Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine

NCT00595127 | Completed Results DMC

• 1 other identifier: 01-062
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this research study is to: (1) determine if the combination of low dose total body irradiation, low dose cyclophosphamide and the addition of fludarabine, and a serum to suppress the immune system can allow selected stem cells to take and grow; (2) determine if selected stem cells from the blood or marrow can take and not cause graft-versus-host disease (GvHD), and; (3) evaluate the side effects of the combination of low dose radiation and chemotherapy drugs used for these transplants.

Conditions

Fanconi Anemia

Keywords

Fanconi; Anemia; Stem Cell; Transplant

Outcome Measures

Primary Outcomes (1)

• Incidence & Quality of Engraftment & Hematopoietic Reconstitution

  Number of patients who engrafted

  8 years

Study Arms (1)

1

EXPERIMENTAL

This is an open-label single arm study of 131I-8H9, injected intravenously at 10 mCi/1.73 m\^2 dose \[intended specific activity of \~20 mCi/mg protein\] preceded by administration of 50mg/1.73m\^2 of unlabeled 8H9.

Drug: Cyclophosphamide; Drug: Fludarabine; Radiation: Total body irradiation (TBI)

Interventions

Cyclophosphamide DRUG

Cyclophosphamide (10mg/kg/day x 4days)

1

Fludarabine DRUG

Fludarabine (30mg/m\^2/day x 5 days)

1

Total body irradiation (TBI) RADIATION

TBI (450 cGY in a single fraction)

1

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis:
• Research participants must be have a diagnosis of Fanconi anemia (confirmed by mitomycin or diepoxybutane \[DEB\] chromosomal breakage testing).
• Hematologic Diagnosis and Status:
• Research participants must have one of the following hematologic diagnoses:
• Severe Aplastic Anemia (SAA)/Severe Isolated Single lineage Cytopenia
• Myelodysplastic Syndrome(MDS)
• Acute leukemia.
• HLA-compatible Unrelated volunteer donors:
• Research participants who do not have a related HLA-matched donor but have an unrelated donor who is either matched at all A, B and DRB1 loci or who is mismatched at 1/6 loci (A, B, or DRB1) as tested by DNA analysis, will be eligible for entry on this protocol.
• HLA-mismatched Related donors:
• Research participants who do not have a related or unrelated HLA-compatible donor must have a healthy family member who is at least HLA-haplotype identical to the recipient. First degree related donors must have a normal DEB test.
• The donor must be healthy and willing and able (1) to receive a 5 day course of G-CSF and undergo 2 daily leukaphereses, or (2) to undergo general anesthesia and bone marrow donation. In order to undergo a Tcell depletion, a donor should be able to have a volume of 15 ml/Kg of the research participant's body weight harvested safely.
• HLA-compatible Cord Blood Units:
• Research participants who do not have a related HLA-matched donor but have an unrelated placental cord blood unit which matched at all A, B and DRB1 loci or who is mismatched at 1/6 or 2/6 loci (A, B, or DRB1) as tested by DNA analysis, will be eligible for entry on this protocol.
• Research participants may be of either gender or any ethnic background.

You may not qualify if:

• Active CNS leukemic involvement
• Female research participants who are pregnant or breast-feeding
• Active viral, bacterial or fungal infection
• Research participant seropositive for HIV-I/II; HTLV -I/II

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center: lead
• National Cancer Institute (NCI): collaborator
• Miltenyi Biotec, Inc.: collaborator

Study Sites (1)

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Related Links

• Memorial Sloan-Kettering Cancer Center

MeSH Terms

Conditions

Fanconi Anemia; Anemia

Interventions

Cyclophosphamide; fludarabine; Whole-Body Irradiation

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, Congenital; Anemia, Aplastic; 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

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Radiotherapy; Therapeutics; Investigative Techniques

Results Point of Contact

• Title: Farid Boulad, MD, Attending
• Organization: Memorial Sloan Kettering Cancer Center

bouladf@mskcc.org

+1212-639-2429

Study Officials

• Farid Boulad, MD

  Memorial Sloan Kettering Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 7, 2008
• First Posted: January 16, 2008
• Study Start: June 1, 2001
• Primary Completion: May 1, 2013
• Study Completion: May 1, 2013
• Last Updated: August 10, 2016
• Results First Posted: August 10, 2016

Record last verified: 2016-06

Locations

US (1)