Transfusion-dependent Beta-ThalassemiaPhase 2NCT07680803
Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia
A Phase 2 study testing FT007 in people with Transfusion-dependent Beta-Thalassemia, run by Fondazione Telethon at 2 sites in Italy.
Trial 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… Read more about this study
- Who is it for?
- Ages 3 to 35 years
- Conditions the study is for
- Transfusion-dependent Beta-Thalassemia
- What is being tested?
- FT007
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 2 sitesItaly
- Study dates
- 6 Jul 2026to Oct 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
- Karnofsky Index or Lansky ≥ 80%.
- Adequate cardiac, renal, hepatic and pulmonary functions resulting in eligibility to undergo autologous HSCT as evidenced by:
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- 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 room air).
- Serum creatinine < 2 x upper limit of normal and estimated GFR > 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 with partners of childbearing potential).
- 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 of the 24 months prior to the DP administration.
Who cannot join
- 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.
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- 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, defined as undetectable HCV RNA at least 12 weeks after completion of therapy) and/or evidence of active infection of Treponema Pallidum or Mycoplasma species.
- 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 study.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Italy2 sites, 2 recruiting
- Recruiting
Ospedale San Raffaele
Milan, Lombardy, 20132
Alessandro Aiuti, +39 02 26439057, aiuti.alessandro@hsr.it
- Recruiting
Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 00165
Franco Locatelli, +39 0668593697, franco.locatelli@opbg.net
About this study
Official title: 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
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:
- 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).
- Baseline phase, carried from the end of the screening phase to the day before the start of the conditioning regimen.
- Treatment phase, from the first day of conditioning regimen until DP administration.
- Follow-up phase: from DP administration until 2 years follow-up.
- Conditions
- Transfusion-dependent Beta-Thalassemia
- Being tested
- FT007
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FT007Genetic
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.
| Study group | Role | What they receive |
|---|---|---|
| subjects with transfusion-dependent beta-thalassemia | Experimental | Not described Receives: Genetic: FT007 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects who achieve TI12 in the 2 years from DP administration
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.
Measured: from 60 days after the IMP infusion, to 24 months of follow-up
Secondary outcomes (19)
Overall survival
Overall survival at 12 and 24 months post IMP infusion
Measured: 12 months and 24 months post IMP infusion
Proportion of Subjects who achieve hematological engraftment ≤ day +60 from DP administration
Proportion of Subjects who achieve hematological engraftment ≤ day +60 from DP administration defined as first day of absolute neutrophil count ≥ 0.5 x 10^9/L on 3 consecutive measurements obtained on different days and platelets ≥20x 10^9/L in the absence of platelet transfusions and/or growth factor support for at least 7 consecutive days.
Measured: from Day 11 to Day 60 post IMP infusion
Frequency and severity of adverse events (AEs) and serious adverse events (SAEs) (Safety and tolerability of the administration of FT007)
Frequency and severity of adverse events (AEs) and serious adverse events (SAEs) attributed to procedures related to harvesting of HSPCs, conditioning regimen, FT007 administration (from screening to 24 months post GT).
Measured: from Baseline to 24 months post IMP infusion
Proportion of Subjects with abnormal clonal proliferation development due to insertional mutagenesis
Proportion of Subjects with abnormal clonal proliferation development due to insertional mutagenesis
Measured: 6, 12, 18 and 24 months post IMP infusion
Polyclonal and multilineage engraftment evaluated by VCN and ISA
ISA will be carried out at 3, 6, 12, 18, 24 months on total nucleated cells and different hematopoietic lineages (granulocytes, lymphocytes, erythroid cells) from PB and/or BM. The criteria to assess "polyclonality" of hematopoiesis is defined as > 1000 unique integration sites retrieved at 3, 6, 12, 18 and 24 months after gene therapy from PB and/or BM cells when available. Clonal dominance will be assessed from 3 months after GT and will be defined as the presence of a vector integration that constitutes > 30% with respect to the total integrations retrieved from whole peripheral blood cells in at least 3 consecutive timepoints and with an increase in the relative contribution over time. Vector-specific quantitative PCR will be performed on PB and/or BM erythroid cells at screening, 1, 2, 3, 6, 12,18 and 24 months after GT (at 1 and 18 months only on PB, at 24 months after GT. Adequate engraftment is defined as ≥ 0.8 VCN/genome on BM erythroid cells and CD15+ cells.
Measured: screening, 1, 2, 3, 6, 12, 18 and 24 months after IMP infusion
Proportion of Subjects with immune reconstitution
Proportion of Subjects with immune reconstitution defined as CD4+ >200 at 6 and 12 months post GT.
Immune-monitoring will be evaluated at baseline and at follow-up time points (6 and 12 months of follow-up). The frequency and functionality of lymphoid compartment (B, T, NK cells) in PB and BM, including humoral and cellular responses will be evaluated.
Measured: 6 and 12 months post IMP infusion
Characterization of Subjects achieving TI12
Duration of TI12 and the time from IMP infusion to last RBC transfusion prior to becoming TI12 will be evaluated. Weighted average Hb during TI12 and the proportion of subjects who meet the definition of TI12 at 24 months will be also evaluated.
Measured: 24 months after IMP infusion
Characterization of transfusion reduction (TR) among subjects not achieving TI12
Percentage of treated subjects with a reduction in the ml/kg RBC transfused from Month 12 through Month 24 (a 12-month period) after DP infusion of at least 50%, 60%, 75%, 90% or 100% compared to the average annual ml/kg RBC transfusion requirement during the 2 years prior to enrolment will be recorded. Moreover, the number of transfusions from Month 12 through Month 24 compared to the average number of annual transfusions during the 2 years prior to enrolment will be collected and analyzed. Two years of retrospective pre-transplant data will be collected for each subject in the study, so that each subject may serve as his/her own control for the parameters of RBC transfusion requirements.
Measured: 12 and 24 months post IMP infusion
Health-related quality of life (HRQoL) - PedsQL
HRQoL will be assessed by the use of standardized questionnaires: PedsQL quality of life version 4.0 up to 25 years of age, according to patient's age. PedsQL quality of life will be assessed at 12 and 24 months of follow-up compared to baseline.
Measured: baseline, 12 and 24 months
Health-related quality of life (HRQoL) - FACT BMT
HRQoL will be assessed by the use of standardized questionnaires: FACT BMT version 4 for adults. FACT BMT will be assessed at 12 and 24 months of follow-up compared to baseline.
Measured: baseline, 12 and 24 months
Health-related quality of life (HRQoL) - EQ-5D-5L
HRQoL will be assessed by the use of standardized questionnaires: EuroQol Quality of Life Scale-5 dimensions-5 levels of severity (EQ-5D-5L) for adults
Measured: baseline, 12 and 24 months
Health-related quality of life (HRQoL) - EQ-5D-Y
HRQoL will be assessed by the use of standardized questionnaires: EuroQol Quality of Life Scale-5 dimensions-5 levels of severity the EuroQol Quality of Life Scale-5 dimensions youth (EQ-5D-Y) for children and adolescents.
Measured: baseline, 12 and 24 months
Proportion of Subjects with engraftment of genetically corrected cells
Proportion of Subjects with engraftment of genetically corrected cells (expressed as VCN ≥ 0.8 on bone marrow erythroid cells and CD15+ cells). Engraftment will be assessed by vector-specific quantitative PCR on bone marrow erythroid cells at 6, 12, and 24 months after IMP infusion.
Measured: 6, 12, and 24 months after IMP infusion
Levels of biomarkers indicative of ineffective erythropoiesis before and after gene therapy at different time points.
Evaluation of plasma iron level, erythropoietin (EPO), soluble serum transferrin receptor (sTfR), Lactate Dehydrogenase (LDH) and Bilirubin will be evaluated as surrogate of ineffective erythropoiesis at screening and 1, 3, 6, 12, 24 months. Decreased levels of these parameters correlate with ineffective erythropoiesis improvement.
Measured: screening and 1, 3, 6, 12, 24 months after IMP infusion
Shear Wave Elastography and Liver Iron Concentration (LIC) over time
Shear Wave Elastography and LIC will be performed at screening and at 12, 24 months after infusion of DP (T2*MRI at screening can be avoided if performed less than 6 months before enrolment at treatment sites); in selected cases liver biopsy can be performed.
Measured: screening, 12 and 24 months after IMP infusion
Cardiac T2* value over time
Cardiac T2* value will be assessed through T2*MRI at screening and at 12, 24 months after infusion of DP (T2*MRI at screening can be avoided if performed less than 6 months before enrolment at treatment sites).
Measured: screening, 12 and 24 months after IMP infusion
Ferritin serum levels over time
Serum ferritin will be evaluated at screening, 3, 6, 12, 18, 24 months after infusion of DP.
Measured: screening, 3, 6, 12, 18 and 24 months after IMP infusion
Proportion of patients free from any iron-chelating therapy, including regular phlebotomies at month 24
The proportion of patients free from iron-chelation therapy, including regular phlebotomies in the entire cohort will be evaluated at month 24 after infusion of DP.
Measured: 24 months after IMP infusion
Duration of iron-chelating treatment after DP infusion
The duration of iron chelation therapy following DP infusion will be assessed. This includes the time from DP infusion to the last documented administration of iron chelation treatment.
Measured: from Day 1 to 24 months post IMP infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jul 2026
- Study start6 Jul 2026
- Primary completionOct 2028 (estimated)
- Study completionOct 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transfusion-dependent Beta-ThalassemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fondazione Telethon
- Sponsor type
- University, hospital or other
- Collaborators
- Ospedale San Raffaele
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07680803
- Sponsor's protocol ID
- BTHAL-FT007-01
- Other IDs
- 2025-522160-32-00, 2025
- Status last verified
- Jun 2026
- First submitted
- 25 Jun 2026
- Last updated
- 27 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07680803
- Acronym / sponsor ID
- BTHAL-FT007-01
- Conditions
- Transfusion-dependent Beta-Thalassemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Fondazione TelethonUniversity, hospital or other
- Collaborators
- Ospedale San Raffaele
- Study start
- 6 Jul 2026 (actual)
- Primary completion
- Oct 2028 (estimated)
- Study completion
- Oct 2030 (estimated)
- First posted
- 2 Jul 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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).
- Baseline phase, carried from the end of the screening phase to the day before the start of the conditioning regimen.
- Treatment phase, from the first day of conditioning regimen until DP administration.
- Follow-up phase: from DP administration until 2 years follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| subjects with transfusion-dependent beta-thalassemia | Experimental | Genetic: FT007 |
Who may qualify
- Ages
- 3 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (15 listed)
- 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.
- Karnofsky Index or Lansky ≥ 80%.
Key exclusion criteria (17 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FT007Autologous 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. | Genetic | subjects with transfusion-dependent beta-thalassemia |
Primary outcome
Proportion of subjects who achieve TI12 in the 2 years from DP administration
Time frame: from 60 days after the IMP infusion, to 24 months of follow-up
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.
19 secondary outcomes
Overall survival
Time frame: 12 months and 24 months post IMP infusion
Proportion of Subjects who achieve hematological engraftment ≤ day +60 from DP administration
Time frame: from Day 11 to Day 60 post IMP infusion
Frequency and severity of adverse events (AEs) and serious adverse events (SAEs) (Safety and tolerability of the administration of FT007)
Time frame: from Baseline to 24 months post IMP infusion
Proportion of Subjects with abnormal clonal proliferation development due to insertional mutagenesis
Time frame: 6, 12, 18 and 24 months post IMP infusion
Polyclonal and multilineage engraftment evaluated by VCN and ISA
Time frame: screening, 1, 2, 3, 6, 12, 18 and 24 months after IMP infusion
Proportion of Subjects with immune reconstitution
Time frame: 6 and 12 months post IMP infusion
Characterization of Subjects achieving TI12
Time frame: 24 months after IMP infusion
Characterization of transfusion reduction (TR) among subjects not achieving TI12
Time frame: 12 and 24 months post IMP infusion
Health-related quality of life (HRQoL) - PedsQL
Time frame: baseline, 12 and 24 months
Health-related quality of life (HRQoL) - FACT BMT
Time frame: baseline, 12 and 24 months
Health-related quality of life (HRQoL) - EQ-5D-5L
Time frame: baseline, 12 and 24 months
Health-related quality of life (HRQoL) - EQ-5D-Y
Time frame: baseline, 12 and 24 months
Proportion of Subjects with engraftment of genetically corrected cells
Time frame: 6, 12, and 24 months after IMP infusion
Levels of biomarkers indicative of ineffective erythropoiesis before and after gene therapy at different time points.
Time frame: screening and 1, 3, 6, 12, 24 months after IMP infusion
Shear Wave Elastography and Liver Iron Concentration (LIC) over time
Time frame: screening, 12 and 24 months after IMP infusion
Cardiac T2* value over time
Time frame: screening, 12 and 24 months after IMP infusion
Ferritin serum levels over time
Time frame: screening, 3, 6, 12, 18 and 24 months after IMP infusion
Proportion of patients free from any iron-chelating therapy, including regular phlebotomies at month 24
Time frame: 24 months after IMP infusion
Duration of iron-chelating treatment after DP infusion
Time frame: from Day 1 to 24 months post IMP infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing FT007 in people with Transfusion-dependent Beta-Thalassemia, run by Fondazione Telethon at 2 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 27 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Alessandro Aiuti, Professor (+39 02 26439057, aiuti.alessandro@hsr.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of subjects who achieve TI12 in the 2 years from DP administration (time frame: from 60 days after the IMP infusion, to 24 months of follow-up). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Jul 2026.