# CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease

> **NCT04091737** · PHASE1 · TERMINATED · sponsor: **CSL Behring** · enrollment: 1 (actual)

## Conditions studied

- Anemia, Sickle Cell

## Interventions

- **BIOLOGICAL:** Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734

## Key facts

- **NCT ID:** NCT04091737
- **Lead sponsor:** CSL Behring
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2019-10-02
- **Primary completion:** 2021-05-05
- **Final completion:** 2021-05-05
- **Target enrollment:** 1 (ACTUAL)
- **Why stopped:** Unanticipated delays, not for safety reasons
- **Last updated:** 2021-06-18


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04091737

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04091737, "CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT04091737. Licensed CC0.

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