Clinical trial NCT03578029: Evaluation of the Safety and Efficacy Study of RGN-137 Topical Gel for Junctional and Dystrophic Epidermolysis Bullosa
Official 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)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Junctional Epidermolysis Bullosa
Dystrophic Epidermolysis Bullosa
Keywords: Epidermolysis Bullosa
Lead sponsor: Lenus Therapeutics, LLC
Sponsor class: INDUSTRY
First posted: 2018-07-05
Enrollment: 4
Sex: ALL
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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).

Detailed description: RGN-137 will be evaluated for efficacy and safety compared to a Placebo. A matched-pair design will be used to evaluate RGN-137 treatment versus placebo for treatment of 15 subjects with JEB or DEB. Eligible subject must have 1 set of matched-pair wound. The investigator will assign pair of index wounds, each wound with an area between 5cm2 and 50cm2, inclusive, for the eligible subject on Day 1, and for each pair, one wound will be randomized to receive RGN-137 gel and the other to receive Placebo gel. Subjects and independent evaluator will be blinded to the treatment assignments for each wound.

Eligibility criteria: 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

Primary outcomes:
measure: Time to achieving 50% reduction in area of each index wound from Day 1 up to Day 84.
timeFrame: up to Day 84
Secondary outcomes:
measure: 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.
timeFrame: up to Day 84
measure: Incidence of 50%, 75%, and 100% reduction and complete re-epithelialization without drainage in area of each index wound at the scheduled visits.
timeFrame: up to Day 84
measure: Change from Baseline and percent change from Baseline in surface area of each index wound at the scheduled visits.
timeFrame: up to Day 84
Other outcomes:
measure: Index wound characteristics at the scheduled visits.
timeFrame: up to Day 84
Locations:
city: Evanston
country: United States
facility: Northwestern University
state: Illinois
city: Brooklyn
country: United States
facility: SUNY Downstate Medical Center
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: San Antonio
country: United States
facility: Texas Dermatology and Laser Specialists
state: Texas
Study officials:
References: