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

October 31, 2019 updated by: Scioderm, Inc.

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

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

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

42

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Palo Alto, California, United States, 94304
    • Illinois
      • Chicago, Illinois, United States, 60611
    • Missouri
      • Saint Louis, Missouri, United States, 63110
    • New Jersey
      • Hackensack, New Jersey, United States, 07601
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27516
    • Texas
      • San Antonio, Texas, United States, 78218
    • Washington
      • Seattle, Washington, United States, 98105

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: SD-101 Dermal Cream (6%)
All participants applied SD-101 dermal cream topically, once a day, to the entire body for the duration of the study.
Drug: SD-101 dermal cream (6%)
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.
Other Names:
  • SD-101
  • Zorblisa
  • SD-101-6.0

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number Of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time Frame: From baseline to 30 days after last application of study drug (up to a maximum of 54 months)
Treatment-emergent adverse events were defined as adverse events that started or worsened on or after baseline visit, which occurred at the final visit date for the SD-003 study. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
From baseline to 30 days after last application of study drug (up to a maximum of 54 months)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline In Body Surface Area Index (BSAI) Of Lesional Skin At Month 24
Time Frame: Baseline, Month 24
Lesional skin was defined as areas that contained any of the following: blisters, erosions, ulcerations, scabbing, bullae, or eschars, as well as areas that were weeping, sloughing, oozing, crusted, or denuded. The percentage, ranging from 0% to 100%, of affected body surface area (BSA) was recorded for each defined body region (that is, head/neck, upper limbs, trunk [includes groin], and lower limbs), multiplied by the weighting factor, and then summed for all body regions to calculate the BSAI. The BSA for lesional skin was to be assessed by the same study physician on each visit for a particular participant. The mean change from baseline (final visit from the SD-003 study) in BSAI was assessed every 3 months. Only participants with data available for analysis at the specified time point are presented.
Baseline, Month 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Scioderm, Inc.

Collaborators

Food and Drug Administration (FDA)
Amicus Therapeutics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

  • Paller A., Browning J., Aslam R., et al. Efficacy and Safety Results From a 24-Month, Open-Label Extension of a Phase 2b Dose-Ranging Study of SD-101 Cream in Patients With Epidermolysis Bullosa. Abstract presented at European Academy of Dermatology and Venereology, 26th Congress, Geneva, Switzerland, September 13-17, 2017.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 26, 2014

Primary Completion (Actual)

September 14, 2018

Study Completion (Actual)

September 14, 2018

Study Registration Dates

First Submitted

March 12, 2014

First Submitted That Met QC Criteria

March 16, 2014

First Posted (Estimate)

March 18, 2014

Study Record Updates

Last Update Posted (Actual)

November 4, 2019

Last Update Submitted That Met QC Criteria

October 31, 2019

Last Verified

October 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SD-004
  • 1R01FD005093-01 (U.S. FDA Grant/Contract)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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