Brief Title
Neurokinin-1 Receptor Antagonist for the Treatment of Itch in EB Patients
Official Title
A Phase 2 Trial of Neurokinin-1 Receptor Antagonist for the Treatment of Itch in Epidermolysis Bullosa Patients
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).
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 Phase
Phase 2
Study Type
Interventional
Primary Outcome
Comparative Weekly Change in NRS Itch Score Over the 8-week Active Treatment Period
Secondary Outcome
Wound Healing Determination
Condition
Epidermolysis Bullosa
Intervention
VPD-737
Study Arms / Comparison Groups
5 mg VPD-737
Description: 5 mg tablets of VPD-737 to be taken daily by mouth for 56 days
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
14
Start Date
August 31, 2016
Completion Date
July 27, 2018
Primary Completion Date
June 28, 2018
Eligibility Criteria
Inclusion Criteria:
- Clinical diagnosis of Epidermolysis Bullosa (EB) and pruritus
Exclusion Criteria:
- Chronic liver or renal disease
Gender
All
Ages
13 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Jean Tang, MD, PhD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT02654483
Organization ID
34182
Responsible Party
Sponsor-Investigator
Study Sponsor
Jean Yuh Tang
Collaborators
Epidermolysis Bullosa Research Partnership
Study Sponsor
Jean Tang, MD, PhD, Principal Investigator, Stanford University
Verification Date
March 2020