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T Cell Depletion

Stem Cell Transplant for Bone Marrow Failure Syndrome

N/A

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 with inherited bone marrow failure conditions associated with predominant single lineage failure, such as severe congenital neutropenia, isolated disorders of erythropoiesis, and congenital thrombocytopenia syndromes

Patients with acquired aplastic anemia meeting criteria for severe or very severe aplastic anemia

Must not have

Patients without suitable closely Human leukocyte antigen (HLA)-matched unrelated or related haploidentical matched donor available

Patients with a clinical diagnosis of myelodysplastic syndrome (MDS)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the use of TCR alpha/beta+ T cell-depleted peripheral blood stem cells from closely matched unrelated donors or partially matched/haploidentical related donors for HSCT in patients with acquired and inherited BMF syndromes.

Who is the study for?

This trial is for patients with various bone marrow failure syndromes who have suitable organ function and no active infections. It's open to those able to consent or minors with guardian consent, including specific conditions like Fanconi anemia, severe aplastic anemia, and others linked to bone marrow failure. Pregnant women or those without a closely matched donor are excluded.

What is being tested?

The study tests the use of TCR alpha/beta+ T cell-depleted peripheral blood stem cells from unrelated donors or partially matched relatives in treating acquired and inherited bone marrow failures. This single-arm pilot study aims to assess the safety and effectiveness of this transplant method.

What are the potential side effects?

While not explicitly listed here, potential side effects may include reactions related to stem cell transplantation such as infection risk due to immune suppression, graft-versus-host disease where donor cells attack the patient's body, bleeding complications, and organ damage.

Eligibility Criteria

Inclusion Criteria

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

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I have a genetic condition that mainly affects one type of blood cell.

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I have been diagnosed with severe or very severe aplastic anemia.

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I have a condition that causes my red blood cells to break down at night.

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I have Fanconi anemia with signs of bone marrow failure.

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I have Shwachman-Diamond syndrome with a confirmed SBDS gene mutation and bone marrow failure.

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I have a telomere disorder like dyskeratosis congenita and my tests show bone marrow failure.

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I have a condition that affects the production of blood cells in my bone marrow.

Exclusion Criteria

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

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I don't have a closely matched donor for a transplant.

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I have been diagnosed with myelodysplastic syndrome.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2017 Phase 2 trial • 93 Patients • NCT00579124

33%

Non-relapsed mortality

100%

80%

60%

40%

20%

Study treatment Arm

AEs for Total Number of Participants: Stratum 2

AEs for Total Number of Participants: Stratum 1

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: TCRalpha/beta Tcell Depletion for BMF with trilineage aplasiaExperimental Treatment1 Intervention

Patients with acquired or inherited bone marrow failure (iBMF) with trilineage aplasia excluding Fanconi Anemia will be given previously established, disease-specific chemotherapy and/or radiation based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from unrelated or partially matched related donors.

Group II: TCRalpha/beta Tcell Depletion for BMF w/o trilineage aplasiaExperimental Treatment1 Intervention

Patients with acquired or inherited bone marrow failure (iBMF) without trilineage aplasia will be given previously established, disease-specific chemotherapy and/or radiation based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from unrelated or partially matched related donors.

Group III: TCRalpha/beta Tcell Depletion for BMF w/ Fanconi AnemiaExperimental Treatment1 Intervention

Patients with acquired or inherited bone marrow failure (iBMF) with Fanconi Anemia and related DNA Repair Disorders will be given previously established, disease-specific chemotherapy and/or radiation based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from unrelated or partially matched related donors.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

CliniMACs

2005

Completed Phase 2

~100

0 / 9Eligibility criteria met