Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa

NCT02670330 | Terminated Phase 3 Results

• 2 other identifiers: SD-0062014-005679-96
• Study Type: interventional
• Target: 152
• Locations: 11 countries
• Sites: 29

Brief Summary

The study aimed to assess the long-term safety of topical use of Zorblisa (SD-101-6.0) in participants with Epidermolysis Bullosa (EB).

Conditions

Epidermolysis Bullosa

Keywords

Amicus Therapeutics; Scioderm, Inc.; Simplex; Recessive Dystrophic; Junctional non-Herlitz; Epidermolysis Bullosa; SD-101 6%; SD-101-6.0; Allantoin 6%; Zorblisa

Outcome Measures

Primary Outcomes (1)

• Number Of Participants With Treatment Emergent Adverse Events (TEAEs)

  TEAEs were defined as adverse events that started or worsened on or after baseline visit.

  From baseline to 30 days after last application of study drug (up to a maximum of 37 months)

Secondary Outcomes (2)

• Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin Up To Month 30

  Baseline, up to Month 30

• Change From Baseline In BSAI Of Total Body Wound Burden Up To Month 30

  Baseline, up to Month 30

Study Arms (1)

Experimental: SD-101-6.0 cream

EXPERIMENTAL

All participants (or their caregivers) applied SD-101-6.0 cream topically once a day to the entire body for a period of up to 36 months.

Drug: SD-101-6.0 cream

Interventions

SD-101-6.0 cream DRUG

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.

Also known as: Zorblisa, SD-101

Experimental: SD-101-6.0 cream

Eligibility Criteria

• Age: 1 Month+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Informed Consent Form signed by the participant or participant's legal representative; if the participant was under the age of 18 but capable of providing assent, signed assent from the participant.
• Participant (or caretaker) must have been willing to comply with all protocol requirements.
• Participants who completed the SD-005 study (on study drug at Visit 5, approximately 90 days from baseline).

You may not qualify if:

• Participants who did not meet the entry criteria outlined above.
• Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at the final visit for Study SD-005 for female participants of childbearing potential and repeated at screening/baseline visit of Study SD-006 if these visits did not occur on the same day).
• Female participants of childbearing potential who were not abstinent or not practicing a medically acceptable method of contraception.

Sponsors & Collaborators

• Scioderm, Inc.: lead
• Amicus Therapeutics: collaborator

Study Sites (30)

Unknown Facility

Phoenix, Arizona, 85016, United States

Location

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Redwood City, California, 94063, United States

Location

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Aurora, Colorado, 80045, United States

Location

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Washington D.C., District of Columbia, 20016, United States

Location

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Chicago, Illinois, 60611, United States

Location

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Minneapolis, Minnesota, 55455, United States

Location

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Columbia, Missouri, 65212, United States

Location

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St Louis, Missouri, 63110, United States

Location

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East Setauket, New York, 11733, United States

Location

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Chapel Hill, North Carolina, 27516, United States

Location

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Cincinnati, Ohio, 45229-3039, United States

Location

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Philadelphia, Pennsylvania, 19104, United States

Location

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Charleston, South Carolina, 29425-5780, United States

Location

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San Antonio, Texas, 78218, United States

Location

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Seattle, Washington, 98105, United States

Location

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Kogarah, New South Wales, 2217, Australia

Location

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Parkville, Victoria, 3052, Australia

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Salzburg, 5020, Austria

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Nice, 1-06202, France

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Paris, 75015, France

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Toulouse, France

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Freiburg im Breisgau, 79104, Germany

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Hanover, 30173, Germany

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Tel Aviv, Israel

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Kaunas, LT-50009, Lithuania

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Groningen, 9713 GZ, Netherlands

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Warsaw, Poland

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Belgrade, Serbia

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Madrid, Spain

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London, WC1N 3JH, United Kingdom

Location

MeSH Terms

Conditions

Epidermolysis Bullosa

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

Limitations and Caveats

Results are presented for all participants who received SD-101-6.0 before the early termination of the study on 4 June 2018. The maximum study duration completed by at least 1 participant was 37 months.

Results Point of Contact

• Title: Patient Advocacy
• Organization: Amicus Therapeutics, Inc.

clinicaltrials@amicusrx.com

+1-609-662-2000

Study Officials

• Medical Monitor

  Amicus Therapeutics

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 28, 2015
• First Posted: February 1, 2016
• Study Start: June 9, 2015
• Primary Completion: September 3, 2018
• Study Completion: September 3, 2018
• Last Updated: September 27, 2019
• Results First Posted: August 2, 2019

Record last verified: 2019-09

Locations

LI (1); NL (1); ES (1); GB (1); AU (2); DE (2); IL (1); US (15); PL (1); SE (1); FR (3)