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Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia

NCT07680803 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2026-07-27

No results posted yet for this study

Summary

This is a prospective, dual-centre, single dose, Phase IIb, single arm, open label study. The proposed clinical trial involves a single infusion of autologous HSPCs genetically modified with the GLOBE lentiviral vector, using an improved transduction protocol in 9 patients affected by transfusion dependent Beta-Thalassemia.

Four study phases are foreseen:

1. Screening phase, during which the conditions required by the clinical protocol for patients' inclusion/exclusion will be assessed after the signature of the informed consents/assents. Patients will be recruited from the two participating sites: IRCCS Ospedale San Raffaele (OSR), Department of Pediatric Immunohematology and adult Hematology (OSR Stem Cells Programme) (Milan) and IRCCS Ospedale Pediatrico Bambino Gesù (OPBG), Department of Haematology, Oncology and Gene and Cell Therapy (Rome).
2. Baseline phase, carried from the end of the screening phase to the day before the start of the conditioning regimen.
3. Treatment phase, from the first day of conditioning regimen until DP administration.
4. Follow-up phase: from DP administration until 2 years follow-up.

Conditions

Interventions

GENETIC

FT007

Autologous hematopoietic stem and progenitor cells (HSPCs) transduced ex vivo with the lentiviral vector GLOBE encoding for the human beta globin using an improved transduction protocol, re-suspended in their final formulation medium and cryopreserved.

Sponsors & Collaborators

  • Ospedale San Raffaele

    collaborator OTHER

  • Fondazione Telethon

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-07-06
Primary Completion
2028-10-31
Completion
2030-10-31

Countries

  • Italy

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07680803 on ClinicalTrials.gov