Epidermolysis Bullosa SimplexPhase 2NCT03154333
Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS)
A Phase 2 study testing diacerein 1% ointment in people with Epidermolysis Bullosa Simplex, run by Castle Creek Pharmaceuticals, LLC at 22 sites in 8 countries.
Trial summary
Epidermolysis bullosa simplex (EBS) is a rare genetic skin disease characterized by fragility of the skin and mucous membranes resulting in painful blisters and erosions after minor trauma. Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Epidermolysis Bullosa Simplex
- What is being tested?
- diacerein 1% ointment
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 54 participants
- Where
- 22 sitesin 8 countries
- Study dates
- 1 Jun 2017to 31 Oct 2018
- 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 4 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject is at least 4 years of age at Screening
- Subject has a documented genetic mutation consistent with EBS. Gene mutations acceptable for inclusion are as follows: KRT5, KRT14, PLEC1, TGM5, PKP1, DSP, FERMT1, EXPH5, DST, KLHL24.
- Subject has an Assessment Area of EBS lesions to be treated, that is ≥2% body surface area (BSA) and the EBS lesions are in one or both of the following body areas:
- Localized: plantar and/or palmar areas
- Generalized: arms, legs, torso, hands and feet
- Subject's EBS lesions in the Assessment Area have an Investigator's Global Assessment (IGA) score of ≥3
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- Subject/caregiver agrees to not use any topical therapies other than the study medication that might influence the status of the EBS lesions during the duration of the study
- Subject is non-pregnant as confirmed by a negative urine pregnancy screen, non-lactating and is not planning for pregnancy during the study period
- If the subject is a woman of childbearing potential, agrees to use an approved effective method of birth control
- Subject is in good general health and free of any known disease state or physical condition which might impair evaluation of the EBS lesions or which exposes the subject to an unacceptable risk by study participation
Who cannot join
- Subject has EBS lesions to be treated that are infected
- Subject has used any diacerein containing product within 6 months prior to Screening
- Subject has used systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to Screening
- Subject has used systemic steroidal therapy or has used topical steroidal therapy on the EBS lesions to be treated within 30 days prior to Baseline
- Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to Screening
- Subject is currently using systemic analgesics and/or anti-histamine therapy, for treatment of EBS lesions unless on a stable regimen (i.e., the same dosing regimen) for at least 4 weeks prior to Screening
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- Subject has used any systemic diuretics or cardiac glycosides or any systemic product that might put the subject at undue risk
- Subject has used any topical product containing allantoin on the EBS lesions to be treated within 30 days prior to Screening
- Subject has a current malignancy, or a history of treatment for a malignancy within 2 years prior to Screening
- Subject currently has diabetes mellitus (HbA1c ≥6.5%) or controlled diabetes (HbA1c < 6.5%)
- Subject has a history of cardiac, hepatic (ALT and or AST >2x ULN, Total bilirubin >1.5x ULN at Screening), or renal disease (eGFR<30 ml/min/1.73 m^2)
- Subject has a non-EBS skin disease or condition (e.g., sunburn) that might put the subject at undue risk by study participation or interferes with the study medication application or the study assessments
Trial locations and contacts
22 sites in 8 countries.
22 sites in 8 countries
United States12 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
University of North Carolina - Chapel Hill
Chapel Hill, North Carolina, 27516
Medical University of South Carolina (MUSC)
Charleston, South Carolina, 29425-5780
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Northwestern University
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University of Missouri Healthcare
Columbia, Missouri, 65212
University of Minnesota
Minneapolis, Minnesota, 55455
Stanford University
Palo Alto, California, 94304
Phoenix Childrens Hospital
Phoenix, Arizona, 85016
Children's Hospital of San Antonio ; Texas Dermatology and Laser Specialists
San Antonio, Texas, 78218
Stony Brook University Medical Center
Stony Brook, New York, 11790
France3 sites
CHU de NICE - Hopital de l'Archet II - Service de Dermatologie
Nice, 06202
Hopital Saint Louis
Paris, Cedex, 75010
Hopital Necker-Enfants Malades
Paris, Cedex, 75015
United Kingdom2 sites
Great Ormond Street Hospital
London, England, WC1N 3JH
St. Thomas' Hospital - St Johns Institute of Dermatology
London, SE1 7EH
Australia1 site
Premier Specialists Pty Ltd; The Church
Kogarah, New South Wales, 2217
Netherlands1 site
University Medical Center Groningen
Groningen, AB20;30,001
About this study
Official title: An International, Multicenter, Randomized, Double-Blind, Parallel-Group Phase 2 Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex
Epidermolysis bullosa simplex (EBS) is a rare genetic skin disease characterized by fragility of the skin and mucous membranes resulting in painful blisters and erosions after minor trauma. The purpose of this study is to compare the efficacy of diacerein 1% ointment to vehicle ointment when applied once-daily for 8 weeks in subjects with EBS.
Read the detailed description
The study is an international, multicenter, randomized, double-blind, vehicle-controlled, parallel group study to evaluate the safety and efficacy of diacerein 1% ointment for the treatment of subjects with EBS. Participants were assigned to study groups receiving either diacerein 1% ointment or vehicle ointment for 8 weeks, followed by an 8 week follow-up period. Approximately 80 subjects were to be randomized to one of the 2 treatment groups.
The primary objective of this study is to compare the efficacy of diacerein 1% ointment to vehicle ointment based on reduction in body surface area (BSA) of EBS lesions being treated when applied once-daily for 8 weeks in subjects with EBS. The secondary objectives are to compare the effects of diacerein 1% ointment to vehicle ointment in subjects with EBS in changes in Investigator Global Assessment (IGA) scores, pain, pruritus, mobility, and safety and tolerability.
- Conditions
- Epidermolysis Bullosa Simplex
- Being tested
- diacerein 1% ointment, A placebo ointment
- Keywords
- Epidermolysis, bullosa, simplex, EBS
- Medical subject headings
- 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
diacerein 1% ointment administered topically
A placebo ointmentDrug
vehicle ointment administered topically
| Study group | Role | What they receive |
|---|---|---|
| diacerein 1% ointment | Experimental | diacerein 1% ointment will be used for 8 weeks Receives: Drug: diacerein 1% ointment |
| vehicle ointment | Placebo comparator | vehicle ointment will be used for 8 weeks Receives: Drug: A placebo ointment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Subjects Who Achieved ≥ 60% Reduction in Body Surface Area (BSA) of EBS Lesions Within Assessment Area
Analysis of the proportion of subjects who achieved a ≥60% reduction in Body Surface Area (BSA) of EBS lesions within Assessment Area from Baseline to Week 8
Measured: Baseline to Week 8
Secondary outcomes (1)
The Proportion of Subjects Who Achieved Success on the Investigator's Global Assessment (IGA)
The investigator's global assessment (IGA) is a five-point scale that is used for overall clinical assessment of severity of disease and classifies EBS-involved skin with a score ranging from 0-4. Success on the IGA was defined as ≥2-point reduction from Baseline to Visit 6 (Week 8).
IGA Scoring:
0 = Clear; 1 = Near Clear; 2 = Mild; 3 = Moderate; 4 = Severe
Minimum score = 0 Maximum score = 4; higher score = worse outcome
Measured: Baseline to Week 8
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2017
- Study start1 Jun 2017
- Primary completion15 Oct 2018
- Study completion31 Oct 2018
Why it stopped: Futility
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 Bullosa SimplexStudies 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 54 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 director, Castle Creek Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03154333
- Sponsor's protocol ID
- CCP-020-301
- Status last verified
- Oct 2019
- First submitted
- 10 May 2017
- Last updated
- 5 Nov 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- 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 5 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An International, Multicenter, Randomized, Double-Blind, Parallel-Group Phase 2 Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex
- ClinicalTrials.gov ID
- NCT03154333
- Acronym / sponsor ID
- CCP-020-301
- Conditions
- Epidermolysis Bullosa Simplex
- Keywords
- Epidermolysis, bullosa, simplex, EBS
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Futility
- Enrolment
- 54 participants (actual)
- Lead sponsor
- Castle Creek Pharmaceuticals, LLCIndustry (pharma, biotech, devices)
- Study start
- 1 Jun 2017 (actual)
- Primary completion
- 15 Oct 2018 (actual)
- Study completion
- 31 Oct 2018 (actual)
- First posted
- 16 May 2017 (actual)
- Results
- Posted 5 Nov 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Epidermolysis bullosa simplex (EBS) is a rare genetic skin disease characterized by fragility of the skin and mucous membranes resulting in painful blisters and erosions after minor trauma. The purpose of this study is to compare the efficacy of diacerein 1% ointment to vehicle ointment when applied once-daily for 8 weeks in subjects with EBS.
Detailed description
The study is an international, multicenter, randomized, double-blind, vehicle-controlled, parallel group study to evaluate the safety and efficacy of diacerein 1% ointment for the treatment of subjects with EBS. Participants were assigned to study groups receiving either diacerein 1% ointment or vehicle ointment for 8 weeks, followed by an 8 week follow-up period. Approximately 80 subjects were to be randomized to one of the 2 treatment groups.
The primary objective of this study is to compare the efficacy of diacerein 1% ointment to vehicle ointment based on reduction in body surface area (BSA) of EBS lesions being treated when applied once-daily for 8 weeks in subjects with EBS. The secondary objectives are to compare the effects of diacerein 1% ointment to vehicle ointment in subjects with EBS in changes in Investigator Global Assessment (IGA) scores, pain, pruritus, mobility, and safety and tolerability.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| diacerein 1% ointment | Experimental | Drug: diacerein 1% ointmentdiacerein 1% ointment will be used for 8 weeks |
| vehicle ointment | Placebo comparator | Drug: A placebo ointmentvehicle ointment will be used for 8 weeks |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Subject is at least 4 years of age at Screening
- Subject has a documented genetic mutation consistent with EBS. Gene mutations acceptable for inclusion are as follows: KRT5, KRT14, PLEC1, TGM5, PKP1, DSP, FERMT1, EXPH5, DST, KLHL24.
- Subject has an Assessment Area of EBS lesions to be treated, that is ≥2% body surface area (BSA) and the EBS lesions are in one or both of the following body areas:
- Subject's EBS lesions in the Assessment Area have an Investigator's Global Assessment (IGA) score of ≥3
- Subject/caregiver agrees to not use any topical therapies other than the study medication that might influence the status of the EBS lesions during the duration of the study
Key exclusion criteria (12 listed)
- Subject has EBS lesions to be treated that are infected
- Subject has used any diacerein containing product within 6 months prior to Screening
- Subject has used systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to Screening
- Subject has used systemic steroidal therapy or has used topical steroidal therapy on the EBS lesions to be treated within 30 days prior to Baseline
- Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to Screening
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-020diacerein 1% ointment administered topically | Drug | diacerein 1% ointment |
| A placebo ointmentvehicle ointment administered topically | Drug | vehicle ointment |
Primary outcome
Proportion of Subjects Who Achieved ≥ 60% Reduction in Body Surface Area (BSA) of EBS Lesions Within Assessment Area
Time frame: Baseline to Week 8
Analysis of the proportion of subjects who achieved a ≥60% reduction in Body Surface Area (BSA) of EBS lesions within Assessment Area from Baseline to Week 8
1 secondary outcome
The Proportion of Subjects Who Achieved Success on the Investigator's Global Assessment (IGA)
Time frame: Baseline to Week 8
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Nov 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Mary Spellman, MD study director
Castle Creek Pharmaceuticals
22 sites in 8 countries: 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 Simplex, run by Castle Creek Pharmaceuticals, LLC at 22 sites in 8 countries.
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 10 inclusion and 12 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 5 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in United States, France, United Kingdom, Australia, Austria and 3 more countries. 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?
Yes. Results are posted in the registry (first posted 5 Nov 2019). 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?
Proportion of Subjects Who Achieved ≥ 60% Reduction in Body Surface Area (BSA) of EBS Lesions Within Assessment Area (time frame: Baseline to Week 8). 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 5 Nov 2019.