Phase I Pilot Study of CD34 Enriched, Fanconi's Anemia Complementation Group C Gene Transduced Autologous Peripheral Blood Stem Cell Transplantation in Patients With Fanconi's Anemia

NCT00005896 | Unknown Phase 1

• 3 other identifiers: 199/15107UMN-MT-1997-10UNM-MT-9710
• Study Type: interventional
• Target: 6
• Locations: 0 countries
• Sites: N/A

Brief Summary

OBJECTIVES: I. Determine the safety of transferring the Fanconi anemia complementation group C (FACC) gene to hematopoietic progenitors by retroviral mediated gene transfer in patients with Fanconi's anemia, complementation group C. II. Determine the extent of engraftment following this treatment regimen without prior ablation of recipient marrow in these patients. III. Determine the ability of this treatment regimen to correct the cell phenotype and improve hematopoietic function in these patients.

Conditions

Fanconi's Anemia

Keywords

Fanconi's anemia; aplastic anemia; hematologic disorders; rare disease

Interventions

filgrastim DRUG

Autologous stem cells transduced with FACC retroviral vector GENETIC

Autologous Stem Cell Transplantation PROCEDURE

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- * Fanconi's anemia, complementation group C (FACC) Confirmed by diepoxybutane or mitomycin testing AND DNA analysis indicating FACC mutations * Patients at least 25 kg weight * No acute leukemia OR Bone marrow aspirate with greater than 10% blasts * No patients who elect bone marrow transplantation --Prior/Concurrent Therapy-- * At least 14 days since prior therapy for any acute viral, bacterial, or fungal infection --Patient Characteristics-- * Performance status: Karnofsky 40-100% * Hepatic: SGOT, SGPT, and alkaline phosphatase no greater than 5 times upper limit of normal (ULN) PT/PTT no greater than 1.5 times ULN Serum amylase no greater than 1.5 times ULN Bilirubin no greater than 2.5 mg/dL Triglyceride less than 400 mg/dL * Renal: Creatinine clearance greater than 50 mL/min * Cardiovascular: Normal cardiac function No ischemic heart disease that may be considered an anesthetic or operative risk * Pulmonary: No lung disease that may be considered an anesthetic or operative risk Resting transcutaneous oxygen saturation greater than 90% on room air * Other: HIV negative Hepatitis B surface antigen negative No underlying condition that may preclude study therapy (e.g., allergies to study reagents) * No acute viral, bacterial, or fungal infection Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• University of Minnesota: lead

MeSH Terms

Conditions

Fanconi Anemia; Anemia, Aplastic; Hematologic Diseases; Rare Diseases

Interventions

Filgrastim

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, Congenital; Anemia; 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; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Granulocyte Colony-Stimulating Factor; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• John E. Wagner, Jr.

  Masonic Cancer Center, University of Minnesota

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Purpose: TREATMENT
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: June 2, 2000
• First Posted: June 5, 2000
• Study Start: March 1, 2000
• Last Updated: June 24, 2005

Record last verified: 2001-03