A single arm, open label, multicenter, single-dose, phase 2b clinical study evaluating efficacy and safety of gene therapy using autologous CD34+ hematopoietic stem cells transduced with the GLOBE lentiviral vector using an improved transduction protocol in subjects with transfusion-dependent beta-thalassemia.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Fondazione Telethon Ets, San Raffaele Hospital

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15], Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Decision date (initial)

2025-12-18

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To evaluate the clinical efficacy of FT007 infusion in terms of transfusion independence in pediatric and adult patients with transfusion dependent beta-thalassemia.

Secondary objectives 2

1. To evaluate the safety and tolerability of FT007 infusion in pediatric and adult patients with transfusion dependent beta thalassemia
2. To evaluate the clinical and biological efficacy of FT007 infusion in pediatric and adult patients with transfusion dependent beta-thalassemia

Conditions and MedDRA coding

transfusion-dependent beta-thalassemia

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-001933-PIP01-16

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Male and female adults/adolescents/children diagnosed with transfusion-dependent β-thalassemia (homozygous or compound heterozygous). At least 2 out of the 9 patients must have B0/B0 or B0/B0- like genotype. In case the genetic diagnosis available at screening wasn’t performed in a certified laboratory (check under PI’s or delegated investigator’s responsibility), the genetic diagnosis will be repeated at clinical sites during the screening phase
2. Documented history of at least 100 mL/kg/year or 10 U/year of packed red blood cell transfusions in each of the 2 years prior to signing informed consent.
3. Age ≥ 18 years and ≤ 35 years for Group 1, Age ≥ 3 years and ≤ 35 years for Group 2.
4. Karnofsky Index or Lansky ≥ 80%
5. Adequate cardiac, renal, hepatic and pulmonary functions resulting in eligibility to undergo autologous HSCT
6. Low risk thrombophilic screen and negative history of significant previous thrombotic events

Exclusion criteria 7

1. Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long active agents)
2. Severe, active viral, bacterial or fungal infection at eligibility evaluation
3. Current or prior malignant neoplasia (except local skin cancer or cervical intraepithelial neoplasia) or exceptional family history of familial cancer syndromes
4. Current or prior immunodeficiency disorder.
5. For patients until the age of 14 years only: availability of an HLA-matched family donor
6. Previous allogeneic hematopoietic stem cell transplantation.
7. Previous gene therapy treatment (gene addition or gene editing).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Proportion of Subjects achieving transfusion independence defined as a weighted average Hb ≥ 9.0 gr/dL without any red blood cell transfusion for a continuous period of ≥ 12 months (TI12) at any time during the study after the drug product administration. The assessment of TI12 starts 60 days after last RBC transfusion for post-transplant support or BTHAL standard of care.

Secondary endpoints 3

1. Overall survival at 12 and 24 months post GT.
2. Evaluation of the safety of treatment
3. Evaluation of biological correlates of safety

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Auxiliary 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Fondazione Telethon Ets

Sponsor organisation

Fondazione Telethon Ets

Address

Via Varese 16 B

City

Rome

Postcode

00185

Country

Italy

Scientific contact point

Organisation

Fondazione Telethon Ets

Contact name

Regulatory Affairs

Public contact point

Organisation

Fondazione Telethon Ets

Contact name

Clinica Development & Operations

San Raffaele Hospital

Sponsor organisation

San Raffaele Hospital

Address

Via Olgettina 58

City

Milan

Postcode

20132

Country

Italy

Sponsor responsibilities

Article 77 compliance

Fondazione Telethon Ets

Contact point sponsor

Fondazione Telethon Ets

Article 77 implementation

Fondazione Telethon Ets

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 50 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications