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NCT06655662

Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.

Recruiting now Phase 1 Last updated 23 October 2024
What this trial tests

Phase 1 trial testing β-globin restored autologous hematopoietic stem cells in Β-thalassemia in 8 participants. Currently enrolling.

Timeline
12 June 2024
Primary endpoint
31 December 2025
31 December 2026

Quick facts

Lead sponsorShenzhen Hemogen
PhasePhase 1
StatusRecruiting now
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment8
Start date12 June 2024
Primary completion31 December 2025
Estimated completion31 December 2026
Sites3 locations across China

Drugs / interventions tested

Conditions studied

Sponsor

Shenzhen Hemogen — full company profile →

Who can join

Adults 6 to 35, any sex, with Β-thalassemia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is a single-arm, open label, multi-center, single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia. The study aims to evaluate the safety and efficacy of autologous lentiviral-transduced CD34+ human hematopoietic stem cells (hHSCs) using the gene therapy product HGI-001.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Clinical hematopoietic stem cell-based gene therapy.
    John T, Czechowicz A. · · 2025 · cited 6× · PMID 40285354 · DOI 10.1016/j.ymthe.2025.04.029
  2. 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
  3. Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche.
    Aprile A, Lidonnici MR, Ferrari G. · · 2025 · PMID 41253147 · DOI 10.1016/j.xcrm.2025.102419

Verify or expand the search:

Other trials of β-globin restored autologous hematopoietic stem cells

Trials testing the same drug.

Other Shenzhen Hemogen trials

Trials by the same sponsor.

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Data sources for this page

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/NCT06655662.

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