Neurokinin-1 Receptor Antagonist for the Treatment of Itch in EB Patients

Jean Yuh Tang

Status and phase: Completed; Phase 2

Conditions: Pruritus; Epidermolysis Bullosa

Treatments: Drug: VPD-737; Other: Placebo

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02654483

34182

Details and patient eligibility

About

Our goal is to determine whether daily oral administration of VPD-737 (5 mg) is effective and safe in treating moderate to severe pruritus in patients with Epidermolysis Bullosa (EB).

Full description

Itch is the most common complaint reported by patients with EB of all subtypes, and there is no current effective treatment. Itch often triggers scratching that creates new wounds and increases EB disease severity.

This study aims to target the physiological mechanisms of pruritus (itch) in patients with EB. Substance P is a major mediator of pruritus and binds to the receptor neurokinin-1 (NK1), which is expressed in the central nervous system and the skin.

VPD-737 (serlopitant), a novel drug that inhibits the NK1 receptor, has been shown to reduce severe itch in a previous study of 257 adult patients with chronic pruritus.

Each patient will be un-blinded individually after completing 3 months of study period.

All patients who complete the study will be offered a 2-month period on active drug. Patient who received active drug in the first period will be contacted and asked if they would like to continue on active drug for an additional 2 months.

Patients who received placebo in the first period will be contacted and asked if they would like to repeat the study on open label for 2 months.

Enrollment: 14 patients

Sex: All

Ages: 13+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Clinical diagnosis of Epidermolysis Bullosa (EB) and pruritus

Exclusion criteria

• Chronic liver or renal disease

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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14 participants in 2 patient groups, including a placebo group

5 mg VPD-737

Experimental group

Description:

5 mg tablets of VPD-737 to be taken daily by mouth for 56 days

Treatment:

Drug: VPD-737

Placebo

Placebo Comparator group

Description:

Placebo tablets to be taken daily by mouth for 56 days

Treatment:

Other: Placebo