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Cell Therapy

Hematopoietic Cell Transplant for Aplastic Anemia

Phase 2
Recruiting
Research Sponsored by Masonic Cancer Center, University of Minnesota
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Early myelodysplastic features (bone marrow (BM) blasts <5%) without history of MDS/AML pre-treatment.
Early myelodysplastic features (bone marrow (BM) blasts <5%), without history of MDS/AML pre-treatment.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 and 2 years post hct
Awards & highlights

Summary

"This trial is testing a new treatment approach for severe aplastic anemia, paroxysmal nocturnal hemoglobinuria, acquired pure red cell aplasia, or acquired amegakaryocytic

Who is the study for?
This trial is for people with severe bone marrow failure conditions like aplastic anemia or paroxysmal nocturnal hemoglobinuria. Participants must have specific blood and bone marrow characteristics, such as low reticulocyte count or evidence of one to three-lineage aplasia, and adequate organ function. Those who've had a previous transplant that didn't work may also qualify.
What is being tested?
The study tests a less intense cell transplant method combined with post-transplant cyclophosphamide medication in patients with certain bone marrow failures. It includes individualized dosing based on pharmacokinetics and varying radiation doses by age and disease features.
What are the potential side effects?
Possible side effects include reactions to medications like Rituximab or Cyclophosphamide (e.g., nausea, hair loss), complications from total body irradiation (e.g., fatigue, skin changes), and risks associated with stem cell infusion (e.g., infection risk).

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My bone marrow has less than 5% blasts and I've never had MDS/AML.
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My bone marrow has less than 5% blasts and I've never had MDS/AML.
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My platelet count is below 20,000/uL or I need regular transfusions.
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My white blood cell count is very low without taking any growth factor medications.
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My blood tests show low reticulocyte count or I need regular blood transfusions, and I have bone marrow issues or a significant PNH clone.
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I had a bone marrow transplant that didn't work and need another one.
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I have a rare blood disorder and need a second bone marrow transplant.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 and 2 years post hct
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 and 2 years post hct for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of chronic GvHD-free, failure-free survival (GFFS)
Secondary outcome measures
Incidence of any chronic GvHD
Incidence of chronic GvHD-free survival
Incidence of failure-free survival (GFFS)
+4 more

Trial Design

2Treatment groups
Experimental Treatment
Group I: Arm B: Clonal hematopoiesisExperimental Treatment9 Interventions
Participants 25-75 years old and/or with clonal hematopoiesis. Active study treatment includes the conditioning regimen followed by the stem cell infusion and GvHD prophylaxis through day +180. Supportive care and follow up activities continue through two years post HCT.
Group II: Arm A: No clonal hematopoiesisExperimental Treatment9 Interventions
Participants 25 years of age and younger with no clonal hematopoiesis. Active study treatment includes the conditioning regimen followed by the stem cell infusion and GvHD prophylaxis through day +180. Supportive care and follow up activities continue through two years post HCT.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Rituximab
1999
Completed Phase 4
~2200
Rabbit ATG
2001
Completed Phase 2
~60
Total Body Irradiation
2006
Completed Phase 3
~820
Cyclophosphamide
2010
Completed Phase 4
~2320
Fludarabine
2012
Completed Phase 4
~1860
Tacrolimus
2019
Completed Phase 4
~5510
Mycophenolate Mofetil
1997
Completed Phase 4
~2380

Find a Location

Who is running the clinical trial?

Masonic Cancer Center, University of MinnesotaLead Sponsor
278 Previous Clinical Trials
15,531 Total Patients Enrolled
2 Trials studying Aplastic Anemia
93 Patients Enrolled for Aplastic Anemia
~40 spots leftby May 2035
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