Hope for rare overgrowth disease: first treatment trial launches

NCT ID NCT04316546

Recruiting now Disease control Sponsor: National Human Genome Research Institute (NHGRI) Source: ClinicalTrials.gov ↗

Summary

This study is testing whether a drug called miransertib can safely slow or stop the abnormal tissue overgrowth caused by Proteus syndrome, a rare genetic condition with no current treatments. Children and adults aged 3 and older with a confirmed diagnosis will take a daily pill for up to four years while researchers monitor changes in their condition, pain levels, and quality of life. The main goal is to see if the drug can prevent the growth of specific skin lesions on the feet.

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

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Study contacts

Contact

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

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

Locations

National Institutes of Health Clinical Center
RECRUITING

Bethesda, Maryland, 20892, United States

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

Conditions

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PROTEUS SYNDROME