Skip to main content
OpenTrials
Completed

NCT Number: NCT02654483

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

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Epidermolysis Bullosa Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Pathological Conditions, Signs and Symptoms Pruritus Signs and Symptoms Skin Abnormalities Skin Diseases Skin Diseases, Genetic Skin Diseases, Vesiculobullous Skin Manifestations Skin and Connective Tissue Diseases

Age range

13 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Stanford

Stanford, California, 94305, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Clinical diagnosis of Epidermolysis Bullosa (EB) and pruritus

Exclusion criteria

  • Chronic liver or renal disease

Treatment and study plan

VPD-737

Drug

VPD-737 inhibits the receptor neurokinin-1.

Other names: Serlopitant

Placebo

Other

Matching tablets to VPD-737 tablets without active drug

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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.

Sponsors and collaborators

Lead sponsor

Jean Yuh Tang

Other

Collaborators

  • Epidermolysis Bullosa Research Partnership
  • Vyne Therapeutics Inc.

Registry information

Official study title

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

Important dates

Study start
2016
Primary completion
2018
Study completion
2018
First posted
Jan 13, 2016
Registry last updated
Mar 23, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.