Cystic FibrosisPhase 2NCT03905642
Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) - Extension Phase
A Phase 2 study testing Arikayce™ 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) patients is acquisition of chronic Pseudomonas (P.) aeruginosa infections. The infection rate with P. aeruginosa increases with age and by age 18 years, 80% of patients with CF 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?
- Arikayce™
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 49 participants
- Where
- 11 sitesin 7 countries
- Study dates
- 8 Jan 2009to 2 Nov 2010
- Phase
- Phase 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 the 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 defined as a positive sweat chloride > 60 milliequivalents (mEq)/liter (by pilocarpine iontophoresis) and/or a genotype with 2 identifiable mutations consistent with CF accompanied by one or more clinical features of the CF phenotype.
- 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.
- 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.
- FEV1 ≥ 40% of predicted at Screening.
Show all 13Show 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 0.5 grams 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 or history of pulmonary exacerbation within the 4 weeks prior to Screening.
- Main criteria for inclusion of patients participating in the 18-month extension period:
- Written informed consent obtained from the patient or designated legal guardian prior to the performance of any study-related procedures in the extension period.
- Patient meets all of the above listed inclusion criteria (1-10) of the main protocol.
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 the 4 weeks prior to Screening.
- History of alcohol, medication, or illicit drug abuse within the 1 year prior to Screening.
- History of lung transplantation.
- 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. All women of childbearing potential will be tested.
Show all 19Show fewer
- Use of any anti-pseudomonal antibiotics (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 the 28 days prior to Screening.
- History of sputum or throat swab culture yielding Burkholderia cepacia within 2 years of Screening.
- History of mycobacterial and/or Aspergillus infection requiring treatment within 2 years prior to Screening, and/or history of allergic bronchopulmonary aspergillosis (ABPA).
- History of biliary cirrhosis with portal hypertension, or splenomegaly (refer to study manual).
- GGT, AST, or ALT ≥ 3 times the upper limit of normal at Screening visit.
- ANC ≤ 1000 performed at Screening visit.
- Serum creatinine > 1.5 times normal performed at Screening visit.
- 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) 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 18 months extension period:
- Patient meets any criteria for exclusion as listed above in the main protocol.
- Patient who met any criteria for study drug discontinuation in the main protocol (TR02-105).
Trial locations and contacts
11 sites in 7 countries.
11 sites in 7 countries
About this study
Official title: Multidose Safety and Tolerability Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) in Cystic Fibrosis Patients With Chronic Infections Due to Pseudomonas Aeruginosa
A major factor in the respiratory health of cystic fibrosis (CF) patients is acquisition of chronic Pseudomonas (P.) aeruginosa infections. The infection rate with P. aeruginosa increases with age and by age 18 years, 80% of patients with CF in the U.S. are infected. Liposomal amikacin for inhalation (LAI; Arikayce™) 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 infected patients when delivered via a nebulizer. Because liposome particles are small enough to penetrate and diffuse through sputum into the bacterial biofilm, they deposit drug close to the bacterial colonies (Meers, et al., 2008) (Clancy, et al., 2013), 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 patients with CF with chronic infection caused by P. aeruginosa.
Read the detailed description
Cystic fibrosis (CF) is a genetic disease resulting from mutations in a 230 kb gene on chromosome 7 known as the cystic fibrosis transmembrane conductance regulator (CFTR). Patients 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 patients with CF is to slow the chronic deterioration of lung function.
This study is a Phase 2 study to evaluate the longer term safety, tolerability and efficacy of 560 mg once daily dose of Arikayce™ administered for 6 cycles over 18 months. Each cycle comprises 28 days of treatment followed by 56 days off treatment.
All study patients will receive study drug by inhalation via a PARI® eFlow nebulizer. All study patients will be followed for safety, pharmacokinetics (PK), clinical, and microbiologic activity for 28 days post completion of study treatment.
The original TR02-105 study was a Phase 2a study of safety and tolerability of 28 days of daily dosing of two dose cohorts (280 mg and 560 mg) of Arikayce™ versus placebo. Study subjects were randomized to receive either study drug or placebo (1.5% NaCl) by inhalation via a PARI® eFlow nebulizer. Cohort 1 (280 mg) completed 28 days of daily dosing with Arikayce™ and 14-day post-dosing safety evaluation by the Safety Committee before initiation of enrollment in Cohort 2 (560 mg). Cohort 2 completed 28 days of daily dosing, and a 14-day post-dosing safety assessment by the Data Safety and Monitoring Board (DSMB) to evaluate safety data. All study patients were followed for safety, PK and clinical and microbiologic activity for 28 days post completion of study treatment. Details of the original study are provided at ClinicalTrials.gov ID NCT00777296.
DSMB has recommended the amendment of the main study to evaluate safety and efficacy of additional cycles of treatment with Arikayce™. 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 Arikayce™ administered for 6 cycles over 18 months. Each cycle comprises 28 days of treatment followed by 56 days off treatment.
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 Arikayce™. 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 Cystic Fibrosis Questionnaire-Revised (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) may be used interchangeably throughout this study and other studies evaluating amikacin liposome inhalation suspension.
- Conditions
- Cystic Fibrosis
- Being tested
- Arikayce™
- 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.
Arikayce™Drug
Also called Liposomal amikacin inhalation, LAI
Amikacin (aminoglycoside) in a liposomal formulation.
| Study group | Role | What they receive |
|---|---|---|
| 560 mg Arikayce™ | Experimental | Subjects in this cohort will receive 560 mg of Arikayce™ Receives: Drug: Arikayce™ |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Event Profile of 560 mg Once Daily Dose of Arikayce™ Administered for Six Cycles Over Eighteen Months.
Number of Participants with indicated Adverse Events in subjects receiving 560 mg once daily dose of Arikayce™ administered for 6 cycles over 18 months.
Measured: 18 Months
Secondary outcomes (5)
FEV1 % Predicted
A summary of relative change from extension study baseline time points in FEV1 % predicted is presented for the overall safety population and by treatment received.
Measured: Baseline, Days1, 14, 28, 56, 70, 85, 98, 112, 140, 154, 169, 182,196, 224, 238, 253, 266, 280, 308, 322, 337, 350, 364, 392, 406, 421, 434, 448, 476, 490, and 504
Absolute Change in Sputum Density
A summary of change from extension study baseline to all post-baseline time points during the treatment periods and at the end of the off-treatment periods in P aeruginosa sputum density (log10 CFU/mL) is presented for the overall safety population and by treatment received.
Measured: Baseline, Days 14, 28, 85, 98, 112, 140, 169, 182,196, 253, 266, 280, 337, 350, 364, 421, 434, and 448
Antipseudomonal Rescue Therapy - Duration of Therapy
The duration of IV and all systemic or inhaled antipseudomonal rescue therapy is presented for the overall safety population and by treatment received.
Measured: 18 Months
Antipseudomonal Rescue Therapy - Time to Therapy
The time to IV and all systemic or inhaled antipseudomonal rescue therapy is presented for the overall safety population and by treatment received.
Measured: 18 Months
Analysis of Cystic Fibrosis Questionnaire - Revised (CFQ-R) for Absolute Change in Score
A summary of absolute change from baseline in the CFQ-R scales at each on-treatment assessment between Day 14 and Day 448 is presented for all patients and by main study treatment group for the safety population. CFQ-R is a disease specific instrument designed to measure impact on overall health, daily life, perceived well-being and symptoms on a scale from 0 to 100 points. Higher values represent a more favorable outcome.
Measured: Days 14, 28, 85, 98, 112, 169, 182,196, 253, 266, 280, 337, 350, 364, 421, 434, and 448
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2019
- Study start8 Jan 2009
- Primary completion2 Nov 2010
- Study completion2 Nov 2010
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 (1)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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Extension phase for evaluation of safety, tolerability and efficacy. Single dose group of 560 mg of Arikase TM
- Masking
- None (open label)
- Enrolment
- 49 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
- NCT03905642
- Sponsor's protocol ID
- TR02-105 Extension
- Status last verified
- Jul 2020
- First submitted
- 2 Apr 2019
- Last updated
- 30 Jul 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
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 23749840
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
- Multidose Safety and Tolerability Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) in Cystic Fibrosis Patients With Chronic Infections Due to Pseudomonas Aeruginosa
- ClinicalTrials.gov ID
- NCT03905642
- Acronym / sponsor ID
- TR02-105 Extension
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, Respiratory Infections, Pulmonary Cystic Fibrosis, CFTR
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 49 participants (actual)
- Lead sponsor
- Insmed IncorporatedIndustry (pharma, biotech, devices)
- Study start
- 8 Jan 2009 (actual)
- Primary completion
- 2 Nov 2010 (actual)
- Study completion
- 2 Nov 2010 (actual)
- First posted
- 5 Apr 2019 (actual)
- Results
- Posted 14 May 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) patients is acquisition of chronic Pseudomonas (P.) aeruginosa infections. The infection rate with P. aeruginosa increases with age and by age 18 years, 80% of patients with CF in the U.S. are infected. Liposomal amikacin for inhalation (LAI; Arikayce™) 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 infected patients when delivered via a nebulizer. Because liposome particles are small enough to penetrate and diffuse through sputum into the bacterial biofilm, they deposit drug close to the bacterial colonies (Meers, et al., 2008) (Clancy, et al., 2013), 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 patients with CF with chronic infection caused by P. aeruginosa.
Detailed description
Cystic fibrosis (CF) is a genetic disease resulting from mutations in a 230 kb gene on chromosome 7 known as the cystic fibrosis transmembrane conductance regulator (CFTR). Patients 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 patients with CF is to slow the chronic deterioration of lung function.
This study is a Phase 2 study to evaluate the longer term safety, tolerability and efficacy of 560 mg once daily dose of Arikayce™ administered for 6 cycles over 18 months. Each cycle comprises 28 days of treatment followed by 56 days off treatment.
All study patients will receive study drug by inhalation via a PARI® eFlow nebulizer. All study patients will be followed for safety, pharmacokinetics (PK), clinical, and microbiologic activity for 28 days post completion of study treatment.
The original TR02-105 study was a Phase 2a study of safety and tolerability of 28 days of daily dosing of two dose cohorts (280 mg and 560 mg) of Arikayce™ versus placebo. Study subjects were randomized to receive either study drug or placebo (1.5% NaCl) by inhalation via a PARI® eFlow nebulizer. Cohort 1 (280 mg) completed 28 days of daily dosing with Arikayce™ and 14-day post-dosing safety evaluation by the Safety Committee before initiation of enrollment in Cohort 2 (560 mg). Cohort 2 completed 28 days of daily dosing, and a 14-day post-dosing safety assessment by the Data Safety and Monitoring Board (DSMB) to evaluate safety data. All study patients were followed for safety, PK and clinical and microbiologic activity for 28 days post completion of study treatment. Details of the original study are provided at ClinicalTrials.gov ID NCT00777296.
DSMB has recommended the amendment of the main study to evaluate safety and efficacy of additional cycles of treatment with Arikayce™. 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 Arikayce™ administered for 6 cycles over 18 months. Each cycle comprises 28 days of treatment followed by 56 days off treatment.
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 Arikayce™. 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 Cystic Fibrosis Questionnaire-Revised (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) may be used interchangeably throughout this study and other studies evaluating amikacin liposome inhalation suspension.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Extension phase for evaluation of safety, tolerability and efficacy. Single dose group of 560 mg of Arikase TM
| Arm | Type | Receives |
|---|---|---|
| 560 mg Arikayce™ | Experimental | Drug: Arikayce™Subjects in this cohort will receive 560 mg of Arikayce™ |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (13 listed)
- Written informed consent obtained from the 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 defined as a positive sweat chloride > 60 milliequivalents (mEq)/liter (by pilocarpine iontophoresis) and/or a genotype with 2 identifiable mutations consistent with CF accompanied by one or more clinical features of the CF phenotype.
- 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.
- 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.
Key exclusion criteria (19 listed)
- Administration of any investigational drug within 8 weeks prior to Screening.
- Emergency room visit or hospitalization for CF or respiratory-related illness within the 4 weeks prior to Screening.
- History of alcohol, medication, or illicit drug abuse within the 1 year prior to Screening.
- History of lung transplantation.
- 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 |
|---|---|---|
| Arikayce™Also: Liposomal amikacin inhalation, LAIAmikacin (aminoglycoside) in a liposomal formulation. | Drug | 560 mg Arikayce™ |
Primary outcome
Adverse Event Profile of 560 mg Once Daily Dose of Arikayce™ Administered for Six Cycles Over Eighteen Months.
Time frame: 18 Months
Number of Participants with indicated Adverse Events in subjects receiving 560 mg once daily dose of Arikayce™ administered for 6 cycles over 18 months.
5 secondary outcomes
FEV1 % Predicted
Time frame: Baseline, Days1, 14, 28, 56, 70, 85, 98, 112, 140, 154, 169, 182,196, 224, 238, 253, 266, 280, 308, 322, 337, 350, 364, 392, 406, 421, 434, 448, 476, 490, and 504
Absolute Change in Sputum Density
Time frame: Baseline, Days 14, 28, 85, 98, 112, 140, 169, 182,196, 253, 266, 280, 337, 350, 364, 421, 434, and 448
Antipseudomonal Rescue Therapy - Duration of Therapy
Time frame: 18 Months
Antipseudomonal Rescue Therapy - Time to Therapy
Time frame: 18 Months
Analysis of Cystic Fibrosis Questionnaire - Revised (CFQ-R) for Absolute Change in Score
Time frame: Days 14, 28, 85, 98, 112, 169, 182,196, 253, 266, 280, 337, 350, 364, 421, 434, and 448
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.
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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 2 study testing Arikayce™ 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 13 inclusion and 19 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
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 14 May 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?
Adverse Event Profile of 560 mg Once Daily Dose of Arikayce™ Administered for Six Cycles Over Eighteen Months. (time frame: 18 Months). 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 NCT03905642, which also lists 1 linked publication.
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.