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New treatment option for Osteopetrosis
Official title Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO)
ClinicalTrials.gov ID: NCT07665021
What this study is testing
What is FT024?
FT024 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for osteopetrosis.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a non-randomized, one-arm, open label, single-center, phase I/II, prospective study, to assess safety, feasibility and efficacy of FT024 in 8 children (Age: ≥ 28 days and ≤ 2 years old, Body weight: ≥ 4 kg) affected by ARO-1. Once written informed consent has been obtained, and subsequently screening procedures have been completed, harvesting of HSPCs will occur.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 30 months
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 28 to 2
You may be able to join if
- Diagnosis of autosomal recessive osteopetrosis caused by mutations in the TCIRG1 gene, defined by one of the following:
- Clinical features of osteopetrosis and documented pathogenic/likely pathogenic biallelic variants (homozygosity or compound heterozygosity, whereby...
- If a patient presents with clinical features suggestive of severe osteopetrosis (i.e. generalized osteosclerosis, club-shaped long bones, skull base...
- Patient's parents/legal guardians' capacity to understand the study goals, the study requirements (i.e., attending study visits, completing...
- Age: ≥ 28 days and ≤ 2 years old.
You likely can't join if
- Availability of a medically appropriate, logistically feasible, fully HLA-matched (10/10) sibling or unrelated donor. The chances of finding a...
- History of uncontrolled seizures or severe psychiatric symptoms.
- Clinically relevant active viral, bacterial or fungal infection.
- Positivity for HIV (serology or RNA), and/or HbsAg and/or HBV DNA and/or HCV RNA and/or active infection for Treponema Pallidum or Mycoplasma.
- Known hypersensitivity to the drugs required for conditioning chemotherapy, or any excipients used in these products.
- Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if long-acting agents).
See the full eligibility criteria
- Diagnosis of autosomal recessive osteopetrosis caused by mutations in the TCIRG1 gene, defined by one of the following:
- Clinical features of osteopetrosis and documented pathogenic/likely pathogenic biallelic variants (homozygosity or compound heterozygosity, whereby at least 1 allele must contain a known pathogenic mutation) in the...
- If a patient presents with clinical features suggestive of severe osteopetrosis (i.e. generalized osteosclerosis, club-shaped long bones, skull base sclerosis, recurrent fractures and osteomyelitis, cranial nerve...
- Patient's parents/legal guardians' capacity to understand the study goals, the study requirements (i.e., attending study visits, completing questionnaires, taking study medications), potential risks associated with...
- Age: ≥ 28 days and ≤ 2 years old.
- Body weight: ≥ 4 kg.
- Adequate cardiac, pulmonary, renal and hepatic function as evidenced by:
- Left ventricular ejection fraction (LVEF) ≥45% by echo and normal electrocardiogram (ECG) or presence of abnormalities not significant for cardiac disease. Absence of clinically significant heart valve disease.
- Pulse oximetry ≥90% in room air and no evidence for parenchymal lung disease on chest X-ray or CT scan.
- serum creatinine \<1.5x upper limit normal in the absence of any form of renal replacement therapy.
- Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) ≤2.5 x and total bilirubin ≤1.5x upper limit of normal (ULN). Normal coagulation tests (INR \<1.5).
- Availability of a medically appropriate, logistically feasible, fully HLA-matched (10/10) sibling or unrelated donor. The chances of finding a suitable, fully matched unrelated donor should be estimated through a...
- History of uncontrolled seizures or severe psychiatric symptoms.
- Clinically relevant active viral, bacterial or fungal infection.
- Positivity for HIV (serology or RNA), and/or HbsAg and/or HBV DNA and/or HCV RNA and/or active infection for Treponema Pallidum or Mycoplasma.
- Known hypersensitivity to the drugs required for conditioning chemotherapy, or any excipients used in these products.
- Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if long-acting agents).
- Previous allogeneic HSCT or gene therapy with a different product.
- Patients affected by neoplasia, a familial predisposition to hematologic malignancies or any hematologic/cytogenetic alterations that may suggest a high risk of developing hematologic malignancies.
- Patients with end-organ damage or any other severe condition which, in the judgment of the investigator, would make the patient inappropriate for either HSPC collection or autologous transplant.
The study team makes the final eligibility decision.
Where it's taking place
- Milan, Italy, Italy
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 30 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 28 days to 2 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Milan, Italy, Italy. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.