New hope for babies with rare calcification disease: first drug trial launches
NCT ID NCT05734196
First seen Jun 27, 2026 · Last updated Aug 11, 2026 · Updated 2 times
Summary
This study tests a new drug called INZ-701 in up to 16 infants (up to 1 year old) with rare genetic conditions (ENPP1 or ABCC6 deficiency) that cause dangerous calcium buildup in arteries and other problems. The main goal is to check the drug's safety and how the body handles it, while also looking at heart function and blood markers. This is an early-stage (Phase 1) trial, so it focuses on safety first, with potential for future treatments.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Hospital Sant Joan de Déu
Barcelona, Spain
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Rady Children's Hospital
San Diego, California, 92123, United States
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Royal Manchester Children's Hospital
Manchester, M13 9WL, United Kingdom
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can medical records unlock the secrets of rare calcification diseases?
- Rare disease detectives: new study maps how two genetic conditions unfold in children
- New care pathways could improve life for rare disease patients
- New hope for babies: drug targets rare genetic disorder that hardens arteries
- New study aims to unlock secrets of rare calcification disease
- New registry aims to unlock secrets of rare genetic diseases