# ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID

> **NCT00598481** · PHASE2 · COMPLETED · sponsor: **Fondazione Telethon** · enrollment: 12 (actual)

## Conditions studied

- Immunologic Deficiency Syndromes

## Interventions

- **GENETIC:** Gene Therapy
- **DRUG:** Busulfan

## Key facts

- **NCT ID:** NCT00598481
- **Lead sponsor:** Fondazione Telethon
- **Sponsor class:** OTHER
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2002-10-02
- **Primary completion:** 2011-07-10
- **Final completion:** 2019-06-19
- **Target enrollment:** 12 (ACTUAL)
- **Last updated:** 2024-01-29


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT00598481

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT00598481, "ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID". Retrieved via AI Analytics 2026-06-22 from https://api.ai-analytics.org/clinical/NCT00598481. Licensed CC0.

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