MedTrace Logo

Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)

NCT03406611 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 39

Last updated 2026-08-10

No results posted yet for this study

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.

Conditions

  • Homocystinuria

Interventions

DRUG

Pegtibatinase

Pegtibatinase sterile solution for subcutaneous injection

DRUG

Placebo

Normal saline for subcutaneous injection

Sponsors & Collaborators

  • Travere Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Michael Imperiale, MD · Travere Therapeutics, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
5 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-01-22
Primary Completion
2027-07-31
Completion
2027-07-31
FDA Drug
Yes

Countries

  • United States
  • France
  • Qatar

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03406611 on ClinicalTrials.gov