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

Cystic Fibrosis Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00004287
• Other study ID #: 199/11715
• Secondary ID: UPHS-28051
• Status: Completed
• Phase: Phase 1
• First received: October 18, 1999
• Last updated: June 23, 2005
• Start date: November 1995

Study information

• Verified date: January 2000
• Source: Office of Rare Diseases (ORD)
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial Summary

OBJECTIVES:

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

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 14
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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 Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Genetic:
• H5.001CBCFTR

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
National Center for Research Resources (NCRR)	University of Pennsylvania

References & Publications (1)

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. — View Citation