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

Primary Purpose

Epidermolysis Bullosa, Epidermolysis Bullosa Simplex

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
diacerein 1% ointment
Sponsored by
Castle Creek Pharmaceuticals, LLC
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Epidermolysis Bullosa focused on measuring Epidermolysis Bullosa (EB), Epidermolysis Bullosa Simplex (EBS)

Eligibility Criteria

6 Months - 99 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  • 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.

Key Exclusion Criteria:

  • 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.

Sites / Locations

  • Ann and Robert H. Lurie Children's Hospital of Chicago
  • University of North Carolina (UNC) - Chapel Hill
  • Children's Hospital of San Antonio

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment period

Arm Description

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.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Full Information

First Posted
December 19, 2017
Last Updated
August 26, 2020
Sponsor
Castle Creek Pharmaceuticals, LLC
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1. Study Identification

Unique Protocol Identification Number
NCT03389308
Brief Title
Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex
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)
Study Type
Interventional

2. Study Status

Record Verification Date
August 2020
Overall Recruitment Status
Completed
Study Start Date
December 1, 2017 (Actual)
Primary Completion Date
April 17, 2020 (Actual)
Study Completion Date
April 17, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Castle Creek Pharmaceuticals, LLC

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Epidermolysis Bullosa, Epidermolysis Bullosa Simplex
Keywords
Epidermolysis Bullosa (EB), Epidermolysis Bullosa Simplex (EBS)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
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)
Masking Description
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.
Allocation
N/A
Enrollment
51 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Treatment period
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
diacerein 1% ointment
Other Intervention Name(s)
CCP-020
Intervention Description
Topically apply study medication to lesions identified by the Investigator that require. treatment
Primary Outcome Measure Information:
Title
Treatment-emergent Adverse Events
Description
Number and percentage of participants with any treatment-emergent adverse event.
Time Frame
up to 52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
99 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: 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. Key Exclusion Criteria: 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mary Spellman, MD
Organizational Affiliation
Castle Creek Pharmaceuticals
Official's Role
Study Chair
Facility Information:
Facility Name
Ann and Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
University of North Carolina (UNC) - Chapel Hill
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27516
Country
United States
Facility Name
Children's Hospital of San Antonio
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78218
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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