Abatacept for Thalassemia in Children

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
The Hospital for Sick Children, Toronto, Canada
Thalassemia in Children+1 More
Abatacept - Drug
Eligibility
< 65
All Sexes
What conditions do you have?
Select

Study Summary

The purpose of this study is to evaluate a novel transplant strategy for the long-term benefit of patients with transfusion dependent high-risk thalassemia.

Eligible Conditions

  • Thalassemia in Children

Treatment Effectiveness

Study Objectives

3 Primary · 1 Secondary · Reporting Duration: Day +100 until Day +365

Day +100 until Day +365
Number of patients who develop Chronic GVHD
Until Day +100
Number of patients who develop Grade II to IV acute GVHD at Day +100
Number of patients who have WBC engraftment by day +100
Until Day +365
Cost effectiveness
Immune reconstitution
Length of stay
Number of patients who will wean Sirolimus at 1 year post transplant

Trial Safety

Trial Design

1 Treatment Group

PTIS followed by abatacept and sirolimus
1 of 1
Experimental Treatment

20 Total Participants · 1 Treatment Group

Primary Treatment: Abatacept · No Placebo Group · Phase 1 & 2

PTIS followed by abatacept and sirolimusExperimental Group · 2 Interventions: Sirolimus, Abatacept · Intervention Types: Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Sirolimus
2013
Completed Phase 4
~2750
Abatacept
2005
Completed Phase 4
~111470

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: day +100 until day +365

Trial Background

Ky Chiang, Staff Physician
Principal Investigator
The Hospital for Sick Children
Closest Location: The Hospital for Sick Children · Toronto, Canada
Photo of Toronto  1Photo of Toronto  2Photo of Toronto  3
2014First Recorded Clinical Trial
1 TrialsResearching Thalassemia in Children
399 CompletedClinical Trials

Eligibility Criteria

Age < 65 · All Participants · 10 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
Patients must have had a complete evaluation of their iron status including measurement of serum ferritin, MRI of the heart and liver (within the preceding 6 months prior to referral).
You have thalassemia genotype.
You have hepatomegaly.
You have a liver iron content of more than 7mg/g dry weight.
You have a history of severe alloimmunization.
You are able to take oral medication and are willing to adhere to the study regimen.
You have a performance status of at least 70% prior to transplantation.\n
You are unable to tolerate iron chelation.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.