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Stem Cell Transplant

Bone Marrow Transplant for Aplastic Anemia (BMT CTN CureAA Trial)

Phase 2
Waitlist Available
Research Sponsored by Medical College of Wisconsin
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Adequate organ function defined by institutional transplant standards or defined as below: 1. Cardiac: Left ventricular ejection fraction (LVEF) at rest > 40% with no clinical signs of cardiac failure. For participants aged < 13 years, shortening fraction (SF) ≥ 26% by echocardiogram or multigated acquisition (MUGA) may be substituted for LVEF. 2. Hepatic: Total bilirubin < 2.0 mg/dL unless Gilbert's disease is present 3. Renal: For participants > 13.0 years of age at the time of enrollment: estimated creatinine clearance (CrCl) > 60 mL/minute (per institutional standard). For participants < 13.0 years of age at enrollment: glomerular filtration rate (GFR) estimated by the updated Schwartz formula ≥ 90 mL/min/1.73 m2. If the estimated GFR is < 90 mL/min/1.73 m2, then renal function must be measured by 24-hour creatinine clearance or nuclear GFR, and must be > 50 mL/min/1.73 m2. 4. Pulmonary: i. For participants > 13.0 years of age: Diffusing capacity of the lung for carbon monoxide (DLCO, corrected/adjusted for hemoglobin [Hb]) > 50%, or Spirometry with forced expiratory volume 1 (FEV1) > 50% predicted (without administration of bronchodilator) and forced vital capacity (FVC) > 50% predicted. ii. For participants < 13.0 years of age unable to perform pulmonary function tests (PFTs) due to age or developmental ability: (1) no evidence of dyspnea at rest and (2) no need for supplemental oxygen and (3) O2 saturation > 92% on room air at sea level (with lower levels allowed at higher elevations per established center standard of care [e.g., Utah, 4,200 feet above sea level, does not give supplemental oxygen unless below 90%]).
Karnofsky or Lansky performance status ≥ 60%
Must not have
Prior desensitization attempt for HLA antibodies to chosen donor. Any intervention with the sole intent to reduce the level of HLA DSA, (e.g., plasmapheresis, intravenous immunoglobulin [IVIG], MMF, etc.) would constitute a desensitization attempt
Prior allogeneic stem cell transplant
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year post-hsct
Awards & highlights
No Placebo-Only Group

Summary

"This trial will study if bone marrow transplantation can be used to treat severe aplastic anemia in patients who have not received treatment before."

Who is the study for?
This trial is for individuals with severe aplastic anemia who haven't been treated before. It's open to those eligible for bone marrow transplants from half-matched (haploidentical) or completely unrelated donors.
What is being tested?
The study compares two types of bone marrow transplants in treating severe aplastic anemia: one from a half-matched family member and another from a non-related donor.
What are the potential side effects?
Bone marrow transplant side effects can include infection risk, graft-versus-host disease, bleeding complications, organ damage, and reactions to the new marrow.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I can care for myself but may need occasional help.
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I am between 3 and 75 years old.
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I have severe aplastic anemia with very low blood cell counts.
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I agree to use two forms of birth control or remain abstinent.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have tried treatments to reduce my body's reaction to a transplant.
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I have had a stem cell transplant from a donor.
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I have had a solid organ transplant.
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I have been officially diagnosed with MDS according to WHO or ICC standards.
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I do not have an ongoing serious infection that isn't getting better with treatment.
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I have antibodies against my donor's HLA, confirmed by a specific test.
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My bone marrow test shows changes that suggest a pre-MDS or MDS condition.
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I have never had a life-threatening reaction to Thymoglobulin®.
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I am not pregnant or breastfeeding.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year post-hsct
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year post-hsct for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Graft versus host disease (GVHD)-free failure-free survival (GFFS) at One year
Secondary study objectives
Participants Alive and Engrafted at One year post conditioning
Participants with Hematologic Response post-transplant
Percentage of Participants with Failure-Free Survival (FFS) at One year post conditioning
+9 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Active Control
Group I: Unrelated donor transplantationActive Control1 Intervention
Patients receiving bone marrow transplanted from an unrelated donor will be included in this arm.
Group II: Haploidentical transplantationActive Control1 Intervention
Patients receiving bone marrow transplanted from a haploidentical related donor will be included in this arm.

Find a Location

Who is running the clinical trial?

National Heart, Lung, and Blood Institute (NHLBI)NIH
3,934 Previous Clinical Trials
47,792,216 Total Patients Enrolled
11 Trials studying Aplastic Anemia
922 Patients Enrolled for Aplastic Anemia
SanofiIndustry Sponsor
2,204 Previous Clinical Trials
4,036,718 Total Patients Enrolled
Medical College of WisconsinLead Sponsor
631 Previous Clinical Trials
1,181,896 Total Patients Enrolled
~40 spots leftby Aug 2028
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