New study aims to track rare childhood disease for future treatments

NCT ID NCT07432880

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

methylmalonic acidemia methylmalonic aciduria due to methylmalonyl-CoA mutase deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

More trials for these conditions

Other studies related to the condition(s) this trial covers.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • CHOP (Children's hospital of Philadelphia)

    Philadelphia, Pennsylvania, 19104, United States

    Contact Email: •••••@•••••

    Contact

  • GOSH NHS (Great Ormond Street Hospital for Children)

    London, WC1N 3JH, United Kingdom

    Contact Email: •••••@•••••

    Contact

  • Hospital Universitario 12 de Octubre

    Madrid, 28041, Spain

    Contact Email: •••••@•••••

    Contact

  • OBGP (Bambino Gesu Ospedale Pediatrico)

    Roma, 00146, Italy

    Contact Email: •••••@•••••

    Contact

  • OSR_San Raffaele

    Milan, 20132, Italy

    Contact Email: •••••@•••••

    Contact

  • Royal Manchester Children Hospital

    Manchester, M13 9WL, United Kingdom

    Contact Email: •••••@•••••

    Contact

  • SJD_San Joan de Deù Children's Hospital

    Barcelona, 08950, Spain

    Contact Email: •••••@•••••

    Contact

  • UPMC (Children's hospital of Pittsburgh)

    Pittsburgh, Pennsylvania, 15224, United States

    Contact Email: •••••@•••••

    Contact