CI

At a glance

ClinicalIndex Comparison Record
N/ARecruiting· 10 target
Drug / intervention
Gene-modified autologous stem cellsgenetic
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

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NCT03351868N/ARecruitingMonitor
Slow Enrollment
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Gene Transfer for Fanconi Anemia Using a Self-inactivating Lentiviral Vector

Shenzhen Geno-Immune Medical Institute·interventional·Posted Nov 24, 2017·Updated Jun 23, 2026

In Brief

A clinical study evaluating Gene-modified autologous stem cells for Fanconi Anemia. Currently recruiting, targeting 10 participants across 1 site.

Signals

Enrolling slower than its timeline implies

Detailed Summary

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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsFanconi Anemia
CountriesChina
Collaborators--

Timeline

N/ARecruiting
20182019202020212022202320242025202620272028202920302031
First PostedNov 24, 2017
Enrollment StartJun 1, 2026
Primary CompletionDec 31, 2029
Study CompletionDec 31, 2030
TodayJul 1, 2026
Enrollment to primary: 3.6 yearsPosted 8.6 years agoPrimary completion in 3.5 years

Arms & Interventions

Gene-modified autologous stem cellsexperimental

Autologous hematopoeitic stem cells and mesenchymal stem cells transduced with lentiviral vector carrying the FANCA gene ex vivo

Genetic: Gene-modified autologous stem cells

Interventions

Gene-modified autologous stem cellsgenetic

Infusion for 5x10\^6\~1x10\^7 per kilogram of body weight of gene-modified cells; or more infusions depending on the circumstances