Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa

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Brief Title

Study of Effectiveness and Safety of SD-101 in Participants With Epidermolysis Bullosa

Official Title

A Phase 2b, Multi-Center, Randomized, Double-Blind, Vehicle-Controlled Study of the Effectiveness and Safety of SD-101 Cream in Subjects With Epidermolysis Bullosa

Brief Summary

      The purpose of this study was to assess whether the topical use of SD-101 cream (3% or 6%)
      was effective in treating wounds in participants with Simplex, Recessive Dystrophic, or
      Junctional non-Herlitz Epidermolysis Bullosa (EB).
    

Detailed Description

      This was a Phase 2b, multi-center, randomized, double-blind, vehicle-controlled study to
      assess the efficacy and safety of SD-101 dermal cream (3% or 6%) on skin lesions in
      participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.

      SD-101 cream [containing 3% or 6% or vehicle (0%)] was applied topically, once a day to the
      entire body for a period of 90 days. Eligible participants had a target wound assessed at
      baseline. Selected target wound had to be at least a certain age and within a prespecified
      size range at study entry. Photographic confirmation of the target wound location was
      collected at baseline, and the picture saved from the first visit was used to confirm
      location of the target wound at subsequent visits. The participant returned to the study site
      at Week 2, Month 1, Month 4, and Month 5 (at 14, 30, 60, and 90 days, respectively) to have
      the target wound previously identified at baseline re-assessed for the level of healing. In
      addition, changes in itching, pain, body surface area coverage of blisters and lesions, and
      scaring of the healed target wound were also assessed at each visit.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Participants With Documented Complete Closure Of The Target Wound Within 1 Month After Initiation Of Treatment

Secondary Outcome

 Participants With Documented Complete Closure Of The Target Wound Within 2 And 3 Months After Initiation Of Treatment

Condition

Epidermolysis Bullosa

Intervention

SD-101 dermal cream (3%)

Study Arms / Comparison Groups

 SD-101 Dermal Cream (6%)
Description:  SD-101 dermal cream (6%) applied topically once daily over the entire body for 90 days.

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

48

Start Date

January 6, 2014

Completion Date

June 24, 2014

Primary Completion Date

June 24, 2014

Eligibility Criteria

        Inclusion Criteria:

          -  Informed Consent form signed by the participant or the participant's legal
             representative; if the participant is under the age of majority but capable of
             providing assent, signed assent from the participant.

          -  Participant (or caretaker) was willing to comply with all protocol requirements.

          -  Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.

          -  Participants 6 months of age and older.

          -  Participants had 1 target wound within a prespecified size range at study entry.

          -  Target wound was at least 21 days or older.

        Exclusion Criteria:

          -  Participants who did not meet all the entry criteria outlined in inclusion criteria.

          -  Selected target wound had clinical evidence of local infection.

          -  Use of any investigational drug within 30 days before enrollment.

          -  Use of immunotherapy or cytotoxic chemotherapy within 60 days before enrollment.

          -  Use of systemic or topical steroidal therapy within 30 days before enrollment (inhaled
             steroids and ophthalmic drops containing steroids were allowed).

          -  Use of systemic antibiotics within 7 days before enrollment.

          -  Current or former malignancy.

          -  Arterial or venous disorder resulting in ulcerated wounds.

          -  Diabetes mellitus.

          -  Pregnancy or breastfeeding during the study (a urine pregnancy test was performed at
             screening for female participants of childbearing potential).

          -  Females of childbearing potential who were not abstinent and not practicing a
             medically acceptable method of contraception.

          -  Known history of cardiac, hepatic, or renal disease.
      

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

NCT02014376

Organization ID

SD-003


Responsible Party

Sponsor

Study Sponsor

Scioderm, Inc.

Collaborators

 Amicus Therapeutics

Study Sponsor

Medical Monitor, Study Director, Amicus Therapeutics


Verification Date

January 2020