# Gene Therapy for Transfusion Dependent Beta-thalassemia

> **NCT02453477** · PHASE1,PHASE2 · UNKNOWN · sponsor: **IRCCS San Raffaele** · enrollment: 10 (actual)

## Conditions studied

- Beta-Thalassemia

## Interventions

- **GENETIC:** Autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene

## Key facts

- **NCT ID:** NCT02453477
- **Lead sponsor:** IRCCS San Raffaele
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** UNKNOWN
- **Start date:** 2015-05
- **Primary completion:** 2019-08
- **Final completion:** 2019-08
- **Target enrollment:** 10 (ACTUAL)
- **Last updated:** 2019-06-28

## Collaborators

- [object Object]
- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT02453477

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT02453477, "Gene Therapy for Transfusion Dependent Beta-thalassemia". Retrieved via AI Analytics 2026-06-15 from https://api.ai-analytics.org/clinical/NCT02453477. Licensed CC0.

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