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Trial record 2 of 27 for:    epidermolysis bullosa | Recruiting, Not yet recruiting, Available Studies

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

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ClinicalTrials.gov Identifier: NCT03578029
Recruitment Status : Recruiting
First Posted : July 4, 2018
Last Update Posted : November 15, 2018
Sponsor:
Information provided by (Responsible Party):
Lenus Therapeutics, LLC

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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 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
Estimated Study Start Date : November 2019
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : August 2020


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




Primary Outcome Measures :
  1. Time to complete re-epithelialization of index wounds up to Day 84. [ Time Frame: up to Day 84 ]
    This primary endpoint will be the time to complete re-epithelialization of each index wound up to Day 84, defined as date of the first study visit on which complete-re-epithelialization is achieved minus date of Baseline visit.


Secondary Outcome Measures :
  1. Change from Baseline in surface area of index wounds 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.

  2. 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.


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)



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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)
  • Neurological, cardiovascular, respiratory, hepatic, renal, or metabolic disease likely to interfere with the subject's participation in or completion of the study
  • 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

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


Contacts
Contact: Shinwook Kang 609-734-4310 swkang@thelenus.com

Locations
United States, Texas
Texas Dermatology and Laser Specialists Recruiting
San Antonio, Texas, United States, 78218
Contact: Gabriel Leal    210-660-1157      
Principal Investigator: John C. Browning, MD         
Sponsors and Collaborators
Lenus Therapeutics, LLC

Responsible Party: Lenus Therapeutics, LLC
ClinicalTrials.gov Identifier: NCT03578029     History of Changes
Other Study ID Numbers: RGN-137-EB-202
First Posted: July 4, 2018    Key Record Dates
Last Update Posted: November 15, 2018
Last Verified: November 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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:
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