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

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Brief Title

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

Official Title

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

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.
    

Study Phase

Phase 3

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 Up To Month 30

Condition

Epidermolysis Bullosa

Intervention

SD-101-6.0 cream

Study Arms / Comparison Groups

 Experimental: SD-101-6.0 cream
Description:  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.

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

152

Start Date

June 9, 2015

Completion Date

September 3, 2018

Primary Completion Date

September 3, 2018

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.
      

Gender

All

Ages

1 Month - N/A

Accepts Healthy Volunteers

No

Contacts

Medical Monitor, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT02670330

Organization ID

SD-006

Secondary IDs

2014-005679-96

Responsible Party

Sponsor

Study Sponsor

Scioderm, Inc.

Collaborators

 Amicus Therapeutics

Study Sponsor

Medical Monitor, Study Director, Amicus Therapeutics


Verification Date

September 2019