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
- Conditions
- Homocystinuria
- Registration Number
- NCT06247085
- Lead Sponsor
- Travere Therapeutics, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 70
Inclusion Criteria:<br><br> - Must be =12 to =65 years of age, at the time of signing the informed consent<br><br> - Must have a diagnosis of classical HCU based on clinical, biochemical, and/or<br> molecular genetic testing<br><br> - Plasma tHcy =80 µM at Screening visit, with allowance for up to 18 participants who<br> may be enrolled with a Screening plasma tHcy =50 to <80 µM<br><br> - Willing to maintain a generally stable diet for the duration of the study (unless<br> changes are required based on medical/safety reasons)<br><br> - Willing to maintain generally stable intake and doses of betaine, pyridoxine, and<br> Met free formula for the duration of the study (unless changes are required based on<br> medical/safety reasons)<br><br>Exclusion Criteria:<br><br> - Diagnosis of Marfan syndrome, methylenetetrahydrofolate reductase (MTHFR)<br> deficiency, or disorder of cobalamin metabolism<br><br> - Concurrent disease or condition (eg, history or presence of clinically significant<br> cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine,<br> immunologic, dermatologic, neurological, oncologic, or psychiatric disease) that<br> would interfere with study participation or safety (excluding complications of HCU).<br><br> - History of major thrombotic event (eg, cerebrovascular accident, myocardial<br> infarction, pulmonary embolism) in the previous 6 months.<br><br> - Body weight =160 kg.<br><br> - Use or planned use of any injectable drugs containing PEG (excluding PEG-containing<br> vaccines)<br><br> - Any previous exposure to pegtibatinase and/or previous participation in a clinical<br> study that included administration of pegtibatinase or pegtarviliase<br><br> - Prior severe immune reaction to a PEG-containing product
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change from baseline in plasma tHcy levels
- Secondary Outcome Measures
Name Time Method Plasma tHcy levels