Clinical trial for Transfusion-dependent Beta-Thalassemia
Official title Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia
ClinicalTrials.gov ID: NCT07680803
What this study is testing
What is FT007?
FT007 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for transfusion-dependent beta-thalassemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 to 35
You may be able to join if
- Must be willing to adhere to the protocol as evidenced by written informed consent for adults or parental informed consent and subject assent for...
- Male and female adults/adolescents/children diagnosed with transfusion-dependent β-thalassemia (homozygous or compound heterozygous). At least 2 out...
- 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...
- Age ≥ 18 years and ≤ 35 years for Group 1, Age ≥ 3 years and ≤ 35 years for Group 2.
- Karnofsky Index or Lansky ≥ 80%.
You likely can't join if
- 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...
- Current or prior immunodeficiency disorder.
- History of uncontrolled seizures.
- Aspartate transaminase (AST), alanine transaminase (ALT) \>3 × the upper limit of normal (ULN), or direct bilirubin value \>2.5 × ULN.
See the full eligibility criteria
- 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...
- 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.
- Karnofsky Index or Lansky ≥ 80%.
- Adequate cardiac, renal, hepatic and pulmonary functions resulting in eligibility to undergo autologous HSCT as evidenced by:
- Left ventricular ejection fraction (LVEF) greater than 45% by echo and normal ECG or presence of abnormalities not significant for cardiac disease. Absence of severe pulmonary hypertension.
- Diffusing capacity of the lung for carbon monoxide (DLCO) \> 50% and forced expiratory volume in 1 sec (FEV1) and forced expiratory vital capacity (FVC) \> 60% predicted (if non cooperative: pulse oximetry \> 95 % in...
- Serum creatinine \ 60 ml/min/1.73m2, calculated using the CKD-EPI formula (Levey 2009) for adults and the modified Schwartz formula (Schwartz 2009) for pediatric patients.
- Absent-mild-moderate liver iron overload on T2\ MRI (i.e. LIC \< 15 mg Fe/gr dry weight assessed at screening. T2\ MRI at screening can be avoided if performed less than 6 months before enrolment at treatment centers).
- Absent-mild-moderate cardiac iron overload T2\ MRI (i.e. \> 20 msec assessed at screening. T2\ MRI at screening can be avoided if performed less than 6 months before enrolment at treatment centers).
- Absence of severe liver fibrosis or cirrhosis on Shear Wave (less than 6 months before enrolment) or of other advanced liver disorder.
- For all patients in reproductive age, agreement to use highly effective and adequate method of contraception for at least 12 months following DP administration (including both females of childbearing potential and males...
- Good adherence to transfusion and chelation programme, as indirect evidence of good adherence to treatment and follow-up evaluations for the current trial.
- Availability of an adequate and well documented transfusion history (at least previous 24 months) or availability to follow a regular transfusion regimen according to guidelines and provide a detailed transfusion record...
- 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.
- History of uncontrolled seizures.
- Aspartate transaminase (AST), alanine transaminase (ALT) \>3 × the upper limit of normal (ULN), or direct bilirubin value \>2.5 × ULN.
- Baseline prothrombin time (International Normalized Ratio; INR) \>1.5 × ULN.
- Other clinical conditions judged non compatible with the procedure and/or the treatment.
- Positivity for HIV (serology or RNA), and/or HbsAg and/or HBV DNA and/or HCV RNA (patients who have completed antiviral treatment for HCV may only be enrolled if they have achieved a sustained virologic response...
- Positive history of significant previous thrombotic events. In case of a positive thrombophilic screening, patient's inclusion will be evaluated according to national guidelines.
- Active alcohol or substance abuse within 6 months of the study.
- Pregnancy or lactation.
- Previous allogeneic hematopoietic stem cell transplantation.
- Previous gene therapy treatment (gene addition or gene editing).
- For patients until the age of 14 years only: availability of an HLA-matched family donor.
- Patients with associated α-thalassemia and \>1 alpha deletion, or alpha multiplications.
- Any other condition that would not allow the potential subject to complete follow-up examinations during the course of the study and, in the opinion of the investigator, makes the potential subject unsuitable for the...
The study team makes the final eligibility decision.
Where it's taking place
- Rome, Lazio, Italy
- Milan, Lombardy, Italy
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 3 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Rome, Lazio, Italy; Milan, Lombardy, Italy. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.