New hope for kids with rare copper deficiency disease

NCT ID NCT04074512

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This program gives children under 6 with Menkes disease access to copper histidinate injections. Menkes disease is a rare genetic disorder where the body cannot properly use copper, leading to severe developmental problems. The treatment aims to supply the missing copper to help manage the condition.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

copper histidinate (CUTX-101)

What this could lead to

If this treatment works, it could help manage Menkes disease by providing the copper the body needs, potentially improving survival and development in young children.

What could go wrong

This is an expanded access program, not a controlled trial, so results are not definitive. The treatment may not work for all patients, and side effects from copper therapy are possible.

Disclaimer Read more

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.

Menkes disease

As listed by the trial registrant

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

Contacts and locations

Locations

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • Connecticut Children's Medical Center

    Hartford, Connecticut, 06106, United States

  • Cook Children's Health Care System

    Fort Worth, Texas, 76104, United States

  • Duke University Hospital

    Durham, North Carolina, 27710, United States

  • Emory University

    Atlanta, Georgia, 30322, United States

  • Helen DeVos Children's Hospital

    Grand Rapids, Michigan, 49503, United States

  • Johns Hopkins All Children's Hospital

    St. Petersburg, Florida, 33701, United States

  • Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Maine Medical Center

    Portland, Maine, 04102, United States

  • Memorial Healthcare Systems

    Hollywood, Florida, 33021, United States

  • Monroe Carrell Jr. Children's Hospital at Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

  • Oregon Health & Science University

    Portland, Oregon, 97239, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • Rady Children's Hospital

    San Diego, California, 92123, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Shodair Children's Hospital

    Helena, Montana, 59601, United States

  • University of California Davis Medical Center

    Sacramento, California, 95817, United States

  • University of Iowa Stead Family Children's Hospital

    Iowa City, Iowa, 52242, United States