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

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)

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).

Study Overview

• Status: Terminated
• Conditions: Dystrophic Epidermolysis Bullosa; Junctional Epidermolysis Bullosa
• Intervention / Treatment: Drug: RGN-137; Drug: Placebo

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.

• Study Type: Interventional
• Enrollment (Actual): 4
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Illinois
    • Evanston, Illinois, United States, 60208
      • Northwestern University
  • New York
    • Brooklyn, New York, United States, 11203
      • SUNY Downstate Medical Center
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • The Children's Hospital of Philadelphia
  • Texas
    • San Antonio, Texas, United States, 78218
      • Texas Dermatology and Laser Specialists

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: RGN-137; It is formulated as a gel for topical administration.	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
Placebo Comparator: Placebo; It is composed of the same excipients as RGN-137 formulation without the active ingredient.	Drug: Placebo; It will be applied topically to the appropriate wound once a day for up to 84 days.; Other Names: Vehicle Control

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Index wound characteristics at the scheduled visits.	(inflammation/erythema, induration, crusting, exudate, cellulitis, and other abnormalities)	up to Day 84

Collaborators and Investigators

• Sponsor: Lenus Therapeutics, LLC

Study record dates

Study Major Dates

• Study Start (Actual): May 22, 2019
• Primary Completion (Actual): September 1, 2021
• Study Completion (Actual): November 23, 2021

Study Registration Dates

• First Submitted: June 14, 2018
• First Submitted That Met QC Criteria: June 24, 2018
• First Posted (Actual): July 5, 2018

Study Record Updates

• Last Update Posted (Actual): August 22, 2022
• Last Update Submitted That Met QC Criteria: August 18, 2022
• Last Verified: August 1, 2022

More Information

Terms related to this study

• Keywords: Epidermolysis Bullosa
• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Connective Tissue Diseases; Skin Diseases, Genetic; Skin Diseases, Vesiculobullous; Skin Abnormalities; Collagen Diseases; Epidermolysis Bullosa; Epidermolysis Bullosa Dystrophica; Epidermolysis Bullosa, Junctional
• Other Study ID Numbers: RGN-137-EB-202

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No