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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03578029
Recruitment Status : Terminated (Business Decision)
First Posted : July 5, 2018
Last Update Posted : August 22, 2022
Sponsor:
Information provided by (Responsible Party):
Lenus Therapeutics, LLC

Study Description
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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).

Condition or disease Intervention/treatment Phase
Junctional Epidermolysis Bullosa Dystrophic Epidermolysis Bullosa Drug: RGN-137 Drug: Placebo Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Outcomes Assessor)
Primary Purpose: Treatment
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)
Actual Study Start Date : May 22, 2019
Actual Primary Completion Date : September 1, 2021
Actual Study Completion Date : November 23, 2021

Resource links provided by the National Library of Medicine


Arms and Interventions
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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 Name: 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 Name: Vehicle Control


Outcome Measures
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Primary Outcome Measures :
  1. Time to achieving 50% reduction in area of each index wound from Day 1 up to Day 84. [ Time Frame: 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.


Secondary Outcome Measures :
  1. 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. [ Time Frame: 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.

  2. Incidence of 50%, 75%, and 100% reduction and complete re-epithelialization without drainage in area of each index wound at the scheduled visits. [ Time Frame: up to Day 84 ]
    This secondary endpoint will be summarized by treatment using frequency counts and percentages.

  3. Change from Baseline and percent change from Baseline in surface area of each index wound at the scheduled visits. [ Time Frame: up to Day 84 ]
    This secondary endpoint will be computed for each treatment group as well as for treatment difference at each visit.


Other Outcome Measures:
  1. Index wound characteristics at the scheduled visits. [ Time Frame: up to Day 84 ]
    (inflammation/erythema, induration, crusting, exudate, cellulitis, and other abnormalities)


Eligibility Criteria
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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03578029


Locations
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United States, Illinois
Northwestern University
Evanston, Illinois, United States, 60208
United States, New York
SUNY Downstate Medical Center
Brooklyn, New York, United States, 11203
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
Texas Dermatology and Laser Specialists
San Antonio, Texas, United States, 78218
Sponsors and Collaborators
Lenus Therapeutics, LLC
More Information
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Responsible Party: Lenus Therapeutics, LLC
ClinicalTrials.gov Identifier: NCT03578029    
Other Study ID Numbers: RGN-137-EB-202
First Posted: July 5, 2018    Key Record Dates
Last Update Posted: August 22, 2022
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Lenus Therapeutics, LLC:
Epidermolysis Bullosa
Additional relevant MeSH terms:
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Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Epidermolysis Bullosa, Junctional
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
 Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Collagen Diseases
Connective Tissue Diseases