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Alkylating Agent

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

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 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

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.

Find a Location

Who is running the clinical trial?

Pediatric Transplantation and Cellular Therapy ConsortiumUNKNOWN
Boston Children's HospitalLead Sponsor
778 Previous Clinical Trials
5,580,778 Total Patients Enrolled
2 Trials studying Aplastic Anemia
85 Patients Enrolled for Aplastic Anemia
Center for International Blood and Marrow Transplant ResearchNETWORK
38 Previous Clinical Trials
200,193,196 Total Patients Enrolled

Media Library

Cyclophosphamide (Alkylating Agent) Clinical Trial Eligibility Overview. Trial Name: NCT05600426 — Phase 3
Aplastic Anemia Research Study Groups: Immunosuppressive Therapy, Matched Unrelated Stem Cell Transplant
Aplastic Anemia Clinical Trial 2023: Cyclophosphamide Highlights & Side Effects. Trial Name: NCT05600426 — Phase 3
Cyclophosphamide (Alkylating Agent) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05600426 — Phase 3
~156 spots leftby Dec 2029
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