Health TrialsbyHealthPanel

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

ConditionEpidermolysis Bullosa (EB)
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age28-12
SponsorParadigm Therapeutics

About this trial

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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 (intermediate) EB.

Patient must have 1 target wound (size 10 to 50 cm2).

Disqualifiers

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.

Trial design

Design model

Parallel

Treatments tested in this trial

  • 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.

  • 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.

Treatment groups

80 Participants
are divided into 2 treatment groups
Group A: SD-101 Dermal Cream (6%)Active comparator 1 intervention
Group B: Vehicle (SD-101 0%)Placebo comparator 1 intervention

Trial outcomes

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.

Time frame
1 month

Secondary outcomes

1

Change in wound burden

Change in wound burden based on BSA measurements at Month 2, compared to Baseline.

Time frame
2 months
2

Change in itching

The Itch Man Pruritus Assessment Tool, which is a validated 5-point Likert scale that goes from 0 to 4, where 0 is comfortable and 4 is the most terrible. The scale is used to assess itching at Week 1 (Days 1-7), Week 2, Month 1 and Month 2, compared to Baseline.

Time frame
2 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Paradigm Therapeutics

Lead sponsor

Source ClinicalTrials.gov
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