Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age3-35
SponsorFondazione Telethon
About this trial
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.
Eligibility criteria
Qualifiers
Must be willing to adhere to the protocol as evidenced by written informed consent for adults or parental informed consent and subject assent for adolescents and children.
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.
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.
Age ≥ 18 years and ≤ 35 years for Group 1, Age ≥ 3 years and ≤ 35 years for Group 2.
Disqualifiers
Use of other investigational agents within 4 weeks prior to study enrolment (within 6 weeks if use of long-acting agents).
Severe, active viral, bacterial or fungal infection at eligibility evaluation.
Current or prior malignant neoplasia (except local skin cancer or cervical intraepithelial neoplasia) or exceptional family history of familial cancer syndromes.
Current or prior immunodeficiency disorder.
Trial design
Treatments tested in this trial
- FT007
Treatment groups
9 Participants
are divided into 1 treatment groupSponsors and collaborators
Fondazione Telethon
Lead sponsor
Ospedale San Raffaele
Collaborator
Source ClinicalTrials.gov