Completed

NCT Number: NCT00004533

Phase I Randomized Study of Adeno-Associated Virus-CFTR Vector in Patients With Cystic Fibrosis

OBJECTIVES:

I. Determine the maximum tolerated dose of recombinant adeno-associated virus-CFTR vector in patients with cystic fibrosis.

II. Assess the safety of this gene therapy in these patients.

III. Assess the in vivo gene transfer of this vector in these patients.

IV. Assess the CFTR gene expression and physiologic activity following gene transfer in these patients.

V. Assess the clinical impact of CFTR gene expression following gene transfer in these patients.

VI. Monitor patient immune response directed against CFTR or vector components following vector administration.

Completed

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Disease Attributes Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases Respiratory Tract Diseases

Age range

15 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

About this study

PROTOCOL OUTLINE:

This is a randomized, dose escalation, double blind, placebo controlled, multicenter study. Patients are randomized to receive either adeno-associated virus-CFTR (AAV-CFTR) vector or placebo.

Patients undergo tests on days -10 to -4 to rule out adenovirus and adeno-associated virus infections. Patients then receive AAV-CFTR vector intranasally to the right or left inferior turbinates and placebo to the other side of the nose. The next day, patients receive an endobronchial dose of AAV-CFTR vector to the superior segment of the right lower lobe.

Cohorts of 2-4 patients each receive escalating doses of AAV-CFTR vector until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose level immediately preceding the dose at which at least 2 subjects develop dose limiting toxicity.

Patients are followed at day 10, then at 1, 2, 3, 6, 9, and 12 months.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Diagnosis of cystic fibrosis (CF) with mild to moderate lung disease; FVC at least 60% predicted; Schwachman-Kulczycki score at least 65; Sweat chloride greater than 60 mEq/L

All CF genotypes allowed

No sputum colonization with Burkholderia cepacia or another multiply resistant organism

No recurrent or intermittent hemoptysis; At least 1 year since significant hemoptysis requiring transfusion or embolization OR Coughing of greater than 30 mL of blood more often than once a week

--Prior/Concurrent Therapy--

At least 30 days since hospitalization or home intravenous antibiotic therapy for pulmonary exacerbation

No concurrent investigational drugs or therapy

--Patient Characteristics--

Other: No concurrent cigarette smoking; Not pregnant or nursing; Negative pregnancy test; Fertile patients must use effective contraception

Treatment and study plan

Adeno-associated virus-CFTR vector

Drug

Sponsors and collaborators

Lead sponsor

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Nih

Collaborators

University of Florida

Registry information

Important dates

Study start: 1999
Primary completion: 2002
Study completion: 2002
First posted: Jan 31, 2000
Registry last updated: Jun 1, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed