Skip to content
Pharma Observatory a Scientist.com platform

Clinical trial

A Phase I/II Open Label Study to Assess Safety, Feasibility and Efficacy of ex Vivo Expanded, Autologous Haematopoietic Stem and Progenitor Cell Populations That Contain CD34+ Cells Transduced With a Lentiviral Vector Encoding the TCIRG1 cDNA in Children With Autosomal Recessive Osteopetrosis Caused by Mutations in the TCIRG1 Gene.

NCT07665021 is registered in ClinicalTrials.gov with Fondazione Telethon as lead sponsor.

NCT NCT07665021
Email me this page as a PDF

Search trials, sites, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.

Get the NCT07665021 report as a PDF

The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

Registry record

As filed in ClinicalTrials.gov.

Title
A Phase I/II Open Label Study to Assess Safety, Feasibility and Efficacy of ex Vivo Expanded, Autologous Haematopoietic Stem and Progenitor Cell Populations That Contain CD34+ Cells Transduced With a Lentiviral Vector Encoding the TCIRG1 cDNA in Children With Autosomal Recessive Osteopetrosis Caused by Mutations in the TCIRG1 Gene.
Lead sponsor
Fondazione Telethon
Phase
Phase 1/2
Overall status
Recruiting
Start date
June 20, 2026
Completion date
June 30, 2031
Enrollment
8 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

osteopetrosis