Clinical trials
Homocystinuria clinical trials
Below are recruiting homocystinuria clinical trials, each written for real people, not researchers. We’re tracking 6 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT03406611RecruitingPhase 1/Phase 2
Enzyme replacement for classical homocystinuria in children
This study tests a new enzyme replacement therapy called pegtibatinase for children with classical homocystinuria (HCU). The enzyme helps break down homocysteine, which may lower its levels in the blood and reduce symptoms.
Aurora, ColoradoAges 5–65 - NCT06247085RecruitingPhase 3
A study testing pegtibatinase for classical homocystinuria
This trial tests an injectable drug called pegtibatinase for people with a rare condition called homocystinuria. It aims to lower high levels of homocysteine in the blood compared to a placebo, while you continue your usual treatments.
Phoenix, ArizonaAges 12–65 - NCT06556615Recruiting
Quality of life in classical homocystinuria
This study surveys how classical homocystinuria (CBS deficiency) affects daily life and well-being. It includes patients age 8 and up, parents of younger or cognitively impaired children, and medical experts.
ZurichAges 8 years+ - NCT02998710Recruiting
Natural history study for homocystinuria due to CBS deficiency
This study mainly observes what happens in people with homocystinuria caused by a CBS gene deficiency over time. It helps doctors better understand the condition’s natural course and may guide future treatments.
Culver City, CaliforniaAges 1–65 - NCT06431893Enrolling by invitationPhase 3
Long-term study of pegtibatinase for classical homocystinuria
This trial is a long-term follow-up study examining the safety and effectiveness of pegtibatinase for people with classical homocystinuria (HCU). It is open to those who have recently taken part in or finished the HARMONY or COMPOSE studies.
Phoenix, ArizonaAges 5–65 - NCT06545305Enrolling by invitation
Eye problems in homocystinuria patients, 2018–2022
This study looks at eye problems in people with homocystinuria. It will review medical records from 2018 to 2022 to find out how common eye issues are and what might increase the risk.
JeddahAges 20–30
Hear when a new Homocystinuria trial opens
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Common questions
- Are there clinical trials for homocystinuria?
- Yes. Clin2 currently lists 6 recruiting homocystinuria studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a homocystinuria trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a homocystinuria trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.