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Tests treatment safety and results for Fanconi Anemia
Official title FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector
ClinicalTrials.gov ID: NCT03351868
What this study is testing
What is Gene-modified autologous stem cells?
Gene-modified autologous stem cells is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for fanconi anemia.
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 Phase I/II clinical trial of gene therapy for treating Fanconi anemia using a self-inactivating lentiviral vector to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 20
You may be able to join if
- Diagnosis of Fanconi anemia FANCA type based on DNA sequencing and sensitivity test for chromosomal cleavage by mitomycin C or butylene oxide.
- No cytogenetic abnormalities and the proportion of myelodysplastic abnormalities does not exceed 5% within 3 months prior to stem cell collection.
- Age: ≥ 4 years.
- Karnofsky: ≥ 70%.
- ANC ≥ 5×10\^8/L; PLT ≥ 2×10\^10/L.
You likely can't join if
- Diagnosis of active malignant disease or myelodysplastic syndrome.
- Diagnosis of myeloid leukemia.
- Pregnant or lactating females.
- Existence of an available HLA-identical related donor.
- Subject infected with HBV (HBsAg positive), HIV (HIV antibody positive), HTLV (HTLV antibody positive), Treponema pallidum antibody positive or TB...
- Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.
See the full eligibility criteria
- Diagnosis of Fanconi anemia FANCA type based on DNA sequencing and sensitivity test for chromosomal cleavage by mitomycin C or butylene oxide.
- No cytogenetic abnormalities and the proportion of myelodysplastic abnormalities does not exceed 5% within 3 months prior to stem cell collection.
- Age: ≥ 4 years.
- Karnofsky: ≥ 70%.
- ANC ≥ 5×10\^8/L; PLT ≥ 2×10\^10/L.
- Hemoglobin ≥ 8g/dL.
- Proper renal and hepatic functions (ULN denotes "upper limit of normal range") with
- serum creatinine ≤ 1.5×ULN;
- serum bilirubin ≤ 3×ULN;
- AST/ALT ≤ 5×ULN.
- Pulmonary function is normal; DLCO \> 50%.
- Written, informed consent obtained prior to any study-specific procedures.
- Diagnosis of active malignant disease or myelodysplastic syndrome.
- Diagnosis of myeloid leukemia.
- Pregnant or lactating females.
- Existence of an available HLA-identical related donor.
- Subject infected with HBV (HBsAg positive), HIV (HIV antibody positive), HTLV (HTLV antibody positive), Treponema pallidum antibody positive or TB culture positive.
- Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.
The study team makes the final eligibility decision.
Where it's taking place
- Shenzhen, Guangdong, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
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 listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 20 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shenzhen, Guangdong, China. 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.