Unrelated HSCT in Patients With Fanconi Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02127905
• Date of registration: 19/10/2012
• Prospective Registration: No
• Primary sponsor: Neena Kapoor, M.D.
• Public title: Unrelated HSCT in Patients With Fanconi Anemia
• Scientific title: A Study of Total Body Irradiation, Cyclophosphamide and Fludarabine Followed by Alternated Donor Hematopoietic Cell Transplantation in Patients With Fanconi Anemia
• Date of first enrolment: March 2011
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT02127905
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: N/A

Countries of recruitment

United States

Contacts

• Name: Neena Kapoor, M.D.
• Affiliation: Children's Hospital Los Angeles, University of Southern California

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients must be > 2 months and < 21 years of age with a diagnosis of Fanconi anemia.

- Patients must have an HLA-A, B, DRB1 identical or 1 antigen mismatched related
(non-sibling) or unrelated donor. Patients and donors will be typed for HLA-A and B
using serological or molecular techniques and for DRB1 using high resolution molecular
typing. (Patients with a 2 antigen mismatched related donor will be eligible for the
protocol but evaluated separately).

- Patients with FA must have high risk genotype or aplastic anemia (AA) or
myelodysplastic syndrome without excess blasts.

- Aplastic anemia is defined as having at least one of the following:

1. platelet count <20 x 109/L

2. ANC <5 x 108/L

3. Hgb <8 g/dL with at least one of the following:

1. transfusion dependence

2. supportive care toxicity

- Myelodysplastic syndrome with multilineage dysplasia with or without chromosomal
anomalies.

- High risk genotype (e.g. IVS-4 or exon 14 FANCC mutations)

- Adequate major organ function including:

- Cardiac: ejection fraction >45%

- Renal: creatinine clearance >40 mL/min.

- Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)

- Karnofsky performance status >70% or Lansky >50%

- Women of child bearing age must be using adequate birth control and have a negative
pregnancy test.

Exclusion Criteria:

- Available HLA-genotypically identical related donor.

- The harvested marrow (prior to TCD) should contain a minimum of 2.5 x 108 nucleated
cells/kg recipient body weight with a goal of >5.0 x 108 nucleated cells/kg recipient
body weight.

- Positive lymphocytotoxic crossmatch against donor (T cells and B cells)

- History of gram negative sepsis or systemic fungal infection (proven or suspected
based on radiographic studies).

- Myelodysplastic syndrome with excess blasts or leukemia.

- Active CNS leukemia at time of HCT.

- Malignant solid tumor (e.g. squamous cell carcinoma of the head/neck/cervix) within 2
years of HCT.

- Pregnant or lactating female.

- Prior radiation therapy preventing use of TBI 450 cGy.



Age minimum: 8 Weeks
Age maximum: 21 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fanconi Anemia

Intervention(s)

Biological: CD34+ selected cells

Primary Outcome(s)

Event free survival post stem cell transplant [Time Frame: 5 years]

Secondary Outcome(s)

Peripheral blood CBC counts for engraftment evaluation [Time Frame: 3 years]

Graft Versus Host Disease (GVHD) surveillance after HSCT [Time Frame: 3 years]

Chimerism assay for engraftment evaluation [Time Frame: 3 years]

Secondary ID(s)

CCI-10-00176

Source(s) of Monetary Support

Please refer to primary and secondary sponsors