Transplantation for inherited bone marrow failure disorders
MT2017-17:T Cell Receptor Alpha/Beta T Cell Depleted Hematopoietic Cell Transplantation in Patients With Inherited Bone Marrow Failure (BMF) Disorders
This study is testing a new way to do stem cell transplants for people with inherited bone marrow failure disorders to see if it can help them recover faster and reduce the risk of infections without needing extra medication.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 48 (estimated) |
| Ages | N/A to 65 Years |
| Sex | All |
| Sponsor | Masonic Cancer Center, University of Minnesota Academic / other |
| Locations | 1 site (Minneapolis, Minnesota) |
| Trial ID | NCT03579875 on ClinicalTrials.gov |
What this trial studies
This phase II trial investigates the use of T cell receptor alpha/beta depletion in peripheral blood stem cell transplantation for patients with inherited bone marrow failure disorders. The approach aims to eliminate the need for routine graft-versus-host disease immune suppression, which could lead to earlier immune recovery and a reduced risk of severe infections post-transplantation. The study includes patients with conditions such as Fanconi Anemia and Severe Aplastic Anemia, and involves interventions like total body irradiation and chemotherapy.
Who should consider this trial
Good fit: Ideal candidates include patients under 65 years with Fanconi Anemia or under 70 years with other specified inherited bone marrow failure disorders.
Not a fit: Patients with advanced age or those without the specified inherited bone marrow failure disorders may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could significantly improve immune recovery and reduce infection risks for patients undergoing stem cell transplantation.
How similar studies have performed: Other studies have shown promise with similar approaches in stem cell transplantation, indicating potential for success in this trial.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Minneapolis, Minnesota
- Masonic Cancer Center at University of Minnesota — Minneapolis, Minnesota, United States (Recruiting)
Study contacts
- Principal investigator: Margaret MacMillan, MD, Msc, FRCPC — Masonic Cancer Center, University of Minnesota
- Study coordinator: Margaret MacMillan, MD, Msc, FRCPC
- Email: macmi002@umn.edu
- Phone: 612-626-2961
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.