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Alkylating Agent

IST + BMT for Aplastic Anemia (TransIT Trial)

Phase 3

Recruiting

Led By Bronwen Shaw, 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

TransIT Trial 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.Check my eligibility

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.See study design

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.

TransIT Trial 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

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

Primary outcome measures

Adrenal gland hypofunction

Secondary outcome measures

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

TransIT 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.

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Pediatric Transplantation and Cellular Therapy ConsortiumUNKNOWN

Boston Children's HospitalLead Sponsor

761 Previous Clinical Trials

5,579,496 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

Frequently Asked Questions

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

What risks may be associated with Immunosuppressive Therapy?

"Immunosuppressive Therapy is assessed as safe, garnering a score of 3. This can be attributed to evidence from both Phase 3 trials and further clinical research that shows efficacy for this treatment option."

Answered by AI

Could you provide the upper limit of participants involved in this experiment?

"Affirmative. Clinicaltrials.gov confirms that this investigation, which was first published on January 1st 2023, is actively recruiting participants. A total of 234 individuals need to be enrolled from one medical center."

Answered by AI

Does this study have an age restriction, and if so, what is the cutoff for participation?

"By examining the inclusion criteria, it becomes apparent that this trial is available to those between 0 and 25 years of age. For younger patients (under 18) there are 100 clinical studies while for elderly individuals (over 65), 169 trials exist."

Answered by AI

Is this experiment currently accepting participants?

"Affirmative. Based on the information located on clinicaltrials.gov, this research is actively enrolling patients. The trial was first posted on January 1st 2023 and its data points have been updated as recently as September 1st of the same year. A total of 234 people will be admitted at one site for participation in this study."

Answered by AI

Do I fulfill the requirements to partake in this medical experiment?

"This trial seeks 234 individuals aged 0 to 25 with severe aplastic anemia. Furthermore, they must provide signed consent, possess no fully matched related donor, have at least two unrelated donors noted on their National Marrow Donor Program search that are well-matched (9/10 or 10/10 for HLA-A, B, C., DRB1 and DQB1 using high resolution), demonstrate bone marrow cellularity below 25% or hematopoietic cells <30%, display two of the following three in peripheral blood: neutrophils lower than 0.5 x 10^9/L; platelets"

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

David Williams 2023. "A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05600426.

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