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Anti-metabolites

Fludarabine RIC for Bone Marrow Failure Syndrome

Phase < 1

Recruiting

Led By Timothy Olson, MD, PhD

Research Sponsored by Children's Hospital of Philadelphia

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients 0-22 years with acquired aplastic anemia or a diagnosed inherited bone marrow failure syndrome

Patients must have a fully Human leukocyte antigen (HLA)-matched (10/10) related donor

Must not have

Uncontrolled bacterial, viral or fungal infections

Patients with a clinical diagnosis of Myelodysplastic syndrome

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 1 year post transplant

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing whether a less intense form of chemotherapy can help patients with aplastic anemia or inherited bone marrow failure syndromes to receive a bone marrow transplant from a related donor.

Who is the study for?

This trial is for young people (ages 0-22) with bone marrow failure syndromes like aplastic anemia or inherited conditions causing low blood counts. They need a fully matched related bone marrow donor, good organ function, and no uncontrolled infections. Pregnant females or those without a suitable donor are excluded.

What is being tested?

The study tests if fludarabine-based reduced intensity conditioning helps patients with acquired aplastic anemia or inherited bone marrow failures successfully accept transplants from matched donors.

What are the potential side effects?

Fludarabine may cause side effects such as nausea, fatigue, weakened immune system leading to increased infection risk, liver issues, and rarely severe lung problems.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 22 or younger with a rare disorder affecting my bone marrow.

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I have a relative who is a perfect match for my transplant needs.

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I have severe aplastic anemia confirmed by specific tests and blood counts.

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My kidney, liver, and heart functions are within normal ranges.

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I can do most activities but may need help.

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I have a family member who is a match for a bone marrow donation.

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I do not have any untreated infections.

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I am 22 years old or younger.

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I need regular blood or platelet transfusions, or I have a low white blood cell count.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have any untreated serious infections.

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I have been diagnosed with Myelodysplastic syndrome.

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I do not have a fully matched family donor for a transplant.

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I don't have a family donor for a bone marrow transplant.

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I have PNH without bone marrow failure.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 1 year post transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 1 year post transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 1 year post transplant for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Rate of graft failure

Time to neutrophil engraftment

Awards & Highlights

No Placebo-Only Group

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

Trial Design

3Treatment groups

Experimental Treatment

Group I: Inherited Bone Marrow Failure Syndrome no Trilineage AplasiaExperimental Treatment1 Intervention

Patients with inherited bone marrow failure (iBMF) syndromes without trilineage aplasia includes those with diagnoses of Severe Congenital Neutropenia, Diamond-Blackfan Anemia, and related conditions. Patients will receive a matched related donor bone marrow transplant following conditioning with thymoglobulin, busulfan and fludarabine.

Group II: Inherited Bone Marrow Failure Syndrome + Trilineage AplasiaExperimental Treatment1 Intervention

Patients with inherited bone marrow failure (iBMF) syndromes with trilineage aplasia includes those with diagnoses of Fanconi Anemia, Dyskeratosis Congenita, and related conditions. Patients will receive a matched related donor bone marrow transplant following conditioning with fludarabine, cyclophosphamide, thymoglobulin.

Group III: Acquired Aplastic Anemia (AA)Experimental Treatment1 Intervention

Patients with severe or very severe acquired aplastic anemia (AA). Patients will receive a matched related donor bone marrow transplant following reduced intensity conditioning (RIC) including thymoglobulin (ATG), fludarabine and dose-reduced cyclophosphamide.

0 / 14Eligibility criteria met