NCT00271089

Brief Summary

Fanconi anemia (FA) is a disease that affects an individual's bone marrow. It is caused by a defective gene in the CD34+ cells, which are responsible for producing various types of blood cells. Individuals with FA may experience fatigue, bleeding, and increased infections. The purpose of this study is to collect and purify blood cells from individuals with FA and store them for future therapeutic use.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
40

participants targeted

Target at P25-P50 for not_applicable

Timeline
Completed

Started Aug 2004

Typical duration for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2004

Completed
1.4 years until next milestone

First Submitted

Initial submission to the registry

December 29, 2005

Completed
1 day until next milestone

First Posted

Study publicly available on registry

December 30, 2005

Completed
1.8 years until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2007

Completed
Last Updated

July 12, 2016

Status Verified

December 1, 2007

First QC Date

December 29, 2005

Last Update Submit

July 11, 2016

Conditions

Fanconi Anemia

Outcome Measures

Primary Outcomes (1)

  • CD34+ cell collection

Interventions

PBSC CollectionPROCEDURE
Bone Marrow HarvestPROCEDURE
CliniMacs Cell Selection SystemDEVICE

Eligibility Criteria

Age1 Year - 35 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Diagnosis of FA
  • Normal bone marrow cytogenetics within 3 months of study entry
  • Absolute neutrophil count (ANC) level greater than 750
  • Hemoglobin level greater than 8 without transfusion
  • Platelet level greater than 30,000 without transfusion
  • Must weigh at least 7.5 kg

You may not qualify if:

  • Myloid or lymphoid leukemia
  • Cytogenic abnormalities
  • HIV infected
  • Neoplastic or non-neoplastic disease of any major organ system that would compromise the ability to withstand the collection procedure
  • Uncontrolled infection
  • Unable to tolerate general anesthesia
  • Known adverse reaction to E. Coli
  • Pregnant or breastfeeding

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Related Publications (1)

  • Kelly PF, Radtke S, von Kalle C, Balcik B, Bohn K, Mueller R, Schuesler T, Haren M, Reeves L, Cancelas JA, Leemhuis T, Harris R, Auerbach AD, Smith FO, Davies SM, Williams DA. Stem cell collection and gene transfer in Fanconi anemia. Mol Ther. 2007 Jan;15(1):211-9. doi: 10.1038/sj.mt.6300033.

    PMID: 17164793BACKGROUND

MeSH Terms

Conditions

Fanconi Anemia

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, CongenitalAnemia, AplasticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesCongenital Bone Marrow Failure SyndromesBone Marrow Failure DisordersBone Marrow DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDNA Repair-Deficiency DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Stella Davies, MBBS

    Children's Hospital Medical Center, Cincinnati

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
NIH

Study Record Dates

First Submitted

December 29, 2005

First Posted

December 30, 2005

Study Start

August 1, 2004

Study Completion

October 1, 2007

Last Updated

July 12, 2016

Record last verified: 2007-12

Locations

US(1)