Last reviewed · How we verify
NCT04091737
CSL200 Gene Therapy in Adults With Severe Sickle Cell Disease
Phase 1 trial testing Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734 in Anemia, Sickle Cell in 1 participant. Terminated before completion.
5 May 2021
Quick facts
| Lead sponsor | CSL Behring |
|---|---|
| Phase | Phase 1 |
| Status | Terminated |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 1 |
| Start date | 2 October 2019 |
| Primary completion | 5 May 2021 |
| Estimated completion | 5 May 2021 |
| Sites | 1 location across United States |
Drugs / interventions tested
- Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
Conditions studied
- Anemia, Sickle Cell — all drugs for Anemia, Sickle Cell →
Sponsor
CSL Behring — full company profile →
Who can join
Adults 18 to 45, any sex, with Anemia, Sickle Cell. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
This is a phase 1 pilot study of CSL200 in adult subjects with severe sickle cell disease. The primary objectives of this study are to evaluate the safety of the following: collection of CD34+ hematopoietic stem / progenitor cells by apheresis after mobilization with plerixafor, reduced intensity conditioning with melphalan, and administration of CSL200.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
-
Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases.
Tucci F, Scaramuzza S, Aiuti A, Mortellaro A. · · 2021 · cited 72× · PMID 33221437 · DOI 10.1016/j.ymthe.2020.11.020 -
Gene therapy for sickle cell disease: moving from the bench to the bedside.
Abraham AA, Tisdale JF. · · 2021 · cited 66× · PMID 34232993 · DOI 10.1182/blood.2019003776 -
Large-Scale Production of Lentiviral Vectors: Current Perspectives and Challenges.
Martínez-Molina E, Chocarro-Wrona C, Martínez-Moreno D, Marchal JA, et al · · 2020 · cited 55× · PMID 33153183 · DOI 10.3390/pharmaceutics12111051 -
Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease.
Germino-Watnick P, Hinds M, Le A, Chu R, et al · · 2022 · cited 33× · PMID 35681538 · DOI 10.3390/cells11111843 -
Research in Sickle Cell Disease: From Bedside to Bench to Bedside.
Salinas Cisneros G, Thein SL. · · 2021 · cited 19× · PMID 34095767 · DOI 10.1097/hs9.0000000000000584 -
A Small Key for a Heavy Door: Genetic Therapies for the Treatment of Hemoglobinopathies.
Zittersteijn HA, Harteveld CL, Klaver-Flores S, Lankester AC, et al · · 2020 · cited 14× · PMID 34713239 · DOI 10.3389/fgeed.2020.617780 -
The EHA Research Roadmap: Hematopoietic Stem Cell Gene Therapy.
Naldini L, Cicalese MP, Bernardo ME, Gentner B, et al · · 2022 · cited 10× · PMID 35198856 · DOI 10.1097/hs9.0000000000000671 -
Current and future treatments for sickle cell disease: From hematopoietic stem cell transplantation to in vivo gene therapy.
Ball J, Bradley A, Le A, Tisdale JF, et al · · 2025 · cited 5× · PMID 40083162 · DOI 10.1016/j.ymthe.2025.03.016
Verify or expand the search:
- PubMed search for NCT04091737
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Anemia, Sickle Cell
Currently open trials in the same condition.
- NCT06144749 — A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects Wit · Phase 1, PHASE2 · recruiting
- NCT06290401 — A Socio-ecological Approach for Improving Self-management in Adolescents With SCD · Phase 2 · recruiting
- NCT06481306 — A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease · Phase 1, PHASE2 · recruiting
- NCT05087303 — Telemedicine for Children With Sickle Cell Disease · NA · active not recruiting
- NCT04406818 — Metabolic and Hemodynamic Reserve in Pediatric SCA · NA · recruiting
Other CSL Behring trials
Trials by the same sponsor.
- NCT07326592 — Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza · Phase 4 · not yet recruiting
- NCT07224360 — Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD) · Phase 2 · recruiting
- NCT07332091 — Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary He · Phase 2 · recruiting
- NCT07094087 — Efficacy and Safety of 4F-PCC (4-Factor Prothrombin Complex Concentrate) in Adult Patients Undergoing Complex Cardiovasc · Phase 3 · recruiting
- NCT07048262 — Dose Range Finding, Efficacy, and Safety Study of Nebulized CSL787 in Adults With Non-cystic Fibrosis Bronchiectasis (NC · Phase 2 · recruiting
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT04091737 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by CSL Behring
- Last refreshed: 18 June 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04091737.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing