Epidermolysis Bullosa (EB)Phase 1NCT03472287
To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use in Patients With Epidermolysis Bullosa (EB)
A Phase 1 study testing Diacerein 1% Ointment in people with Epidermolysis Bullosa (EB) and Epidermolysis Bullosa Simplex, run by Castle Creek Pharmaceuticals, LLC at 3 sites in United States.
Trial summary
A pharmacokinetic (PK) study in 16-20 EB subjects to be allocated to two cohorts. Cohort 1 to include 8-10 subjects (ages 12 yrs and older); Cohort 2 to include 8-10 subjects (ages 6 months-11 yrs, inclusive). Cohort 2 only included subjects 4 yrs and older. Serial PK blood sampling collected on Days 1 and 10. Read more about this study
- Who is it for?
- Ages 4 years and older
- Conditions the study is for
- Epidermolysis Bullosa (EB), Epidermolysis Bullosa Simplex, Dystrophic Epidermolysis Bullosa
- What is being tested?
- Diacerein 1% Ointment
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 3 sitesUnited States
- Study dates
- 18 May 2018to 14 Feb 2019
- Phase
- Phase 1
- 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
- At least 12 years of age (Cohort 1) or at least 6 months of age to 11 years, inclusive (Cohort 2) at screening. For US only: at least 4 years of age to 11 years, inclusive (Cohort 2)
- The subject must weigh at least 9 kg (19.8 lbs) at Screening.
- Subject has a documented genetic mutation consistent with EB.
- Subject has EB lesions on ≥ 2% body surface area (BSA) and the EB lesions are in the following body areas: a. Localized: plantar and/or palmar areas, b. Generalized: arms, legs, torso, hands and feet.
Who cannot join
- Subject has EB lesions where drug will be applied that are infected
- Subject has used any diacerein containing product within 1 month prior to Visit 1
- Subject has used systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to dosing.
- Subject has used systemic steroidal therapy or has used topical steroidal therapy on the EB lesions in the application area within 14 days prior to dosing
- Subject has participated in an investigational drug trial in which administration of an investigational study medication occurred within 30 days prior to dosing
Trial locations and contacts
3 sites in 1 country.
United States3 sites
UNC Dermatology
Chapel Hill, North Carolina, 27599
Northwestern University Medical Center
Chicago, Illinois, 60654
Stanford School of Medicine
Palo Alto, California, 94304
About this study
Official title: A Multi-Center Study to Evaluate the Pharmacokinetics of Diacerein and Rhein and the Safety of Diacerein After Maximum Use, Topical Administration of CCP-020 (Diacerein 1% Ointment) to Patients With Epidermolysis Bullosa (EB)
A pharmacokinetic (PK) study in 16-20 EB subjects to be allocated to two cohorts. Cohort 1 to include 8-10 subjects (ages 12 yrs and older); Cohort 2 to include 8-10 subjects (ages 6 months-11 yrs, inclusive). Cohort 2 only included subjects 4 yrs and older. Serial PK blood sampling collected on Days 1 and 10. Analyses were performed to determine the concentrations of diacerein and rhein.
Read the detailed description
This was an open-label, single-period study that intended to include subjects with EB consisting of infants/ children (ages 6 months to 11 years, inclusive) and adolescents/ adults (ages 12 years and older). The study included 2 cohorts.
Cohort 1 was to include adolescent/adult subjects with EB (ages 12 years and older);
Cohort 2 was to include infants/children with EB (ages 4 to 11 years, inclusive).
For both cohorts, the study intended to enroll subjects with epidermolysis bullosa simplex (EBS), dystrophic epidermolysis bullosa (DEB), and junctional epidermolysis bullosa (JEB) with lesions encompassing >/=2% BSA. The diacerein application area was >/=5% BSA and included lesioned and non-lesioned skin (if lesions accounted for <5% BSA); however, topical administration was </=30% BSA. On Days 1 and 10, the topical dose of study drug was applied by study staff, and blood was sampled for PK analyses of diacerein and rhein pre-dose and through 8 hours post-application. On Days 2 to 9, study drug was applied to the application area(s) at home. For Cohort 1, trough PK samples were collected on any 2 available days from Days 3 through 9, and study drug was applied after blood draw, while for Cohort 2 trough PK analyses were not performed.
- Conditions
- Epidermolysis Bullosa (EB), Epidermolysis Bullosa Simplex, Dystrophic Epidermolysis Bullosa, Junctional Epidermolysis Bullosa
- Being tested
- Diacerein 1% Ointment
- Keywords
- EB, EBS, DEB, JEB
- Medical subject headings
- Epidermolysis Bullosa, Epidermolysis Bullosa Simplex, Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa, Junctional
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
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 (Adolescents, Adults) | Experimental | Adolescent and adult subjects with EB (aged 12 years and older) received diacerein 1% ointment daily for 10 days. Receives: Drug: Diacerein 1% Ointment |
| Cohort 2 (Children) | Experimental | Children with EB (aged 4 to 11 years, inclusive) received diacerein 1% ointment daily for 10 days. Receives: Drug: Diacerein 1% Ointment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Detectable Plasma Concentrations of Diacerein and Rhein
Bioanalytical analyses were performed to determine concentrations levels of diacerein and rhein in plasma using validated bioanalytical methods.
For Cohort 1, blood samples were taken at pre-dose and 0.5, 1, 2, 3, 4, 6, and 8 hours post-dose.
For Cohort 2, blood samples were taken at pre-dose and 1, 2, 4, 6, and 8 hours post-dose.
Trough PK samples were collected on any 2 available days from Days 3 through 9 for Cohort 1 only.
Summary statistics for each scheduled time were only reported if at least 50% of subjects had quantifiable concentrations.
Measured: Days 1-10, at select time points per protocol
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2018
- Study start18 May 2018
- Primary completion31 Jan 2019
- Study completion14 Feb 2019
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 (EB)Studies 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 1
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Castle Creek Pharmaceuticals, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mary Spellman, Study chair, Castle Creek Pharmaceuticals, LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03472287
- Sponsor's protocol ID
- CCP-020-101
- Status last verified
- Feb 2020
- First submitted
- 14 Mar 2018
- Last updated
- 10 Feb 2020
- Results posted
- Yes, on ClinicalTrials.gov
- 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 10 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center Study to Evaluate the Pharmacokinetics of Diacerein and Rhein and the Safety of Diacerein After Maximum Use, Topical Administration of CCP-020 (Diacerein 1% Ointment) to Patients With Epidermolysis Bullosa (EB)
- ClinicalTrials.gov ID
- NCT03472287
- Acronym / sponsor ID
- CCP-020-101
- Conditions
- Epidermolysis Bullosa (EB), Epidermolysis Bullosa Simplex, Dystrophic Epidermolysis Bullosa, Junctional Epidermolysis Bullosa
- Keywords
- EB, EBS, DEB, JEB
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Castle Creek Pharmaceuticals, LLCIndustry (pharma, biotech, devices)
- Study start
- 18 May 2018 (actual)
- Primary completion
- 31 Jan 2019 (actual)
- Study completion
- 14 Feb 2019 (actual)
- First posted
- 21 Mar 2018 (actual)
- Results
- Posted 10 Feb 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A pharmacokinetic (PK) study in 16-20 EB subjects to be allocated to two cohorts. Cohort 1 to include 8-10 subjects (ages 12 yrs and older); Cohort 2 to include 8-10 subjects (ages 6 months-11 yrs, inclusive). Cohort 2 only included subjects 4 yrs and older. Serial PK blood sampling collected on Days 1 and 10. Analyses were performed to determine the concentrations of diacerein and rhein.
Detailed description
This was an open-label, single-period study that intended to include subjects with EB consisting of infants/ children (ages 6 months to 11 years, inclusive) and adolescents/ adults (ages 12 years and older). The study included 2 cohorts.
Cohort 1 was to include adolescent/adult subjects with EB (ages 12 years and older);
Cohort 2 was to include infants/children with EB (ages 4 to 11 years, inclusive).
For both cohorts, the study intended to enroll subjects with epidermolysis bullosa simplex (EBS), dystrophic epidermolysis bullosa (DEB), and junctional epidermolysis bullosa (JEB) with lesions encompassing >/=2% BSA. The diacerein application area was >/=5% BSA and included lesioned and non-lesioned skin (if lesions accounted for <5% BSA); however, topical administration was </=30% BSA. On Days 1 and 10, the topical dose of study drug was applied by study staff, and blood was sampled for PK analyses of diacerein and rhein pre-dose and through 8 hours post-application. On Days 2 to 9, study drug was applied to the application area(s) at home. For Cohort 1, trough PK samples were collected on any 2 available days from Days 3 through 9, and study drug was applied after blood draw, while for Cohort 2 trough PK analyses were not performed.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 (Adolescents, Adults) | Experimental | Drug: Diacerein 1% OintmentAdolescent and adult subjects with EB (aged 12 years and older) received diacerein 1% ointment daily for 10 days. |
| Cohort 2 (Children) | Experimental | Drug: Diacerein 1% OintmentChildren with EB (aged 4 to 11 years, inclusive) received diacerein 1% ointment daily for 10 days. |
Who may qualify
- Ages
- 4 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- At least 12 years of age (Cohort 1) or at least 6 months of age to 11 years, inclusive (Cohort 2) at screening. For US only: at least 4 years of age to 11 years, inclusive (Cohort 2)
- The subject must weigh at least 9 kg (19.8 lbs) at Screening.
- Subject has a documented genetic mutation consistent with EB.
- Subject has EB lesions on ≥ 2% body surface area (BSA) and the EB lesions are in the following body areas: a. Localized: plantar and/or palmar areas, b. Generalized: arms, legs, torso, hands and feet.
Key exclusion criteria (5 listed)
- Subject has EB lesions where drug will be applied that are infected
- Subject has used any diacerein containing product within 1 month prior to Visit 1
- Subject has used systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to dosing.
- Subject has used systemic steroidal therapy or has used topical steroidal therapy on the EB lesions in the application area within 14 days prior to dosing
- Subject has participated in an investigational drug trial in which administration of an investigational study medication occurred within 30 days prior to dosing
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 | Cohort 1 (Adolescents, Adults), Cohort 2 (Children) |
Primary outcome
Detectable Plasma Concentrations of Diacerein and Rhein
Time frame: Days 1-10, at select time points per protocol
Bioanalytical analyses were performed to determine concentrations levels of diacerein and rhein in plasma using validated bioanalytical methods. For Cohort 1, blood samples were taken at pre-dose and 0.5, 1, 2, 3, 4, 6, and 8 hours post-dose. For Cohort 2, blood samples were taken at pre-dose and 1, 2, 4, 6, and 8 hours post-dose. Trough PK samples were collected on any 2 available days from Days 3 through 9 for Cohort 1 only. Summary statistics for each scheduled time were only reported if at least 50% of subjects had quantifiable concentrations.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Feb 2020.
- 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 study chair
Castle Creek Pharmaceuticals, LLC
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Diacerein 1% Ointment in people with Epidermolysis Bullosa (EB) 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 4 inclusion and 5 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 10 Feb 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 10 Feb 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?
Detectable Plasma Concentrations of Diacerein and Rhein (time frame: Days 1-10, at select time points per protocol). 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 10 Feb 2020.