Junctional Epidermolysis BullosaPhase 2CELEBNCT03578029
Evaluation of the Safety and Efficacy Study of RGN-137 Topical Gel for Junctional and Dystrophic Epidermolysis Bullosa
A Phase 2 study testing RGN-137 in people with Junctional Epidermolysis Bullosa and Dystrophic Epidermolysis Bullosa, run by Lenus Therapeutics, LLC at 5 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Junctional Epidermolysis Bullosa, Dystrophic Epidermolysis Bullosa
- What is being tested?
- RGN-137
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 4 participants
- Where
- 5 sitesUnited States
- Study dates
- 22 May 2019to 23 Nov 2021
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
SUNY Downstate Medical Center
Brooklyn, New York, 11203
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Northwestern University
Evanston, Illinois, 60208
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Texas Dermatology and Laser Specialists
San Antonio, Texas, 78218
About this study
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)
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).
Read the 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.
- Being tested
- RGN-137, Placebo
- Keywords
- Epidermolysis Bullosa
- Medical subject headings
- Epidermolysis Bullosa, Junctional, Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RGN-137Drug
Also called Dermal Topical Gel
It will be applied topically to the appropriate wound once a day for up to 84 days.
PlaceboDrug
Also called Vehicle Control
It will be applied topically to the appropriate wound once a day for up to 84 days.
| Study group | Role | What they receive |
|---|---|---|
| RGN-137 | Experimental | It is formulated as a gel for topical administration. Receives: Drug: RGN-137 |
| Placebo | Placebo comparator | It is composed of the same excipients as RGN-137 formulation without the active ingredient. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: up to Day 84
Secondary outcomes (3)
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.
Measured: up to Day 84
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.
Measured: up to Day 84
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.
Measured: up to Day 84
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jul 2018
- Study start22 May 2019
- Primary completion1 Sep 2021
- Study completion23 Nov 2021
Why it stopped: Business Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Junctional Epidermolysis BullosaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Outcomes assessor
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Lenus Therapeutics, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03578029
- Sponsor's protocol ID
- RGN-137-EB-202
- Acronym
- CELEB
- Status last verified
- Aug 2022
- First submitted
- 14 Jun 2018
- Last updated
- 22 Aug 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT03578029
- Acronym / sponsor ID
- CELEB / RGN-137-EB-202
- Keywords
- Epidermolysis Bullosa
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Business Decision
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Lenus Therapeutics, LLCIndustry (pharma, biotech, devices)
- Study start
- 22 May 2019 (actual)
- Primary completion
- 1 Sep 2021 (actual)
- Study completion
- 23 Nov 2021 (actual)
- First posted
- 5 Jul 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| RGN-137 | Experimental | Drug: RGN-137It is formulated as a gel for topical administration. |
| Placebo | Placebo comparator | Drug: PlaceboIt is composed of the same excipients as RGN-137 formulation without the active ingredient. |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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
- 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
Key exclusion criteria (10 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RGN-137Also: Dermal Topical GelIt will be applied topically to the appropriate wound once a day for up to 84 days. | Drug | RGN-137 |
| PlaceboAlso: Vehicle ControlIt will be applied topically to the appropriate wound once a day for up to 84 days. | Drug | Placebo |
Primary outcome
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.
3 secondary outcomes
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
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
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
Results and safety
The study was stopped early (reason given: Business Decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Aug 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RGN-137 in people with Junctional Epidermolysis Bullosa and Dystrophic Epidermolysis Bullosa, run by Lenus Therapeutics, LLC at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 22 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Business Decision). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to achieving 50% reduction in area of each index wound from Day 1 up to Day 84. (time frame: up to Day 84). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Aug 2022.