Efficacy and Safety Study to Evaluate SD-101 in Epidermolysis Bullosa

NCT07482787 | Recruiting Phase 3 FDA Drug

• 1 other identifier: SD-007
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 1

Brief Summary

The upcoming trial is for EB patients is a topically applied whole-body treatment of patients with either Simplex, RDEB or Junctional (nH) ages 1 month to 12 years old at study entry. There are only 4 site visits by patients with minimal assessments over a 2-month period, and patients completing this study will have the ability to continue receiving SD-101-6.0 at home in an open-label extension study. The drug product and placebo require no special preparation or storage conditions (room temperature).

Conditions

Epidermolysis Bullosa (EB)

Keywords

Wound closure; Itching; Wound burden

Outcome Measures

Primary Outcomes (1)

• Target wound closure

  The primary endpoint is the proportion of patients in each treatment group who experience complete closure of their target wound by Month 1 on study. Complete target wound closure is defined as skin re-epithelialization without drainage.

  1 month

Secondary Outcomes (2)

• Change in wound burden

  2 months

• Change in itching

  2 months

Study Arms (2)

SD-101 Dermal Cream (6%)

ACTIVE COMPARATOR

Drug: SD-101 Dermal Cream (6%)

Vehicle (SD-101 0%)

PLACEBO COMPARATOR

Drug: Vehicle (SD-101 0%)

Interventions

SD-101 Dermal Cream (6%) 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 (6%)

Vehicle (SD-101 0%) DRUG

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

Vehicle (SD-101 0%)

Eligibility Criteria

• Age: 28 Days - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Informed Consent (ICF) form signed by the patient or patient's legal representative. Since all patients with be 12 years old or younger at study entry, signed assent from the patient's legal representative will be required.
• Patient (or caretaker) must be willing to comply with all protocol requirements.
• Diagnosis of Simplex, Recessive Dystrophic, or JEB-nH EB.
• Patient must have 1 target wound (size 10 to 50 cm2).
• Patients must be between 28 days to 12 years at study entry
• Target wound must be present for 21 days or more.
• Patients must have wound burden based on BSA of ≥5% at baseline

You may not qualify if:

• Patients who do not meet the entry criteria outlined above.
• Selected target wound cannot have clinical evidence of local infection.
• Use of any investigational drug or approved EB therapy 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 are allowed)
• Use of systemic antibiotics within 7 days before enrollment.
• Current or former malignancy.
• Participant is one of the following:
• Not of childbearing potential (premenarchal or male/not in possession of a uterus).
• If of childbearing potential, is nonpregnant (negative urine pregnancy test results at Screening), nonlactating, and practicing 1 of the following medically acceptable methods of birth control:
• Abstinence from heterosexual intercourse as a lifestyle choice.
• Hormonal methods such as oral, implantable, injectable, or transdermal contraceptives for a minimum of 1 full cycle (based on the participant's usual menstrual cycle period) before product administration.
• Intrauterine device. If male, is willing to use a highly effective method of contraception throughout the study period.

Sponsors & Collaborators

• Paradigm Therapeutics: lead

Study Sites (1)

Mission Dermatology

Rancho Santa Margarita, California, 92688, United States

RECRUITING

Related Links

• Paradigm Therapeutics Website
• EB Research Partnership Website
• Debra US Website

MeSH Terms

Conditions

Epidermolysis Bullosa; Pruritus

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous; Skin Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Kathleen Watts

CONTACT

352-586-4083; Cure@Kapadi.com

Robert P Ryan, PhD

CONTACT

919-274-0703; rryan@paradigmtherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 4, 2026
• First Posted: March 19, 2026
• Study Start: July 21, 2026
• Primary Completion (Estimated): May 17, 2027
• Study Completion (Estimated): June 17, 2027
• Last Updated: July 23, 2026

Record last verified: 2026-07

Locations

US (1)