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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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 1 year post-transplantation

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 1 year post-transplantation

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 1 year post-transplantation

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

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence of acute graft vs. host disease (GVHD)

Incidence of chronic graft vs. host disease (GVHD)

Rate of graft failure

+1 more

Secondary outcome measures

Probability of event-free survival (EFS)

Probability of overall survival (OS)

Reactivation/Infection from CMV, EBV, adenovirus

+1 more

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

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 / 7Eligibility criteria met

Find a Location

Who is running the clinical trial?

Children's Hospital of PhiladelphiaLead Sponsor

708 Previous Clinical Trials

8,581,009 Total Patients Enrolled

Timothy Olson, MD, PhDPrincipal Investigator - Children's Hospital of Philadelphia

Children's Hospital of Philadelphia

2 Previous Clinical Trials

45 Total Patients Enrolled

Media Library

CliniMACs (T Cell Depletion) Clinical Trial Eligibility Overview. Trial Name: NCT03047746 — N/A

Paroxysmal Nocturnal Hemoglobinuria Research Study Groups: TCRalpha/beta Tcell Depletion for BMF with trilineage aplasia, TCRalpha/beta Tcell Depletion for BMF w/o trilineage aplasia, TCRalpha/beta Tcell Depletion for BMF w/ Fanconi Anemia

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial 2023: CliniMACs Highlights & Side Effects. Trial Name: NCT03047746 — N/A

CliniMACs (T Cell Depletion) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03047746 — N/A

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the current sample size of this research endeavor?

"Indeed. According to the information on clinicaltrials.gov, this medical trial is still recruiting participants and has been since February 1st 2017 when it was first posted. 40 patients are needed from one single location; this study was last updated on March 1st 2022."

Answered by AI

Are researchers presently enrolling participants for this trial?

"Affirmative, the details on clinicaltrials.gov indicate that this investigation is actively enrolling volunteers. This study was first published in February 1st 2017 and revised March 1st 2022. Aspiring participants should note that 40 individuals are needed at one medical facility to complete the trial's requirements."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Timothy Olson 2017. "Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03047746.

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