Clinical trial 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)?
This is an investigational radiation treatment being studied as part of a transplant procedure for Fanconi Anemia. It is designed to use low-dose radiation to help prepare the body for new stem cells while protecting the thymus.
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
- The study wants to see if removing alpha/beta T cells from the transplant reduces the need for drugs that prevent graft‑versus‑host disease and speeds up immune recovery.
- Who it's for
- It is looking for adults or children who have inherited bone marrow failure disorders such as Fanconi anemia, severe aplastic anemia, or telomere biology disorders.
- What's involved
- Participants will receive a peripheral blood stem cell transplant that has had alpha/beta T cells removed. They will have regular clinic visits and blood tests to monitor recovery and any complications.
- 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
- Diagnosis of Fanconi anemia
- Age \<65 years of age
- Severe aplastic anemia (SAA)
- Myelodysplastic features
- High risk genotype
You likely can't join if
- Pregnant or breastfeeding as the treatment used in this study are Pregnancy Category D.
- Active, uncontrolled infection within 1 week prior to starting study therapy
- 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...
See the full eligibility criteria
- Diagnosis of Fanconi anemia
- Age \<65 years of age
- 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
- Diagnosis of TBD
- Age \<70 years of age
- 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
- 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
- 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 is enrolling
- 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, but 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?
This study is enrolling now at Minneapolis, Minnesota, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.