NCT03579875 Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
| NCT ID | NCT03579875 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Masonic Cancer Center, University of Minnesota |
| Condition | Fanconi Anemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 48 participants |
| Start Date | 2018-11-13 |
| Primary Completion | 2027-01-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 48 participants in total. It began in 2018-11-13 with a primary completion date of 2027-01-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.
Eligibility Criteria
Patient Selection: Inclusion Criteria: For FA patients: * Diagnosis of Fanconi anemia * Age \<65 years of age * Has one of the following risk factors: * Severe aplastic anemia (SAA) * Myelodysplastic features * High risk genotype * Immunodeficiency associated with history of recurrent infections * Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score ≥ 50% for patients \<16 years of age * Adequate pulmonary, cardiac and liver function * Voluntary written consent (minor assent if appropriate) prior to the performance of any study related procedures not part of standard medical care For TBD patients: • Diagnosis of TBD * Age \<70 years of age * Has one of the following risk factors: * Severe aplastic anemia (SAA) * Myelodysplastic features * Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score ≥ 50% for patients \<16 years of age * Adequate pulmonary, cardiac and liver function * Voluntary written consent (minor assent if appropriate) prior to the performance of any study related procedures not part of standard medical care Exclusion Criteria: * 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 * Malignant solid tumor cancer within previous 2 years Donor Selection (Inclusion Criteria): meets one of the following match criteria: * an HLA-A, B, DRB1 matched sibling donor (matched sibling) * an HLA-A, B, DRB1 matched related donor (other than sibling) * a related donor mismatched at 1 HLA-A, B, C and DRB1 antigen * 7-8/8 HLA-A,B,C,DRB1 allele matched unrelated donor per current institutional guidelines Patients and donors are typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing. If a donor has been selected on the basis of HLA-A, B, C and DRB1 typing as above, preference will be made for donors matched at the HLA-C locus. * Body weight of at least 40 kilograms and at least 12 years of age * Willing and able to undergo mobilized peripheral blood apheresis * In general good health as determined by the medical provider * Adequate organ function defined as: * Hematologic: hemoglobin, WBC, platelet within 10% of upper and lower limit of normal range of test (gender based for hemoglobin) * Hepatic: ALT \< 2 x upper limit of normal * Renal: serum creatinine \< 1.8 mg/dl * Performance of a donor infectious disease screen panel including CMV Antibody, Hepatitis B Surface Antigen, Hepatitis B Core Antibody, Hepatitis C Antibody, HIV 1/2 Antibody, HTLVA 1/2 Antibody, Treponema, and Trypanosoma Cruzi (T. Cruzi) plus HBV, HCV, WNV, HIV by nucleic acid testing (NAT); and screening for evidence of and risks factors for infection with Zika virus, or per current standard institutional donor screen - must be negative for HIV and active hepatitis B * Not pregnant - females of childbearing potential must have a negative pregnancy test within 7 days of mobilization start * Voluntary written consent (parent/guardian and minor assent, if \< 18 years) prior to the performance of any research related procedure
Contact & Investigator
Margaret MacMillan, MD, Msc, FRCPC
PRINCIPAL INVESTIGATOR
Masonic Cancer Center, University of Minnesota
Frequently Asked Questions
Who can join the NCT03579875 clinical trial?
This trial is open to participants of all sexes, up to 65 Years, studying Fanconi Anemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT03579875 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT03579875 currently recruiting?
Yes, NCT03579875 is actively recruiting participants. Contact the research team at macmi002@umn.edu for enrollment information.
Where is the NCT03579875 trial being conducted?
This trial is being conducted at Minneapolis, United States.
Who is sponsoring the NCT03579875 clinical trial?
NCT03579875 is sponsored by Masonic Cancer Center, University of Minnesota. The principal investigator is Margaret MacMillan, MD, Msc, FRCPC at Masonic Cancer Center, University of Minnesota. The trial plans to enroll 48 participants.