Actively Recruiting
Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia
Led by The Hospital for Sick Children · Updated on 2023-12-19
20
Participants Needed
1
Research Sites
249 weeks
Total Duration
On this page
Sponsors
T
The Hospital for Sick Children
Lead Sponsor
T
Thalassemia Foundation of Canada
Collaborating Sponsor
AI-Summary
What this Trial Is About
The purpose of this study is to evaluate a novel transplant strategy for the long-term benefit of patients with transfusion dependent high-risk thalassemia.
CONDITIONS
Official Title
Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 1 to 18 years with transfusion dependent beta or alpha thalassemia (3 or 4 gene deletion)
- Thalassemia confirmed by molecular genetic testing
- At least one high-risk feature: age >7 years, hepatomegaly (2 cm below costal margin), inadequate iron chelation (liver iron content >7 mg/g dry weight), severe alloimmunization, or inability to tolerate iron chelation
- Complete iron status evaluation including serum ferritin, heart and liver MRI within 6 months, and liver elastography within 3 months (not required)
- Ability to take oral medication and willingness to adhere to study regimen
- Performance status of at least 70% Karnofsky or Lansky prior to transplantation
- Acceptable candidates for marrow transplantation based on evaluation
- Have histocompatibility sibling or HLA haploidentical family donor approved as hematopoietic progenitor cell donor
- Not candidates for gene therapy
- Signed informed consent by patient or legal guardian
You will not qualify if you...
- Significant cardiac dysfunction (resting left ventricular ejection fraction < 50% without improvement, marked cardiomegaly, or uncontrollable hypertension)
- Renal dysfunction with > 50% reduction in creatinine clearance or GFR < 60 mL/min/1.73m2
- Hepatic dysfunction with serum direct bilirubin > 2.5 mg/dl or ALT > 5 times upper limit of normal
- Pulmonary infection or pneumonitis with FEV1 < 50% or DLCO < 50%, not requiring ventilation
- Presence of donor specific antibody with mean fluorescence intensity > 3000
- Previous stem cell transplant or gene therapy
- Cardiomyopathy with T2* < 10 ms on cardiac MRI
- Significant liver iron deposition > 15 mg/g dry weight unless improved with chelation
- Active HIV, hepatitis B or C infection
- Severe liver cirrhosis or bridging fibrosis if biopsy done
- Prior or current malignancy, myeloproliferative or immunodeficiency disorder
- Active, deep-seated, life-threatening infections despite therapy
- Women of childbearing potential who are pregnant or not using contraception
- Any condition preventing serial follow-up
- Known life-threatening allergy to study drugs (fludarabine, treosulfan, cyclophosphamide, anti-thymocyte globulin, abatacept, sirolimus)
- Conditions interfering with compliance or study interpretation as judged by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Yogi Chopra
Toronto, Ontario, Canada
Actively Recruiting
Research Team
Y
Yogi Chopra, MD
CONTACT
E
Erilda Kapllani
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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