Groundbreaking trial aims to treat rare diseases in the womb

NCT ID NCT04532047

Recruiting now Disease control Sponsor: University of California, San Francisco Source: ClinicalTrials.gov ↗

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

Summary

This study tests whether giving enzyme replacement therapy to fetuses with certain rare genetic diseases (like MPS I, Gaucher, or Pompe) before birth is safe and feasible. About 10 pregnant participants will receive the treatment through the umbilical vein. The goal is to see if early treatment can reduce disease effects, but lifelong management will still be needed after birth.

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Conditions

The condition(s) this trial relates to.

Gaucher disease Gaucher disease type II Gaucher disease type III glycogen storage disease due to acid maltase deficiency, infantile onset mucopolysaccharidosis I, dog mucopolysaccharidosis II, dog Mucopolysaccharidosis IV mucopolysaccharidosis type 4A mucopolysaccharidosis type 6 mucopolysaccharidosis type 7 Sudden Infant Death Wolman disease

As listed by the trial registrant

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

GAUCHER DISEASE, TYPE 2 GAUCHER DISEASE, TYPE 3 MPS I MPS II MPS IVA MPS VI MPS VII POMPE DISEASE INFANTILE-ONSET WOLMAN DISEASE

Contacts and locations

Locations

University of California
RECRUITING

San Francisco, California, 94158, United States

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