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

NCT ID: NCT02384460

Last Updated: 2020-04-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 169 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-03-11
• Study Completion Date: 2017-07-05

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.

Conditions

Epidermolysis Bullosa

Study Design

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

Study Groups

SD-101-6.0 cream

SD-101-6.0 cream applied topically, once a day to the entire body for a period of 90 days

Group Type: EXPERIMENTAL

SD-101-6.0 cream

Intervention Type: DRUG

applied topically once a day for 90 days

Placebo (SD-101-0.0) cream

SD-101-0.0 (placebo) cream applied topically, once a day to the entire body for a period of 90 days

Group Type: PLACEBO_COMPARATOR

Placebo (SD-101-0.0) cream

Intervention Type: DRUG

applied topically once a day for 90 days

Interventions

SD-101-6.0 cream

applied topically once a day for 90 days

Intervention Type: DRUG

Placebo (SD-101-0.0) cream

applied topically once a day for 90 days

Intervention Type: DRUG

Other Intervention Names

SD-101; placebo

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.

• Minimum Eligible Age: 1 Month
• 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

Phoenix, Arizona, United States

Redwood City, California, United States

Aurora, Colorado, United States

Washington D.C., District of Columbia, United States

Miami, Florida, United States

Miami, Florida, United States

Chicago, Illinois, United States

Minneapolis, Minnesota, United States

Columbia, Missouri, United States

St Louis, Missouri, United States

Lebanon, New Hampshire, United States

Hackensack, New Jersey, United States

East Setauket, New York, United States

New York, New York, United States

Chapel Hill, North Carolina, United States

Cincinnati, Ohio, United States

Philadelphia, Pennsylvania, United States

Charleston, South Carolina, United States

Austin, Texas, United States

San Antonio, Texas, United States

Seattle, Washington, United States

Kogarah, New South Wales, Australia

Parkville, Victoria, Australia

Parkville, Victoria, Australia

Salzburg, , Austria

Nice, , France

Paris, , France

Toulouse, , France

Freiburg im Breisgau, , Germany

Hanover, , Germany

Tel Aviv, , Israel

Milan, , Italy

Kaunas, , Lithuania

Groningen, , Netherlands

Warsaw, Koszykowa, Poland

Belgrade, , Serbia

Madrid, , Spain

London, , United Kingdom

Countries

United States; Australia; Austria; France; Germany; Israel; Italy; Lithuania; Netherlands; Poland; Serbia; Spain; United Kingdom

References

Murrell DF, Paller AS, Bodemer C, Browning J, Nikolic M, Barth JA, Lagast H, Krusinska E, Reha A; ESSENCE Study Group. Wound closure in epidermolysis bullosa: data from the vehicle arm of the phase 3 ESSENCE Study. Orphanet J Rare Dis. 2020 Jul 21;15(1):190. doi: 10.1186/s13023-020-01435-3.

Reference Type: DERIVED

PMID: 32693833 (View on PubMed)

Paller AS, Browning J, Nikolic M, Bodemer C, Murrell DF, Lenon W, Krusinska E, Reha A, Lagast H, Barth JA; ESSENCE Study Group. Efficacy and tolerability of the investigational topical cream SD-101 (6% allantoin) in patients with epidermolysis bullosa: a phase 3, randomized, double-blind, vehicle-controlled trial (ESSENCE study). Orphanet J Rare Dis. 2020 Jun 23;15(1):158. doi: 10.1186/s13023-020-01419-3.

Reference Type: DERIVED

PMID: 32576219 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2014-002288-14

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

R01-005095-01

Identifier Type: OTHER

Identifier Source: secondary_id

SD-005

Identifier Type: -

Identifier Source: org_study_id