Actively Recruiting
Phase 3 Study of Pegtibatinase Injection Versus Placebo for Classical Homocystinuria in Patients Aged 12 to 65 Years Receiving Standard Care
Led by Travere Therapeutics, Inc. · Updated on 2026-08-07
70
Participants Needed
52
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the efficacy and safety of pegtibatinase treatment compared with placebo in people aged 12 to 65 years who have classical homocystinuria HCU due to cystathionine beta synthase deficiency. This phase 3, randomized, blinded, placebo-controlled study includes participants who continue to have elevated total homocysteine tHcy levels despite receiving standard care. The goal is to better understand the impact of pegtibatinase on reducing tHcy levels in this population. Participants will be randomly assigned to receive either pegtibatinase or a placebo administered subcutaneously twice weekly during a 24-week blinded treatment period. Before treatment, participants undergo a screening process lasting up to 10 weeks, including an initial screening and a pre-treatment diet standardization period of up to 6 weeks to stabilize protein intake and supplement use. After the treatment period, a 4-week safety follow-up is conducted for those not continuing in the long-term extension study. During the study, participants diet and compliance with HCU-related treatments are closely monitored using a specialized diet tool. Study visits, including some home visits for drug administration and possible remote visits, occur regularly. The primary measurement is the change in plasma tHcy levels from baseline during weeks 6 to 12, with secondary measurements extending to weeks 16 to 24. Safety and treatment adherence are continually assessed throughout the study duration, which can last up to 38 weeks in total.
CONDITIONS
Brief Title
A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Be between 12 and 65 years old at the time of informed consent
- Have a diagnosis of classical homocystinuria based on clinical, biochemical, or genetic testing
- Have plasma total homocysteine (tHcy) levels of 80 µM or higher at screening, with some allowance for levels between 50 and less than 80 µM
- If able to become pregnant, have a negative pregnancy test before starting and use highly effective birth control during the study and for 4 weeks after last dose
- Willing to maintain a generally stable diet throughout the study unless medically required to change
- Willing to keep stable intake and doses of betaine, pyridoxine, and medical food during the study unless medically required to change
You will not qualify if you...
- Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase deficiency, or cobalamin metabolism disorders
- Presence of significant concurrent diseases that may interfere with participation or safety (cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurological, oncologic, or psychiatric conditions), except HCU complications
- History of major thrombotic events like stroke, heart attack, or pulmonary embolism within the past 6 months
- Body weight of 160 kg or more
- Current or planned use of injectable drugs containing polyethylene glycol (PEG), excluding PEG-containing vaccines
- Previous use of pegtibatinase or participation in a study involving pegtibatinase or pegtarviliase
- Prior severe immune reaction to PEG-containing products
Research Team
T
Travere Call Center
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