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Sponsors and Collaborators: |
National Center for Research Resources (NCRR) University of Pennsylvania |
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Information provided by: | Office of Rare Diseases (ORD) |
ClinicalTrials.gov Identifier: | NCT00004287 |
OBJECTIVES:
I. Assess the safety and feasibility of gene transfer with the third generation adenovirus H5.001CBCFTR in patients with cystic fibrosis.
Condition | Intervention | Phase |
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Cystic Fibrosis |
Gene Transfer: H5.001CBCFTR |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment |
Estimated Enrollment: | 14 |
Study Start Date: | November 1995 |
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.
Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
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
Study ID Numbers: | 199/11715, UPHS-28051 |
Study First Received: | October 18, 1999 |
Last Updated: | June 23, 2005 |
ClinicalTrials.gov Identifier: | NCT00004287 History of Changes |
Health Authority: | United States: Federal Government |
cardiovascular and respiratory diseases cystic fibrosis genetic diseases and dysmorphic syndromes rare disease |
Digestive System Diseases Genetic Diseases, Inborn Respiratory Tract Diseases Cystic Fibrosis Fibrosis Lung Diseases |
Adenoviridae Infections Respiration Disorders Rare Diseases Infant, Newborn, Diseases Pancreatic Diseases |
Pathologic Processes Digestive System Diseases Genetic Diseases, Inborn Respiratory Tract Diseases Cystic Fibrosis |
Fibrosis Lung Diseases Infant, Newborn, Diseases Pancreatic Diseases |