NCT00004806

Brief Summary

OBJECTIVES: Evaluate the efficacy and safety of lipid-mediated transfer of the cystic fibrosis transmembrane conductance regulator gene to nasal epithelium in patients with cystic fibrosis.

Trial Health

100
On Track

Trial Health Score

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

Enrollment
9

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jun 1995

Longer than P75 for phase_1

Status
completed

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

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 1995

Completed
4.7 years until next milestone

First Submitted

Initial submission to the registry

February 24, 2000

Completed
1 day until next milestone

First Posted

Study publicly available on registry

February 25, 2000

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2002

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2002

Completed
Last Updated

March 30, 2011

Status Verified

March 1, 2011

Enrollment Period

7.4 years

First QC Date

February 24, 2000

Last Update Submit

March 29, 2011

Conditions

Cystic Fibrosis

Keywords

cardiovascular and respiratory diseasescystic fibrosisgenetic diseases and dysmorphic syndromesrare disease

Interventions

Cystic fibrosis transmembrane conductance regulatorGENETIC

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Cystic fibrosis (CF), i.e.: Sweat chloride greater than 60 mEq/L Clinical manifestations of CF Homozygous delta F508 mutation preferred Compound heterozygotes for F508 mutation with pancreatic insufficiency eligible FEV1 greater than 40% of predicted PO2 at least 60 mm Hg on room air No acute or recurrent sinusitis No obstructive nasal polyposis No pneumothorax or hemoptysis, e.g., more than 250 mL blood within 24-hour period, within past year No unstable lung disease with worsening pulmonary symptoms, arterial blood gas, or pulmonary function tests within 2 weeks prior to entry No pulmonary exacerbation within 4 weeks prior to entry --Prior/Concurrent Therapy-- At least 2 months since participation in any other clinical study At least 2 months since DNase At least 4 months since corticosteroids --Patient Characteristics-- Cardiac: No severe cardiac disease Other: No other severe organ system disease, e.g., juvenile-type diabetes mellitus No pregnant women

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

MeSH Terms

Conditions

Cystic FibrosisRespiratory Tract DiseasesGenetic Diseases, InbornRare Diseases

Interventions

Cystic Fibrosis Transmembrane Conductance Regulator

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Multidrug Resistance-Associated ProteinsATP-Binding Cassette TransportersMembrane Transport ProteinsCarrier ProteinsProteinsAmino Acids, Peptides, and ProteinsChloride ChannelsIon ChannelsOrganic Anion Transporters, ATP-DependentOrganic Anion TransportersAnion Transport ProteinsIon PumpsMembrane GlycoproteinsMembrane Proteins

Study Officials

  • Eric J. Sorscher

    University of Alabama at Birmingham

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 1
Purpose
TREATMENT
Sponsor Type
OTHER

Study Record Dates

First Submitted

February 24, 2000

First Posted

February 25, 2000

Study Start

June 1, 1995

Primary Completion

November 1, 2002

Study Completion

November 1, 2002

Last Updated

March 30, 2011

Record last verified: 2011-03