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

This study is ongoing, but not recruiting participants.

First Received: June 2, 2000 Last Updated: June 23, 2005 History of Changes

Sponsored by:	University of Minnesota
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00005896

Purpose

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.

Condition	Intervention	Phase
Fanconi's Anemia	Drug: filgrastim Gene Transfer: Autologous stem cells transduced with FACC retroviral vector Procedure: Autologous Stem Cell Transplantation	Phase I

MedlinePlus related topics: Anemia

Drug Information available for: Filgrastim

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment:	6
Study Start Date:	March 2000

Detailed Description:

PROTOCOL OUTLINE: Patients receive filgrastim (G-CSF) subcutaneously daily on days 0-6 followed by apheresis to collect peripheral blood stem cells (PBSC) on days 5-7. PBSCs are processed in vitro for enrichment of CD34 cells and transduced with a Fanconi's anemia complementation C (FACC) retroviral vector on days 5-10. Patients receive transduced PBSCs IV over no more than 2 hours on days 8-10. PBSC infusions may be repeated no more than every 2 months for up to 4 courses total. Patients are followed monthly for 3 months, every 3 months for 9 months, every 6 months for the next year, and then yearly thereafter.

Eligibility

Ages Eligible for Study:	5 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00005896

Sponsors and Collaborators

University of Minnesota

Investigators

Study Chair:

John E. Wagner, Jr.

University of Minnesota

More Information

No publications provided

Study ID Numbers:	199/15107, UMN-MT-1997-10, UNM-MT-9710
Study First Received:	June 2, 2000
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00005896 History of Changes
Health Authority:	Unspecified

Keywords provided by Office of Rare Diseases (ORD):

Fanconi's anemia
aplastic anemia
hematologic disorders
rare disease

Study placed in the following topic categories:

Metabolic Diseases
Genetic Diseases, Inborn
Aplastic Anemia
Hematologic Diseases
Fanconi Anemia
Rare Diseases

Anemia, Aplastic
Anemia
Bone Marrow Diseases
Metabolic Disorder
Fanconi's Anemia

Additional relevant MeSH terms:

Metabolic Diseases
Anemia, Hypoplastic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Fanconi Anemia

DNA Repair-Deficiency Disorders
Anemia, Aplastic
Anemia
Bone Marrow Diseases

ClinicalTrials.gov processed this record on May 07, 2009