Thalassemia Major
Conditions
Brief summary
Hypothesis: Taurine, in combination with standard iron chelation therapy, is more effective than chelation therapy alone in reducing cardiac iron overload, oxidative stress and cardiac damage in β-Thalassemia. Protocol: Sixty subjects with transfusion dependent β-Thalassemia receiving deferasirox iron chelation therapy will be recruited and randomized in a 1:1 ratio to either (1) placebo and continuation of their iron chelation or (2) a combination of iron chelation plus taurine. Transfusion and safety visits will be scheduled monthly with clinical/biochemical assessment visits every three months. The efficacy of taurine combined with standard chelation therapy will be assessed at baseline and 12 months posttreatment by both cardiac T2\*MRI, and cardiac function. The recruitment period is projected to be 12 months from initiation.
Interventions
DIETARY_SUPPLEMENTTaurine
675mg taurine four times daily
OTHERPlacebo
placebo four times daily
Sponsors
University Health Network, Toronto
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Masking description
Both taurine and placebo powder inside the capsules are a white crystalline, odorless powder. The powder is loaded into clear, vegetarian, odorless capsules. Placebo and Investigational Product capsules will be packaged in 500 cc HDPE bottles.
Intervention model description
Prospective, double-blind, randomized control trial. Subjects are randomized to receive either taurine or placebo in addition to their standard chelation regimen for a 12 month period.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Participant inclusion criteria Participants who meet all of the following criteria will qualify for entry into the study: 1. Diagnosis of transfusion-dependent Thalassemia being followed at the RBC clinic at TGH 2. Age 18 or older 3. On a stable dose of iron chelation for \>30 days. Combination iron chelation regimen is allowed in the study 4. Cardiac MRI T2\* ≥8ms measured within 3 months prior to randomization. 5. Preserved left ventricular ejection fraction (LVEF) \>50% as measured by cardiac MRI measured within 3 months prior to randomization. Participant
Exclusion criteria
Participants who meet any of the following criteria will be excluded from the study: 1. More than 16 transfusions in the past 12 months or those who are anticipated to be on a 3 week transfusion schedule during the study period 2. Serum ferritin \< 500 ng/mL at screening 3. Liver iron concentration \> 40 mg/g dw as measured by liver R2 MRI (FerriScan) measured within 3 months prior to randomization 4. Signs and symptoms consistent with congestive heart failure in the opinion of the investigator 5. As a result of medical review, physical examination or screening investigations, the investigator considers the subject unfit for the study. 6. No fixed address 7. Interval advent of general contraindications to MRI. 8. Taking another investigational product within 30 days of anticipated date of randomization 9. Women who are currently pregnant or plan to become pregnant during the study period
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Cardiac iron overload | 12 months | Differences in ratio (T2\* at year 1)/(T2\* at baseline) between the taurine and standard chelation therapy arm |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Blood taurine level | 12 months | Differences in blood taurine level between the taurine and standard chelation therapy arm |
| C-reactive protein | 12 months | Differences in CRP between the taurine and standard chelation therapy arm |
| Left ventricular ejection fraction | 12 months | Differences in LVEF between the taurine and standard chelation therapy arm |
| Plasma MDA | 12 months | Differences in Plasma MDA between the taurine and standard chelation therapy arm |
| Reduced glutathione | 12 months | Differences in GSH between the taurine and standard chelation therapy arm |
| Interleukin-6 | 12 months | Differences in IL-6 between the taurine and standard chelation therapy arm |
Countries
Canada
Contacts
Primary ContactAnneliesse Justiniano
Backup ContactRed blood Cell Disorders Clinical Trials Program
Outcome results
None listed