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

NCT03578029 | Terminated Phase 2 FDA Drug

• 1 other identifier: RGN-137-EB-202
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 5

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).

Conditions

Junctional Epidermolysis Bullosa; Dystrophic Epidermolysis Bullosa

Keywords

Epidermolysis Bullosa

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• 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.

  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.

  up to Day 84

• Change from Baseline and percent change from Baseline in surface area of each index wound at the scheduled visits.

  up to Day 84

Other Outcomes (1)

• Index wound characteristics at the scheduled visits.

  up to Day 84

Study Arms (2)

RGN-137

EXPERIMENTAL

It is formulated as a gel for topical administration.

Drug: RGN-137

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

RGN-137 DRUG

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

Also known as: Dermal Topical Gel

RGN-137

Placebo DRUG

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

Also known as: Vehicle Control

Placebo

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Lenus Therapeutics, LLC: lead

Study Sites (5)

Northwestern University

Evanston, Illinois, 60208, United States

Location

SUNY Downstate Medical Center

Brooklyn, New York, 11203, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Texas Dermatology and Laser Specialists

San Antonio, Texas, 78218, United States

Location

MeSH Terms

Conditions

Epidermolysis Bullosa, Junctional; Epidermolysis Bullosa Dystrophica; Epidermolysis Bullosa

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous; Collagen Diseases; Connective Tissue Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 14, 2018
• First Posted: July 5, 2018
• Study Start: May 22, 2019
• Primary Completion: September 1, 2021
• Study Completion: November 23, 2021
• Last Updated: August 22, 2022

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)