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Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)
Sponsor: Travere Therapeutics, Inc.
Summary
Researchers are looking for a better way to treat people who have classical homocystinuria (HCU), a rare condition that is passed down by parents (or "genetic condition"). It is caused by changes in the cystathionine beta-synthase (or "CBS") gene and prevents an enzyme from working correctly in the body. This enzyme breaks down a substance called homocysteine (from dietary methionine found in protein) and keeps both homocysteine and methionine at normal levels. When this enzyme is not working, homocysteine and methionine build up in the blood, which spreads into different tissues of the body and stops these body tissues from working normally. People with HCU can experience problems with vision, bones, blood vessels, and cognitive function (the ability to think, learn, and remember). Treatments available for HCU, such as a low protein diet and betaine (Cystadane®), help reduce homocysteine levels. The diet is a low methionine diet and a methionine-free protein supplement (a product that provides extra protein to help meet daily protein needs). These treatments are either not sufficient or are hard to take for many patients. Pegtibatinase was developed by scientists to be a version of the CBS enzyme that can be given to people with HCU. Researchers believe that giving pegtibatinase to people with HCU already getting medical treatment (or "standard of care") may reduce their homocysteine levels. This study is split into 7 different groups getting different amounts of drug. The first 6 groups have already finished the study. Group 7 plans to enroll participants from the US (virtual and in-person), France, and Qatar.
Official title: A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Effects on Clinical Outcomes of Pegtibatinase (TVT-058) Administered Subcutaneously in Subjects With Cystathionine Beta Synthase-Deficient Homocystinuria (COMPOSE)
Key Details
Gender
All
Age Range
5 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
39
Start Date
2019-01-22
Completion Date
2027-07
Last Updated
2026-08-10
Healthy Volunteers
No
Conditions
Interventions
Pegtibatinase
Pegtibatinase sterile solution for subcutaneous injection
Placebo
Normal saline for subcutaneous injection
Locations (12)
Travere Investigational Site
Aurora, Colorado, United States
Travere Investigational Site
Miami, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Travere Investigational Site
Indianapolis, Indiana, United States
Travere Investigational Site
Portland, Maine, United States
Travere Investigational Site
Boston, Massachusetts, United States
The Mount Sinai Hospital
New York, New York, United States
Travere Investigational Site
New York, New York, United States
Science 37 - Virtual Site
Morrisville, North Carolina, United States
Travere Investigational Site
Philadelphia, Pennsylvania, United States
Hospital Necker-Enfants Malades, Neurologie Pediatrique
Paris, France
Sidra Medicine
Doha, Qatar