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

NCT ID: NCT02670330

Last Updated: 2019-09-27

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 152 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-06-09
• Study Completion Date: 2018-09-03

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).

Detailed Description

This was an open label, multi-center extension study to assess the long-term safety of topically applied SD-101-6.0 in participants with simplex, recessive dystrophic, and junctional non-Herlitz EB. SD-101-6.0 was applied topically once a day to the entire body. The planned duration of treatment with SD-101-6.0 for Study SD-006 was up to 48 months, with a safety follow-up period of 30 days; however, the study was terminated early by the sponsor. The maximum study duration completed by at least 1 participant, treatment and safety follow-up, was 37 months.

Participants who successfully completed Study SD-005 had the option to rollover into Study SD-006. The screening/baseline visit (Visit 1) occurred at Visit 5 (approximately 90 days from baseline) of Study SD-005. The Body Surface Area (BSA) assessments of lesional skin and wound burden performed at Visit 5 (approximately 90 days from baseline) for Study SD-005 were utilized as the baseline assessments for Study SD-006. Participants returned for follow-up visits at Month 1 then every 3 months.

At each visit, assessments included BSA of lesional skin and wound burden. For target wounds that are not closed by the end of Study SD-005, the ARANZ picture and calculation of target wound area at the final visit for Study SD-005 was used as the baseline area size of the target wound for Study SD-006. These unhealed target wounds from Study SD-005 were assessed via ARANZ SilhouetteStar™ at each subsequent scheduled visit until the target wound was documented as closed. Closed wounds were assessed for scarring.

Conditions

Epidermolysis Bullosa

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Experimental: SD-101-6.0 cream

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.

Group Type: EXPERIMENTAL

SD-101-6.0 cream

Intervention Type: 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.

Interventions

SD-101-6.0 cream

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.

Intervention Type: DRUG

Other Intervention Names

Zorblisa; SD-101

Eligibility Criteria

Inclusion Criteria

• 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).

Exclusion Criteria

• 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.

• Minimum Eligible Age: 1 Month
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Amicus Therapeutics

INDUSTRY

Sponsor Role: collaborator

Scioderm, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Monitor

Role: STUDY_DIRECTOR

Amicus Therapeutics

Locations

Phoenix, Arizona, United States

Redwood City, California, United States

Aurora, Colorado, United States

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

Chicago, Illinois, United States

Minneapolis, Minnesota, United States

Columbia, Missouri, United States

St Louis, Missouri, United States

East Setauket, New York, United States

Chapel Hill, North Carolina, United States

Cincinnati, Ohio, United States

Philadelphia, Pennsylvania, United States

Charleston, South Carolina, United States

San Antonio, Texas, United States

Seattle, Washington, United States

Kogarah, New South Wales, Australia

Parkville, Victoria, Australia

Salzburg, , Austria

Nice, , France

Paris, , France

Toulouse, , France

Freiburg im Breisgau, , Germany

Hanover, , Germany

Tel Aviv, , Israel

Kaunas, , Lithuania

Groningen, , Netherlands

Warsaw, , Poland

Belgrade, , Serbia

Madrid, , Spain

London, , United Kingdom

Countries

United States; Australia; Austria; France; Germany; Israel; Lithuania; Netherlands; Poland; Serbia; Spain; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2014-005679-96

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

SD-006

Identifier Type: -

Identifier Source: org_study_id