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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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ClinicalTrials.gov Identifier: NCT00005896
Recruitment Status : Unknown
Verified March 2001 by Office of Rare Diseases (ORD).
Recruitment status was:  Active, not recruiting
First Posted : June 5, 2000
Last Update Posted : June 24, 2005
Sponsor:
Information provided by:

Study Description
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.


Condition or disease Intervention/treatment Phase
Fanconi's Anemia Drug: filgrastim Genetic: Autologous stem cells transduced with FACC retroviral vector Procedure: Autologous Stem Cell Transplantation Phase 1

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.


Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Primary Purpose: Treatment
Study Start Date : March 2000


Arms and Interventions


Outcome Measures

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00005896


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

ClinicalTrials.gov Identifier: NCT00005896     History of Changes
Other Study ID Numbers: 199/15107
UMN-MT-1997-10
UNM-MT-9710
First Posted: June 5, 2000    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: March 2001

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

Additional relevant MeSH terms:
Fanconi Anemia
Fanconi Syndrome
Anemia
Hematologic Diseases
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Bone Marrow Diseases
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metabolism, Inborn Errors