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New treatment option for Fanconi Anemia
Official title Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
ClinicalTrials.gov ID: NCT03579875
What this study is testing
What is Total Body Irradiation (TBI) (Plan 1)?
Total Body Irradiation (TBI) (Plan 1) is an investigational medicine, being studied as a potential treatment for fanconi anemia.
Also referred to as TBI.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 65
You may be able to join if
- Patient Selection: For FA patients:
- Diagnosis of Fanconi anemia
- Age \<65 years of age
- Has one of the following risk factors:
- Severe aplastic anemia (SAA)
You likely can't join if
- Pregnant or breastfeeding as the treatment used in this study are Pregnancy Category D. Females of childbearing potential must have a negative...
- 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
See the full eligibility criteria
- Patient Selection: 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
- 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...
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Minneapolis, Minnesota, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, up to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Minneapolis, Minnesota, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.