Thalassemia in Children study: Abatacept vs Sirolimus
Official title Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia
ClinicalTrials.gov ID: NCT05426252
What this study is testing
What is Abatacept?
Abatacept is an investigational medicine, being studied as a potential treatment for thalassemia in children.
Also referred to as Orencia.
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
- The study aims to see if a new transplant conditioning method can give long‑term benefit to these patients.
- Who it's for
- Children who need regular blood transfusions and have high‑risk thalassemia.
- What's involved
- Participants will receive the experimental conditioning treatment before a stem‑cell transplant, followed by scheduled clinic visits and medical tests.
- 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 1 to 18
You may be able to join if
- Patients with a diagnosis of transfusion dependent beta or alpha thalassemia (3 or 4 gene deletion) between the age of 1-18 years.
- Thalassemia genotype must be confirmed by molecular genetic testing.
- Age \>7 years
- Hepatomegaly (2 cm below costal margin)
- Inadequate iron chelation (liver iron content \>7mg/g dry weight)
You likely can't join if
- Patients will not be excluded based on sex, race, or ethnic background.
- Cardiac: Evidence of significant cardiac dysfunction (resting left ventricular ejection fraction of \< 50% with absence of improvement with...
- Renal: Evidence of \> 50% reduction in expected creatinine clearance or GFR \< 60mL/min/1.73m2
- Hepatic: Evidence of hepatic dysfunction evidenced by a serum direct (conjugate) bilirubin of \> 2.5 mg/dl, or ALT \> 5 times the upper limit of...
- Pulmonary: Evidence of focal or diffuse active infection or pneumonitis and the patient demonstrates a FEV1 \< 50% or carbon monoxide diffusing...
- Presence of donor specific antibody (DSA) with mean fluorescence intensity (MFI) greater than 3,000.
See the full eligibility criteria
- Patients with a diagnosis of transfusion dependent beta or alpha thalassemia (3 or 4 gene deletion) between the age of 1-18 years.
- Thalassemia genotype must be confirmed by molecular genetic testing.
- Age \>7 years
- Hepatomegaly (2 cm below costal margin)
- Inadequate iron chelation (liver iron content \>7mg/g dry weight)
- Severe alloimmunization
- Unable to tolerate iron chelation 3\. Patients must have had a complete evaluation of their iron status including measurement of serum ferritin, MRI of the heart and liver (within the previous 6 months prior to...
- Patients will not be excluded based on sex, race, or ethnic background.
- Cardiac: Evidence of significant cardiac dysfunction (resting left ventricular ejection fraction of \< 50% with absence of improvement with exercise), marked cardiomegaly or uncontrollable hypertension.
- Renal: Evidence of \> 50% reduction in expected creatinine clearance or GFR \< 60mL/min/1.73m2
- Hepatic: Evidence of hepatic dysfunction evidenced by a serum direct (conjugate) bilirubin of \> 2.5 mg/dl, or ALT \> 5 times the upper limit of normal for age.
- Pulmonary: Evidence of focal or diffuse active infection or pneumonitis and the patient demonstrates a FEV1 \< 50% or carbon monoxide diffusing capacity (DLCO) of \< 50% predicted value (adjusted for hemoglobin).
- Presence of donor specific antibody (DSA) with mean fluorescence intensity (MFI) greater than 3,000.
- Previous stem cell transplant or gene therapy.
- Presence of cardiomyopathy with a T2\ \< 10ms per Cardiac MRI.
- Presence of significant liver iron deposition defined as liver iron content \>15mg/g liver dry weight.
- Active HIV, hepatitis B or hepatitis C disease.
- Severe liver cirrhosis or bridging fibrosis on liver biopsy if previously done.
- Prior or current malignancy or myeloproliferative or immunodeficiency disorder.
- Evidence of active, deep seated, life-threatening infections despite therapy (e.g., certain fungal species, HIV, etc.).
- Patients will be excluded if they are women of childbearing potential who are currently pregnant (b-HCG+) or who are not practicing adequate contraception.
- Any condition that would preclude serial follow up.
- Patients with a known life-threatening allergy to components of the pre transplant immunosuppression (fludarabine), conditioning (treosulfan, cyclophosphamide or anti-thymocyte globulin) or graft versus host...
- May have thalassemia or sickle trait.
- Will also consider ABO match and lack of donor specific anti-HLA antibodies.
- Donors must be minimal of 15 kg weight and have completed routine donor evaluations as per our standard of care.
- Donors must have signed (by patient or legal guardian) informed consent for the protocol approved by the Research Ethical Board of the Hospital for Sick Children/University of Toronto.
- No evidence of transmissible diseases in compliance with the Health Canada CTO regulations
- Not pregnant or lactating
- Must not be allergic to granulocyte colony stimulating factor (G-CSF)
The study team makes the final eligibility decision.
Where it is enrolling
- Toronto, Ontario, Canada
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, but 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, 1 year to 18 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Toronto, Ontario, Canada. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.