← Back to Search

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

No Placebo-Only Group

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

Select...

My bone marrow has less than 5% blasts and I've never had MDS/AML.

Select...

My bone marrow has less than 5% blasts and I've never had MDS/AML.

Select...

My platelet count is below 20,000/uL or I need regular transfusions.

Select...

My white blood cell count is very low without taking any growth factor medications.

Select...

My blood tests show low reticulocyte count or I need regular blood transfusions, and I have bone marrow issues or a significant PNH clone.

Select...

I had a bone marrow transplant that didn't work and need another one.

Select...

I have a rare blood disorder and need a second bone marrow transplant.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 and 2 years post hct

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

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

Primary study objectives

Incidence of chronic GvHD-free, failure-free survival (GFFS)

Secondary study objectives

Incidence of any chronic GvHD

Incidence of chronic GvHD-free survival

Incidence of failure-free survival (GFFS)

+4 more

Awards & Highlights

No Placebo-Only Group

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

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

~2990

Rabbit ATG

2001

Completed Phase 2

~60