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

• ClinicalTrials.gov Identifier: NCT03578029
• Recruitment Status: Terminated
• First Posted: July 5, 2018
• Last Update Posted: August 22, 2022
• Sponsor: Lenus Therapeutics, LLC
• Information provided by (Responsible Party): Lenus Therapeutics, LLC

Tracking Information

• First Submitted Date: June 14, 2018
• First Posted Date: July 5, 2018
• Last Update Posted Date: August 22, 2022
• Actual Study Start Date: May 22, 2019
• Actual Primary Completion Date: September 1, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 5, 2019)

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.

Original Primary Outcome Measures (submitted: June 24, 2018)

Time to complete re-epithelialization of index lesions up to Day 84. [ Time Frame: up to Day 84 ]

This primary endpoint will be the time to complete re-epithelialization of each index lesion up to Day 84, defined as date of the first study visit on which complete-re-epithelialization is achieved minus date of Baseline visit.

Current Secondary Outcome Measures (submitted: March 5, 2019)

• 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.
• 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.
• 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.

Original Secondary Outcome Measures (submitted: June 24, 2018)

• Change from Baseline in surface area of index lesions at the EOT Visit and the other 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.
• Incidence of complete re-epithelialization at the EOT Visit and the other scheduled visits. [ Time Frame: up to Day 84 ]
  This secondary endpoint will be summarized by treatment using frequency counts and percentages.

Current Other Pre-specified Outcome Measures (submitted: June 24, 2018)

Index wound characteristics at the scheduled visits. [ Time Frame: up to Day 84 ]

(inflammation/erythema, induration, crusting, exudate, cellulitis, and other abnormalities)

• Original Other Pre-specified Outcome Measures: Same as current

Descriptive Information

• Brief Title: Evaluation of the Safety and Efficacy Study of RGN-137 Topical Gel for Junctional and Dystrophic Epidermolysis Bullosa
• 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)
• 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.
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Outcomes Assessor); Primary Purpose: Treatment

Condition

• Junctional Epidermolysis Bullosa
• Dystrophic Epidermolysis Bullosa

Intervention

• 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
• Drug: Placebo
  It will be applied topically to the appropriate wound once a day for up to 84 days.
  Other Name: Vehicle Control

Study Arms

• Experimental: RGN-137
  It is formulated as a gel for topical administration.
  Intervention: Drug: RGN-137
• Placebo Comparator: Placebo
  It is composed of the same excipients as RGN-137 formulation without the active ingredient.
  Intervention: Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Terminated
• Actual Enrollment (submitted: August 18, 2022): 4
• Original Estimated Enrollment (submitted: June 24, 2018): 15
• Actual Study Completion Date: November 23, 2021
• Actual Primary Completion Date: September 1, 2021 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 4 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03578029
• Other Study ID Numbers: RGN-137-EB-202
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Lenus Therapeutics, LLC
• Original Responsible Party: GtreeBNT Co., Ltd.
• Current Study Sponsor: Lenus Therapeutics, LLC
• Original Study Sponsor: GtreeBNT Co., Ltd.
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Lenus Therapeutics, LLC
• Verification Date: August 2022