Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa

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Brief Title

Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa

Official Title

An Open Label Extension, Multi-Center, Study to Evaluate the Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa

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
    

Detailed Description

      This was an open-label extension study to assess the continued safety of topically applied
      SD-101 dermal cream (6%) in participants with Simplex, Recessive Dystrophic, and Junctional
      non-Herlitz EB.

      SD-101 dermal cream (6%) was applied topically, once a day to the entire body for the
      duration of the study. Participants who successfully completed the entire SD-003 study
      (NCT02014376) had the option to roll over into the SD-004 study (NCT02090283). The baseline
      visit occurred at the final visit date for the SD-003 study. The body surface area (BSA)
      coverage of blisters and lesions assessment made at the final SD-003 study visit were used as
      the baseline information at the baseline visit for the SD-004 study. Participants returned to
      the study site for the following 13 visits (36 months) to have BSA assessed. BSA was assessed
      at all subsequent scheduled study center visits. Scheduled study center visits occurred every
      6 months after Month 36 (Month 42, 48, and so on). After completion of Month 36, the next
      participant visit (Month 39) was a phone call from the site to the participant. Telephone
      visits occurred every 6 months thereafter (Month 45, 51, and so on) and included assessment
      of adverse events and concomitant medications only. For female participants of childbearing
      potential, a urine pregnancy test was performed at Month 6 and every 6 months up to and
      including the final study visit.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

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

Secondary Outcome

 Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 24

Condition

Epidermolysis Bullosa

Intervention

SD-101 dermal cream (6%)

Study Arms / Comparison Groups

 SD-101 Dermal Cream (6%)
Description:  All participants applied SD-101 dermal cream topically, once a day, to the entire body for the duration of the study.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

42

Start Date

March 26, 2014

Completion Date

September 14, 2018

Primary Completion Date

September 14, 2018

Eligibility Criteria

        Inclusion Criteria:

          -  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.

        Exclusion Criteria:

          -  Participants who do not meet the inclusion criteria.

          -  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.
      

Gender

All

Ages

6 Months - N/A

Accepts Healthy Volunteers

No

Contacts

Medical Monitor, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02090283

Organization ID

SD-004

Secondary IDs

1R01FD005093-01

Responsible Party

Sponsor

Study Sponsor

Scioderm, Inc.

Collaborators

 Amicus Therapeutics

Study Sponsor

Medical Monitor, Study Director, Amicus Therapeutics


Verification Date

October 2019