Condition
Homocystinuria clinical trials and research
As of November 12, 2026, Find a Trial lists 3 Homocystinuria clinical trials that are recruiting, at sites in 15 countries (most in France (2), Qatar (2), United States (2)). By phase: Phase 3 (1), Phase 1/2 (1). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 3Clinical trials
- 15Countries
Newest Homocystinuria clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| Assessing Quality of Life in Patients with Classical Homocystinuria | — | Zurich | Recruiting | Aug 16, 2024 |
| Pegtibatinase versus placebo for people 12–65 with classical CBS‑deficient homocystinuria on standard care | Phase 3 | Phoenix, Arizona and 51 other locations | Recruiting | Feb 7, 2024 |
| Pegtibatinase for Classical Homocystinuria | Phase 1/2 | Aurora, Colorado and 11 other locations | Recruiting | Jan 23, 2018 |
All recruiting trials (3)
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Assessing Quality of Life in Patients with Classical Homocystinuria
This study is trying to understand how classical homocystinuria affects the daily lives of patients and their families by talking to them and creating a questionnaire to measure their quality of life.
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Pegtibatinase versus placebo for people 12–65 with classical CBS‑deficient homocystinuria on standard care
This trial tests whether pegtibatinase is safe and can lower homocysteine levels in people aged 12–65 with classical homocystinuria who are already receiving standard treatment.
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Pegtibatinase for Classical Homocystinuria
This study tests pegtibatinase to see if it is safe and tolerable for people with classical homocystinuria, including children aged 5 and up who have high levels of homocysteine.
Quick answers
How many Homocystinuria clinical trials are recruiting right now?
3, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 15 countries. The most sites are in France (2 trials), Qatar (2 trials), United States (2 trials), Switzerland (1 trial), Australia (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 3: 1, Phase 1/2: 1. Trials with no phase listed are usually device, behavioural or observational studies.
How do I join one?
Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.
Find the studies that fit you
A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.