Osteopetrosis – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Thu, 28 May 2026 11:29:33 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Osteopetrosis – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study of gene-modified stem cells (FT024) in children with TCIRG1-related osteopetrosis using treosulfan, thiotepa, and plerixafor for treatment https://clinicaltrials.eu/trial/study-of-gene-modified-stem-cells-ft024-in-children-with-tcirg1-related-osteopetrosis-using-treosulfan-thiotepa-and-plerixafor-for-treatment/ Wed, 29 Apr 2026 15:06:55 +0000 https://clinicaltrials.eu/trial/study-of-gene-modified-stem-cells-ft024-in-children-with-tcirg1-related-osteopetrosis-using-treosulfan-thiotepa-and-plerixafor-for-treatment/ This clinical trial is studying a treatment for children with autosomal recessive osteopetrosis, a rare genetic bone disease caused by mutations in the TCIRG1 gene. The condition causes bones to become unusually dense and prone to breaks. The study aims to evaluate a new treatment that uses modified stem cells from the patient’s own body. The treatment involves collecting blood-forming stem cells from the patient, modifying them in a laboratory to correct the genetic defect, and then returning them to the patient through an infusion.

The main treatment being tested is called FT024, which consists of specially processed stem cells that have been modified using a lentiviral vector (a tool that delivers genetic material into cells). Several other medications will be used as part of the treatment process, including Trecondi (treosulfan), Tepadina (thiotepa), and Mozobil (plerixafor). These medications help prepare the body for the stem cell treatment.

The study will monitor patients for two years after treatment to check if the modified stem cells successfully integrate into the body and improve the condition. Doctors will track various health measures, including blood cell counts, need for blood transfusions, bone health, and overall survival. The treatment’s safety will be closely monitored throughout the study period.

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