Cystic FibrosisPhase 1/2NCT00777296
Multidose Safety and Tolerability Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKACE™)
A Phase 1/2 study testing ARIKACE™ in people with Cystic Fibrosis, run by Insmed Incorporated at 11 sites in 7 countries.
Trial summary
A major factor in the respiratory health of cystic fibrosis (CF) subjects is acquisition of chronic Pseudomonas aeruginosa infections. The infection rate with P. aeruginosa increases with age and by age 18 years, 80% of CF subjects in the U.S. are infected. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- ARIKACE™
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 66 participants
- Where
- 11 sitesin 7 countries
- Study dates
- 22 Feb 2007to 27 Feb 2008
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Written informed consent obtained from patient or designated legal guardian prior to the performance of any study related procedures.
- Male or female study subjects ≥6 years of age or older
- Confirmed diagnosis of CF
- History of chronic infection with P. aeruginosa
- Study subjects must produce a screening specimen that is positive for growth of P. aeruginosa
- FEV1 ≥ 40% predicted at Screening
Show all 10Show fewer
- SaO2 ≥ 90% at Screening while breathing room air
- Ability to comply with study medication use, study visits and study procedures as judged by the investigator
- Ability to produce sputum or be willing to undergo an induction to produce sputum for clinical evaluation
- Clinically stable with no evidence of acute upper or lower respiratory tract infection of history of pulmonary exacerbation within 4 weeks prior to screening
Who cannot join
- Administration of any investigational drug within 8 weeks prior to Screening
- 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 the 1 year prior to screening
- History of lung transplant
- 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)
- Positive pregnancy test
Show all 13Show fewer
- Use of any anti-pseudomonal anitbiotics (IV antibiotics, all inhalation antibiotics, oral fluoroquinolones)within the 28 days prior to screening
- Initiation of chronic therapy (i.e. TOBI®, high-dose ibuprofen, rhDNase, macrolide antibiotics) within 28 days prior to screening
- History of sputum or throat swab culture yielding Burkholderia cepacia within 2 years of Screening
- History of mycobacterial or Aspergillus infection
- Requiring treatment within 2 years prior to screening, and/or history of allergic bronchopulmonary aspergillosis.
- History of biliary cirrhosis with portal hypertension, or splenomegaly
- History of daily, continuous oxygen supplementation or requirement for more than 2 L/min at night Change in chest x-ray at screening (or within the 3 months prior to screening)
Trial locations and contacts
11 sites in 7 countries.
11 sites in 7 countries
About this study
Official title: Phase 2a 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.
A major factor in the respiratory health of cystic fibrosis (CF) subjects is acquisition of chronic Pseudomonas aeruginosa infections. The infection rate with P. aeruginosa increases with age and by age 18 years, 80% of CF subjects in the U.S. are infected. Liposomal Amikacin for Inhalation (Arikace™) is a sterile aqueous liposomal suspension consisting of amikacin sulfate encapsulated in liposomes. This formulation of amikacin maximizes the achievable dose and delivery to the lungs of subjects infected via a nebulizer. Because liposome particles are small enough to penetrate and diffuse through sputum into the bacterial biofilm, they deposit drug in close proximity to the bacterial colonies, thus improving the bioavailability of amikacin at the infection site. The clinically achievable doses of amikacin in the LAI formulation can effectively increase the half-life of the drug in the lungs, and decrease the potential for systemic toxicity. LAI offers several advantages over current therapies in treating CF subjects with chronic infection caused by P. aeruginosa.
Read the detailed description
Cystic fibrosis is a genetic 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 of organs that express CFTR. The lungs are frequently affected, the sequelae being chronic infections and airway inflammation. The principal goal of treatment of subjects with CF is to slow the chronic deterioration of lung function.
This is a Phase 2a study of safety, and tolerability of 28 days of daily dosing of two dose (280 mg, and 560 mg) cohorts of Arikace™ versus placebo. Study subjects will be randomized to receive either study drug or placebo (1.5% NaCl) by inhalation via a PARI eFlow® nebulizer. Cohort 1 (280mg) will complete 28 days of daily dosing with Arikace™ and 14 day post dosing safety evaluation by the Safety Committee (DSMB) before initiation of enrollment in Cohort 2 (560mg). Cohort 2 will complete 28 days of daily dosing, and a 14 day post dosing safety assessment by the DSMB to evaluate safety data. All study patients will be followed for safety, pharmacokinetics, clinical, and microbiologic activity for 28 days post completion of study treatment.
The total study period will be up to 56 days, with screening visit occurring within the preceding 14 days prior to randomization. Patients will be clinically evaluated during the first 48 hours post-randomization, and weekly for the 28 days treatment period, and during the follow up visits at study days 35, 42, 49, and 56 days to determine safety, 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 Arikace™ 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.
DSMB has recommended the amendment of the main study to evaluate safety and efficacy of additional cycles of treatment with Arikace™. All patients who were randomized in the main study, were compliant with the study protocol, and continue meeting study eligibility criteria can be consented to participate in the open-label extension to evaluate the safety, tolerability and efficacy of 560 mg once daily dose of Arikace™ administered for six cycles over eighteen months. Each cycle will comprise of 28 days of treatment followed by 56 days off treatment. The total extension period will be up to 518 days (74 weeks, about 18 months).
Clinical laboratory parameters, audiology testing, clinical adverse events, and pulmonary function will be evaluated for all study subjects in order to determine the longer term safety, tolerability, and efficacy of Arikace™. Serum specimens will be collected at periodic intervals to assess PK for safety. 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. Arikace™, Arikayce™, Liposomal Amikacin for Inhalation (LAI) and Amikacin Liposome Inhalation Suspension (ALIS) are all the same may be used interchangeably throughout the study and other studies evaluating amikacin liposome inhalation suspension.
- Conditions
- Cystic Fibrosis
- Being tested
- ARIKACE™, Placebo
- Keywords
- Cystic Fibrosis, Respiratory Infections, Pulmonary Cystic Fibrosis, CFTR
- Medical subject headings
- Cystic Fibrosis, Respiratory Tract Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARIKACE™Drug
Also called liposomal amikacin for inhalation, Arikayce
Study start date is before Jan 18, 2017.
PlaceboDrug
Study start date is before Jan 18, 2017.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 - 280 mg ARIKACE™ | Experimental | Subjects in this cohort will receive 280 mg of ARIKACE™ Receives: Drug: ARIKACE™ |
| Cohort 1 - Placebo | Placebo comparator | Subjects in this arm of cohort 1 will receive matching placebo Receives: Drug: Placebo |
| Cohort 2 - 560 mg ARIKACE™ | Experimental | Subjects in this cohort will receive 560 mg of ARIKACE™ Receives: Drug: ARIKACE™ |
| Cohort 2 - Placebo | Placebo comparator | Subjects in this arm of cohort 2 will receive matching placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinically Significant Laboratory Abnormalities.
Changes in chemistry and hematology lab tests (clinically significant value of CTCAE grade ≥ 3).
Measured: 28 Days
Secondary outcomes (11)
Pharmacokinetics (PK) of Arikace™ in Serum.
Measure PK parameters (AUC0-infinity) of Arikace™ in serum.
Measured: Day 1, Day 14 and Day 28
Pharmacokinetic (PK) of Arikace in Serum (Cmax).
Measure PK parameter (Cmax) of Arikace™ in serum.
Measured: Day 1, Day 14 and Day 28
Pharmacokinetics (PK) of Arikace™ in Sputum (AUC).
Measure PK parameter (AUC0-24) of Arikace™ in sputum.
Measured: 28 days
Pharmacokinetics (PK) of Arikace™ in Urine.
Measure PK parameter (Ae0-24 (mg) of Arikace™.
Measured: Day 1, Day 14 and Day 28
Sputum Amikacin Levels of Arikace™.
Measure PK parameter (sputum amicakin concentration) of Arikace™ in sputum.
Measured: Day 1, Day 14 and Day 28
Pulmonary Function: FEV1 %-Predicted.
Relative Change (%) from Baseline to End of treatment (Day 28) and Day 56 in Pulmonary Function.
Measured: Baseline, Day 28, and Day 56
Pulmonary Function: FEV1.
Mean Percent Change (%) from Baseline to End of treatment (Day 28) and Day 56 in Pulmonary Function.
Measured: Baseline, Day 28, and Day 56
Change From Baseline in Log10CFU Per Gram (Density) of Pseudomonas Aeruginosa in Sputum.
End-of-treatment (Day 28) from baseline in density of P. aeruginosa (log10 CFU/g) in sputum.
Measured: Day 7, Day 14, Day 21, Day 28 and Day 35
Duration of Systemic Antipseudomonal Rescue Therapy.
Duration of systemic antipseudomonal rescue therapy during the study in both the ARIKACE™ and placebo groups.
Measured: Through study duration, approximately 56 days
Number of Subjects Requiring Antipseudomonal Rescue Therapy.
Number of Subjects requiring systemic antipseudomonal rescue therapy during the study in both the ARIKACE™ and placebo groups.
Measured: Through study duration, approximately 56 days
CFQ-R Respiratory Scale (Absolute Change From Baseline).
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 symptoms in patients with a diagnosis of cystic fibrosis. Scores range from 0 to 100, with higher scores indicating better health. Scores for each Health Related Quality of Life (HRQoL) domain; after recoding, each item is summed to generate a domain score and standardized.
Measured: Baseline/Day 1, Day 15, Day 28 and Day 42
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2008 (estimated)
- Study start22 Feb 2007
- Primary completion27 Feb 2008
- Study completion27 Feb 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 66 participants (actual)
Sponsor and investigators
- Lead sponsor
- Insmed Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Gina Eagle, MD, Study director, Insmed Incorporated
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00777296
- Sponsor's protocol ID
- TR02-105
- Status last verified
- Jul 2020
- First submitted
- 15 Oct 2008
- Last updated
- 30 Jul 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Clancy JP, Dupont L, Konstan MW, Billings J, Fustik S, Goss CH, Lymp J, Minic P, Quittner AL, Rubenstein RC, Young KR, Saiman L, Burns JL, Govan JR, Ramsey B, Gupta R; Arikace Study Group. Phase II studies of nebulised Arikace in CF patients with Pseudomonas aeruginosa infection. Thorax. 2013 Sep;68(9):818-25. doi: 10.1136/thoraxjnl-2012-202230. Epub 2013 Jun 8.
PubMed 23749840Okusanya 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.
PubMed 24687506Meers P, Neville M, Malinin V, Scotto AW, Sardaryan G, Kurumunda R, Mackinson C, James G, Fisher S, Perkins WR. Biofilm penetration, triggered release and in vivo activity of inhaled liposomal amikacin in chronic Pseudomonas aeruginosa lung infections. J Antimicrob Chemother. 2008 Apr;61(4):859-68. doi: 10.1093/jac/dkn059. Epub 2008 Feb 27.
PubMed 18305202
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2a 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.
- ClinicalTrials.gov ID
- NCT00777296
- Acronym / sponsor ID
- TR02-105
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, Respiratory Infections, Pulmonary Cystic Fibrosis, CFTR
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 66 participants (actual)
- Lead sponsor
- Insmed IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 22 Feb 2007 (actual)
- Primary completion
- 27 Feb 2008 (actual)
- Study completion
- 27 Feb 2008 (actual)
- First posted
- 22 Oct 2008 (estimated)
- Results
- Posted 16 Jul 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A major factor in the respiratory health of cystic fibrosis (CF) subjects is acquisition of chronic Pseudomonas aeruginosa infections. The infection rate with P. aeruginosa increases with age and by age 18 years, 80% of CF subjects in the U.S. are infected. Liposomal Amikacin for Inhalation (Arikace™) is a sterile aqueous liposomal suspension consisting of amikacin sulfate encapsulated in liposomes. This formulation of amikacin maximizes the achievable dose and delivery to the lungs of subjects infected via a nebulizer. Because liposome particles are small enough to penetrate and diffuse through sputum into the bacterial biofilm, they deposit drug in close proximity to the bacterial colonies, thus improving the bioavailability of amikacin at the infection site. The clinically achievable doses of amikacin in the LAI formulation can effectively increase the half-life of the drug in the lungs, and decrease the potential for systemic toxicity. LAI offers several advantages over current therapies in treating CF subjects with chronic infection caused by P. aeruginosa.
Detailed description
Cystic fibrosis is a genetic 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 of organs that express CFTR. The lungs are frequently affected, the sequelae being chronic infections and airway inflammation. The principal goal of treatment of subjects with CF is to slow the chronic deterioration of lung function.
This is a Phase 2a study of safety, and tolerability of 28 days of daily dosing of two dose (280 mg, and 560 mg) cohorts of Arikace™ versus placebo. Study subjects will be randomized to receive either study drug or placebo (1.5% NaCl) by inhalation via a PARI eFlow® nebulizer. Cohort 1 (280mg) will complete 28 days of daily dosing with Arikace™ and 14 day post dosing safety evaluation by the Safety Committee (DSMB) before initiation of enrollment in Cohort 2 (560mg). Cohort 2 will complete 28 days of daily dosing, and a 14 day post dosing safety assessment by the DSMB to evaluate safety data. All study patients will be followed for safety, pharmacokinetics, clinical, and microbiologic activity for 28 days post completion of study treatment.
The total study period will be up to 56 days, with screening visit occurring within the preceding 14 days prior to randomization. Patients will be clinically evaluated during the first 48 hours post-randomization, and weekly for the 28 days treatment period, and during the follow up visits at study days 35, 42, 49, and 56 days to determine safety, 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 Arikace™ 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.
DSMB has recommended the amendment of the main study to evaluate safety and efficacy of additional cycles of treatment with Arikace™. All patients who were randomized in the main study, were compliant with the study protocol, and continue meeting study eligibility criteria can be consented to participate in the open-label extension to evaluate the safety, tolerability and efficacy of 560 mg once daily dose of Arikace™ administered for six cycles over eighteen months. Each cycle will comprise of 28 days of treatment followed by 56 days off treatment. The total extension period will be up to 518 days (74 weeks, about 18 months).
Clinical laboratory parameters, audiology testing, clinical adverse events, and pulmonary function will be evaluated for all study subjects in order to determine the longer term safety, tolerability, and efficacy of Arikace™. Serum specimens will be collected at periodic intervals to assess PK for safety. 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. Arikace™, Arikayce™, Liposomal Amikacin for Inhalation (LAI) and Amikacin Liposome Inhalation Suspension (ALIS) are all the same may be used interchangeably throughout the study and other studies evaluating amikacin liposome inhalation suspension.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 - 280 mg ARIKACE™ | Experimental | Drug: ARIKACE™Subjects in this cohort will receive 280 mg of ARIKACE™ |
| Cohort 1 - Placebo | Placebo comparator | Drug: PlaceboSubjects in this arm of cohort 1 will receive matching placebo |
| Cohort 2 - 560 mg ARIKACE™ | Experimental | Drug: ARIKACE™Subjects in this cohort will receive 560 mg of ARIKACE™ |
| Cohort 2 - Placebo | Placebo comparator | Drug: PlaceboSubjects in this arm of cohort 2 will receive matching placebo |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Written informed consent obtained from patient or designated legal guardian prior to the performance of any study related procedures.
- Male or female study subjects ≥6 years of age or older
- Confirmed diagnosis of CF
- History of chronic infection with P. aeruginosa
- Study subjects must produce a screening specimen that is positive for growth of P. aeruginosa
Key exclusion criteria (13 listed)
- Administration of any investigational drug within 8 weeks prior to Screening
- 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 the 1 year prior to screening
- History of lung transplant
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ARIKACE™Also: liposomal amikacin for inhalation, ArikayceStudy start date is before Jan 18, 2017. | Drug | Cohort 1 - 280 mg ARIKACE™, Cohort 2 - 560 mg ARIKACE™ |
| PlaceboStudy start date is before Jan 18, 2017. | Drug | Cohort 1 - Placebo, Cohort 2 - Placebo |
Primary outcome
Clinically Significant Laboratory Abnormalities.
Time frame: 28 Days
Changes in chemistry and hematology lab tests (clinically significant value of CTCAE grade ≥ 3).
11 secondary outcomes
Pharmacokinetics (PK) of Arikace™ in Serum.
Time frame: Day 1, Day 14 and Day 28
Pharmacokinetic (PK) of Arikace in Serum (Cmax).
Time frame: Day 1, Day 14 and Day 28
Pharmacokinetics (PK) of Arikace™ in Sputum (AUC).
Time frame: 28 days
Pharmacokinetics (PK) of Arikace™ in Urine.
Time frame: Day 1, Day 14 and Day 28
Sputum Amikacin Levels of Arikace™.
Time frame: Day 1, Day 14 and Day 28
Pulmonary Function: FEV1 %-Predicted.
Time frame: Baseline, Day 28, and Day 56
Pulmonary Function: FEV1.
Time frame: Baseline, Day 28, and Day 56
Change From Baseline in Log10CFU Per Gram (Density) of Pseudomonas Aeruginosa in Sputum.
Time frame: Day 7, Day 14, Day 21, Day 28 and Day 35
Duration of Systemic Antipseudomonal Rescue Therapy.
Time frame: Through study duration, approximately 56 days
Number of Subjects Requiring Antipseudomonal Rescue Therapy.
Time frame: Through study duration, approximately 56 days
CFQ-R Respiratory Scale (Absolute Change From Baseline).
Time frame: Baseline/Day 1, Day 15, Day 28 and Day 42
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jul 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 23749840Clancy JP, Dupont L, Konstan MW, Billings J, Fustik S, Goss CH, Lymp J, Minic P, Quittner AL, Rubenstein RC, Young KR, Saiman L, Burns JL, Govan JR, Ramsey B, Gupta R; Arikace Study Group. Phase II studies of nebulised Arikace in CF patients with Pseudomonas aeruginosa infection. Thorax. 2013 Sep;68(9):818-25. doi: 10.1136/thoraxjnl-2012-202230. Epub 2013 Jun 8.
- Background: PubMed 24687506Okusanya 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.
- Background: PubMed 18305202Meers P, Neville M, Malinin V, Scotto AW, Sardaryan G, Kurumunda R, Mackinson C, James G, Fisher S, Perkins WR. Biofilm penetration, triggered release and in vivo activity of inhaled liposomal amikacin in chronic Pseudomonas aeruginosa lung infections. J Antimicrob Chemother. 2008 Apr;61(4):859-68. doi: 10.1093/jac/dkn059. Epub 2008 Feb 27.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gina Eagle, MD study director
Insmed Incorporated
11 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ARIKACE™ in people with Cystic Fibrosis, run by Insmed Incorporated at 11 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Hungary, Poland, Slovakia, Ukraine, Belgium and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Jul 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Clinically Significant Laboratory Abnormalities. (time frame: 28 Days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00777296, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jul 2020.