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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified February 2001 by Office of Rare Diseases (ORD).
Recruitment status was  Active, not recruiting
University of Florida
Information provided by:
Office of Rare Diseases (ORD) Identifier:
First received: January 28, 2000
Last updated: June 23, 2005
Last verified: February 2001


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.

Condition Intervention Phase
Cystic Fibrosis
Drug: Adeno-associated virus-CFTR vector
Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Masking: Double-Blind
Primary Purpose: Treatment

Resource links provided by NLM:

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment: 19
Study Start Date: August 1999
Detailed Description:


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.


Ages Eligible for Study:   15 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


--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

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00004533

Sponsors and Collaborators
University of Florida
Study Chair: Terence Flotte University of Florida
  More Information

No publications provided Identifier: NCT00004533     History of Changes
Other Study ID Numbers: 199/14137, UF-G-037-96, TGC-AAV-9502, UF-G-37-1996
Study First Received: January 28, 2000
Last Updated: June 23, 2005
Health Authority: United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):
cardiovascular and respiratory diseases
cystic fibrosis
genetic diseases and dysmorphic syndromes
rare disease

Additional relevant MeSH terms:
Cystic Fibrosis
Digestive System Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Lung Diseases
Pancreatic Diseases
Pathologic Processes
Respiratory Tract Diseases processed this record on November 27, 2014