Thalassemia MajorTICATSNCT04291352
Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation
A study testing Taurine in people with Thalassemia Major, run by University Health Network, Toronto at 1 site in Canada.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thalassemia Major
- What is being tested?
- Taurine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteCanada
- Study dates
- 1 Jun 2020to 31 Oct 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Participant inclusion criteria
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.
Participant exclusion criteria
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
Trial locations and contacts
1 site in 1 country.
Red blood Cell Disorders Clinical Trials Program contact
Canada1 site, 1 recruiting
- Recruiting
University Health Network
Toronto, Ontario, M5G 2C4
Anneliesse Justiniano, +1-416-715-6485, RBCD.clinicaltrials@uhn.ca
Kevin Kuo, MD, MSc, FRCPC
About this study
Official title: 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.
- Conditions
- Thalassemia Major
- Being tested
- Taurine, Placebo
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TaurineDietary supplement
675mg taurine four times daily
PlaceboOther
placebo four times daily
| Study group | Role | What they receive |
|---|---|---|
| Taurine | Experimental | 675mg taurine four times daily Receives: Dietary Supplement: Taurine |
| Placebo | Placebo comparator | placebo four times daily Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cardiac iron overload
Differences in ratio (T2* at year 1)/(T2* at baseline) between the taurine and standard chelation therapy arm
Measured: 12 months
Secondary outcomes (6)
Left ventricular ejection fraction
Differences in LVEF between the taurine and standard chelation therapy arm
Measured: 12 months
Blood taurine level
Differences in blood taurine level between the taurine and standard chelation therapy arm
Measured: 12 months
C-reactive protein
Differences in CRP between the taurine and standard chelation therapy arm
Measured: 12 months
Interleukin-6
Differences in IL-6 between the taurine and standard chelation therapy arm
Measured: 12 months
Plasma MDA
Differences in Plasma MDA between the taurine and standard chelation therapy arm
Measured: 12 months
Reduced glutathione
Differences in GSH between the taurine and standard chelation therapy arm
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Mar 2020
- Study start1 Jun 2020
- Primary completion31 Oct 2024 (estimated)
- Study completion31 Oct 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia MajorStudies of the same condition, open and under way.
- More trials run by University Health Network, TorontoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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 blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Health Network, Toronto
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Kevin H.M. Kuo, MD, MSc, FRCPC, University Health Network, Toronto
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04291352
- Sponsor's protocol ID
- 18-5613
- Acronym
- TICATS
- Status last verified
- Sep 2023
- First submitted
- 28 Feb 2020
- Last updated
- 3 Oct 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Undetermined at this time
Study at a glance
From the registry record, last updated by the sponsor on 3 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation (TICATS Study)
- ClinicalTrials.gov ID
- NCT04291352
- Acronym / sponsor ID
- TICATS / 18-5613
- Conditions
- Thalassemia Major
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- University Health Network, TorontoUniversity, hospital or other
- Study start
- 1 Jun 2020 (actual)
- Primary completion
- 31 Oct 2024 (estimated)
- Study completion
- 31 Oct 2025 (estimated)
- First posted
- 2 Mar 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| Taurine | Experimental | Dietary Supplement: Taurine675mg taurine four times daily |
| Placebo | Placebo comparator | Other: Placeboplacebo four times daily |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Taurine675mg taurine four times daily | Dietary supplement | Taurine |
| Placeboplacebo four times daily | Other | Placebo |
Primary outcome
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
6 secondary outcomes
Left ventricular ejection fraction
Time frame: 12 months
Blood taurine level
Time frame: 12 months
C-reactive protein
Time frame: 12 months
Interleukin-6
Time frame: 12 months
Plasma MDA
Time frame: 12 months
Reduced glutathione
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Oct 2023.
- Individual participant dataSharing planned: Undecided. Undetermined at this time
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Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
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A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Red blood Cell Disorders Clinical Trials Program contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Taurine in people with Thalassemia Major, run by University Health Network, Toronto at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 3 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Anneliesse Justiniano (416-340-4800 ext. 6729, anneliesse.justiniano@uhn.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cardiac iron overload (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Oct 2023.