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

NCT ID: NCT02654483

Last Updated: 2020-03-23

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

14 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-08-31

Study Completion Date

2018-07-27

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Epidermolysis Bullosa Pruritus

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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 Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Research personnel not involved in this study was responsible for blinding. This was the only personnel with access to the code linking study drug bottle numbers to treatment (i.e. active or placebo). The code was broken when all data was captured and locked on an electronic data capture system.

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

5 mg VPD-737

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

Group Type EXPERIMENTAL

VPD-737

Intervention Type DRUG

VPD-737 inhibits the receptor neurokinin-1.

Placebo

Placebo tablets to be taken daily by mouth for 56 days

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

Matching tablets to VPD-737 tablets without active drug

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

VPD-737

VPD-737 inhibits the receptor neurokinin-1.

Intervention Type DRUG

Placebo

Matching tablets to VPD-737 tablets without active drug

Intervention Type OTHER

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

Serlopitant

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Clinical diagnosis of Epidermolysis Bullosa (EB) and pruritus

Exclusion Criteria

* Chronic liver or renal disease
Minimum Eligible Age

13 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Epidermolysis Bullosa Research Partnership

OTHER_GOV

Sponsor Role collaborator

Vyne Therapeutics Inc.

INDUSTRY

Sponsor Role collaborator

Jean Yuh Tang

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Jean Yuh Tang

Associate Professor

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Jean Tang, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Stanford

Stanford, California, United States

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type RESULT
PMID: 31541747 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Informed Consent Form

View Document

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

Access external resources that provide additional context or updates about the study.

https://www-ncbi-nlm-nih-gov.laneproxy.stanford.edu/pubmed/31541747

Phase 2 trial of a neurokinin-1 receptor antagonist for the treatment of chronic itch in patients with epidermolysis bullosa: A randomized clinical trial.

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

34182

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Gentamicin for RDEB
NCT03012191 COMPLETED PHASE1/PHASE2
Xerotic Dermatitis in Aged People
NCT01806935 COMPLETED NA
Gene Therapy for Netherton Syndrome
NCT01545323 UNKNOWN PHASE1