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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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03578029
Recruitment Status : Recruiting
First Posted : July 5, 2018
Last Update Posted : August 28, 2019
Sponsor:
Information provided by (Responsible Party):
Lenus Therapeutics, LLC

Tracking Information
First Submitted Date  ICMJE June 14, 2018
First Posted Date  ICMJE July 5, 2018
Last Update Posted Date August 28, 2019
Actual Study Start Date  ICMJE May 22, 2019
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (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  ICMJE
 (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.
Change History
Current Secondary Outcome Measures  ICMJE
 (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  ICMJE
 (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  ICMJE Evaluation of the Safety and Efficacy Study of RGN-137 Topical Gel for Junctional and Dystrophic Epidermolysis Bullosa
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Junctional Epidermolysis Bullosa
  • Dystrophic Epidermolysis Bullosa
Intervention  ICMJE
  • 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  ICMJE
  • 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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 24, 2018)
15
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 2020
Estimated Primary Completion Date June 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 4 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Shinwook Kang 609-734-4310 swkang@thelenus.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03578029
Other Study ID Numbers  ICMJE 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  ICMJE
Plan to Share IPD: No
Responsible Party Lenus Therapeutics, LLC
Study Sponsor  ICMJE Lenus Therapeutics, LLC
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Lenus Therapeutics, LLC
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP