Trial | NCT03578029  Report issue

Title

Evaluation of the Safety and Efficacy Study of RGN-137 Topical Gel for Junctional and Dystrophic Epidermolysis Bullosa
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)

  • Phase

    Phase 2

  • Study Type

    Interventional

  • Status

    Terminated

  • Intervention/Treatment

    RGN-137 ...

  • Study Participants

    4
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).
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.
Study Started
May 22
2019
Primary Completion
Sep 01
2021
Study Completion
Nov 23
2021
Last Update
Aug 22
2022

Drug RGN-137

It will be applied topically to the appropriate wound once a day for up to 84 days.

  • Other names: Dermal Topical Gel

Drug Placebo

It will be applied topically to the appropriate wound once a day for up to 84 days.

  • Other names: Vehicle Control

RGN-137 Experimental

It is formulated as a gel for topical administration.

Placebo Placebo Comparator

It is composed of the same excipients as RGN-137 formulation without the active ingredient.

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

Index wounds must have a surface area between 5 cm2 and 50 cm2, inclusive at the screening visit
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
No Results Posted