Transplantation for inherited bone marrow failure disorders

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• 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

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Understanding long-term effects of bone marrow failure syndromes

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.