HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

• Register: ClinicalTrials.gov
• Last refreshed on: 22 January 2024
• Main ID: NCT02143830
• Date of registration: 28/04/2014
• Prospective Registration: No
• Primary sponsor: Children's Hospital Medical Center, Cincinnati
• Public title: HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy RAFA
• Scientific title: 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
• Date of first enrolment: April 2014
• Target sample size: 70
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02143830
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Jamie Wilhelm

• Name: Sheri Ballard

• Name: Parinda Mehta, MD
• Affiliation: CCHMC

Key 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



Age minimum: 3 Months
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Acute Myelogenous Leukemia (AML)

Myelodysplastic Syndrome (MDS)

Fanconi Anemia

Severe Marrow Failure

Intervention(s)

Drug: Fludarabine

Drug: G-CSF

Drug: Busulfan

Drug: Cyclophosphamide

Drug: rabbit ATG

Biological: Peripheral blood stem cell

Primary Outcome(s)

Graft Failure or Rejection [Time Frame: 5 years]

Secondary Outcome(s)

Post-transplant severe morbidity and mortality [Time Frame: 2 years post-transplant]

Secondary ID(s)

2013-7501

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Fred Hutchinson Cancer Center

Memorial Sloan Kettering Cancer Center