Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation

NCT04291352 | Unknown DMC

• 1 other identifier: 18-5613
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

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.

Conditions

Thalassemia Major

Outcome Measures

Primary Outcomes (1)

• Cardiac iron overload

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

  12 months

Secondary Outcomes (6)

• Left ventricular ejection fraction

  12 months

• Blood taurine level

  12 months

• C-reactive protein

  12 months

• Interleukin-6

  12 months

• Plasma MDA

  12 months

Study Arms (2)

Taurine

EXPERIMENTAL

675mg taurine four times daily

Dietary Supplement: Taurine

Placebo

PLACEBO COMPARATOR

placebo four times daily

Other: Placebo

Interventions

Taurine DIETARY_SUPPLEMENT

675mg taurine four times daily

Taurine

Placebo OTHER

placebo four times daily

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants who meet all of the following criteria will qualify for entry into the study:
• Diagnosis of transfusion-dependent Thalassemia being followed at the RBC clinic at TGH
• Age 18 or older
• On a stable dose of iron chelation for \>30 days. Combination iron chelation regimen is allowed in the study
• Cardiac MRI T2\* ≥8ms measured within 3 months prior to randomization.
• Preserved left ventricular ejection fraction (LVEF) \>50% as measured by cardiac MRI measured within 3 months prior to randomization.

You may not qualify if:

• Participants who meet any of the following criteria will be excluded from the study:
• 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
• Serum ferritin \< 500 ng/mL at screening
• Liver iron concentration \> 40 mg/g dw as measured by liver R2 MRI (FerriScan) measured within 3 months prior to randomization
• Signs and symptoms consistent with congestive heart failure in the opinion of the investigator
• As a result of medical review, physical examination or screening investigations, the investigator considers the subject unfit for the study.
• No fixed address
• Interval advent of general contraindications to MRI.
• Taking another investigational product within 30 days of anticipated date of randomization
• Women who are currently pregnant or plan to become pregnant during the study period

Sponsors & Collaborators

• University Health Network, Toronto: lead

Study Sites (1)

University Health Network

Toronto, Ontario, M5G 2C4, Canada

RECRUITING

MeSH Terms

Conditions

beta-Thalassemia

Interventions

Taurine

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Organic Chemicals; Sulfonic Acids; Sulfur Acids; Sulfur Compounds

Central Study Contacts

Anneliesse Justiniano

CONTACT

416-340-4800; anneliesse.justiniano@uhn.ca

Red blood Cell Disorders Clinical Trials Program

CONTACT

rbcd.clinicaltrials@uhn.ca

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: 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.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Model Details: 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.

Study Record Dates

• First Submitted: February 28, 2020
• First Posted: March 2, 2020
• Study Start: June 1, 2020
• Primary Completion: October 31, 2024
• Study Completion: October 31, 2025
• Last Updated: October 3, 2023

Record last verified: 2023-09

Locations

CA (1)