New study aims to track rare childhood disease for future treatments
NCT ID NCT07432880
First seen Jun 27, 2026 · Last updated Aug 17, 2026 · Updated 3 times
Summary
This study follows up to 30 children (up to age 16) with a rare genetic condition called methylmalonic acidemia (MMA), which affects how the body processes food. The goal is to observe how the disease progresses over time by tracking specific blood tests and health signs. This information will help researchers develop better treatments in the future. Some children in the study have had a liver transplant, and some have not.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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CHOP (Children's hospital of Philadelphia)
NOT_YET_RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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GOSH NHS (Great Ormond Street Hospital for Children)
RECRUITINGLondon, WC1N 3JH, United Kingdom
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Hospital Universitario 12 de Octubre
NOT_YET_RECRUITINGMadrid, 28041, Spain
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OBGP (Bambino Gesu Ospedale Pediatrico)
NOT_YET_RECRUITINGRoma, 00146, Italy
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OSR_San Raffaele
NOT_YET_RECRUITINGMilan, 20132, Italy
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SJD_San Joan de Deù Children's Hospital
NOT_YET_RECRUITINGBarcelona, 08950, Spain
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Saint Mary's Hospital
NOT_YET_RECRUITINGManchester, M13 9WL, United Kingdom
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UPMC (Children's hospital of Pittsburgh)
NOT_YET_RECRUITINGPittsburgh, Pennsylvania, 15224, United States
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