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CAR T-cell Therapy

TCD HCT for Fanconi Anemia

Q: What therapeutic benefits have been observed from G-CSF?

Granulocyte-colony stimulating factor (G-CSF) is a viable therapy for treating leukaemia, ophthalmia, sympathetic disorders, and scalp structure abnormalities.

Q: Is enrollment open for this research experiment?

The clinical research project detailed on clinicaltrials.gov is presently recruiting volunteers, having initially been posted on 13th November 2018 and updated for the final time on 2nd November 2022.

Q: What regulatory approval has G-CSF obtained?

As this study is of the Phase 2 variety, meaning there is evidence for safety but not efficacy, G-CSF has been rated with a score of 2.

Q: What is the current sample size of this experiment?

Confirmed. According to clinicaltrials.gov, this medical trial that was initially posted on November 13th 2018 is actively searching for participants across one site and seeks 48 patients in total.

Q: What findings have other researchers made regarding G-CSF?

Currently, 1337 trials are running to study the efficacy of G-CSF. 265 of those studies have reached Phase 3. Although most experiments for this drug are located in Bethesda, <a href="https://www.withpower.com/clinical-trials/maryland">Maryland</a>, 42190 sites globally offer research on its effectiveness.

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year after transplant

Awards & highlights

Study 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.Check my eligibility

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.See study design

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.

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

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

Primary outcome measures

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

Secondary outcome measures

Acute graft versus host disease (aGVHD)

Bacterial, viral and fungal infections

Chronic graft versus host disease (cGVHD)

+5 more

Trial Design

3Treatment groups

Experimental Treatment

Group I: Treatment Plan 3: BU, Cy, FLU, MP and RituximabExperimental 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 II: Treatment Plan 2: CY, FLU and MPExperimental Treatment6 Interventions

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

Group III: Treatment Plan 1: TBI 300 with Thymic Shielding, CY, FLU, MPExperimental 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

Busulfan

FDA approved

Filgrastim

FDA approved

Rituximab

FDA approved

0 / 5Eligibility criteria met

Find a Location

Who is running the clinical trial?

Masonic Cancer Center, University of MinnesotaLead Sponsor

271 Previous Clinical Trials

14,577 Total Patients Enrolled

6 Trials studying Fanconi Anemia

357 Patients Enrolled for Fanconi Anemia

Margaret MacMillan, MD, Msc, FRCPCPrincipal InvestigatorMasonic Cancer Center, University of Minnesota

Media Library

T Cell Receptor α/β TCD HCT (CAR T-cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03579875 — Phase 2

Fanconi Anemia Research Study Groups: Treatment Plan 2: CY, FLU and MP, Treatment Plan 3: BU, Cy, FLU, MP and Rituximab, Treatment Plan 1: TBI 300 with Thymic Shielding, CY, FLU, MP

Fanconi Anemia Clinical Trial 2023: T Cell Receptor α/β TCD HCT Highlights & Side Effects. Trial Name: NCT03579875 — Phase 2

T Cell Receptor α/β TCD HCT (CAR T-cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03579875 — Phase 2

Frequently Asked Questions

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

What therapeutic benefits have been observed from G-CSF?

"Granulocyte-colony stimulating factor (G-CSF) is a viable therapy for treating leukaemia, ophthalmia, sympathetic disorders, and scalp structure abnormalities."

Answered by AI

Is enrollment open for this research experiment?

"The clinical research project detailed on clinicaltrials.gov is presently recruiting volunteers, having initially been posted on 13th November 2018 and updated for the final time on 2nd November 2022."

Answered by AI

What regulatory approval has G-CSF obtained?

"As this study is of the Phase 2 variety, meaning there is evidence for safety but not efficacy, G-CSF has been rated with a score of 2."

Answered by AI

What is the current sample size of this experiment?

"Confirmed. According to clinicaltrials.gov, this medical trial that was initially posted on November 13th 2018 is actively searching for participants across one site and seeks 48 patients in total."

Answered by AI

What findings have other researchers made regarding G-CSF?

"Currently, 1337 trials are running to study the efficacy of G-CSF. 265 of those studies have reached Phase 3. Although most experiments for this drug are located in Bethesda, Maryland, 42190 sites globally offer research on its effectiveness."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

T Cell Receptor α/β TCD HCT in Patients With Fanconi Anemia

2018. "T Cell Receptor α/β TCD HCT in Patients With Fanconi Anemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03579875.

All > Aplastic Anemia > Myelodysplastic Syndrome