Clinical Trials for Inborn Errors of Metabolism

Find clinical trials for inborn errors of metabolism. Discover research opportunities and treatment options in your area.

7 Active Trials Found

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Clinical and Laboratory Study of Methylmalonic Acidemia

NCT ID: NCT00078078

This clinical trial is studying a condition called methylmalonic acidemia (MMA), which affects how the body processes certain fats and proteins. People with MMA may experience serious health proble...

Recruiting

Type: observational Locations: Bethesda, Maryland, United States, Bethesda, Maryland, United States +3 more

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

NCT ID: NCT06839456

This clinical trial is studying a new approach to help prevent serious infections in patients with certain types of blood cancers, such as leukemia and lymphoma, after they receive a stem cell tran...

Recruiting

Phase: PHASE1 Type: interventional Locations: Philadelphia, Pennsylvania, United States

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

NCT ID: NCT02356653

This clinical trial aims to help patients with serious blood disorders like leukemia and certain genetic conditions who need a special type of transplant called allogeneic hematopoietic stem cell t...

Recruiting

Phase: EARLY_PHASE1 Type: interventional Locations: Philadelphia, Pennsylvania, United States

Growth and Development of Children With Inborn Errors of Metabolism in Assiut Governorate

NCT ID: NCT06654765

This clinical trial is looking at how children with inborn errors of metabolism (IEM) grow and develop in the Assiut Governorate. Inborn errors of metabolism are conditions where the body has troub...

Not yet recruiting

Type: observational Locations: Assiut, , Egypt

Institutional Registry of Rare Diseases

NCT ID: NCT06573723

This clinical trial, called the Institutional Registry of Rare Diseases, aims to gather information about various rare diseases, including conditions like amyloidosis, sarcoidosis, and Wilson disea...

Recruiting

Type: observational Locations: Buenos Aires, , Argentina

Targeted Interventions for Successful Transition and Transfer of Adolescents With Inborn Errors of Metabolism to Adult Services

NCT ID: NCT05413278

This clinical trial is focused on helping teenagers with inborn errors of metabolism transition smoothly from pediatric to adult healthcare services. The researchers aim to understand how well thes...

Recruiting

Phase: NA Type: interventional Locations: Basel, , Switzerland, Zürich, , Switzerland +6 more

Blood Spot and Urine Metabolomic Screening Applied to Rare Diseases

NCT ID: NCT06360913

This clinical trial is focused on understanding rare diseases, particularly those involving metabolism, which are often caused by genetic factors. The main goal is to collect samples of dried blood...

Recruiting

Phase: NA Type: interventional Locations: Charleroi, , Belgium, Brussels, , Belgium +2 more

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