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Study of Diacerein After Maximum Use in Patients With EB

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ClinicalTrials.gov Identifier: NCT03472287
Recruitment Status : Recruiting
First Posted : March 21, 2018
Last Update Posted : May 11, 2018
Sponsor:
Information provided by (Responsible Party):
Castle Creek Pharmaceuticals, LLC

Brief Summary:
The study is designed as an open label, single period study in approximately 16-20 patients with EB ranging in age from infants/children (ages 6 months - 11 years, inclusive) and adolescents/adults (ages 12 and up) with at least 8-10 subjects between the aged 6 months to 11 years, inclusive (infants/children). Note: US sites will only enroll patients ages 4 and up. The study is 10 days total and will consist of serial PK blood sampling on Days 1 and 10.

Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa Drug: CCP-020 (Diacerein 1% Ointment) Phase 1

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Other
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)
Actual Study Start Date : May 10, 2018
Estimated Primary Completion Date : January 2019
Estimated Study Completion Date : January 2019


Arm Intervention/treatment
Experimental: Cohort 1 (Adolescents, Adults)
8-10 adolescent and adult patients with EB (aged 12 and older) to receive CCP-020 daily for 10 days
Drug: CCP-020 (Diacerein 1% Ointment)
Diacerein 1% Ointment administered topically

Experimental: Cohort 2 (Children, Infants)
8-10 infants/children with EB (aged 6 months to 11 years, inclusive) to receive CCP-020 daily for 10 days
Drug: CCP-020 (Diacerein 1% Ointment)
Diacerein 1% Ointment administered topically




Primary Outcome Measures :
  1. Pharmacokinetics of CCP-020 [ Time Frame: Days 1-10, at select time points per protocol ]
    Concentrations of diacerein (if measurable) and rhein in plasma will be determined after each treatment using validated bioanalytical methods.


Secondary Outcome Measures :
  1. Adverse Events [ Time Frame: 10 Days + 30 days after last product application ]
    Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)



Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subject/caregiver is able to comprehend and willing to sign an Informed Consent and/or Assent Form.
  • Subject is male or female at least 12 years of age (Cohort 1) or at least 6 months of age to 11 years, inclusive (Cohort 2) at screening. US only: subjects must be at least 4 years of age to 11 years, inclusive (Cohort 2) at time of consent.
  • The subject must weigh at least 9 kg (19.8 lbs) at Screening.
  • Subject has a documented genetic mutation consistent with EB. A blood or saliva sample will be collected for genetic confirmation if no documented gene mutation data is available.
  • Subject has EB lesions on ≥ 2% body surface area (BSA) and the EB lesions are in the following body areas:

    1. Localized: plantar and/or palmar areas
    2. Generalized: arms, legs, torso, hands and feet.

Exclusion Criteria:

  • Subject has EB lesions where drug will be applied that are infected (i.e., EB lesions that require anti-microbial therapy to treat an infection)
  • 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 (Note: inhaled, nasal sprays, and ophthalmic products containing steroids are allowed)
  • Subject has participated in an investigational drug trial in which administration of an investigational study medication occurred within 30 days prior to dosing

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03472287


Contacts
Contact: Castle Creek Pharmaceuticals (862) 286-0400 contact@castlecreekpharma.com

Locations
United States, Arizona
Phoenix Children's Hospital Not yet recruiting
Phoenix, Arizona, United States, 85016
Contact: Harper Price, MD         
Contact       kbadger@phoenixchildrens.com   
United States, California
Stanford School of Medicine Not yet recruiting
Palo Alto, California, United States, 94304
Contact: Joyce Teng, MD         
Contact       etafoya@stanford.edu   
United States, Illinois
Northwestern University Medical Center Not yet recruiting
Chicago, Illinois, United States, 60654
Contact: Amy Paller, MD         
Contact       rema.zebda@northwestern.edu   
United States, Minnesota
University of Minnesota Not yet recruiting
Minneapolis, Minnesota, United States, 55454
Contact: Kristen Hook, MD         
Contact       amhanson@umn.edu   
United States, North Carolina
UNC Dermatology Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Aida Lugo-Somolinos, MD         
Contact       'laura_mcleod@med.unc.edu   
France
Hopital Universitaire, Necker-Enfants-Malades Not yet recruiting
Paris, France
Contact: Christine Bodemer, MD       wiam.bhia-ext@aphp.fr   
Netherlands
University Medical Center Groningen Not yet recruiting
Groningen, Netherlands
Contact: Marcel Jonkman, MD         
Contact       r.bozoglan@umcg.nl   
United Kingdom
Great Ormond Street Hospital Not yet recruiting
London, United Kingdom
Contact: Anna Martinez, MD         
Contact       Sorcha.L.Osullivan@gstt.nhs.uk   
Sponsors and Collaborators
Castle Creek Pharmaceuticals, LLC

Responsible Party: Castle Creek Pharmaceuticals, LLC
ClinicalTrials.gov Identifier: NCT03472287     History of Changes
Other Study ID Numbers: CCP-020-101
First Posted: March 21, 2018    Key Record Dates
Last Update Posted: May 11, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Epidermolysis Bullosa
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Diacetylrhein
Anti-Inflammatory Agents