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

• ClinicalTrials.gov Identifier: NCT04291352
• Recruitment Status: Unknown
• First Posted: March 2, 2020
• Last Update Posted: March 2, 2020
• Sponsor: University Health Network, Toronto
• Information provided by (Responsible Party): University Health Network, Toronto

Tracking Information

• First Submitted Date: February 28, 2020
• First Posted Date: March 2, 2020
• Last Update Posted Date: March 2, 2020
• Estimated Study Start Date: June 1, 2020
• Estimated Primary Completion Date: May 31, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: February 28, 2020)

Cardiac iron overload [ Time Frame: 12 months ]

Differences in ratio (T2* at year 1)/(T2* at baseline) between the taurine and standard chelation therapy arm

• Original Primary Outcome Measures: Same as current
• Change History: No Changes Posted

Current Secondary Outcome Measures (submitted: February 28, 2020)

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

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation
• Official Title: Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation (TICATS Study)

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.

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Not Applicable

Study Design

Allocation: Randomized
Intervention Model: Parallel Assignment
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.

Masking: Triple (Participant, Care Provider, 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.

Primary Purpose: Treatment

• Condition: Thalassemia Major

Intervention

• Dietary Supplement: Taurine
  675mg taurine four times daily
• Other: Placebo
  placebo four times daily

Study Arms

• Experimental: Taurine
  675mg taurine four times daily
  Intervention: Dietary Supplement: Taurine
• Placebo Comparator: Placebo
  placebo four times daily
  Intervention: Other: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Unknown status
• Estimated Enrollment (submitted: February 28, 2020): 60
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: May 31, 2023
• Estimated Primary Completion Date: May 31, 2023 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Not Provided

Administrative Information

• NCT Number: NCT04291352
• Other Study ID Numbers: 18-5613
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Undecided
• Plan Description: Undetermined at this time

• Current Responsible Party: University Health Network, Toronto
• Original Responsible Party: Same as current
• Current Study Sponsor: University Health Network, Toronto
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: University Health Network, Toronto
• Verification Date: February 2020