Expired Study
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Iowa City, Iowa


Purpose:

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.


Study summary:

CF is a gentic disease resulting from mutations in a 230 kb gene on chromosome 7 known as the cystic fibrosis transmembrane conductance regulator (CFTR). Study subjects with CF manifest pathological changes in a variety or organs that express CFTR. The lungs are frequently affected, the sequelae being chronic infections and airway inflammation. The principal goal of both treatment of subjects with CF is to slow the chronic deterioration of lung function. Study subjects will be randomized to receive either study drug or placebo (1.5% NaCl) by inhalation via a PARI eFlow nebulizer. Each subject will complete 28 days of daily dosing. All study patients will be followed for safety, pharmacokinetics, clinical and microbiologic activity for 56 days post completion of study treatment. For the two lower doses (70 mg and 140 mg): patients received drug for 28 days, followed by a 28 day safety evaluation. For 560 mg: patients received drug for 28 days, followed by a 56 day safety evaluation. The total study period will be up to 84 days, with screening visit occurring within the preceding 14 days prior to study day 1. Patients will be clinically evaluated during the first 48 hours post first study dose and weekly for the 28 day treatment period and during the follow up visits at study days 35, 42, 49, 56, 70 and 85 days to determine safety and tolerability, pharmacokinetics (PK) and clinical and microbiologic activity. Clinical laboratory parameters, audiology testing, clinical adverse events and pulmonary function will be evaluated for all study subjects in order to determine the qualitative and quantitative safety and tolerability of Arikayce™ compared to placebo. Serum, urine and sputum specimens will be collected at periodic intervals to assess PK. Additionally, sputum samples will be collected to determine changes in bacterial density. Pulmonary function testing and CFQ-R measurements will be assessed at selected time points throughout the study. An exploratory evaluation of a Cystic Fibrosis Symptom Diary (CFSD) will also be implemented. Arikace™,Arikayce™, Liposomal Amikacin for Inhalation (LAI), and Amikacin Liposome Inhalation Suspension (ALIS) may be used interchangeably throughout this study and other studies evaluating amikacin liposomal inhalation suspension.


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


Study is Available At:


Original ID:

TR02-106


NCT ID:

NCT00558844


Secondary ID:


Study Acronym:


Brief Title:

Safety/Tolerability Study of Arikayce™ in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa


Official Title:

Phase 1b/2a Multidose Safety and Tolerability Study of Liposomal Amikacin for Inhalation (Arikayce™) in Cystic Fibrosis Patient With Chronic Infections Due to Pseudomonas Aeruginosa.


Overall Status:

Completed


Study Phase:

Phase 1/Phase 2


Genders:

N/A


Minimum Age:

6 Years


Maximum Age:

N/A


Quick Facts

Healthy Volunteers
Oversight Has DMC
Study Is FDA Regulated
Study Is Section 801
Has Expanded Access

Study Source:

Insmed Incorporated


Oversight Authority:

United States: Food and Drug Administration


Reasons Why Stopped:


Study Type:

Interventional


Study Design:


Number of Arms:

5


Number of Groups:

0


Total Enrollment:

41


Enrollment Type:

Actual


Overall Contact Information

Official Name:Gina Eagle, MD
Study Director
Insmed Incorporated

Study Dates

Start Date:January 2008
Completion Date:June 2009
Completion Type:Actual
Primary Completion Date:June 2009
Primary Completion Type:Actual
Verification Date:May 2019
Last Changed Date:May 14, 2019
First Received Date:November 13, 2007
First Results Date:April 3, 2019

Study Outcomes

Outcome Type:Secondary Outcome
Measure:CFQ-R Respiratory Scale (Relative Change % From Baseline)
Time Frame:Day 15, Day 28 and Day 42
Safety Issues:False
Description:Quality of Life was measured by the absolute change from baseline in the Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory scale. Disease specific instrument designed to measure impact on overall health, daily life, perceived well-being and sympto
Outcome Type:Secondary Outcome
Measure:Duration of Systemic Anti-Pseudomonal Rescue Therapy
Time Frame:Through study duration, approximately 84 days
Safety Issues:False
Outcome Type:Secondary Outcome
Measure:Density of Pseudomonas Aeruginosa in Sputum
Time Frame:Day 7, Day 14, Day 21, Day 28 and Day 35
Safety Issues:False
Description:Change (log10 CFU) from Baseline by Study Day and Treatment Arm
Outcome Type:Secondary Outcome
Measure:Pulmonary Function: Pre-Dose FEV1 (%-Predicted)
Time Frame:Baseline, Day 28, Day 56, Day 70 and Day 84
Safety Issues:False
Description:Relative Change (%) from Baseline to Day 28, Day 56, Day 70, and Day 84 in Pulmonary Function
Outcome Type:Secondary Outcome
Measure:Pharmacokinetics (PK) of Arikayce™ in Serum
Time Frame:Day 1, Day 14 and Day 28
Safety Issues:False
Description:Measure PK parameter (AUC) of Arikayce in Serum
Outcome Type:Secondary Outcome
Measure:Pharmacokinetics (PK) of Arikayce™ in Urine
Time Frame:Day 1, Day 14 and Day 28
Safety Issues:False
Description:Measure PK parameter (Ae0-24) of Arikayce in urine
Outcome Type:Secondary Outcome
Measure:Pharmacokinetics (PK) of Arikayce™ in Sputum
Time Frame:Day 1 post-dose, Day 14 pre- and post-dose, Day 28 pre- and post-dose
Safety Issues:False
Description:Measure PK parameters (sputum concentration) of Arikayce in sputum, pre- and post-dose
Outcome Type:Secondary Outcome
Measure:Pharmacokinetics of Arikayce™ in Serum
Time Frame:Day 1, Day 14 and Day 28
Safety Issues:False
Description:Measure PK parameter (Cmax) of Arikayce in serum
Outcome Type:Primary Outcome
Measure:Number of Participants With Treatment-Emergent Adverse Events
Time Frame:56 days
Safety Issues:False
Description:To evaluate the safety and tolerability of 28 days of daily dosing of nebulized Arikayce™, liposomal amikacin for inhalation.

Study Interventions

Intervention Type:Drug
Name:Arikayce™ 560 mg
Description:Arikayce™ at 560 mg Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 2:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.
Arm Name:A
Intervention Type:Drug
Name:Placebo for 560 mg
Description:Matching placebo Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 2:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.
Arm Name:B
Intervention Type:Drug
Name:Arikayce™ 70 mg
Description:Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.
Arm Name:C
Intervention Type:Drug
Name:Arikayce™ 140 mg
Description:Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.
Arm Name:D
Intervention Type:Drug
Name:Placebo for 70 mg / 140 mg
Description:Matching placebo Subjects will be randomly assigned to study drug dose of of Arikayce™ or placebo in accordance with a code provided by the Sponsor/CRO. Randomization will be made in a 1:1:1 allocation between Arikayce™ and placebo. They will be blinded whether they receive Arikayce™ or Placebo Study subjects will receive Arikayce™ or placebo on Days 1 through Day 28. Drug is administered once a day via a nebulizer.
Arm Name:E

Study Arms

Study Arm Type:Placebo Comparator
Arm Name:E
Description:Matching placebo for 70 mg/140 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo.
Study Arm Type:Active Comparator
Arm Name:D
Description:Arikayce™ at 140 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo.
Study Arm Type:Active Comparator
Arm Name:C
Description:Arikayce™ at 70 mg Subjects randomized 1:1:1 to receive Arikayce 70 mg, Arikayce 140 mg or Placebo.
Study Arm Type:Placebo Comparator
Arm Name:B
Description:Matching placebo for 560 mg Subjects randomized 2:1 to receive Arikayce 560 mg or Placebo.
Study Arm Type:Active Comparator
Arm Name:A
Description:Arikayce™ at 560 mg Subjects randomized 2:1 to receive Arikayce 560 mg or Placebo.

Study Agencies

Agency Class:Industry
Agency Type:Lead Sponsor
Agency Name:Insmed Incorporated

Sample and Retention Information

There are no available Sample and Retention Information

Study References

Reference Type:Reference
Citation:Okusanya OO, Bhavnani SM, Hammel JP, Forrest A, Bulik CC, Ambrose PG, Gupta R. Evaluation of the pharmacokinetics and pharmacodynamics of liposomal amikacin for inhalation in cystic fibrosis patients with chronic pseudomonal infections using data from two phase 2 clinical studies. Antimicrob Agents Chemother. 2014 Sep;58(9):5005-15. doi: 10.1128/AAC.02421-13. Epub 2014 Mar 31.
PMID:24687506

Data Source: ClinicalTrials.gov

Date Processed: January 21, 2020

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