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CAR T-cell Therapy

TCD HCT for Fanconi Anemia

Phase 2

Recruiting

Led By Margaret MacMillan, MD, Msc, FRCPC

Research Sponsored by Masonic Cancer Center, University of Minnesota

Eligibility Criteria Checklist

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

Must have

Less than 65 years of age

Presence of at least one of the following risk factors: Severe aplastic anemia (SAA) defined as: platelet count <20 x 109/L, absolute neutrophil count of <5 x 108/L, hemoglobin <8 g/dL, Myelodysplastic syndrome (MDS) or acute leukemia, High risk genotype

Must not have

Pregnant or breastfeeding as the treatment used in this study are Pregnancy Category D. Females of childbearing potential must have a negative pregnancy test (serum or urine) within 14 days of study registration

Active, uncontrolled infection within 1 week prior to starting study therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year after transplant

Awards & highlights

All Individual Drugs Already Approved

Approved for 50 Other Conditions

No Placebo-Only Group

Summary

This trial is testing whether a new transplant method can help people with Fanconi anemia by reducing the risk of severe infections.

Who is the study for?

This trial is for patients with Fanconi anemia who have a suitable donor for blood cell transplant, are under 65 years old, and have good heart and lung function. They must not be pregnant or breastfeeding, have had solid tumor cancer in the last 2 years, or active infections. Participants need to agree to use contraception during treatment.

What is being tested?

The study tests T cell receptor α/β depletion (α/β TCD) hematopoietic cell transplantation (HCT) in Fanconi anemia patients. It aims to reduce graft-versus-host disease without routine immune suppression drugs, hoping for faster immune recovery and fewer severe infections post-transplant.

What are the potential side effects?

Potential side effects include reactions related to the infusion of cells from donors, effects from chemotherapy like Busulfan and Fludarabine such as nausea or hair loss, risk of infection due to G-CSF usage, hormonal changes from Methylprednisolone, and tissue damage from Total Body Irradiation.

Eligibility Criteria

Inclusion Criteria

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

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I am under 65 years old.

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I have severe anemia, MDS, acute leukemia, or a high-risk genotype.

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My liver, heart, and lung functions meet the required health standards.

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I have been diagnosed with Fanconi anemia.

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I can care for myself but may not be able to do active work or play.

Exclusion Criteria

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

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I am not pregnant or breastfeeding and have a negative pregnancy test.

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I have not had an uncontrolled infection in the last week.

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I was diagnosed with a solid tumor cancer in the last 2 years.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 30 days after transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~30 days after transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and 30 days after 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

Grade II-IV acute graft versus host disease (GVHD)

Secondary study objectives

Acute graft versus host disease (aGVHD)

Bacterial, viral and fungal infections

Chronic graft versus host disease (cGVHD)

+5 more

Awards & Highlights

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Approved for 50 Other Conditions

This treatment demonstrated efficacy for 50 other conditions.

No Placebo-Only Group

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

Trial Design

5Treatment groups

Experimental Treatment

Group I: Treatment Plan 5: CY, FLU, melphalan (MEL), and alemtuzumab.Experimental Treatment4 Interventions

given to TBD patients with: * Early MDS features (with or without cytogenetic abnormalities) AND * Any donor type including haploidentical (4/8) to 8/8-HLA matched related donor, or 7-8/8 HLA-matched unrelated donor Based on historical numbers, it is expected approximately 2 patients would be treated per year. Statistical outcomes will be descriptive.

Group II: Treatment Plan 4: CY, FLU, and alemtuzumabExperimental Treatment3 Interventions

given to TBD patients with: * Bone marrow failure AND * Any donor type including haploidentical (4/8) to 8/8-HLA matched related donor, or 7-8/8 HLA-matched unrelated donor Based on historical numbers, it is expected approximately 3 patients would be treated per year. Statistical outcomes will be descriptive.

Group III: Treatment Plan 3: BU, Cy, FLU, MP and Rituximab in patients with Fanconi AnemiaExperimental Treatment5 Interventions

Given to: * Patients with an unrelated donor or HLA mismatched related donor, regardless of disease type who cannot tolerate TBI * Patients with an HLA- identical sibling donor recipient and MDS or acute leukemia who cannot tolerate TBI * Per treating physician preference

Group IV: Treatment Plan 2: CY, FLU and MP in patients with Fanconi AnemiaExperimental Treatment6 Interventions

Given to: • HLA-identical sibling donor recipients with aplastic anemia

Group V: Treatment Plan 1: TBI 300 with Thymic Shielding, CY, FLU, MP in patients with Fanconi AnemiaExperimental Treatment7 Interventions

Given to: * Patients with an unrelated donor or HLA mismatched related donor, regardless of disease type OR * Patients with an HLA- identical sibling donor recipient and MDS or acute leukemia

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Rituximab

FDA approved

Filgrastim

FDA approved

Busulfan

FDA approved

Alemtuzumab

2004