A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03836001|
Recruitment Status : Recruiting
First Posted : February 11, 2019
Last Update Posted : March 17, 2021
|Condition or disease||Intervention/treatment||Phase|
|Epidermolysis Bullosa||Drug: Serlopitant Tablet Drug: Placebo Oral Tablet||Phase 2|
The investigator will determine whether more patients taking serlopitant 5 mg daily as compared to placebo can achieve a 3 point or greater reduction in itch severity as measured by numeric rating scale (NRS) score following two months of treatment.
Secondary objectives include;
- comparative weekly change in daily worst-itch NRS,
- comparative weekly change in average daily NRS itch severity,
- the proportion of patients who achieve at a least 30% or 50% reduction in NRS severity from baseline at the end of two months of treatment,
- proportion of patients achieving 2 point and 4 point reductions in average daily itch severity following two months of treatment,
- change in participant-reported outcomes of global assessment of change in itch and overall improvement as measured by static participant assessment of itch, participant global assessment of change in itch severity, and caregiver global assessment of change in itch severity, and
- assessment on the safety of Serlopitant in adolescents (≥13 y.o.) and adults with pruritus associated with epidermolysis bullosa (EB).
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||This is an investigator-initiated, single-center, randomized, double-blind, placebo controlled, parallel arm trial evaluating the effects of serlopitant at 5 mg by mouth daily on EB-related itch.|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Masking Description:||This is a double-blind study.|
|Official Title:||A Neurokinin-1 Receptor Antagonist for the Treatment of Pruritus in Patients With Epidermolysis Bullosa|
|Actual Study Start Date :||April 18, 2019|
|Estimated Primary Completion Date :||April 30, 2022|
|Estimated Study Completion Date :||May 30, 2022|
Placebo Comparator: Placebo Oral Tablet
We aim to recruit at least 20 patients who will undergo two months of dosing with placebo (inactive drug or sugar pill), followed by one month of wash-out. All patients will be offered the option of participating in a 12-month open label extension with serlopitant at 5 mg (taken by mouth) daily for continued safety monitoring.
Drug: Placebo Oral Tablet
The placebo is a tablet that looks like a drug but has no drug or other active ingredient in it.
Other Name: Sugar pill
Active Comparator: Serlopitant Tablet
We aim to recruit at least 20 patients who will undergo two months of Serlopitant dosing, followed by one month of wash-out. All patients will be offered the option of participating in a 12-month open label extension with serlopitant 5 mg (taken by mouth) daily for continued safety monitoring.
Drug: Serlopitant Tablet
Serlopitant is a small molecule, highly selective NK1-R (neurokinin-1 receptor) antagonist. Two critical mediators of the urge to scratch are Substance P, or SP, and its receptor, NK1-R. SP is a naturally occurring peptide in the tachykinin neuropeptide family. Tachykinins have a broad range of functions in the nervous and immune systems. SP binding of NK1-R has been shown to be a key mediator of sensory nerve signaling, including the itch-scratch reflex and the vomiting reflex.
Other Name: VPD-737
- The proportion of patients who achieve at least a 3-point reduction in NRS itch severity from baseline and after two months of treatment. [ Time Frame: 2 months ]Participants will be asked to complete a daily itch diary with their average itch score (NRS) over the past 24 hours and their itch score during dressing changes or bathing (NRS).
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): NCT03836001
|Contact: Clinical Trial Coodinatorfirstname.lastname@example.org|
|Contact: Clinical Trial Coordinatoremail@example.com|
|United States, California|
|Redwood City, California, United States, 94063|
|Contact: Clinical Trial Coordinator 650-721-7149 firstname.lastname@example.org|
|Principal Investigator:||Albert S Chiou, MD/MBA||Stanford University|