MULTIDOSE SAFETY AND TOLERABILITY STUDY OF DOSE ESCALATION OF LIPOSOMAL AMIKACIN FOR INHALATION (ARIKACE™) IN CYSTIC FIBROSIS PATIENTS WITH CHRONIC INFECTIONS DUE TO PSEUDOMONAS AERUGINOSA

Conditions: Cystic fibrosis
MedDRA version: 9.1Level: LLTClassification code 10011762Term: Cystic fibrosis

Registration Number: EUCTR2006-006980-22-HU

Lead Sponsor: Transave, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 60

Inclusion Criteria

CF study subjects with mild to moderate obstructive lung disease (FEV1% predicted = 40%) will be eligible for enrollment. Study subjects must be 6 years old or older. To be eligible, study subjects must also have history of sputum containing Pseudomonas aeruginosa.

1) Written informed consent prior to the performance of any study related
procedures
2) Male or female study subjects = 6 years of age or older
3) Confirmed diagnosis of CF defined as a positive sweat chloride > 60 mEq/liter
(by pilocarpine iontophoresis) and/or a genotype with two identifiable mutations
consistent with CF accompanied by one or more clinical features of the CF
phenotype
4) History of chronic infection with P. aeruginosa (defined as 3 documented positive
cultures in the prior 2 years of which at least one was obtained in the 3 months
prior to randomization. The cultures could be obtained from the following
respiratory secretions:
sputum, throat swabs, nasopharyngeal swabs or broncho-alveolar lavage fluid
specimens)
5) Study subjects must produce a screening specimen (expectorated or induced
sputum, throat swabs, nasopharyngeal swabs or broncho-alveolar lavage fluid)
that is positive for growth of P. aeruginosa
6) FEV1 = 40% of predicted at Screening
7) SaO2 = 90% at Screening while breathing room air
8) Ability to comply with study medication use, study visits, and study procedures as
judged by the investigator
9) Ability to produce 0.5 grams sputum or be willing to undergo an induction to
produce sputum for clinical evaluation
10)Clinically stable with no evidence of acute upper or lower respiratory tract
infection or history of pulmonary exacerbation within the 4 weeks prior to
randomization

Main criteria for inclusion of patients participating in the 6 months extension period:
1) Written informed consent obtained from the patient or designated legal guardian prior to the performance of any study related procedures in the extension period
2) Patient meets all of the above listed inclusion criteria (1-10) of the main protocol
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1) Administration of any investigational drug within 8 weeks prior to Screening
2) Emergency room visit or hospitalization for CF or respiratory-related illness within
the 4 weeks prior to screening
3) History of alcohol, medication, or illicit drug abuse within the 1 year prior to
screening
4) History of lung transplantation
5) Female of childbearing potential who is lactating or is not practicing an acceptable
method of birth control (e.g., abstinence, hormonal or barrier methods, partner
sterilization, or IUD)
6) Positive pregnancy test. All women of child bearing potential will be tested
7) Use of any anti-pseudomonal antibiotics (IV antibiotics, all inhalation antibiotics,
oral fluoroquinolones) within the 28 days prior to screening.
8) Initiation of chronic therapy (i.e. TOBI®, high-dose ibuprofen, rhDNase, macrolide
antibiotics) within the 28 days prior to screening
9) History of sputum or throat swab culture yielding Burkholderia cepacia within 2
years of Screening
10)History of mycobacterial infection
11)History of biliary cirrhosis, portal hypertension, or splenomegaly (refer to study
manual)
12)GGT, AST, or ALT = 3 times the upper limit of normal at Screening visit
13)ANC = 1000 performed at Screening visit
14)Serum creatinine > 1.5 times normal performed at Screening visit
15)History of daily, continuous oxygen supplementation or requirement for more
than 2 L/min at night
16)Change in chest x-ray at screening (or within the 3 months prior to screening)
with new onset infiltrates or that which compromise the safety of the study
patient or the quality of the study data

Main criteria for exclusion of patients participating in the 6 months extension period:
1)Patient meets all criteria for exclusion as listed above in the main protocol
2)Patient which met any criteria for study drug discontinuation in the main protocol (see section 5.3 Criteria for Study Drug Discontinuation)

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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