Clinical trials
Inherited Metabolic Disorders clinical trials
Below are recruiting inherited metabolic disorders clinical trials, each written for real people, not researchers. We’re tracking 13 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06471842Recruiting
Eating issues in kids with metabolic diseases on special diets
This trial studies eating disorders in young children with certain inherited metabolic diseases that require a special diet. It aims to understand how often these eating problems happen and what they look like.
ParisAges 1–6 - NCT04414046RecruitingPhase 2
Stem cell transplant using T-cell cleanup for immune and metabolism disorders
This Phase 2 study tests a specialized stem cell transplant that removes certain “T-cells” (a type of immune cell) to treat children with serious immune system or inherited metabolic problems. The goal is to improve or stabilize disease by resetting the immune system and supporting the body’s function.
St. Petersburg, FloridaAges Up to 21 years - NCT01351545Recruiting
Cryopreserved cord blood access program for transplant patients
This trial studies an organized way to obtain and distribute unlicensed (not FDA-approved for general use) stored cord blood units that have been frozen and kept. It may help people who need a cord blood transplant but don’t fit the “licensed cord blood only” options.
Birmingham, AlabamaAges Any age - NCT04644016RecruitingPhase 2
Cord blood transplant for children and young adults with blood cancers
This Phase 2 trial studies using a cord blood transplant after finding it is risky enough that standard treatment may not be sufficient. It may help certain children and young adults with specific leukemia, lymphoma, and some non-cancer blood/immune or inherited disorders who do not have a well-matched donor available in time.
New York, New YorkAges Up to 21 years - NCT04880356Recruiting
Study of very rare inherited brain diseases over time
This study follows adults over time who have a very rare inherited brain disease, or whose illness seems like it may run in families. The goal is to better understand these conditions and how they progress.
Milan, MilanoAges 18 years+ - NCT01962415RecruitingPhase 2
Reduced-intensity stem cell transplant for non-cancer disorders
This trial tests whether a gentler (reduced-intensity) conditioning treatment before a stem cell transplant works for non-cancer diseases. It uses specific donor stem cell matches and checks heart, lung, liver, and kidney function to help lower transplant stress while still aiming for benefit.
Pittsburgh, PennsylvaniaAges 2 months–55 years - NCT04528355Recruiting
Study of hospital data after stem cell transplant for non-cancer illness
This study collects medical information from people with certain non-cancer conditions who receive a stem cell transplant. It may help doctors better understand transplant outcomes and care, since the study focuses on data collection rather than adding new treatment.
Pittsburgh, PennsylvaniaAges 2 months–60 years - NCT05007990Recruiting
Interview study of caregiving networks over a person’s life
This study uses interviews and questionnaires to better understand who makes up a caregiver’s support network when caring for someone with a long-term health condition. Your answers may help researchers understand how caregiving changes over time and across different health situations.
Bethesda, MarylandAges 18–100 - NCT04394871Recruiting
Study of ALS and related genetic nerve disorders using scans and fluid tests
This study looks at signs and biology (biomarkers) in people with certain inherited nerve conditions caused by RNA-processing gene changes. It may help researchers understand disease patterns and measure health changes using MRI scans and sometimes a spinal fluid test.
Bethesda, MarylandAges 5–120 - NCT02254863RecruitingPhase 1
Stem cell transplant into the spinal fluid for rare brain diseases
This Phase 1 trial tests a transplant using special donated stem-cell–like cells placed into the spinal fluid to treat certain inherited brain diseases. It is mainly designed to check safety, and it may offer benefit by targeting brain and nerve damage.
Durham, North CarolinaAges 1 week–22 years - NCT06839456RecruitingPhase 1/Phase 2
Stem cell addback to prevent infections after transplant
This trial tests whether adding back certain immune cells after a stem cell transplant can help prevent serious viral or fungal infections. It's for children and young adults up to age 25 who need a transplant for a blood cancer or other curable disease.
Philadelphia, PennsylvaniaAges 1 month–25 years - NCT06090669RecruitingPhase 1
Trial of imatinib for people with RUNX1 deficiency
This trial tests if imatinib can help people with a genetic condition called RUNX1 deficiency, which causes bleeding problems. It aims to increase the activity of the RUNX1 gene to improve blood clotting.
Bethesda, MarylandAges 18–120 - NCT01793168Recruiting
Rare disease registry and history study
This study keeps a registry (a structured list) of people with rare diseases and may also track how the condition changes over time. It can help researchers better understand rare illnesses and find gaps in care, which may improve future treatments.
Sioux Falls, South DakotaAges Any age
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Common questions
- Are there clinical trials for inherited metabolic disorders?
- Yes. Clin2 currently lists 13 recruiting inherited metabolic disorders 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 inherited metabolic disorders 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 inherited metabolic disorders 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.