Phase I Study of the Third Generation Adenovirus H5.001CBCFTR in Patients With Cystic Fibrosis

NCT ID: NCT00004287

Last Updated: 2005-06-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

14 participants

Study Classification

INTERVENTIONAL

Study Start Date

1995-11-30

Brief Summary

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OBJECTIVES:

I. Assess the safety and feasibility of gene transfer with the third generation adenovirus H5.001CBCFTR in patients with cystic fibrosis.

Detailed Description

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PROTOCOL OUTLINE: H5.001CBCFTR, an adenovirus vector containing the cystic fibrosis transmembrane conductance regulator gene, is administered endobronchially.

Cohort of 2 patients receive 1 of 6 H5.001CBCFTR concentrations. There is no intrapatient dose escalation.

Conditions

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Cystic Fibrosis

Study Design

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Primary Study Purpose

TREATMENT

Interventions

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H5.001CBCFTR

Intervention Type GENETIC

Eligibility Criteria

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Inclusion Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics-- Cystic fibrosis diagnosed as follows: Sweat sodium or chloride greater than 60 mEq/L by pilocarpine iontophoresis or cystic fibrosis genotype Clinical manifestations Estimated 2-year survival greater than 50%, i.e.: FEV1 at least 30% of predicted PaO2 greater than 55 mm Hg on room air PaCO2 less than 50 mm Hg on room air --Prior/Concurrent Therapy-- At least 2 months since systemic glucocorticoids At least 90 days since participation in investigational therapeutic study --Patient Characteristics-- Pulmonary: No pneumothorax within 12 months No asthma or allergic bronchopulmonary aspergillosis requiring glucocorticoids within 2 months No sputum pathogens unless sensitive to at least 2 antibiotics No hemoptysis of more than 250 mL blood over 24 hours within 1 year Other: No active adenoviral infection Ad5 (or similar type) antibody seropositive No other contraindication to protocol participation, e.g.: Drug abuse Alcoholism Psychiatric instability Inadequate motivation Documented azoospermia (men) Bilateral tubal ligation or hysterectomy (women) Screening exams within 4 weeks prior to registration
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University of Pennsylvania

OTHER

Sponsor Role collaborator

National Center for Research Resources (NCRR)

NIH

Sponsor Role lead

Principal Investigators

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James M. Wilson

Role: STUDY_CHAIR

University of Pennsylvania

References

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Zuckerman JB, Robinson CB, McCoy KS, Shell R, Sferra TJ, Chirmule N, Magosin SA, Propert KJ, Brown-Parr EC, Hughes JV, Tazelaar J, Baker C, Goldman MJ, Wilson JM. A phase I study of adenovirus-mediated transfer of the human cystic fibrosis transmembrane conductance regulator gene to a lung segment of individuals with cystic fibrosis. Hum Gene Ther. 1999 Dec 10;10(18):2973-85. doi: 10.1089/10430349950016384.

Reference Type BACKGROUND
PMID: 10609658 (View on PubMed)

Other Identifiers

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UPHS-28051

Identifier Type: -

Identifier Source: secondary_id

199/11715

Identifier Type: -

Identifier Source: org_study_id