Unrelated HSCT in Patients With Fanconi Anemia

NCT02127905 | Withdrawn DMC

• 1 other identifier: CCI-10-00176
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The protocol is designed for the compassionate treatment of patients with Fanconi Anemia who do not have an HLA-matched sibling donor. The purpose of this study is to determine the likelihood of engraftment in Fanconi Anemia patients using total body irradiation (TBI), cyclophosphamide (CY), fludarabine (FLU) and antithymocyte globulin (ATG) followed by an unrelated donor hematopoietic cell transplant with T-cell depletion using the CliniMACS device.

Conditions

Fanconi Anemia

Keywords

Fanconi Anemia; compassionate treatment; BMT; HSCT; unrelated; bone marrow transplantation; PBSC; peripheral blood; CD34+

Outcome Measures

Primary Outcomes (1)

• Event free survival post stem cell transplant

  Patients who are alive at 5 years post transplants with assessments at 30, 60, 90, 180 and yearly up to 5 years

  5 years

Secondary Outcomes (3)

• Peripheral blood CBC counts for engraftment evaluation

  3 years

• Chimerism assay for engraftment evaluation

  3 years

• Graft Versus Host Disease (GVHD) surveillance after HSCT

  3 years

Study Arms (1)

CD34+ selected cells

OTHER

use of unrelated bone marrow or peripheral blood for hematopoietic stem cell transplantation with CD34+ selected cells

Biological: CD34+ selected cells

Interventions

CD34+ selected cells BIOLOGICAL

Compassionate treatment of Fanconi Anemia patients with unrelated bone marrow or peripheral blood HSCT followed by the infusion of CD34+ selected cells using CliniMACS

Also known as: CD34+ selected cells using CliniMACS

CD34+ selected cells

Eligibility Criteria

• Age: 8 Weeks - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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:
• platelet count \<20 x 109/L
• ANC \<5 x 108/L
• Hgb \<8 g/dL with at least one of the following:
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• transfusion dependence
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Neena Kapoor, M.D.: lead

Study Sites (1)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

MeSH Terms

Conditions

Fanconi Anemia

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, Congenital; Anemia, Aplastic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Congenital Bone Marrow Failure Syndromes; Bone Marrow Failure Disorders; Bone Marrow Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Neena Kapoor, M.D.

  Children's Hospital Los Angeles, University of Southern California

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Pediatrics, Keck School of Medicine; Division Head, Division of Research Immunology/BMT

Study Record Dates

• First Submitted: October 19, 2012
• First Posted: May 1, 2014
• Study Start: March 1, 2011
• Primary Completion: March 1, 2016
• Study Completion: March 1, 2016
• Last Updated: July 11, 2017

Record last verified: 2017-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, CSR
• Time Frame: End of subject enrollment and following 2 year subject follow-up
• Access Criteria: Researchers/Investigators interested in severe combined immune deficiency disease treatment and outcomes.

Locations

US (1)