Matched Unrelated Stem Cell Transplant for Severe Aplastic Anemia

Phase-Based Progress Estimates
Cohen Children's Medical Center of New York, New Hyde Park, NYSevere Aplastic AnemiaMethotrexate - Drug
No minimum age - 25
All Sexes
What conditions do you have?

Study 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.

Eligible Conditions
  • Severe Aplastic Anemia

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

1 Primary · 27 Secondary · Reporting Duration: Randomization to one year, randomization to two years

Year 2
Upper arm
Year 2
Blood Platelets
Randomization through Day 100
Bone Marrow Transplantation
Estimate the time from randomization to initiation of IST or BMT.
Randomization through two years post randomization
Year 5
Upper arm
Year 2
Upper arm
Year 2
Red Blood Cell Transfusion
Blood Platelets
Estimate the rates of failure of patients randomized to IST
Estimate the rates of response of patients randomized to IST
Year 2
Therapeutic procedure
Bone Marrow Transplantation
Comparison of subjects with inadequate counts at 2 years among those who have not died or failed treatment.
Adrenal gland hypofunction
Year 5
Comparison of HR-QoL score between patients randomized to both arms
Randomization to 3-5 years
Leukemia, Myelocytic, Acute
Therapeutic procedure
Estimate the rates of engraftment in patients who are randomized to URD BMT.
Graft-vs-Host Disease
Graft-vs-Host Disease
Graft-vs-Host Disease
Estimate the rates of primary graft failure in patients who are randomized to URD BMT .
Estimate the rates of secondary graft failure in patients who are randomized to URD BMT .
Randomization to one year, randomization to two years
Comparison of overall survival at 1 and 2 years from randomization in both arms.

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Trial Design

2 Treatment Groups

Matched Unrelated Stem Cell Transplant
1 of 2
Immunosuppressive Therapy
1 of 2

Active Control

234 Total Participants · 2 Treatment Groups

Primary Treatment: Matched Unrelated Stem Cell Transplant · No Placebo Group · Phase 3

Matched Unrelated Stem Cell TransplantActiveComparator Group · 7 Interventions: Methotrexate, Matched Unrelated Donor Hematopoetic Stem Cell Transplant, rabbit anti-thymocyte globulin (ATG), cyclosporine, low-dose total body irradiation (TBI), Cyclophosphamide, Fludarabine · Intervention Types: Drug, Procedure, Drug, Drug, Radiation, Drug, Drug
Immunosuppressive TherapyActiveComparator Group · 3 Interventions: Immunosuppressive Therapy (IST), cyclosporine, horse anti-thymocyte globulin (ATG) · Intervention Types: Procedure, Drug, Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: randomization to one year, randomization to two years

Who is running the clinical trial?

Boston Children's HospitalLead Sponsor
718 Previous Clinical Trials
5,324,196 Total Patients Enrolled
Center for International Blood and Marrow Transplant ResearchNETWORK
34 Previous Clinical Trials
200,173,844 Total Patients Enrolled
National Heart, Lung, and Blood Institute (NHLBI)NIH
3,616 Previous Clinical Trials
47,117,977 Total Patients Enrolled
North American Pediatric Aplastic Anemia ConsortiumUNKNOWN
Blood and Marrow Transplant Clinical Trials NetworkNETWORK
49 Previous Clinical Trials
13,807 Total Patients Enrolled
Pediatric Transplantation and Cellular Therapy ConsortiumUNKNOWN
National Institutes of Health (NIH)NIH
2,471 Previous Clinical Trials
24,591,479 Total Patients Enrolled
Michael Pulsipher, MDPrincipal InvestigatorUniversity of Utah
8 Previous Clinical Trials
637 Total Patients Enrolled
David Williams, MDPrincipal InvestigatorBoston Children's Hospital
4 Previous Clinical Trials
935 Total Patients Enrolled
Bronwen Shaw, MDPrincipal InvestigatorCIBMTR/Medical College of Wisconsin (MCW)

Eligibility Criteria

Age No minimum age - 25 · All Participants · 6 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:

Frequently Asked Questions

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." - Anonymous Online Contributor

Unverified Answer

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

"Affirmative. 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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

Is this experiment currently accepting participants?

"Affirmative. Based on the information located on, 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." - Anonymous Online Contributor

Unverified Answer

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" - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.