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All 3 Homocystinuria trials and their US sites
Enter your ZIP code above to sort them by how far their nearest open site is from you.
- Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care TreatmentMost sites in Pennsylvania, Arizona and ConnecticutRecruitingPhase 3Ages 12-65Travere Therapeutics13US sites
12 states - Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)Most sites in Illinois, New York and North CarolinaRecruitingPhase 1/2Ages 5-65Travere Therapeutics3US sites
3 states - Health Related Quality of Life (HrQoL) in Classical Homocystinuria (CBS Deficiency)RecruitingAges 8+University Children's Hospital, ZurichNo US site yet
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Who runs the Homocystinuria trials in the US, and what stage they are in
Of the 2 Homocystinuria trials with a US site, 1 is in Phase 3, the last stage before a company can ask the FDA for approval. One more is an earlier Phase 1 or 2 test of safety and dosing. Companies run all of them, with none led by a university, hospital or government group. Travere Therapeutics runs the most, with 2. The most recently listed one first appeared on ClinicalTrials.gov on February 7, 2024.
Homocystinuria trials in the US, state by state
All 2 Homocystinuria trials with a US site accept children under 18. Their open sites are in 12 states, most often Illinois, New York and North Carolina. A trial counts once in each state where it has an open site, and some trials accept both children and adults.
Show the other 2 states
US hospitals running the most Homocystinuria trials
Centers that run trials for a rare condition often see more people with it than most clinics do, so this list can also help you find doctors who know Homocystinuria.
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, Illinois2 trials
- Science 37 - Virtual SiteMorrisville, North Carolina2 trials
- The Mount Sinai HospitalNew York, New York2 trials
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania1 trial
- Emory UniversityAtlanta, Georgia1 trial
- Maine Health - Maine Medical CenterPortland, Maine1 trial
- Oregon Health and Science UniversityPortland, Oregon1 trial
- Phoenix Children's HospitalPhoenix, Arizona1 trial
- Uncommon CuresChevy Chase, Maryland1 trial
- University of Pittsburgh Medical Center - Children's Hospital of PittsburghPittsburgh, Pennsylvania1 trial
Help getting to a Homocystinuria trial far from home
Ask the study team whether the trial pays back travel, parking and hotel costs, because many sponsors do. The FDA's guidance on paying research volunteers says paying back these costs does not count as pressuring someone to join.
These nonprofits also help patients reach care, and each one sets its own rules.
- Mercy Medical Angels
- Pays for gas cards, ground rides and airline tickets anywhere in the US, and it lists clinical trials as a reason to travel. Household income at or below 300% of the federal poverty guidelines, and usually a trip of 50 miles or more each way.
- Air Charity Network
- Volunteer pilots in all 50 states fly patients for free in small private planes, and members also fly people taking part in clinical trials. You must be able to walk and sit upright for the flight, show financial need and have a backup plan if weather cancels it.
- Ronald McDonald House
- Gives families a room near the hospital while a child gets care, through 394 House programs around the world. Families of a sick child, with rules set by each local chapter.
- Healthcare Hospitality Network
- Keeps a map of hospitality houses near hospitals, and most member houses charge little or nothing to patients and families. Each house sets its own rules, so check with the house near your study site.
Program details were checked on each program's website on October 24, 2026.
Questions about finding a Homocystinuria trial near you
Are there Homocystinuria clinical trials near me?
As of November 13, 2026, 3 Homocystinuria clinical trials are recruiting. In the US, 2 of them have at least 1 open site, across 12 states. Enter your ZIP code on this page to rank every one of them by the distance to its closest open site, with the sites shown on a map.
Which states have Homocystinuria clinical trial sites?
Recruiting Homocystinuria trials list open sites in Arizona, Connecticut, Georgia, Illinois, Maine, Maryland, New York, North Carolina, Oregon, Pennsylvania, Texas and Utah.
Are there Homocystinuria trials for children near me?
All 2 Homocystinuria trials with a US site accept children under 18. Their open sites are in 12 states, most often Illinois, New York and North Carolina. Each trial sets its own age limits, so check the ages listed on a trial before you contact it.
Who pays for travel to a Homocystinuria clinical trial?
Many trial sponsors pay back travel, parking and hotel costs, so ask the study team before you rule out a far-away site. The FDA's guidance says paying back these costs does not count as pressuring someone to join. Charities such as Mercy Medical Angels and Air Charity Network can also help with rides and flights.
What if there is no Homocystinuria trial near me?
Some trials send staff to your home or use a local lab, so fewer visits happen at the study site. You can also sign up on this page to get an email when a Homocystinuria trial opens a new site or changes status.
How is the distance to a trial site measured?
Distances are straight-line miles from the center of your ZIP code to each study site's listed location, not driving distance. Your browser looks up the ZIP code's coordinates with a free map service, Zippopotam.us, and the ZIP code is never sent to or stored by Trial Friend.
FDA-approved for Homocystinuria: Cystadane (betaine).
Clinical trials near you for related conditions
Source: ClinicalTrials.gov, trials with status Recruiting or Not yet recruiting. Refreshed every 6 hours; counts as of November 13, 2026. Sites that ClinicalTrials.gov lists as closed or withdrawn are left out. Children means the trial's age group includes people under 18.