NCT02654483

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

Completed Phase 2 Results posted Last updated 23 March 2020

What this trial tests

Phase 2 trial testing VPD-737 in Epidermolysis Bullosa in 14 participants. Completed in 27 July 2018.

Timeline

31 August 2016

Primary endpoint
28 June 2018

27 July 2018

Quick facts

Lead sponsor	Jean Yuh Tang
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	14
Start date	31 August 2016
Primary completion	28 June 2018
Estimated completion	27 July 2018
Sites	1 location across United States

Drugs / interventions tested

• VPD-737 — full drug profile →
• Placebo

Conditions studied

• Epidermolysis Bullosa — all drugs for Epidermolysis Bullosa →
• Pruritus — all drugs for Pruritus →

Sponsor

Jean Yuh Tang

Who can join

13 and older, any sex, with Epidermolysis Bullosa or Pruritus. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Comparative Weekly Change in NRS Itch Score Over the 8-week Active Treatment Period Primary · 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 t

Group	Value	95% CI
5 mg VPD-737	-2	-5 – 0
Placebo	-1	-7 – 1

Wound Healing Determination Secondary · 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.

Group	Value	95% CI
5 mg VPD-737	154.54	± 227
Placebo	38.39	± 180

Change in Mean NRS Itch Score During Bathing/Dressing Changes Secondary · 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.

Group	Value	95% CI
5 mg VPD-737	-2.5	-4 – 1
Placebo	-2	-5 – 0

Adverse events — posted to ClinicalTrials.gov

Time frame: Up to 10 months; some patients did not proceed to the open-label arm immediately and were monitored for AEs during the period between arms.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Double-blind, 5 mg VPD-737

Serious: 1/7 (14%)

Deaths: 0/7

Double-blind, Placebo

Serious: 1/7 (14%)

Deaths: 1/7

Open-label, 5 mg VPD-737 - 5 mg VPD-737

Serious: 0/5 (0%)

Deaths: 0/5

Open-label, Placebo - 5 mg VPD-737

Serious: 0/3 (0%)

Deaths: 0/3

Serious adverse events (5 terms)

Reaction	System	Double-blind, 5 mg VPD-737	Double-blind, Placebo	Open-label, 5 mg VPD-737 -…	Open-label, Placebo - 5 mg…
pulmonary embolism	Respiratory, thoracic and mediastinal disorders	—	—	—	—
heart failure	Cardiac disorders	—	—	—	—
pleural effusion	Respiratory, thoracic and mediastinal disorders	—	—	—	—
nephropathy	Renal and urinary disorders	—	—	—	—
hematuria	Renal and urinary disorders	—	—	—	—

Other adverse events (21 terms — click to expand)

Reaction	System	Double-blind, 5 mg VPD-737	Double-blind, Placebo	Open-label, 5 mg VPD-737 -…	Open-label, Placebo - 5 mg…
nausea	Gastrointestinal disorders	—	—	—	—
infection (wound)	Infections and infestations	—	—	—	—
diarrhea	Gastrointestinal disorders	—	—	—	—
rash	Skin and subcutaneous tissue disorders	—	—	—	—
otitis	Ear and labyrinth disorders	—	—	—	—
headache	Nervous system disorders	—	—	—	—
somnolence	Nervous system disorders	—	—	—	—
dizziness	Nervous system disorders	—	—	—	—
exacerbation of pruritus	Skin and subcutaneous tissue disorders	—	—	—	—
flu-like symptoms	General disorders	—	—	—	—
cough	Respiratory, thoracic and mediastinal disorders	—	—	—	—
pain	Injury, poisoning and procedural complications	—	—	—	—
toothache	Gastrointestinal disorders	—	—	—	—
infection	Infections and infestations	—	—	—	—
fever	General disorders	—	—	—	—
skin ulceration	Skin and subcutaneous tissue disorders	—	—	—	—
upper respiratory infection	Infections and infestations	—	—	—	—
constipation	Gastrointestinal disorders	—	—	—	—
dysesthesia	Nervous system disorders	—	—	—	—
pain	Musculoskeletal and connective tissue disorders	—	—	—	—
dyspepsia	Gastrointestinal disorders	—	—	—	—

Most-reported serious reactions: pulmonary embolism, heart failure, pleural effusion, nephropathy, hematuria.

Data from ClinicalTrials.gov NCT02654483 adverse events section.

Sponsor's own description

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).

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. NK-1 Receptor Antagonists and Pruritus: Review of Current Literature.
   Pojawa-Gołąb M, Jaworecka K, Reich A. · · 2019 · cited 30× · PMID 31190215 · DOI 10.1007/s13555-019-0305-2