Phase 2
Completed N=41
Safety/Tolerability Study of Arikayce™ in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa
Source: ClinicalTrials.gov NCT00558844 ↗Enrolled (actual)
41
Serious AEs
22.0%
Results posted
Jun 2019
Primary outcomePrimary: Number of Participants With Treatment-Emergent Adverse Events — 14; 7; 7; 4 Participants
Summary
This is a study to determine the safety and tolerability of 28 days of daily dosing of 560 mg of Arikayce™ versus placebo and daily dosing of 70 mg and 140 mg of Arikayce™ versus placebo in patients who have Cystic fibrosis (CF) and chronic infection due to pseudomonas aeruginosa.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events |
14; 7; 7; 4; 6; 7 | — |
| SECONDARY Pharmacokinetics of Arikayce™ in Serum |
1.59; 0.216; 0.375; 2.17; 0.265; 0.447 | — |
| SECONDARY Pharmacokinetics (PK) of Arikayce™ in Sputum |
4417; 1119; 1632; 132; 150; 20.1 | — |
| SECONDARY Pharmacokinetics (PK) of Arikayce™ in Urine |
25.9; 4.80; 15.2; 63.6; 7.76; 21.7 | — |
| SECONDARY Pharmacokinetics (PK) of Arikayce™ in Serum |
11.3; 1.06; 2.90; 15.0; 1.56; 4.32 | — |
| SECONDARY Pulmonary Function: Pre-Dose FEV1 (%-Predicted) |
68.800; 66.143; 59.286; 70.400; 69.286; -0.003 | — |
| SECONDARY Density of Pseudomonas Aeruginosa in Sputum |
-1.605; 1.118; -1.093; -0.637; -0.254; -1.082 | — |
| SECONDARY Duration of Systemic Anti-Pseudomonal Rescue Therapy |
22.800; 30.333; 17.500; 18.000; 30.500 | — |
| SECONDARY CFQ-R Respiratory Scale (Relative Change % From Baseline) |
0.385; -0.063; 0.075; 0.100; 0.042; 0.387 | — |
Eligibility Criteria
Key Inclusion Criteria
- Male or female study subjects must be adults (≥ 6 years of age)
- Confirmed diagnosis of CF
- History of chronic infection with P.aeruginosa
- FEV1 ≥40% of predicted at Screening
- Ability to comply with study medication use, study visits and procedures
- Ability to produce 0.5 grams of sputum
Key Exclusion Criteria
- Administration of any investigational drug within 8 weeks to Study Day 1
- Emergency room visit or hospitalization for CF or respiratory-related illness within 4 weeks prior to screening
- History of alcohol, medication or illicit drug abuse within 1 yr. to screening
- History of lung transplantation
- Female of childbearing potential who are not practicing an acceptable method of birth control or who are lactating
- Positive Pregnancy test
- Use of any anti-pseudomonal antibiotics within 28 days prior to Study Day 1
- Initiation of chronic therapy within 28 days prior to Study Day 1
- History of sputum or throat swab culture yielding Burkholderia cepacia within 2 years prior to screening
- History of mycobacterial and/or Aspergillus infection requiring treatment within 2 years prior to screening
- History of biliary cirrhosis with portal hypertension, or splenomegaly
Data sourced from ClinicalTrials.gov (NCT00558844). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.