FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector
NCT ID: NCT03351868
Last Updated: 2019-09-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
NA
10 participants
INTERVENTIONAL
2017-12-01
2021-12-31
Brief Summary
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Detailed Description
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The primary objectives are to evaluate the safety of the self-inactivating lentiviral vector, the ex vivo gene transfer clinical protocol and the efficacy of immune reconstitution in patients overcoming immune abnormalities present at the time of treatment, assessment of gene correction efficiency, and finally the long-term correction of Fanconi anemia associated disease symptoms.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Gene-modified autologous stem cells
Autologous hematopoeitic stem cells and mesenchymal stem cells transduced with lentiviral vector carrying the FANCA gene ex vivo
Gene-modified autologous stem cells
Infusion for 5x10\^6\~1x10\^7 per kilogram of body weight of gene-modified cells; or more infusions depending on the circumstances
Interventions
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Gene-modified autologous stem cells
Infusion for 5x10\^6\~1x10\^7 per kilogram of body weight of gene-modified cells; or more infusions depending on the circumstances
Eligibility Criteria
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Inclusion Criteria
2. No cytogenetic abnormalities and the proportion of myelodysplastic abnormalities does not exceed 5% within 3 months prior to stem cell collection.
3. Age: ≥ 4 years.
4. Karnofsky: ≥ 70%.
5. ANC ≥ 5×10\^8/L; PLT ≥ 2×10\^10/L.
6. Hemoglobin ≥ 8g/dL.
7. 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.
8. Pulmonary function is normal; DLCO \> 50%.
9. Written, informed consent obtained prior to any study-specific procedures.
Exclusion Criteria
2. Diagnosis of myeloid leukemia.
3. Pregnant or lactating females.
4. Existence of an available HLA-identical related donor.
5. Subject infected with HBV (HBsAg positive), HIV (HIV antibody positive), HTLV (HTLV antibody positive), Treponema pallidum antibody positive or TB culture positive.
6. Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.
2 Years
20 Years
ALL
No
Sponsors
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Shenzhen Geno-Immune Medical Institute
OTHER
Responsible Party
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Lung-Ji Chang
President
Principal Investigators
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Lung-Ji Chang, Ph.D
Role: PRINCIPAL_INVESTIGATOR
Shenzhen Geno-Immune Medical Institute
Xiao-Dong Shi, M.D./Ph. D
Role: STUDY_DIRECTOR
Capital Institute of Pediatrics affiliated Children's hospital
Jie Zheng, M.D./Ph. D
Role: STUDY_DIRECTOR
Beijing Children's Hospital
Locations
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Capital Institute of Pediatrics affiliated Children's hospital
Beijing, Beijing Municipality, China
Beijing Children's Hospital
Beijing, Beijing Municipality, China
Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, China
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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GIMI-IRB-17021
Identifier Type: -
Identifier Source: org_study_id
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