Transplant for Patients with Fanconi Anemia Using 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
This study is testing a new way to use lower doses of chemotherapy before a stem cell transplant to see if it can safely help people with Fanconi anemia and serious blood disorders.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 70 (estimated) |
| Ages | 3 Months and up |
| Sex | All |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Drugs / interventions | cyclophosphamide, fludarabine, chemotherapy |
| Locations | 3 sites (New York, New York and 2 other locations) |
| Trial ID | NCT02143830 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the safety and efficacy of a risk-adjusted chemotherapy regimen combined with a stem cell transplant for patients with Fanconi anemia and severe hematologic conditions. Participants will receive a preparative regimen that includes lower doses of busulfan, fludarabine, anti-thymocyte globulin, and cyclophosphamide, followed by a transplant using either mismatched related or matched unrelated donors. The study is designed to minimize transplant-related side effects while effectively treating severe marrow failure, myelodysplastic syndrome, or acute myelogenous leukemia.
Who should consider this trial
Good fit: Ideal candidates include patients diagnosed with Fanconi anemia who also have severe marrow failure, myelodysplastic syndrome, or acute myelogenous leukemia.
Not a fit: Patients without a diagnosis of Fanconi anemia or those who do not meet the specific hematologic criteria may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could lead to safer transplant procedures with fewer side effects for patients with Fanconi anemia.
How similar studies have performed: Other studies have shown promise with similar chemotherapy approaches in stem cell transplants, suggesting potential for success in this trial.
Eligibility criteria
Show full inclusion / exclusion criteria
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/II
Where this trial is running
New York, New York and 2 other locations
- Memorial Sloan Kettering Cancer Center — New York, New York, United States (Completed)
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Recruiting)
- Fred Hutchinson Cancer Research Center — Seattle, Washington, United States (Recruiting)
Study contacts
- Principal investigator: Parinda Mehta, MD — Cchmc
- Study coordinator: Jamie Wilhelm
- Email: Jamie.Wilhelm@cchmc.org
- Phone: (513)803-1102
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.