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New treatment option for Fanconi Anemia
Official title HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
ClinicalTrials.gov ID: NCT02143830
What this study is testing
What is Busulfan?
Busulfan is an investigational medicine, being studied as a potential treatment for fanconi anemia.
Also referred to as Myleran, Busulfex IV.
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 purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.
- 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 3 and older
You may be able to join if
- Patients must have a diagnosis of Fanconi anemia
- Patients must have one of the following hematologic diagnoses:
- Severe Aplastic Anemia (SAA), with bone marrow cellularity of \<25% OR Severe Isolated Single Lineage Cytopenia and at least one of the following...
- Platelet count \<20 x 109/L or platelet transfusion dependence\
- ANC \<1000 x 109/L
You likely can't join if
- Active CNS leukemia
- Female patients who are pregnant (positive serum or urine HCG) or breast-feeding.
- Active uncontrolled viral, bacterial or fungal infection
- Patient seropositive for HIV-I/II; HTLV -I/II
See the full eligibility criteria
- Patients must have a diagnosis of Fanconi anemia
- Patients must have one of the following hematologic diagnoses:
- Severe Aplastic Anemia (SAA), with bone marrow cellularity of \<25% OR Severe Isolated Single Lineage Cytopenia and at least one of the following features:
- Platelet count \<20 x 109/L or platelet transfusion dependence\
- ANC \<1000 x 109/L
- Hgb \<8 gm/dl or red cell transfusion dependence\
- Myelodysplastic Syndrome (MDS) (based on WHO or IPSS Classification
- Acute Myelogenous Leukemia (untreated, in remission or with refractory or relapsed disease)
- Donors will be either human leukocyte antigen (HLA) compatible unrelated or HLA-genotypically matched related donors (no fully matched sibling donor).
- Patients and donors may be of either gender or any ethnic background.
- Patients must have a Karnofsky adult, or Lansky pediatric performance scale status \> 70%.
- Patients must have adequate physical function measured by:
- Cardiac: asymptomatic or if symptomatic then 1) left ventricular ejection fraction (LVEF) at rest must be \> 50% and must improve with exercise or 2) Shortening Fraction \> 29%
- Hepatic: \< 5 x upper limit of normal (ULN) alanine transaminase (ALT) and \< 2.0 mg/dl total serum bilirubin.
- Renal: serum creatinine \ 50 ml/min/1.73 m2
- Pulmonary: asymptomatic or if symptomatic, DLCO \> 50% of predicted
- Each patient must be willing to participate as a research subject and must sign an informed consent form.
- Female patients and donors must not be pregnant or breastfeeding at the time of signing consent. Women must be willing to undergo a pregnancy test prior to transplant and avoid becoming pregnant while on study.
- Active CNS leukemia
- Female patients who are pregnant (positive serum or urine HCG) or breast-feeding.
- Active uncontrolled viral, bacterial or fungal infection
- Patient seropositive for HIV-I/II; HTLV -I/II
The study team makes the final eligibility decision.
Where it's taking place
- New York, New York, United States
- Cincinnati, Ohio, United States
- Seattle, Washington, 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, 3 months and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include New York, New York, United States; Cincinnati, Ohio, United States; Seattle, Washington, 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.