Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation

Thalassemia Major Clinical Trial

— TICATS  

Official title:

Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation (TICATS Study)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04291352
• Other study ID #: 18-5613
• Status: Recruiting
• Phase: N/A
• Start date: June 1, 2020
• Est. completion date: October 31, 2025

Study information

• Verified date: September 2023
• Source: University Health Network, Toronto
• Contact: Anneliesse Justiniano
• Phone: 416-340-4800
• Email: anneliesse.justiniano[@]uhn.ca
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 60
• Est. completion date: October 31, 2025
• Est. primary completion date: October 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• beta-Thalassemia
• Cardiomyopathies
• Iron Overload
• Thalassemia
• Thalassemia Major

Intervention

Dietary Supplement:
• Taurine
675mg taurine four times daily

Other:
• Placebo
placebo four times daily

Locations

Country	Name	City	State
Canada	University Health Network	Toronto	Ontario

Sponsors (1)

Lead Sponsor	Collaborator
University Health Network, Toronto

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Cardiac iron overload	Differences in ratio (T2* at year 1)/(T2* at baseline) between the taurine and standard chelation therapy arm	12 months
Secondary	Left ventricular ejection fraction	Differences in LVEF between the taurine and standard chelation therapy arm	12 months
Secondary	Blood taurine level	Differences in blood taurine level between the taurine and standard chelation therapy arm	12 months
Secondary	C-reactive protein	Differences in CRP between the taurine and standard chelation therapy arm	12 months
Secondary	Interleukin-6	Differences in IL-6 between the taurine and standard chelation therapy arm	12 months
Secondary	Plasma MDA	Differences in Plasma MDA between the taurine and standard chelation therapy arm	12 months
Secondary	Reduced glutathione	Differences in GSH between the taurine and standard chelation therapy arm	12 months