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Drug & research development » Clinical trials » NCT01937325

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

Official title:

Clinical Trials gov number: NCT01937325

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Purpose: To assess the safety and feasibility of gene transfer with the third generation adenovirus H5.001CBCFTR in CF patients.

Phase: 1

Type: Interventional

Study sponsor: National Center for Research Resources (NCRR); University of Pennsylvania

Disease characteristics

  • Cystic fibrosis diagnosed as follows: Sweat sodium or chloride >60 mEq/L by pilocarpine iontophoresis or cystic fibrosis genotype Clinical manifestations Estimated 2-year survival greater than 50%, i.e.: FEV1 >30% of predicted PaO2 greater than 55 mm Hg on room air PaCO2 <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


  • No pneumothorax within 12 months
  • No asthma or allergic bronchopulmonary aspergillosis requiring glucocorticoids within 2 months
  • No sputum pathogens unless sensitive to ≥2 antibiotics
  • No hemoptysis of >250 mL blood over 24 hours within 1 year


  • 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

Genetic, H5.001CBCFTR

Geographical Location
No contacts or locations provided

Number of Participants
≤50 (≥18 years)

Primary Endpoint

Not provided

Secondary Endpoint

Not provided

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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.



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. PubMed PMID: 10609658.

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