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

NCT ID: NCT02014376

Last Updated: 2020-01-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 48 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-01-06
• Study Completion Date: 2014-06-24

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.

Conditions

Epidermolysis Bullosa

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

SD-101 Dermal Cream (6%)

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

Group Type: EXPERIMENTAL

SD-101 Dermal Cream (6%)

Intervention Type: DRUG

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.

SD-101 Dermal Cream (3%)

SD-101 dermal cream (3%) applied topically once daily over the entire body for 90 days.

Group Type: EXPERIMENTAL

SD-101 dermal cream (3%)

Intervention Type: DRUG

SD-101 is a white, crystalline powder that is formulated within an odorless, soft, white cream base. SD-101-3.0 cream contains allantoin, a diureide glyoxylic acid, at a concentration of 3% and other excipients.

Vehicle (0%)

Vehicle dermal cream (SD-101 0%) applied topically once daily over the entire body for 90 days.

Group Type: PLACEBO_COMPARATOR

Vehicle (SD-101 0%)

Intervention Type: DRUG

A white, crystalline powder that is formulated within an odorless, soft, white cream base. It contains no allantoin, only excipients.

Interventions

SD-101 dermal cream (3%)

SD-101 is a white, crystalline powder that is formulated within an odorless, soft, white cream base. SD-101-3.0 cream contains allantoin, a diureide glyoxylic acid, at a concentration of 3% and other excipients.

Intervention Type: 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.

Intervention Type: DRUG

Vehicle (SD-101 0%)

A white, crystalline powder that is formulated within an odorless, soft, white cream base. It contains no allantoin, only excipients.

Intervention Type: DRUG

Other Intervention Names

SD-101; SD-101-3.0; Zorblisa; SD-101; SD-101-6.0; Zorblisa; Placebo Comparator; SD-101-0.0

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.

• 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.

• Minimum Eligible Age: 6 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Amicus Therapeutics

INDUSTRY

Sponsor Role: collaborator

Scioderm, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Monitor

Role: STUDY_DIRECTOR

Amicus Therapeutics

Locations

Palo Alto, California, United States

Chicago, Illinois, United States

St Louis, Missouri, United States

Hackensack, New Jersey, United States

Chapel Hill, North Carolina, United States

San Antonio, Texas, United States

Seattle, Washington, United States

Countries

United States

Other Identifiers

SD-003

Identifier Type: -

Identifier Source: org_study_id