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

Dystrophic Epidermolysis Bullosa Clinical Trial

— CELEB  

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)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03578029
• Other study ID #: RGN-137-EB-202
• Status: Terminated
• Phase: Phase 2
• Start date: May 22, 2019
• Est. completion date: November 23, 2021

Study information

• Verified date: August 2022
• Source: Lenus Therapeutics, LLC
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• 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

Related Conditions & MeSH terms

• Dystrophic Epidermolysis Bullosa
• Epidermolysis Bullosa
• Epidermolysis Bullosa Dystrophica
• Epidermolysis Bullosa, Junctional
• Junctional Epidermolysis Bullosa

Intervention

Drug:
• RGN-137
It will be applied topically to the appropriate wound once a day for up to 84 days.
• Placebo
It will be applied topically to the appropriate wound once a day for up to 84 days.

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Lenus Therapeutics, LLC

Country where clinical trial is conducted

United States, 

Outcome

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