Clinical trial 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, given as an IV infusion (through a vein), 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 study wants to find out if using a lower dose of busulfan and removing cyclosporine can lower side effects from the transplant.
- Who it's for
- People with Fanconi anemia who have severe marrow failure, myelodysplastic syndrome, or acute myelogenous leukemia and need a stem‑cell transplant.
- What's involved
- Participants will receive a stem‑cell transplant from a mismatched related or matched unrelated donor after a chemotherapy prep that includes busulfan, fludarabine, anti‑thymocyte globulin and cyclophosphamide.
- Time commitment
- The study continues through the transplant and post‑transplant follow‑up, with visits for screening, the hospital stay for the transplant, and later check‑ups.
- 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
- 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
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
- 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%.
- 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 is enrolling
- 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, 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, 3 months and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Cincinnati, Ohio, United States; Seattle, Washington, 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.