Study to Assess the Efficacy of VLY-686 in Relieving Symptoms of Gastroparesis

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Proof of Concept Study to Assess the Efficacy of VLY-686 (Tradipitant) in Relieving Symptoms of Gastroparesis

This is a multicenter, randomized, double-blind, placebo-controlled study to be conducted in the United States. One hundred fifty (150) subjects diagnosed with gastroparesis, who satisfy the selection criteria for the study, will be randomized to one of two treatment groups, active or placebo.

Study Overview

• Status: Completed
• Conditions: Gastroparesis
• Intervention / Treatment: Drug: VLY-686 (Tradipitant); Other: Placebo

Detailed Description

This is a multicenter, randomized, double-blind, placebo-controlled study to be conducted in the United States. One hundred fifty (150) subjects diagnosed with gastroparesis, who satisfy the selection criteria for the study, will be randomized to one of two treatment groups, active or placebo.

The study is divided into two phases: the screening phase and the evaluation phase. The screening phase includes a screening visit to evaluate subjects' preliminary eligibility for the study. During the screening phase, subjects will collect diary data for at least 4 weeks. The evaluation phase includes 4 weeks of randomized double-blind treatment.

• Study Type: Interventional
• Enrollment (Actual): 152
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Dothan, Alabama, United States, 36305
      • Vanda Investigational Site
    • Huntsville, Alabama, United States, 35801
      • Vanda Investigational Site
  • Arizona
    • Tucson, Arizona, United States, 85712
      • Vanda Investigational Site
  • Arkansas
    • Little Rock, Arkansas, United States, 72211
      • Vanda Investigational Site
  • California
    • Chula Vista, California, United States, 91910
      • Vanda Investigational Site
    • Los Angeles, California, United States, 90095
      • Vanda Investigational Site
    • Palo Alto, California, United States, 94305
      • Vanda Investigational Site
    • San Francisco, California, United States, 94115
      • Vanda Investigational Site
  • Florida
    • Jacksonville, Florida, United States, 32256
      • Vanda Investigational Site
    • Largo, Florida, United States, 33777
      • Vanda Investigational Site
    • Miami, Florida, United States, 33134
      • Vanda Investigational Site
  • Georgia
    • Athens, Georgia, United States, 30607
      • Vanda Investigational Site
  • Illinois
    • Elgin, Illinois, United States, 60123
      • Vanda Investigational Site
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Vanda Investigational Site
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • Vanda Investigational Site
  • Louisiana
    • Marrero, Louisiana, United States, 70072
      • Vanda Investigational Site
  • Maryland
    • Chevy Chase, Maryland, United States, 20815
      • Vanda Investigational Site
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Vanda Investigational Site
    • Boston, Massachusetts, United States, 02215
      • Vanda Investigational Site
  • Michigan
    • Wyoming, Michigan, United States, 49519
      • Vanda Investigational Site
  • Minnesota
    • Minneapolis, Minnesota, United States, 55114
      • Vanda Investigational Site
  • Mississippi
    • Pascagoula, Mississippi, United States, 39564
      • Vanda Investigational Site
  • Missouri
    • Saint Louis, Missouri, United States, 63104
      • Vanda Investigational Site
  • New York
    • Great Neck, New York, United States, 11023
      • Vanda Investigational Site
    • New York, New York, United States, 10028
      • Vanda Investigational Site
  • North Carolina
    • Raleigh, North Carolina, United States, 27612
      • Vanda Investigational Site
    • Salisbury, North Carolina, United States, 28144
      • Vanda Investigational Site
  • Ohio
    • Huber Heights, Ohio, United States, 45424
      • Vanda Investigational Site
    • Mentor, Ohio, United States, 44060
      • Vanda Investigational Site
  • Oklahoma
    • Tulsa, Oklahoma, United States, 74104
      • Vanda Investigational Site
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19140
      • Vanda Investigational Site
  • Tennessee
    • Chattanooga, Tennessee, United States, 37421
      • Vanda Investigational Site
    • Jackson, Tennessee, United States, 38305
      • Vanda Investigational Site
    • Nashville, Tennessee, United States, 37211
      • Vanda Investigational Site
  • Texas
    • El Paso, Texas, United States, 79905
      • Vanda Investigational Site
    • Houston, Texas, United States, 77030
      • Vanda Investigational Site
    • Richardson, Texas, United States, 75082
      • Vanda Investigational Site
  • Virginia
    • Norfolk, Virginia, United States, 23502
      • Vanda Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Diagnosed with gastroparesis;
2. Subjects must agree to the use of contraception
3. Ability and acceptance to provide written informed consent;
4. Willing to participate in the pharmacogenomics sample collection;
5. Willing and able to comply with all study requirements and restrictions
6. Willing to not participate in any other interventional trial for the duration of their participation.

Exclusion Criteria:

1. Another active disorder or treatment which could explain or contribute to symptoms in the opinion of the Investigator (including but not limited to gastric malignancy, neurological disorder, or heavy doses of strong anticholinergics);
2. Pregnancy or nursing;
3. History of intolerance and/or hypersensitivity to medications similar to VLY-686 (Tradipitant) and its accompanying excipients;
4. Use of another NK1 antagonist or palonosetron;
5. Exposure to any investigational medication, including placebo, within 60 days of the Baseline Visit;
6. Any other reason as determined by the Investigator which may lead to an unfavorable risk-benefit of study participation, may interfere with study compliance, or may confound study results.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Study Drug; VLY-686 (Tradipitant) oral capsule for 4 weeks.	Drug: VLY-686 (Tradipitant); oral capsule
Placebo Comparator: Placebo; Placebo oral capsule for 4 weeks.	Other: Placebo; placebo oral

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Average Nausea Severity	The Gastroparesis Core Symptom Daily Diary (GCSDD) is a patient reported diary that asks patients to rate the worst occurrence of each cardinal symptom of gastroparesis (nausea severity, early satiety, postprandial fullness, bloating, and abdominal pain) in the past 24 hours on a Likert scale from 0 (no symptoms) to 5 (very severe). Change from baseline in average nausea severity score is calculated as the weekly average post value minus baseline value of the daily nausea severity score from the GCSDD. A negative change indicates improvement.	4 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Weekly % Nausea-Free Days	The Gastroparesis Core Symptom Daily Diary (GCSDD) is a patient reported diary that asks patients if they have had any nausea over the past 24 hours and if yes, to rate the worst occurrence in the past 24 hours on a Likert scale from 1 (very mild) to 5 (very severe). Change in weekly percentage of nausea-free days from baseline is calculated as weekly average post value minus baseline value. Baseline is defined as the average of all nonmissing values in the screening phase (4 weeks).	4 weeks
Daily Average Vomiting Frequency, Change From Baseline	The Gastroparesis Core Symptom Daily Diary (GCSDD) is a patient reported diary that asks patients if they have vomited in the past 24 hours and how many times they vomited in the past 24 hours. Change from baseline in the weekly average of daily vomiting frequency is calculated as the weekly average post value minus baseline value of the daily daily vomiting frequency from the GCSDD. Baseline is defined as the average of all non-missing values in the screening phase (4 weeks). A negative change indicates improvement.	4 weeks
Gastroparesis Cardinal Symptom Index (GCSI), Change From Baseline	The GCSI is a subject reported outcome administered in-clinic with a 2-week recall period to measure the severity of symptoms in gastroparesis. The GCSI is composed of 9 items based on three subscales: post-prandial fullness/early satiety (4 items); nausea/vomiting (3 items), and bloating (2 items). The severity of each symptom is rated on a six-point Likert response scale. The minimal scale value = 0 (none) and the maximum scale value = 5 (very severe). GCSI Total score is constructed as the average of the three symptom sub-scales. Higher score is indicative of greater symptom severity.	4 weeks
PAGI-SYM, Change From Baseline	The PAGI-SYM is a patient reported outcome which asks patients to describe the severity of their symptoms over the last two weeks to measure symptom severity for gastroparesis, functional dyspepsia, and gastroesophageal reflux disease. The measure consists of 20 symptom severity items, which cover the following domains: nausea/vomiting, fullness/early satiety, bloating, upper abdominal pain, heartburn/regurgitation, and lower abdominal pain. This questionnaire includes the Gastroparesis Cardinal Symptom Index (GCSI). The severity of each symptom is rated on a six-point Likert response scale. The minimal scale value = 0 (none) and the maximum scale value= 5 (very severe). PAGI-SYM Total score is constructed as the average of the six symptom sub-scales and a higher score is indicative of greater symptom severity.	4 weeks
CGI-S, Change From Baseline	The Clinician Global Impression of Severity (CGI-S) is a 7-point scale on which the clinician rates the severity of the patient's gastroparesis at the time of the assessment and refers to the degree of illness at the time of the visit and during the 2 weeks before the visit. The CGI-S is rated on the following 7-point scale: 1: normal, not at all ill; 2: borderline ill: 3: mildly ill; 4: moderately ill; 5: markedly ill; 6: severely ill; 7: among the most extremely ill patients. Change from baseline is calculated as post value minus baseline value. A negative change indicates improvement.	4 weeks
PGI-C	The Patient Global Impression of Change (PGI-C) is a patient reported questionnaire with a 7-point rating scale where the participant rates his/her own improvement in overall symptoms relative to the baseline assessment. Is is rated as 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse. Higher scores indicate a worse outcome.	4 weeks

Collaborators and Investigators

• Sponsor: Vanda Pharmaceuticals

Publications and helpful links

General Publications

• Carlin JL, Lieberman VR, Dahal A, Keefe MS, Xiao C, Birznieks G, Abell TL, Lembo A, Parkman HP, Polymeropoulos MH. Efficacy and Safety of Tradipitant in Patients With Diabetic and Idiopathic Gastroparesis in a Randomized, Placebo-Controlled Trial. Gastroenterology. 2021 Jan;160(1):76-87.e4. doi: 10.1053/j.gastro.2020.07.029. Epub 2020 Jul 18.

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2016
• Primary Completion (Actual): November 1, 2018
• Study Completion (Actual): December 1, 2018

Study Registration Dates

• First Submitted: November 15, 2016
• First Submitted That Met QC Criteria: November 18, 2016
• First Posted (Estimated): November 22, 2016

Study Record Updates

• Last Update Posted (Actual): August 6, 2024
• Last Update Submitted That Met QC Criteria: July 15, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Neurologic Manifestations; Gastrointestinal Diseases; Stomach Diseases; Paralysis; Gastroparesis
• Other Study ID Numbers: VP-VLY-686-2301

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO