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

March 10, 2020 updated by: Jean Yuh Tang

A Phase 2 Trial of Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Epidermolysis Bullosa Patients

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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

14

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Stanford, California, United States, 94305
        • Stanford

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

13 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Clinical diagnosis of Epidermolysis Bullosa (EB) and pruritus

Exclusion Criteria:

  • Chronic liver or renal disease

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 5 mg VPD-737
5 mg tablets of VPD-737 to be taken daily by mouth for 56 days
Drug: VPD-737
VPD-737 inhibits the receptor neurokinin-1.
Other Names:
  • Serlopitant
Placebo Comparator: Placebo
Placebo tablets to be taken daily by mouth for 56 days
Other: Placebo
Matching tablets to VPD-737 tablets without active drug

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Comparative Weekly Change in NRS Itch Score Over the 8-week Active Treatment Period
Time Frame: Baseline and 8 weeks

Determine the efficacy of Serlopitant compared with placebo on reducing EB-associated daily itch score as measured by patient self-reports using a numeric rating scale (NRS) for itch severity.The NRS is comprised of one item and represents the numbers 0 ("no itch") to 10 ("worst imaginable itch"). Because itch is subjective and can vary day to day, nightly NRS scores were recorded in patients' Itch Diaries. NRS recorded by subject daily, from screening visit through the end of the study.

This study was designed to detect differences between the two treatment groups. The primary endpoint was the comparative weekly change in Numeric Rating Scale (NRS) itch severity score from baseline over 8 weeks; derived from a linear mixed effects model which utilizes observations from both the treatment and placebo groups to generate an interaction term of interest. Itch severity changes from day to day and this model can more appropriately report trends in patients' itch severity with treatment.
Baseline and 8 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Wound Healing Determination
Time Frame: Baseline and 8 weeks
Wound dimensions, including length, width, and area (in cm2), will be obtained using the Canfield system. Changes in dimensions between visits as well as changes in dimensions from baseline will be recorded. Overall mean % change from baseline is reported as the secondary endpoint.
Baseline and 8 weeks
Change in Mean NRS Itch Score During Bathing/Dressing Changes
Time Frame: Baseline and 8 weeks
Itch is exacerbated by activities such as dressing changes or bathing. NRS itch score during bathing or dressing in the past 24 hours were collected. Numeric rating scale (NRS) for itch severity is comprised of one item and represents the numbers 0 ("no itch") to 10 ("worst imaginable itch"). This study was designed to detect differences between the two treatment groups. This secondary endpoint was the comparative change in Numeric Rating Scale (NRS) itch severity score during bathing/dressing changes from baseline over 8 weeks as determined by application of a linear mixed effects model.
Baseline and 8 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Jean Yuh Tang

Collaborators

Vyne Therapeutics Inc.
Epidermolysis Bullosa Research Partnership

Investigators

  • Principal Investigator: Jean Tang, MD, PhD, Stanford University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 31, 2016

Primary Completion (Actual)

June 28, 2018

Study Completion (Actual)

July 27, 2018

Study Registration Dates

First Submitted

January 11, 2016

First Submitted That Met QC Criteria

January 12, 2016

First Posted (Estimate)

January 13, 2016

Study Record Updates

Last Update Posted (Actual)

March 23, 2020

Last Update Submitted That Met QC Criteria

March 10, 2020

Last Verified

March 1, 2020

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 34182

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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