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

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

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.

Study Overview

• Status: Recruiting
• Conditions: Thalassemia Major
• Intervention / Treatment: Dietary supplement: Taurine; Other: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Anneliesse Justiniano; Phone Number: 6729 416-340-4800; Email: anneliesse.justiniano@uhn.ca
• Study Contact Backup: Name: Red blood Cell Disorders Clinical Trials Program; Email: rbcd.clinicaltrials@uhn.ca

Study Locations

• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 2C4
      • Recruiting
      • University Health Network
      • Principal Investigator: Kevin Kuo, MD, MSc, FRCPC
      • Contact: Anneliesse Justiniano; Phone Number: +1-416-715-6485; Email: RBCD.clinicaltrials@uhn.ca

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Taurine; 675mg taurine four times daily	Dietary supplement: Taurine; 675mg taurine four times daily
Placebo Comparator: Placebo; placebo four times daily	Other: Placebo; placebo four times daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cardiac iron overload	Differences in ratio (T2* at year 1)/(T2* at baseline) between the taurine and standard chelation therapy arm	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Left ventricular ejection fraction	Differences in LVEF between the taurine and standard chelation therapy arm	12 months
Blood taurine level	Differences in blood taurine level between the taurine and standard chelation therapy arm	12 months
C-reactive protein	Differences in CRP between the taurine and standard chelation therapy arm	12 months
Interleukin-6	Differences in IL-6 between the taurine and standard chelation therapy arm	12 months
Plasma MDA	Differences in Plasma MDA between the taurine and standard chelation therapy arm	12 months
Reduced glutathione	Differences in GSH between the taurine and standard chelation therapy arm	12 months

Collaborators and Investigators

• Sponsor: University Health Network, Toronto

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2020
• Primary Completion (Estimated): October 31, 2024
• Study Completion (Estimated): October 31, 2025

Study Registration Dates

• First Submitted: February 28, 2020
• First Submitted That Met QC Criteria: February 28, 2020
• First Posted (Actual): March 2, 2020

Study Record Updates

• Last Update Posted (Actual): October 3, 2023
• Last Update Submitted That Met QC Criteria: September 30, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Heart Diseases; Cardiovascular Diseases; Metabolic Diseases; Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Iron Metabolism Disorders; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Iron Overload; Cardiomyopathies; Thalassemia; beta-Thalassemia
• Other Study ID Numbers: 18-5613

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED
• IPD Plan Description: Undetermined at this time

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No