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

NCT ID: NCT03578029

Last Updated: 2022-08-22

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 4 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-05-22
• Study Completion Date: 2021-11-23

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.

Conditions

Junctional Epidermolysis Bullosa; Dystrophic Epidermolysis Bullosa

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

RGN-137

It is formulated as a gel for topical administration.

Group Type: EXPERIMENTAL

RGN-137

Intervention Type: DRUG

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

Placebo

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

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

Interventions

RGN-137

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

Intervention Type: DRUG

Placebo

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

Intervention Type: DRUG

Other Intervention Names

Dermal Topical Gel; Vehicle Control

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

• Minimum Eligible Age: 4 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Lenus Therapeutics, LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Northwestern University

Evanston, Illinois, United States

SUNY Downstate Medical Center

Brooklyn, New York, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Texas Dermatology and Laser Specialists

San Antonio, Texas, United States

Countries

United States

Other Identifiers

RGN-137-EB-202

Identifier Type: -

Identifier Source: org_study_id