IST + BMT for Aplastic Anemia (TransIT Trial)

Phase 3

Recruiting

Led By David Williams, MD

Research Sponsored by Boston Children's Hospital

Eligibility Criteria Checklist

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

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up randomization to 1 year post-randomization, randomization to 2 years post-randomization, and randomization to up to 5 years post-randomization

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

This trial will compare treatments for Severe Aplastic Anemia, a rare condition where the body stops producing blood cells. Treatment includes immune suppressive therapy or a bone marrow transplant.

Who is the study for?

This trial is for young patients (25 years or younger) with severe aplastic anemia without a matched family bone marrow donor. They must have specific blood cell counts, at least two potential unrelated donors, and no contraindications to bone marrow transplant or immune suppressive therapy. Exclusions include inherited syndromes, prior transplants, certain infections like HIV, pregnancy, breastfeeding, and previous disease-modifying treatments.

What is being tested?

The study compares initial treatment of severe aplastic anemia using either immune suppressive therapy (IST) or a bone marrow transplant from an unrelated donor (URD BMT). It will measure the time until treatment failure or death and examine quality of life, fertility markers, genetic factors related to marrow failure and changes after treatment.

What are the potential side effects?

Potential side effects can include reactions to anti-thymocyte globulin such as fever and chills; cyclosporine may cause kidney damage or high blood pressure; chemotherapy drugs like cyclophosphamide might lead to nausea or hair loss; low-dose total body irradiation could result in fatigue or skin changes.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ randomization to 3-5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~randomization to 3-5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and randomization to 3-5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Adrenal gland hypofunction

Secondary study objectives

Comparison of HR-QoL score between patients randomized to both arms

Upper arm

Comparison of overall survival at 1 and 2 years from randomization in both arms.

+24 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Active Control

Group I: Immunosuppressive TherapyActive Control3 Interventions

Patient will receive standard immunosuppressive therapy combination of drugs: horse anti-thymocyte globulin (ATG) and cyclosporine.

Group II: Matched Unrelated Stem Cell TransplantActive Control7 Interventions

Patient will under go matched unrelated donor transplant of hematopoietic stem cells as their therapy using fludarabine, cyclophosphamide, rabbit anti-thymocyte globulin (ATG), and low-dose total body irradiation (TBI) as preparative regimen and cyclosporine and methotrexate for graft versus host disease (GVHD) prevention.

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