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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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified March 2001 by Office of Rare Diseases (ORD).
Recruitment status was  Active, not recruiting
Sponsor:
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00005896
First received: June 2, 2000
Last updated: June 23, 2005
Last verified: March 2001
History of Changes
  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
Genetic: Autologous stem cells transduced with FACC retroviral vector
Procedure: Autologous Stem Cell Transplantation
 Phase 1

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:

MedlinePlus related topics: Anemia
U.S. FDA Resources

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 - Clinical and Translational Science Institute
Investigators
Study Chair: John E. Wagner, Jr. University of Minnesota - Clinical and Translational Science Institute
  More Information

No publications provided

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

Keywords provided by Office of Rare Diseases (ORD):
Fanconi's anemia
aplastic anemia
hematologic disorders
rare disease

Additional relevant MeSH terms:
Anemia
Fanconi Anemia
Fanconi Syndrome
Hematologic Diseases
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Bone Marrow Diseases
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
 Kidney Diseases
Urologic Diseases
Renal Tubular Transport, Inborn Errors
Metabolism, Inborn Errors
Lenograstim
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 19, 2013