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

Primary Purpose

Junctional Epidermolysis Bullosa, Dystrophic Epidermolysis Bullosa

Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
RGN-137
Placebo
Sponsored by
Lenus Therapeutics, LLC
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Junctional Epidermolysis Bullosa focused on measuring Epidermolysis Bullosa

Eligibility Criteria

4 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Northwestern University
  • SUNY Downstate Medical Center
  • Cincinnati Children's Hospital Medical Center
  • The Children's Hospital of Philadelphia
  • Texas Dermatology and Laser Specialists

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

RGN-137

Placebo

Arm Description

It is formulated as a gel for topical administration.

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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.
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.
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.
This secondary endpoint will be computed for each treatment group as well as for treatment difference at each visit.

Full Information

First Posted
June 14, 2018
Last Updated
August 18, 2022
Sponsor
Lenus Therapeutics, LLC
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1. Study Identification

Unique Protocol Identification Number
NCT03578029
Brief Title
Evaluation of the Safety and Efficacy Study of RGN-137 Topical Gel for Junctional and Dystrophic Epidermolysis Bullosa
Acronym
CELEB
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)
Study Type
Interventional

2. Study Status

Record Verification Date
August 2022
Overall Recruitment Status
Terminated
Why Stopped
Business Decision
Study Start Date
May 22, 2019 (Actual)
Primary Completion Date
September 1, 2021 (Actual)
Study Completion Date
November 23, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Lenus Therapeutics, LLC

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Junctional Epidermolysis Bullosa, Dystrophic Epidermolysis Bullosa
Keywords
Epidermolysis Bullosa

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderOutcomes Assessor
Allocation
Randomized
Enrollment
4 (Actual)

8. Arms, Groups, and Interventions

Arm Title
RGN-137
Arm Type
Experimental
Arm Description
It is formulated as a gel for topical administration.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
It is composed of the same excipients as RGN-137 formulation without the active ingredient.
Intervention Type
Drug
Intervention Name(s)
RGN-137
Other Intervention Name(s)
Dermal Topical Gel
Intervention Description
It will be applied topically to the appropriate wound once a day for up to 84 days.
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
Vehicle Control
Intervention Description
It will be applied topically to the appropriate wound once a day for up to 84 days.
Primary Outcome Measure Information:
Title
Time to achieving 50% reduction in area of each index wound from Day 1 up to Day 84.
Description
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.
Time Frame
up to Day 84
Secondary Outcome Measure Information:
Title
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.
Description
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.
Time Frame
up to Day 84
Title
Incidence of 50%, 75%, and 100% reduction and complete re-epithelialization without drainage in area of each index wound at the scheduled visits.
Description
This secondary endpoint will be summarized by treatment using frequency counts and percentages.
Time Frame
up to Day 84
Title
Change from Baseline and percent change from Baseline in surface area of each index wound at the scheduled visits.
Description
This secondary endpoint will be computed for each treatment group as well as for treatment difference at each visit.
Time Frame
up to Day 84
Other Pre-specified Outcome Measures:
Title
Index wound characteristics at the scheduled visits.
Description
(inflammation/erythema, induration, crusting, exudate, cellulitis, and other abnormalities)
Time Frame
up to Day 84

10. Eligibility

Sex
All
Minimum Age & Unit of Time
4 Years
Accepts Healthy Volunteers
No
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 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 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
Facility Information:
Facility Name
Northwestern University
City
Evanston
State/Province
Illinois
ZIP/Postal Code
60208
Country
United States
Facility Name
SUNY Downstate Medical Center
City
Brooklyn
State/Province
New York
ZIP/Postal Code
11203
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Texas Dermatology and Laser Specialists
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78218
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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