What this study is
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.
What's being tested
- Pegtibatinase
Intervention · DRUG
Pegtibatinase sterile solution for subcutaneous injection
- Placebo
Intervention · DRUG
Normal saline for subcutaneous injection
Study details
- 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)
- Sponsor
- Travere Therapeutics, Inc.
- Conditions
- Homocystinuria
- Enrolling
- 39 people
- Starts
- 2019-01-22
- Est. completion
- 2027-07
- Design
- Quadruple-blind, randomized, parallel groups, Phase 1, Phase 2
- Record
- View on ClinicalTrials.gov ↗
Who can join
Age 5 Years – 65 YearsSex ALLHealthy volunteers Not acceptedYou may qualify if
- Age; Cohort 7 (currently enrolling): ≥5 to <12 years of age.; Completed Cohorts 1-6: ≥12 to 65 years of age.
- Diagnosis of classical homocystinuria (HCU); Cohort 7 (currently enrolling): Diagnosis based on clinical, biochemical, and/or molecular genetic testing.; Completed Cohorts 1-6: Genetically confirmed cystathionine beta-synthase (CBS)-deficient HCU.
- Plasma total homocysteine (tHcy); Cohort 7 (currently enrolling): Plasma tHcy ≥50 μM at Screening.; Completed Cohorts 1-6: Plasma tHcy ≥50 μM at Screening and documented historical plasma tHcy ≥80 μM.
- Willing and able (or parent/legal guardian willing and able) to provide informed consent/assent and comply with study procedures.
- Willing to maintain a generally stable standard-of-care treatment regimen, including dietary management and HCU-related therapies, unless changes are medically necessary.
- Participants of childbearing potential must have a negative pregnancy test before study treatment and agree to use protocol-specified contraception, if applicable.
You can't join if
- Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase (MTHFR) deficiency, or a disorder of cobalamin metabolism.
- History of a major thrombotic event within the previous 6 months.
- Body weight <15 kg.
- Previous treatment with pegtibatinase or pegtarviliase)
- Participation in a pegtibatinase clinical study.
- Receipt of another investigational drug or investigational medical device within 30 days before Screening or planned use during study participation.
- Use of injectable polyethylene glycol (PEG)-containing medications (other than pegtibatinase or PEG-containing vaccines) within 3 months before Screening or during study participation.
- Known hypersensitivity to pegtibatinase or a history of severe hypersensitivity to a PEG-containing product.
- Active HIV, hepatitis B, or hepatitis C infection.
- History of organ transplantation or immunosuppressive therapy.
- Clinically significant medical conditions that could interfere with study participation or participant safety.
- Pregnant or breastfeeding, or planning to become pregnant during study participation.
- Major surgery planned during the study period.
- Any condition that could prevent the participant from complying with study procedures or completing the study.
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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Study locations (12)
5 openAnn & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Not yet recruitingThe Mount Sinai Hospital
New York, New York, United States
Not yet recruitingScience 37 - Virtual Site
Morrisville, North Carolina, United States
RecruitingTravere Investigational Site
Aurora, Colorado, United States
CompletedTravere Investigational Site
Miami, Florida, United States
CompletedTravere Investigational Site
Indianapolis, Indiana, United States
CompletedTravere Investigational Site
Portland, Maine, United States
CompletedTravere Investigational Site
Boston, Massachusetts, United States
CompletedTravere Investigational Site
New York, New York, United States
CompletedTravere Investigational Site
Philadelphia, Pennsylvania, United States
Completed
2 sites outside the US
Hospital Necker-Enfants Malades, Neurologie Pediatrique
Paris, France
Not yet recruitingSidra Medicine
Doha, Qatar
Not yet recruiting
Study team
Sponsor
Travere Therapeutics, Inc.
Study ID CBS-HCY-CT-01
Study director
Michael Imperiale, MD
Travere Therapeutics, Inc.
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.