 60 days, Systemic steroidal therapy within 30 days, and topical steroidal therapy within 14 days) - History of sensitivity to any component of the treatment - Neurological, cardiovascular, respiratory, hepatic, renal, or metabolic disease likely to interfere with the subject's participation in or completion of the study (At the discretion of investigator, participants with cardiomyopathy can participate) - Current or former malignancy, including a history of squamous cell carcinomas - Arterial or venous disorder resulting in ulcerated wounds - Uncontrolled diabetes mellitus - Pregnancy or breastfeeding during the study - Girls or women who have had menarche but have not completed menopause - Any condition or situation likely to cause the subject to be unable or unwilling to participate in the study procedures |
Country | Name | City | State |
---|---|---|---|
United States | SUNY Downstate Medical Center | Brooklyn | New York |
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
United States | Northwestern University | Evanston | Illinois |
United States | The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania |
United States | Texas Dermatology and Laser Specialists | San Antonio | Texas |
Lead Sponsor | Collaborator |
---|---|
Lenus Therapeutics, LLC |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Index wound characteristics at the scheduled visits. | (inflammation/erythema, induration, crusting, exudate, cellulitis, and other abnormalities) | up to Day 84 | |
Primary | Time to achieving 50% reduction in area of each index wound from Day 1 up to Day 84. | This primary endpoint will the time to achieving 50% reduction in area of each index wound from Day 1 up to Day 84, defined as date of the first study visit on which 50% reduction in index wound area is achieved minus date of Day 1 visit. | up to Day 84 | |
Secondary | Time to achieving 50%, 75%, and 100% (with drainage or without drainage) reduction in area of each index wound from Day 1 up to Day 84. | This secondary endpoint will the time to achieving 50%, 75%, and 100% reduction in area of each index wound from Day 1 up to Day 84, defined as date of the first study visit on which 50% reduction in index wound area is achieved minus date of Day 1 visit. | up to Day 84 | |
Secondary | Incidence of 50%, 75%, and 100% reduction and complete re-epithelialization without drainage in area of each index wound at the scheduled visits. | This secondary endpoint will be summarized by treatment using frequency counts and percentages. | up to Day 84 | |
Secondary | Change from Baseline and percent change from Baseline in surface area of each index wound at the scheduled visits. | This secondary endpoint will be computed for each treatment group as well as for treatment difference at each visit. | up to Day 84 |
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