Clinical trial NCT02654483: Neurokinin-1 Receptor Antagonist for the Treatment of Itch in EB Patients
Official title: A Phase 2 Trial of Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Epidermolysis Bullosa Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Epidermolysis Bullosa
Pruritus
Lead sponsor: Stanford University
Sponsor class: OTHER
Collaborators: name: Epidermolysis Bullosa Research Partnership
name: Vyne Therapeutics Inc.
First posted: 2016-01-13
Enrollment: 14
Sex: ALL
Minimum age (years): 13
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Phase 2 investigator-initiated, randomized, double-blind, placebo-controlled, parallel-arm trial evaluating the comparative effect of serlopitant 5 mg by mouth daily versus placebo in treatment of chronic pruritus in pediatric and adult EB patients
Primary purpose: TREATMENT

Brief summary: 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).

Detailed 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.

Eligibility criteria: Inclusion Criteria:

* Clinical diagnosis of Epidermolysis Bullosa (EB) and pruritus

Exclusion Criteria:

* Chronic liver or renal disease

Primary outcomes:
measure: Comparative Weekly Change in NRS Itch Score Over the 8-week Active Treatment Period
timeFrame: Baseline and 8 weeks
Secondary outcomes:
measure: Wound Healing Determination
timeFrame: Baseline and 8 weeks
measure: Change in Mean NRS Itch Score During Bathing/Dressing Changes
timeFrame: Baseline and 8 weeks
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford
state: California
Study officials:
affiliation: Stanford University
name: Jean Tang, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Chiou AS, Choi S, Barriga M, Dutt-Singkh Y, Solis DC, Nazaroff J, Bailey-Healy I, Li S, Shu K, Joing M, Kwon P, Tang JY. Phase 2 trial of a neurokinin-1 receptor antagonist for the treatment of chronic itch in patients with epidermolysis bullosa: A randomized clinical trial. J Am Acad Dermatol. 2020 Jun;82(6):1415-1421. doi: 10.1016/j.jaad.2019.09.014. Epub 2019 Sep 18.