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

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ClinicalTrials.gov Identifier: NCT02654483

Recruitment Status : Active, not recruiting

First Posted : January 13, 2016

Last Update Posted : November 1, 2018

Sponsor:

Collaborators:

Epidermolysis Bullosa Research Partnership

Menlo Therapeutics Inc.

Information provided by (Responsible Party):

Jean Yuh Tang, Stanford University

Study Description

Brief Summary:

Our goal is to determine whether daily oral administration of VPD-737 (5 mg) is effective and safe in adolescents and adults 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.

The investigators are now testing VPD-737 in 14 patients with EB in a Phase II randomized, placebo-controlled, double-blinded clinical trial.

Study Design


Study Type :	Interventional (Clinical Trial)
Estimated Enrollment :	14 participants
Allocation:	Randomized
Intervention Model:	Crossover Assignment
Masking:	Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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 :	July 27, 2018
Estimated Study Completion Date :	February 2019

Resource links provided by the National Library of Medicine

Genetics Home Reference related topics: Epidermolysis bullosa simplex Epidermolysis bullosa with pyloric atresia Junctional epidermolysis bullosa

MedlinePlus related topics: Itching

Genetic and Rare Diseases Information Center resources: Epidermolysis Bullosa

U.S. FDA Resources

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

Change in EB associated itch [ Time Frame: NRS recorded by subject daily, from screening visit through the end of the study ]
Determine the efficacy of Serlopitant compared with placebo on reducing EB itch as measured by patient self-reports using a numeric rating scale (NRS) for itch severity and the Stanford EB itch questionnaire. NRS is included in the Stanford EB Itch survey.

Secondary Outcome Measures :

Wound healing determination [ Time Frame: screening, month 1, month 2 ]
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.

Eligibility Criteria

Information from the National Library of Medicine

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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 and pruritus

Exclusion Criteria:

Have chronic liver or renal disease

Contacts and Locations

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


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

Sponsors and Collaborators

Jean Yuh Tang

Epidermolysis Bullosa Research Partnership

Menlo Therapeutics Inc.

Investigators


Principal Investigator:	Jean Tang, MD, PhD	Stanford University

More Information


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
Last Update Posted:	November 1, 2018
Last Verified:	October 2018

Additional relevant MeSH terms:


Pruritus Epidermolysis Bullosa Skin Diseases Skin Manifestations Signs and Symptoms Skin Abnormalities Congenital Abnormalities Skin Diseases, Genetic	Genetic Diseases, Inborn Skin Diseases, Vesiculobullous Neurokinin A Substance P Neurokinin-1 Receptor Antagonists Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs

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