Epidermolysis BullosaPhase 2NCT03389308
Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex
A Phase 2 study testing diacerein 1% ointment in people with Epidermolysis Bullosa and Epidermolysis Bullosa Simplex, run by Castle Creek Pharmaceuticals, LLC at 3 sites in United States.
Trial summary
The primary objective of this study is to evaluate the long term safety and tolerability of diacerein 1% ointment for 2 treatment cycles in subjects with EBS that previously participated in the CCP-020-301 or the CCP-020-101 studies. Read more about this study
- Who is it for?
- Ages 6 months to 99 years
- Conditions the study is for
- Epidermolysis Bullosa, Epidermolysis Bullosa Simplex
- What is being tested?
- diacerein 1% ointment
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 51 participants
- Where
- 3 sitesUnited States
- Study dates
- 1 Dec 2017to 17 Apr 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 6 months to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- The subject is capable of understanding and complying with protocol requirements.
- The subject or the subject's legally acceptable representative signs and dates a written, informed consent/assent form and any required privacy authorization prior to the initiation of any study procedures.
- Subject has a documented genetic mutation consistent with EBS.
- Subject has completed study CCP-020-301 or participated in study CCP-020-101.
- Subject/caregiver agrees to report use of any topical therapies applied to EBS lesions
- If the subject is a woman of childbearing potential, she has a negative urine pregnancy test and agrees to use an approved effective method of birth control, for the duration of the study.
- Subject is non-lactating and is not planning for pregnancy during the study period.
- Subject is willing and able to follow all study instructions and to attend all study visits.
Who cannot join
- Subject has EBS lesions to be treated that are infected
- Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to Baseline Visit.
- The subject was discontinued from the feeder study due to an adverse event judged to be related or possibly related to the study medication.
- Subject has experienced a change in clinical status from the feeder study that puts the subject at undue risk to participate.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
University of North Carolina (UNC) - Chapel Hill
Chapel Hill, North Carolina, 27516
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Children's Hospital of San Antonio
San Antonio, Texas, 78218
About this study
Official title: An International, Multicenter, Open-label, Long Term Extension Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex (EBS)
The primary objective of this study is to evaluate the long term safety and tolerability of diacerein 1% ointment for 2 treatment cycles in subjects with EBS that previously participated in the CCP-020-301 or the CCP-020-101 studies.
Read the detailed description
This is an international, multicenter, open-label, long term extension study evaluating the safety of topical Diacerein 1% Ointment for the treatment of subjects with EBS. At Visit 1, EBS subjects who completed the CCP-020-301 double-blind safety and efficacy study or participated in the CCP-020-101 PK study (feeder studies) who meet all the inclusion/exclusion criteria will be eligible to complete 2 treatment cycles of CCP-020.
Each treatment cycle will consist of 8 weeks on treatment (once-daily, at home study medication applications) followed by 8 weeks off treatment (only Investigator approved bland, non-medicated emollient/moisturizer, routine cleansing products and sunscreens) with a maximum of 2 treatment cycles allowed for up to 52 weeks.
At Baseline Visit (corresponding to the final study visit of the feeder study), the investigator will perform a clinical assessment and determine if any of the subject's lesions require treatment (up to 30% BSA). If the subject has active lesions as determined by the Investigator's clinical assessment, the subject will initiate a cycle of once-daily, at home study medication application to their EBS lesions for 8 weeks (treatment period) followed by an 8-week period where no treatment will be administered. Subjects presenting with no active lesions (as determined by the Investigator's clinical assessment) will not begin treatment and instead, will be instructed to return to the clinic for re-evaluation in 8 weeks or upon worsening of EBS lesions, whichever happens first.
- Being tested
- diacerein 1% ointment
- Keywords
- Epidermolysis Bullosa (EB), Epidermolysis Bullosa Simplex (EBS)
- Medical subject headings
- Epidermolysis Bullosa, Epidermolysis Bullosa Simplex
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
diacerein 1% ointmentDrug
Also called CCP-020
Topically apply study medication to lesions identified by the Investigator that require.
treatment
| Study group | Role | What they receive |
|---|---|---|
| Treatment period | Experimental | Subjects with active lesions as determined Investigator's clinical assessment, will initiate a cycle of applying diacerein 1% ointment once-daily, study medication, at home to their EBS lesions for 8 weeks.
Following the Treatment Period, subjects will go Off Treatment for 8 weeks using only investigator approved bland, non-medicated emollient/moisturizer, routine cleansing products and sunscreens. As determined Investigator's clinical assessment, subjects may enter into another Treatment Period of 8 weeks.
The duration of a subject's participation in the extension study may be as short as 32 weeks or as long as 52 weeks depending on the cycle initiation schedule for each individual subject. Receives: Drug: diacerein 1% ointment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-emergent Adverse Events
Number and percentage of participants with any treatment-emergent adverse event.
Measured: up to 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jan 2018
- Study start1 Dec 2017
- Primary completion17 Apr 2020
- Study completion17 Apr 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for 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
- Not applicable
- Intervention model
- Single group
- Model description
- This is an international, multicenter, open-label, long term extension study evaluating the safety of topical Diacerein 1% Ointment for the treatment of subjects with EBS. At Baseline Visit, EBS subjects who participated the CCP-020-301 double-blind safety and efficacy study or participated in the CCP-020-101 PK study (feeder studies) and who meet all the inclusion/exclusion criteria will be eligible to enroll in this study.
- Masking
- None (open label)
- Enrolment
- 51 participants (actual)
Sponsor and investigators
- Lead sponsor
- Castle Creek Pharmaceuticals, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mary Spellman, MD, Study chair, Castle Creek Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03389308
- Sponsor's protocol ID
- CCP-020-302
- Status last verified
- Aug 2020
- First submitted
- 19 Dec 2017
- Last updated
- 16 Sep 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An International, Multicenter, Open-label, Long Term Extension Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex (EBS)
- ClinicalTrials.gov ID
- NCT03389308
- Acronym / sponsor ID
- CCP-020-302
- Keywords
- Epidermolysis Bullosa (EB), Epidermolysis Bullosa Simplex (EBS)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 51 participants (actual)
- Lead sponsor
- Castle Creek Pharmaceuticals, LLCIndustry (pharma, biotech, devices)
- Study start
- 1 Dec 2017 (actual)
- Primary completion
- 17 Apr 2020 (actual)
- Study completion
- 17 Apr 2020 (actual)
- First posted
- 3 Jan 2018 (actual)
- Results
- Posted 16 Sep 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to evaluate the long term safety and tolerability of diacerein 1% ointment for 2 treatment cycles in subjects with EBS that previously participated in the CCP-020-301 or the CCP-020-101 studies.
Detailed description
This is an international, multicenter, open-label, long term extension study evaluating the safety of topical Diacerein 1% Ointment for the treatment of subjects with EBS. At Visit 1, EBS subjects who completed the CCP-020-301 double-blind safety and efficacy study or participated in the CCP-020-101 PK study (feeder studies) who meet all the inclusion/exclusion criteria will be eligible to complete 2 treatment cycles of CCP-020.
Each treatment cycle will consist of 8 weeks on treatment (once-daily, at home study medication applications) followed by 8 weeks off treatment (only Investigator approved bland, non-medicated emollient/moisturizer, routine cleansing products and sunscreens) with a maximum of 2 treatment cycles allowed for up to 52 weeks.
At Baseline Visit (corresponding to the final study visit of the feeder study), the investigator will perform a clinical assessment and determine if any of the subject's lesions require treatment (up to 30% BSA). If the subject has active lesions as determined by the Investigator's clinical assessment, the subject will initiate a cycle of once-daily, at home study medication application to their EBS lesions for 8 weeks (treatment period) followed by an 8-week period where no treatment will be administered. Subjects presenting with no active lesions (as determined by the Investigator's clinical assessment) will not begin treatment and instead, will be instructed to return to the clinic for re-evaluation in 8 weeks or upon worsening of EBS lesions, whichever happens first.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is an international, multicenter, open-label, long term extension study evaluating the safety of topical Diacerein 1% Ointment for the treatment of subjects with EBS. At Baseline Visit, EBS subjects who participated the CCP-020-301 double-blind safety and efficacy study or participated in the CCP-020-101 PK study (feeder studies) and who meet all the inclusion/exclusion criteria will be eligible to enroll in this study.
| Arm | Type | Receives |
|---|---|---|
| Treatment period | Experimental | Drug: diacerein 1% ointmentSubjects with active lesions as determined Investigator's clinical assessment, will initiate a cycle of applying diacerein 1% ointment once-daily, study medication, at home to their EBS lesions for 8 weeks. Following the Treatment Period, subjects will go Off Treatment for 8 weeks using only investigator approved bland, non-medicated emollient/moisturizer, routine cleansing products and sunscreens. As determined Investigator's clinical assessment, subjects may enter into another Treatment Period of 8 weeks. The duration of a subject's participation in the extension study may be as short as 32 weeks or as long as 52 weeks depending on the cycle initiation schedule for each individual subject. |
Who may qualify
- Ages
- 6 months to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- The subject is capable of understanding and complying with protocol requirements.
- The subject or the subject's legally acceptable representative signs and dates a written, informed consent/assent form and any required privacy authorization prior to the initiation of any study procedures.
- Subject has a documented genetic mutation consistent with EBS.
- Subject has completed study CCP-020-301 or participated in study CCP-020-101.
- Subject/caregiver agrees to report use of any topical therapies applied to EBS lesions
Key exclusion criteria (4 listed)
- Subject has EBS lesions to be treated that are infected
- Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to Baseline Visit.
- The subject was discontinued from the feeder study due to an adverse event judged to be related or possibly related to the study medication.
- Subject has experienced a change in clinical status from the feeder study that puts the subject at undue risk to participate.
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 |
|---|---|---|
| diacerein 1% ointmentAlso: CCP-020Topically apply study medication to lesions identified by the Investigator that require. treatment | Drug | Treatment period |
Primary outcome
Treatment-emergent Adverse Events
Time frame: up to 52 weeks
Number and percentage of participants with any treatment-emergent adverse event.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Mary Spellman, MD study chair
Castle Creek Pharmaceuticals
3 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 diacerein 1% ointment in people with Epidermolysis Bullosa and Epidermolysis Bullosa Simplex, run by Castle Creek Pharmaceuticals, LLC at 3 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 8 inclusion and 4 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: finished, as of the sponsor's last update on 16 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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. 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?
Yes. Results are posted in the registry (first posted 16 Sep 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Treatment-emergent Adverse Events (time frame: up to 52 weeks). 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 16 Sep 2020; AzTrials last refreshed its copy on 6 Nov 2026.