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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

June 2, 2000
June 23, 2005
March 2000
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Complete list of historical versions of study NCT00005896 on ClinicalTrials.gov Archive Site
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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
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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.

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.

Interventional
Phase 1
Primary Purpose: Treatment
Fanconi's Anemia
  • Drug: filgrastim
  • Genetic: Autologous stem cells transduced with FACC retroviral vector
  • Procedure: Autologous Stem Cell Transplantation
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
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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
Both
5 Years and older
No
Contact information is only displayed when the study is recruiting subjects
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NCT00005896
199/15107, UMN-MT-1997-10, UNM-MT-9710
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University of Minnesota - Clinical and Translational Science Institute
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Study Chair: John E. Wagner, Jr. University of Minnesota - Clinical and Translational Science Institute
Office of Rare Diseases (ORD)
March 2001

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP