Clinical trial NCT04291352: Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation
Official title: Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation (TICATS Study)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Thalassemia Major
Lead sponsor: University Health Network, Toronto
Sponsor class: OTHER
First posted: 2020-03-02
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
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.
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Cardiac iron overload
timeFrame: 12 months
Secondary outcomes:
measure: Left ventricular ejection fraction
timeFrame: 12 months
measure: Blood taurine level
timeFrame: 12 months
measure: C-reactive protein
timeFrame: 12 months
measure: Interleukin-6
timeFrame: 12 months
measure: Plasma MDA
timeFrame: 12 months
measure: Reduced glutathione
timeFrame: 12 months
Other outcomes:
Locations:
city: Toronto
country: Canada
facility: University Health Network
state: Ontario
Study officials:
References: