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

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ClinicalTrials.gov Identifier: NCT02654483
Recruitment Status : Completed
First Posted : January 13, 2016
Results First Posted : July 2, 2019
Last Update Posted : July 2, 2019
Sponsor:
Collaborators:
Epidermolysis Bullosa Research Partnership
Menlo Therapeutics Inc.
Information provided by (Responsible Party):
Jean Yuh Tang, Stanford University

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).

Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa Pruritus Drug: VPD-737 Other: Placebo Phase 2

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.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 14 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
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
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: 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.
Primary Purpose: Treatment
Official Title: A Phase 2 Trial of Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Epidermolysis Bullosa Patients
Actual Study Start Date : August 31, 2016
Actual Primary Completion Date : June 28, 2018
Actual Study Completion Date : July 27, 2018


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 Name: 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




Primary Outcome Measures :
  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 Outcome Measures :
  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.



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Ages Eligible for Study:   13 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Clinical diagnosis of Epidermolysis Bullosa (EB) and pruritus

Exclusion Criteria:

  • Chronic liver or renal disease

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02654483


Locations
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United States, California
Stanford
Stanford, California, United States, 94305
Sponsors and Collaborators
Jean Yuh Tang
Epidermolysis Bullosa Research Partnership
Menlo Therapeutics Inc.
Investigators
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Principal Investigator: Jean Tang, MD, PhD Stanford University
  Study Documents (Full-Text)

Documents provided by Jean Yuh Tang, Stanford University:
Informed Consent Form  [PDF] April 17, 2019


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Responsible Party: Jean Yuh Tang, Associate Professor, Stanford University
ClinicalTrials.gov Identifier: NCT02654483     History of Changes
Other Study ID Numbers: 34182
First Posted: January 13, 2016    Key Record Dates
Results First Posted: July 2, 2019
Last Update Posted: July 2, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Epidermolysis Bullosa
Pruritus
Skin Diseases
Skin Manifestations
Signs and Symptoms
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases, Vesiculobullous
Neurokinin-1 Receptor Antagonists
Neurokinin A
Substance P
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs