NCT02654483

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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
14

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Aug 2016

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 11, 2016

Completed
2 days until next milestone

First Posted

Study publicly available on registry

January 13, 2016

Completed
8 months until next milestone

Study Start

First participant enrolled

August 31, 2016

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 28, 2018

Completed
29 days until next milestone

Study Completion

Last participant's last visit for all outcomes

July 27, 2018

Completed
11 months until next milestone

Results Posted

Study results publicly available

July 2, 2019

Completed
Last Updated

March 23, 2020

Status Verified

March 1, 2020

Enrollment Period

1.8 years

First QC Date

January 11, 2016

Results QC Date

April 24, 2019

Last Update Submit

March 10, 2020

Conditions

Epidermolysis BullosaPruritus

Outcome Measures

Primary Outcomes (1)

  • Comparative Weekly Change in NRS Itch Score Over the 8-week Active Treatment Period

    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 Outcomes (2)

  • Wound Healing Determination

    Baseline and 8 weeks

  • Change in Mean NRS Itch Score During Bathing/Dressing Changes

    Baseline and 8 weeks

Study Arms (2)

5 mg VPD-737

EXPERIMENTAL

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

Drug: VPD-737

Placebo

PLACEBO COMPARATOR

Placebo tablets to be taken daily by mouth for 56 days

Other: Placebo

Interventions

VPD-737DRUG

VPD-737 inhibits the receptor neurokinin-1.

Also known as: Serlopitant
5 mg VPD-737
PlaceboOTHER

Matching tablets to VPD-737 tablets without active drug

Placebo

Eligibility Criteria

Age13 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Clinical diagnosis of Epidermolysis Bullosa (EB) and pruritus

You may not qualify if:

  • Chronic liver or renal disease

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Stanford

Stanford, California, 94305, United States

Location

Related Publications (1)

  • 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.

    PMID: 31541747RESULT

Related Links

MeSH Terms

Conditions

Epidermolysis BullosaPruritus

Interventions

serlopitant

Condition Hierarchy (Ancestors)

Skin AbnormalitiesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin Diseases, GeneticGenetic Diseases, InbornSkin DiseasesSkin and Connective Tissue DiseasesSkin Diseases, VesiculobullousSkin ManifestationsSigns and SymptomsPathological Conditions, Signs and Symptoms

Results Point of Contact

Title
Irene Bailey, CRC
Organization
Stanford University
baileyhi@stanford.edu
650-721-7149

Study Officials

  • Jean Tang, MD, PhD

    Stanford University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Masking Details
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.
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: 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
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Associate Professor

Study Record Dates

First Submitted

January 11, 2016

First Posted

January 13, 2016

Study Start

August 31, 2016

Primary Completion

June 28, 2018

Study Completion

July 27, 2018

Last Updated

March 23, 2020

Results First Posted

July 2, 2019

Record last verified: 2020-03

Data Sharing

IPD Sharing
Will not share

Locations

US(1)