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Researchers seek to unlock mysteries of rare metabolic disease

NCT ID NCT00078078

First seen Nov 21, 2025 · Last updated Apr 30, 2026 · Updated 16 times

Summary

This study aims to learn more about methylmalonic acidemia (MMA), a rare genetic disorder that can cause serious health problems like kidney failure, strokes, and developmental delays. Researchers will observe up to 2275 participants over time using blood tests, imaging, and other exams to track how the disease progresses and identify new markers. The goal is to better understand the condition and pave the way for future treatments.

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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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Contacts and locations

Study contacts

  • Contact

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

  • Contact

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

Locations

  • Children's National Medical Center

    COMPLETED

    Washington D.C., District of Columbia, 20010, United States

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

  • UPMC Children's Hospital of Pittsburgh

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

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

Conditions

Explore the condition pages connected to this study.