Clinical trial · Interventional
HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine
NCT02143830CI-TRIAL-00097136RAFArecruitingPhase 2ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (4)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Acute Myelogenous Leukemia (AML) | Acute Myeloid Leukemia | ALIAS | 0.85 |
| Fanconi Anemia | — | UNRESOLVED | — |
| Myelodysplastic Syndrome (MDS) | Myelodysplastic Syndrome | CURATED_BROADER | 0.80 |
| Severe Marrow Failure | — | UNRESOLVED | — |
Interventions
Interventions (6)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Busulfan | Drug | Busulfan | ALIAS |
| Cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| Fludarabine | Drug | Fludarabine | ALIAS |
| G-CSF | Drug | — | UNRESOLVED |
| Peripheral blood stem cell | Biological | — | UNRESOLVED |
| rabbit ATG | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (3)
- type
- EXPERIMENTAL
- label
- Arm A: Good Risk Patients
- description
- Patients 18 years old or younger with marrow aplasia or single lineage cytopenias will be receive intravenous busulfan (4 doses x 2 days), cyclophosphamide (4 doses x 4 days) and fludarabine (4 doses x 4 days), as used in our most current study that led to \> 90% survival rates . Busulfan pharmacokinetics will not be used. All patients will receive rabbit ATG (4 doses x 4 days) prior to and granulocyte-colony stimulating factor (G-CSF) after transplant to promote engraftment. Cyclosporine will not be used for GVHD prophylaxis. The source of the stem cells for all patients will be peripheral blood stem cells mobilized by treatment of the donor with G-CSF. T-cell depletion will be performed by positive CD34 selection with the use of the Miltenyi system (CliniMACS device). Enrollment on this arm will include up to 50 patients.
- interventionNames
- Drug: Busulfan
- Drug: Cyclophosphamide
- Drug: Fludarabine
- Drug: rabbit ATG
- Drug: G-CSF
- Biological: Peripheral blood stem cell
- type
- EXPERIMENTAL
- label
- Arm B: Intermediate Risk Patients
- description
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 3 Months
Show eligibility criteria text
Inclusion Criteria:
* Patients must have a diagnosis of Fanconi anemia
* Patients must have one of the following hematologic diagnoses:
1. Severe Aplastic Anemia (SAA), with bone marrow cellularity of \<25% OR Severe Isolated Single Lineage Cytopenia and at least one of the following features:
1. Platelet count \<20 x 109/L or platelet transfusion dependence\*
2. ANC \<1000 x 109/L
3. Hgb \<8 gm/dl or red cell transfusion dependence\*
2. Myelodysplastic Syndrome (MDS) (based on WHO or IPSS Classification
3. 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:
1. 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%
2. Hepatic: \< 5 x upper limit of normal (ULN) alanine transaminase (ALT) and \< 2.0 mg/dl total serum bilirubin.
3. Renal: serum creatinine \<1.5 mg/dl or if serum creatinine is outside the normal range, then CrCl \> 50 ml/min/1.73 m2
4. 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.
Exclusion Criteria:
* 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/IIReferences
Publications (0)
Data not yet available
No reference posted for this study.