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All 5 Methylmalonic Acidemia trials and their US sites
Enter your ZIP code above to sort them by how far their nearest open site is from you.
- An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and PediatricsMost sites in District of Columbia, Florida and IllinoisRecruitingRECORDATI GROUP5US sites
5 states - An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705Only in CaliforniaRecruitingPhase 1/2Ages 1+ModernaTX3US sites
- A Prospective Study of Pediatric Participants up to 16 Years of Age With Methylmalonic Acidemia (MMA) Due to Mutations in the MMUT GeneOnly in PennsylvaniaRecruitingUp to age 16Genespire Srl2US sites
- Clinical and Laboratory Study of Methylmalonic AcidemiaMost sites in Maryland and PennsylvaniaRecruitingAges 1 Month-115National Human Genome Research Institute (NHGRI)2US sites
2 states - Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods ApproachRecruitingAges 6 Months-99RECORDATI GROUPNo US site yet
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Who runs the Methylmalonic Acidemia trials in the US, and what stage they are in
None of the 4 Methylmalonic Acidemia trials with a US site 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. The remaining 3 have no drug phase, such as studies that follow people over time or test a device or a way of giving care. Companies run 3 of them, and a university, hospital, foundation or government group runs the other. The most recently listed one first appeared on ClinicalTrials.gov on February 25, 2026.
Methylmalonic Acidemia trials in the US, state by state
All 4 Methylmalonic Acidemia trials with a US site accept children under 18. Their open sites are in 7 states and Washington, DC, most often Pennsylvania, California and District of Columbia. A trial counts once in each state where it has an open site, and some trials accept both children and adults.
US hospitals running the most Methylmalonic Acidemia 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 Methylmalonic Acidemia.
- Altman Clinical and Translational Research InstitutionSan Diego, California1 trial
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, Illinois1 trial
- Children's National HospitalWashington D.C., District of Columbia1 trial
- CHOP (Children's hospital of Philadelphia)Philadelphia, Pennsylvania1 trial
- Icahn School of Medicine at Mt. SinaiNew York, New York1 trial
- Lucile Packard Children's Hospital at StanfordPalo Alto, California1 trial
- National Institutes of Health Clinical CenterBethesda, Maryland1 trial
- Riley Children's HospitalIndianapolis, Indiana1 trial
- UCLA Medical CenterLos Angeles, California1 trial
- University of South FloridaTampa, Florida1 trial
Help getting to a Methylmalonic Acidemia 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 Methylmalonic Acidemia trial near you
Are there Methylmalonic Acidemia clinical trials near me?
As of November 13, 2026, 5 Methylmalonic Acidemia clinical trials are recruiting now and 1 is not yet recruiting. In the US, 4 of them have at least 1 open site, across 7 states and Washington, DC. 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 Methylmalonic Acidemia clinical trial sites?
Recruiting Methylmalonic Acidemia trials list open sites in California, District of Columbia, Florida, Illinois, Indiana, Maryland, New York and Pennsylvania.
Are there Methylmalonic Acidemia trials for children near me?
All 4 Methylmalonic Acidemia trials with a US site accept children under 18. Their open sites are in 7 states and Washington, DC, most often Pennsylvania, California and District of Columbia. 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 Methylmalonic Acidemia 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 Methylmalonic Acidemia 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 Methylmalonic Acidemia 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 Methylmalonic Acidemia: Carbaglu (carglumic acid).
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.
