ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa

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Brief Title

ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa

Official Title

A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients With Epidermolysis Bullosa

Brief Summary

      The aim was to assess the efficacy and safety of SD-101-6.0 cream versus Placebo (SD-101-0.0)
      cream in the treatment of skin lesions in participants with Epidermolysis Bullosa. Funding
      Source - United States Food and Drug Administration (FDA) Office of Orphan Products
      Development (OOPD).
    

Detailed Description

      This was a Phase 3, multi-center, randomized, double-blind, placebo-controlled study to
      assess the efficacy and safety of SD-101-6.0 cream versus placebo (SD-101-0.0) cream on skin
      lesions in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz
      Epidermolysis Bullosa. Epidermolysis Bullosa is a rare group of inherited disorders that
      typically manifest at birth as blistering and lesion formation on the skin in response to
      little or no apparent trauma. In this study, SD-101-6.0 cream or placebo (SD-101-0.0) cream
      was to be applied topically, once a day to the entire body for a period of 90 days.
      Participants had 1 target wound selected at baseline by the investigator. The selected target
      wound was required to have been present for at least 21 days. 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 for Visit 2 (approximately 14 days from baseline), Visit 3
      (approximately 30 days from baseline), Visit 4 (approximately 60 days from baseline), and
      Visit 5 (approximately 90 days from baseline) to have the target wound assessed for the level
      of healing. In addition, itching, pain, body surface area, target wound closure, and scarring
      of healed target wound were assessed at each visit. The ARANZ SilhouetteStar™ was used to
      measure the target wound at all visits.
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Time To Complete Target Wound Closure Within 3 Months

Secondary Outcome

 Percentage Of Participants Experiencing Complete Closure Of Their Target Wound At Month 1 And Month 2 Visits

Condition

Epidermolysis Bullosa

Intervention

SD-101-6.0 cream

Study Arms / Comparison Groups

 SD-101-6.0 cream
Description:  SD-101-6.0 cream applied topically, once a day to the entire body for a period of 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

169

Start Date

March 11, 2015

Completion Date

July 5, 2017

Primary Completion Date

July 5, 2017

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.

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

          -  Participant must have had 1 target wound (size 10 to 50 cm^2) at study entry.

          -  Participants 1 month and older.

          -  Target wound must have been present for at least 21 days.

        Exclusion Criteria:

          -  Participants who did not meet the entry criteria outlined above.

          -  Selected target wound did not have clinical evidence of local infection.

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

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

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

          -  Use of systemic antibiotics within the 7 days before enrollment.

          -  Current or former malignancy.

          -  Arterial or venous disorder resulting in ulcerated lesions.

          -  Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at
             screening and every 30 days until the final visit for female participants of
             childbearing potential).

          -  Females of childbearing potential who were not abstinent and 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

NCT02384460

Organization ID

SD-005

Secondary IDs

2014-002288-14

Responsible Party

Sponsor

Study Sponsor

Scioderm, Inc.

Collaborators

 Amicus Therapeutics

Study Sponsor

Medical Monitor, Study Director, Amicus Therapeutics


Verification Date

April 2020