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Alkylating agents

Stem Cell Transplant for Severe Aplastic Anemia

Phase 2
Recruiting
Led By Richard W Childs, M.D.
Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnosed with severe aplastic anemia with specific criteria
The patient does not have any HLA antibodies detectable against any of the mismatched HLA alleles expressed by the haplo-donor
Must not have
Presence of an active infection not adequately responding to appropriate therapy
Availability of an HLA identical or 9/10 HLA matched relative to serve as a stem cell donor
Timeline
Screening 3 weeks
Treatment Varies
Follow Up one-year
Awards & highlights
No Placebo-Only Group

Summary

This trial is for people with severe aplastic anemia, myelodysplastic syndrome, or paroxysmal nocturnal hemoglobinuria who want to receive a stem cell transplant using peripheral blood stem cells from a family member.

Who is the study for?
This trial is for people aged 4-55 with severe aplastic anemia, myelodysplastic syndrome (MDS), or paroxysmal nocturnal hemoglobinuria (PNH) who haven't responded to standard treatments. They need a family member donor aged 4-75. Participants must understand the study and consent; minors will need guardian consent. Exclusions include certain heart, liver, kidney issues, active infections not responding to treatment, HIV positive individuals, pregnant women or those not using birth control.
What is being tested?
The trial tests if peripheral blood stem cells from a relative plus chemotherapy can treat SAA, MDS and PNH effectively and safely. Patients undergo extensive testing before receiving chemo and radiation followed by the stem cell transplant in hospital for about a month with follow-up visits over five years.
What are the potential side effects?
Potential side effects may include reactions to the stem cell infusion such as fever or pain, complications from chemotherapy like nausea or hair loss, increased risk of infections due to weakened immune system post-transplantation and possible organ inflammation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with severe aplastic anemia.
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I don't have antibodies against the donor's tissue markers.
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My PNH does not respond to eculizumab/ravulizumab or I can't access this treatment.
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My severe aplastic anemia has turned into MDS.
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I have a severe bone marrow condition and standard treatments haven't worked for me.
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I have a family member who can donate stem cells and is a partial HLA match.
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I am between the ages of 4 and 55.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have an infection that isn't getting better with treatment.
Select...
I have a family member who is a near-perfect match for a stem cell donation.
Select...
I have been diagnosed with Fanconi's anemia.
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My heart's pumping ability is significantly reduced.
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I do not have any major illnesses or organ failures that would prevent me from surviving a transplant.
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I am eligible for a stem cell transplant from a fully matched donor.
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I need some help with my daily activities.
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I am not pregnant and willing to use birth control or abstain for a year.
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My kidney function is reduced, with a creatinine clearance rate below 50.

Timeline

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

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Evaluate 1 year chronic GVHD-free survival rate

Awards & Highlights

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

Trial Design

1Treatment groups
Experimental Treatment
Group I: Treatment ArmExperimental Treatment2 Interventions
G-CSF mobilized peripheral stem cells and post haplo-identical transplantation cyclophosphamide
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cyclophosphamide
2010
Completed Phase 4
~2310

Find a Location

Who is running the clinical trial?

National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,929 Previous Clinical Trials
47,765,174 Total Patients Enrolled
Richard W Childs, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
18 Previous Clinical Trials
8,700 Total Patients Enrolled

Media Library

Cyclophosphamide (Alkylating agents) Clinical Trial Eligibility Overview. Trial Name: NCT03520647 — Phase 2
Paroxysmal Nocturnal Hemoglobinuria Research Study Groups: Treatment Arm
Paroxysmal Nocturnal Hemoglobinuria Clinical Trial 2023: Cyclophosphamide Highlights & Side Effects. Trial Name: NCT03520647 — Phase 2
Cyclophosphamide (Alkylating agents) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03520647 — Phase 2
~4 spots leftby Jun 2025
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