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Immunomodulator

Reduced Toxicity Conditioning for Thalassemia

Phase 1 & 2

Recruiting

Led By Yogi Chopra, MD

Research Sponsored by The Hospital for Sick Children

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients with a diagnosis of transfusion dependent beta or alpha thalassemia (3 or 4 gene deletion) between the age of 1-18 years

Patients who are not candidates for gene therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day +100 until day +365

Awards & highlights

Study Summary

This trial is testing a new way to do transplants that may help people with thalassemia who need transfusions.

Who is the study for?

This trial is for kids and teens (1-18 years old) with high-risk thalassemia, which requires regular blood transfusions. They should have a sibling or family donor for bone marrow transplant, not be eligible for gene therapy, and able to take oral meds. Excluded are those with severe organ dysfunction, active infections like HIV/hepatitis B/C, previous transplants or gene therapy, pregnant women or significant allergies to the treatment drugs.Check my eligibility

What is being tested?

The study tests a new transplant method using two drugs: Abatacept and Sirolimus in children with thalassemia who need frequent blood transfusions. The goal is to see if this approach can improve long-term outcomes after bone marrow transplantation from family donors.See study design

What are the potential side effects?

Possible side effects of Abatacept and Sirolimus may include increased risk of infection due to immune system suppression, potential liver or kidney problems, allergic reactions, and other drug-specific adverse effects that will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 1-18 years old and need regular blood transfusions for thalassemia.

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I am not eligible for gene therapy.

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I have a family member who is a match and can donate stem cells to me.

Select...

I have thalassemia with at least one high-risk feature like being over 7, having an enlarged liver, issues with iron removal, severe immune reactions, or can't handle iron removal treatments.

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I can care for myself but cannot carry on normal activity or do active work.

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My thalassemia has been confirmed through genetic testing.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day +100 until day +365

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day +100 until day +365

This trial's timeline: 3 weeks for screening, Varies for treatment, and day +100 until day +365 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Immune reconstitution

Number of patients who develop Grade II to IV acute GVHD at Day +100

Number of patients who have WBC engraftment by day +100

Secondary outcome measures

Number of patients who develop Chronic GVHD

Other outcome measures

Cost effectiveness

Length of stay

Number of patients who will wean Sirolimus at 1 year post transplant

Side effects data

From 2023 Phase 3 trial • 613 Patients • NCT03086343

URINARY TRACT INFECTION

UPPER RESPIRATORY TRACT INFECTION

NASOPHARYNGITIS

HYPERTENSION

100%

80%

60%

40%

20%

Study treatment Arm

Abatacept

Upadacitinib 15 mg

Trial Design

1Treatment groups

Experimental Treatment

Group I: PTIS followed by abatacept and sirolimusExperimental Treatment2 Interventions

Administration of reduced-toxicity conditioning regimen combined with pre-transplant immunosuppression, followed by abatacept and sirolimus as graft-versus-host disease (GVHD) prophylaxis for allogeneic transplant with either Human Leukocyte Antigen (HLA)-matched sibling donors or haploidentical donors

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Abatacept

FDA approved

Sirolimus

FDA approved

0 / 6Eligibility criteria met

Find a Location

Who is running the clinical trial?

The Hospital for Sick ChildrenLead Sponsor

690 Previous Clinical Trials

6,945,458 Total Patients Enrolled

Thalassemia Foundation of CanadaUNKNOWN

Yogi Chopra, MDPrincipal InvestigatorThe Hospital for Sick Children

Media Library

Abatacept (Immunomodulator) Clinical Trial Eligibility Overview. Trial Name: NCT05426252 — Phase 1 & 2

Thalassemia Research Study Groups: PTIS followed by abatacept and sirolimus

Thalassemia Clinical Trial 2023: Abatacept Highlights & Side Effects. Trial Name: NCT05426252 — Phase 1 & 2

Abatacept (Immunomodulator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05426252 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the participant inclusion criterion for this trial restricted to those over 55 years of age?

"The enrolment criteria for this medical trial limits participants to those between the ages of 1 and 18. Accordingly, there are 41 studies available exclusively to minors while 22 trials can be accessed by people over 65 years old."

Answered by AI

What criteria must a patient meet to be eligible for participation in this experiment?

"To be eligible for this research, children suffering from thalassemia must be between the ages of one and eighteen. Approximately 20 patients are needed to complete this clinical study."

Answered by AI

What is the overall enrolment rate for this medical experiment?

"Affirmative. Clinicaltrials.gov's data indicates that this clinical trial, which was first published on March 22nd 2022, is currently enrolling patients in need of treatment. A single site needs to recruit 20 participants for the study."

Answered by AI

Are there still opportunities to partake in this trial?

"Affirmative, the data on clinicaltrials.gov reveals that this research initiative is actively recruiting participants. The trial was launched on March 22nd 2022 and has been updated as recently June 19th of the same year. 20 patients are required at one site for enrollment purposes."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia

Yogi Chopra 2022. "Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05426252.

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