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NCT03964792: DREPAGLOBE
Safety and Efficacy of Gene Therapy of the Sickle Cell Disease by Transplantation of an Autologous CD34+ Enriched Cell Fraction That Contains CD34+ Cells Transduced ex Vivo With the GLOBE1 Lentiviral Vector Expressing the βAS3 Globin Gene in Patients With Sickle Cell Disease (DREPAGLOBE)
Phase 1, PHASE2 trial testing DREPAGLOBE drug product in Sickle Cell Disease in 6 participants. Completed in 23 January 2024.
28 July 2022
Quick facts
| Lead sponsor | Assistance Publique - Hôpitaux de Paris |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 6 |
| Start date | 12 November 2019 |
| Primary completion | 28 July 2022 |
| Estimated completion | 23 January 2024 |
| Sites | 1 location across France |
Drugs / interventions tested
- DREPAGLOBE drug product
Conditions studied
- Sickle Cell Disease — all drugs for Sickle Cell Disease →
Sponsor
Assistance Publique - Hôpitaux de Paris — full company profile →
Who can join
Adults 12 to 20, any sex, with Sickle Cell Disease. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The purpose of this study is to evaluate the Safety and Efficacy of Gene Therapy of the Sickle Cell disease by Transplantation of an Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced ex vivo with the GLOBE1 lentiviral vector expressing the βAS3 globin gene (GLOBE1 βAS3 Modified Autologous CD34+ Cells) in Patients with Sickle Cell Disease (SCD)
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
-
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
Lattanzi A, Camarena J, Lahiri P, Segal H, et al · · 2021 · cited 125× · PMID 34135108 · DOI 10.1126/scitranslmed.abf2444 -
Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission.
Piel FB, Rees DC, DeBaun MR, Nnodu O, et al · · 2023 · cited 100× · PMID 37451304 · DOI 10.1016/s2352-3026(23)00096-0 -
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 -
Autologous gene therapy for hemoglobinopathies: From bench to patient's bedside.
Locatelli F, Cavazzana M, Frangoul H, Fuente J, et al · · 2024 · cited 22× · PMID 38454604 · DOI 10.1016/j.ymthe.2024.03.005 -
Diverse Approaches to Gene Therapy of Sickle Cell Disease.
White SL, Hart K, Kohn DB. · · 2023 · cited 20× · PMID 36067800 · DOI 10.1146/annurev-med-042921-021707
Verify or expand the search:
- PubMed search for NCT03964792
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
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Other Assistance Publique - Hôpitaux de Paris trials
Trials by the same sponsor.
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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 NCT03964792 (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 Assistance Publique - Hôpitaux de Paris
- Last refreshed: 5 March 2026
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/NCT03964792.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing