Study Stopped
The Sponsor voluntarily recalled SD-101 and terminated the study due to GMP deficiencies identified during an FDA inspection at the site of the manufacturer.
Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
An Open Label Extension, Multi-Center, Study to Evaluate the Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa
2 other identifiers
interventional
42
1 country
7
Brief Summary
The purpose of this study was to assess the continued safety of topical use of SD-101 cream (6%) in participants with Epidermolysis Bullosa (EB). Funding Source: FDA Office of Orphan Products Development
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Mar 2014
Typical duration for phase_2
7 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 12, 2014
CompletedFirst Posted
Study publicly available on registry
March 18, 2014
CompletedStudy Start
First participant enrolled
March 26, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 14, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
September 14, 2018
CompletedResults Posted
Study results publicly available
November 4, 2019
CompletedNovember 4, 2019
October 1, 2019
4.5 years
March 12, 2014
September 10, 2019
October 31, 2019
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number Of Participants With Treatment-Emergent Adverse Events (TEAEs)
Treatment-emergent adverse events were defined as adverse events that started or worsened on or after baseline visit, which occurred at the final visit date for the SD-003 study. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
From baseline to 30 days after last application of study drug (up to a maximum of 54 months)
Secondary Outcomes (1)
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 24
Baseline, Month 24
Study Arms (1)
SD-101 Dermal Cream (6%)
EXPERIMENTALAll participants applied SD-101 dermal cream topically, once a day, to the entire body for the duration of the study.
Interventions
SD-101 is a white, crystalline powder that is formulated within an odorless, soft, white cream base. SD-101-6.0 cream contains allantoin, a diureide glyoxylic acid, at a concentration of 6% and other excipients.
Eligibility Criteria
You may qualify if:
- Informed consent form signed by the participant or participant's legal representative; if the participant is under the age of 18 but capable of providing assent, signed assent from the participant.
- Participant (or caretaker) must be willing to comply with all protocol requirements.
- Participant must have successfully completed the SD-003 study.
You may not qualify if:
- Pregnancy or breastfeeding during the study. A urine pregnancy test will be performed at the final visit for SD-003 for female participants of childbearing potential.
- Females of childbearing potential who are not abstinent and not practicing a medically acceptable method of contraception.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Scioderm, Inc.lead
- Amicus Therapeuticscollaborator
- Food and Drug Administration (FDA)collaborator
Study Sites (7)
Unknown Facility
Palo Alto, California, 94304, United States
Unknown Facility
Chicago, Illinois, 60611, United States
Unknown Facility
St Louis, Missouri, 63110, United States
Unknown Facility
Hackensack, New Jersey, 07601, United States
Unknown Facility
Chapel Hill, North Carolina, 27516, United States
Unknown Facility
San Antonio, Texas, 78218, United States
Unknown Facility
Seattle, Washington, 98105, United States
Related Publications (1)
Paller A., Browning J., Aslam R., et al. Efficacy and Safety Results From a 24-Month, Open-Label Extension of a Phase 2b Dose-Ranging Study of SD-101 Cream in Patients With Epidermolysis Bullosa. Abstract presented at European Academy of Dermatology and Venereology, 26th Congress, Geneva, Switzerland, September 13-17, 2017.
RESULT
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Limitations and Caveats
Results are presented for all participants who received SD-101 before the early termination of the study on 4 June 2018. The maximum study duration completed by at least 1 participant was 54 months.
Results Point of Contact
- Title
- Patient Advocacy
- Organization
- Amicus Therapeutics, Inc.
Study Officials
- STUDY DIRECTOR
Medical Monitor
Amicus Therapeutics
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- GT60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 12, 2014
First Posted
March 18, 2014
Study Start
March 26, 2014
Primary Completion
September 14, 2018
Study Completion
September 14, 2018
Last Updated
November 4, 2019
Results First Posted
November 4, 2019
Record last verified: 2019-10