Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex

NCT03389308 | Completed Phase 2 Results FDA Drug

• 1 other identifier: CCP-020-302
• Study Type: interventional
• Target: 51
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

Epidermolysis Bullosa; Epidermolysis Bullosa Simplex

Keywords

Epidermolysis Bullosa (EB); Epidermolysis Bullosa Simplex (EBS)

Outcome Measures

Primary Outcomes (1)

• Treatment-emergent Adverse Events

  Number and percentage of participants with any treatment-emergent adverse event.

  up to 52 weeks

Study Arms (1)

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.

Drug: diacerein 1% ointment

Interventions

diacerein 1% ointment DRUG

Topically apply study medication to lesions identified by the Investigator that require. treatment

Also known as: CCP-020

Treatment period

Eligibility Criteria

• Age: 6 Months - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Castle Creek Pharmaceuticals, LLC: lead

Study Sites (3)

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

University of North Carolina (UNC) - Chapel Hill

Chapel Hill, North Carolina, 27516, United States

Location

Children's Hospital of San Antonio

San Antonio, Texas, 78218, United States

Location

MeSH Terms

Conditions

Epidermolysis Bullosa; Epidermolysis Bullosa Simplex

Interventions

diacerein; Ointments

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous

Intervention Hierarchy (Ancestors)

Dosage Forms; Pharmaceutical Preparations

Results Point of Contact

• Title: Mary C. Spellman, MD
• Organization: Castle Creek Pharmaceuticals

mspellman@castlecreekpharma.com

312-847-1291

Study Officials

• Mary Spellman, MD

  Castle Creek Pharmaceuticals

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Masking Details: Each treatment cycle will consist of 8 weeks on treatment, followed by 8 weeks off treatment with a maximum of 2 treatment cycles allowed for up to 1 year. Subjects should be assessed at minimum every 8 weeks for disease activity. Once a subject completes two cycles of treatment or reaches Week 52, the subject will be discharged from the study. Subjects may not start a new treatment cycle past Week 36 from Baseline Visit without prior sponsor approval. 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.
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: December 19, 2017
• First Posted: January 3, 2018
• Study Start: December 1, 2017
• Primary Completion: April 17, 2020
• Study Completion: April 17, 2020
• Last Updated: September 16, 2020
• Results First Posted: September 16, 2020

Record last verified: 2020-08

Locations

US (3)