Cystic FibrosisPhase 3NCT01315691
Study to Evaluate Arikace™ in CF Patients With Chronic Infection Due to Pseudomonas Aeruginosa
A Phase 3 study testing Liposomal amikacin for inhalation in people with Cystic Fibrosis, run by Insmed Incorporated.
Trial summary
A major factor in the respiratory health of Cystic Fibrosis (CF) subjects is the prevalence of chronic Pseudomonas aeruginosa infections. The Pseudomonas aeruginosa infection rate in CF patients increases with age and by age 18 years approximately 85% of CF patients in the US 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?
- Liposomal amikacin for inhalation
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 or assent
- Confirmed diagnosis of CF
- History of chronic infection with Pseudomonas aeruginosa
- History of documented pulmonary exacerbation requiring treatment with antibiotics in the 12 months prior to Screening
- Sputum culture positive for Pseudomonas aeruginosa at Screening
- FEV1 ≥ 25% of predicted value at Screening
Who cannot join
- FEV1 <25% of predicted value at Screening
- History of hypersensitivity to aminoglycosides
- History of major complications of lung disease (including atelectasis, pneumothorax, major pleural effusion) within 8 weeks prior to Screening
- Hemoptysis of ≥60 mL in a 24-hour period within 4 weeks prior to Screening
- History of pulmonary tuberculosis or non-tuberculous mycobacterial lung disease treated within 2 years prior to Screening or requiring treatment at the time of Screening
- History of Allergic Broncho-Pulmonary Aspergillosis requiring systemic steroid treatment or any other condition requiring systemic steroids at a dose ≥ equivalent of 10 mg/day of prednisone within 3 months prior to Screening
Show all 12Show fewer
- Presence of any clinically significant cardiac disease
- Active pulmonary malignancy (primary or metastatic) or any malignancy requiring chemotherapy or radiation therapy within one year prior to Screening or anticipated during the study period
- History of lung transplantation
- Daily, continuous oxygen supplementation or nighttime supplemental oxygen requirement of greater than 2 L/min
- Administration of any investigational products within 8 weeks prior to study Day 1
- Smoking tobacco or any substance within 6 months prior to Screening or anticipated inability to refrain from smoking throughout the study
About this study
Official title: Randomized, Placebo-Controlled, Double-Blind, Multicenter Study to Assess the Efficacy, Safety and Tolerability of Arikace™ in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa
A major factor in the respiratory health of Cystic Fibrosis (CF) subjects is the prevalence of chronic Pseudomonas aeruginosa infections. The Pseudomonas aeruginosa infection rate in CF patients increases with age and by age 18 years approximately 85% of CF patients in the US are infected. Liposomal amikacin for inhalation (Arikace™) was developed as a possible treatment for chronic infection due to Pseudomonas aeruginosa in CF patients.
The purpose of this double-blind, placebo controlled study is to determine whether Arikace™ is effective in treating chronic lung infections caused by Pseudomonas aeruginosa in Cystic Fibrosis subjects. The study will enroll approximately 300 subjects in clinics in the US, Canada, Europe, Australia and New Zealand. Subjects will be randomized to 590 mg Arikace™ or placebo and will receive treatment for 28 days followed by a 56 day safety follow-up period. The subjects will be required to visit the clinic 8 times (including the Screening visit) over a period of approximately 3 months. No overnight stays at the clinic will be required. At the completion of the TR02-109 protocol, subjects who have consented and meet study safety criteria may enroll in the long-term, open-label, multi-cycle extension study of 590 mg of Arikace™ (under a separate protocol TR02-110).
Read the detailed description
Cystic Fibrosis (CF) is a genetic disease resulting from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Patients with CF manifest pathological changes in a variety of organs that express CFTR. The lungs are frequently affected often resulting in chronic infections by bacteria such as Pseudomonas aeruginosa and airway inflammation. Treatment of chronic lung infections is one of the principal goals of CF therapy. Arikace™ (liposomal amikacin for inhalation) is a sustained-release formulation of amikacin encapsulated inside nanoscale liposomal carriers designed for administration via inhalation. It is hypothesized that the sustained-release pulmonary targeting and biofilm penetration properties of this formulation will have several advantages over current therapies in treating CF patients with chronic lung infection caused by Pseudomonas aeruginosa.
This double blind, placebo controlled Phase 3 study has been designed to evaluate the efficacy, safety and tolerability of Arikace™ in treating CF patients with chronic bronchopulmonary infection. Eligible subjects will be randomized 1:1 to receive 590 mg of Arikace™ or placebo once daily using a PARI Investigational eFlow® Nebulizer. Subjects will receive 28 days of treatment and will then be followed for safety for 56 days. Total study duration is up to 102 days (~3 months) including an up to 18 day Screening period. Subjects will be evaluated for safety, tolerability and efficacy bi-weekly throughout the study. Pharmacokinetics (PK) of Arikace™ in blood, sputum and 24-hour urine will be determined in a subgroup of study subjects who consent to PK evaluation.
At the completion of the TR02-109 protocol, subjects who have consented and meet study safety criteria may enroll in the long-term, open-label, multi-cycle extension study of 590 mg of Arikace™ (under a separate protocol TR02-110).
- Conditions
- Cystic Fibrosis
- Being tested
- Liposomal amikacin for inhalation, Placebo for liposomal amikacin for inhalation
- Keywords
- Cystic Fibrosis, Respiratory Infections, Pulmonary Cystic Fibrosis, Amikacin, Anti-bacterial agents
- 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.
Liposomal amikacin for inhalationDrug
- Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
- 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
- Administration time is approximately 13 minutes.
- Liposomal amikacin for inhalation will be administered for 28 days followed by 56 days off treatment.
Placebo for liposomal amikacin for inhalationDrug
- Placebo is provided as a sterile aqueous lipid dispersion for inhalation via nebulization.
- Administration procedures, volume and administration time are the same as for Arikace™.
- Placebo will be administered for 28 days.
| Study group | Role | What they receive |
|---|---|---|
| Arikace™ | Experimental | Liposomal amikacin for inhalation Receives: Drug: Liposomal amikacin for inhalation |
| Placebo | Placebo comparator | Placebo for liposomal amikacin for inhalation Receives: Drug: Placebo for liposomal amikacin for inhalation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to first protocol defined pulmonary exacerbation
Measured: 84 days
Secondary outcomes (6)
Relative change in FEV1 (liters) and FEV1 (% predicted)
Measured: 84 days
Proportion of subjects experiencing protocol defined exacerbations
Measured: 84 days
Time to first antipseudomonal antibiotic treatment for pulmonary exacerbation
Measured: 84 days
Change in Pseudomonas aeruginosa and Burkholderia sp. density in sputum
Measured: 84 days
Change in patient reported outcomes/symptoms
Measured: 84 days
Evaluation of safety and tolerability
Measured: 84 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Mar 2011 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 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
- NCT01315691
- Sponsor's protocol ID
- TR02-109
- Status last verified
- Jul 2018
- First submitted
- 14 Mar 2011
- Last updated
- 31 Jul 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Placebo-Controlled, Double-Blind, Multicenter Study to Assess the Efficacy, Safety and Tolerability of Arikace™ in Cystic Fibrosis Patients With Chronic Infection Due to Pseudomonas Aeruginosa
- ClinicalTrials.gov ID
- NCT01315691
- Acronym / sponsor ID
- TR02-109
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic Fibrosis, Respiratory Infections, Pulmonary Cystic Fibrosis, Amikacin, Anti-bacterial agents
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Insmed IncorporatedIndustry (pharma, biotech, devices)
- First posted
- 15 Mar 2011 (estimated)
- Results
- Not posted in the source record
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 the prevalence of chronic Pseudomonas aeruginosa infections. The Pseudomonas aeruginosa infection rate in CF patients increases with age and by age 18 years approximately 85% of CF patients in the US are infected. Liposomal amikacin for inhalation (Arikace™) was developed as a possible treatment for chronic infection due to Pseudomonas aeruginosa in CF patients.
The purpose of this double-blind, placebo controlled study is to determine whether Arikace™ is effective in treating chronic lung infections caused by Pseudomonas aeruginosa in Cystic Fibrosis subjects. The study will enroll approximately 300 subjects in clinics in the US, Canada, Europe, Australia and New Zealand. Subjects will be randomized to 590 mg Arikace™ or placebo and will receive treatment for 28 days followed by a 56 day safety follow-up period. The subjects will be required to visit the clinic 8 times (including the Screening visit) over a period of approximately 3 months. No overnight stays at the clinic will be required. At the completion of the TR02-109 protocol, subjects who have consented and meet study safety criteria may enroll in the long-term, open-label, multi-cycle extension study of 590 mg of Arikace™ (under a separate protocol TR02-110).
Detailed description
Cystic Fibrosis (CF) is a genetic disease resulting from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Patients with CF manifest pathological changes in a variety of organs that express CFTR. The lungs are frequently affected often resulting in chronic infections by bacteria such as Pseudomonas aeruginosa and airway inflammation. Treatment of chronic lung infections is one of the principal goals of CF therapy. Arikace™ (liposomal amikacin for inhalation) is a sustained-release formulation of amikacin encapsulated inside nanoscale liposomal carriers designed for administration via inhalation. It is hypothesized that the sustained-release pulmonary targeting and biofilm penetration properties of this formulation will have several advantages over current therapies in treating CF patients with chronic lung infection caused by Pseudomonas aeruginosa.
This double blind, placebo controlled Phase 3 study has been designed to evaluate the efficacy, safety and tolerability of Arikace™ in treating CF patients with chronic bronchopulmonary infection. Eligible subjects will be randomized 1:1 to receive 590 mg of Arikace™ or placebo once daily using a PARI Investigational eFlow® Nebulizer. Subjects will receive 28 days of treatment and will then be followed for safety for 56 days. Total study duration is up to 102 days (~3 months) including an up to 18 day Screening period. Subjects will be evaluated for safety, tolerability and efficacy bi-weekly throughout the study. Pharmacokinetics (PK) of Arikace™ in blood, sputum and 24-hour urine will be determined in a subgroup of study subjects who consent to PK evaluation.
At the completion of the TR02-109 protocol, subjects who have consented and meet study safety criteria may enroll in the long-term, open-label, multi-cycle extension study of 590 mg of Arikace™ (under a separate protocol TR02-110).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arikace™ | Experimental | Drug: Liposomal amikacin for inhalationLiposomal amikacin for inhalation |
| Placebo | Placebo comparator | Drug: Placebo for liposomal amikacin for inhalationPlacebo for liposomal amikacin for inhalation |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Written informed consent or assent
- Confirmed diagnosis of CF
- History of chronic infection with Pseudomonas aeruginosa
- History of documented pulmonary exacerbation requiring treatment with antibiotics in the 12 months prior to Screening
- Sputum culture positive for Pseudomonas aeruginosa at Screening
Key exclusion criteria (12 listed)
- FEV1 <25% of predicted value at Screening
- History of hypersensitivity to aminoglycosides
- History of major complications of lung disease (including atelectasis, pneumothorax, major pleural effusion) within 8 weeks prior to Screening
- Hemoptysis of ≥60 mL in a 24-hour period within 4 weeks prior to Screening
- History of pulmonary tuberculosis or non-tuberculous mycobacterial lung disease treated within 2 years prior to Screening or requiring treatment at the time of Screening
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 |
|---|---|---|
| Liposomal amikacin for inhalation* Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization. * 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer. * Administration time is approximately 13 minutes. * Liposomal amikacin for inhalation will be administered for 28 days followed by 56 days off treatment. | Drug | Arikace™ |
| Placebo for liposomal amikacin for inhalation* Placebo is provided as a sterile aqueous lipid dispersion for inhalation via nebulization. * Administration procedures, volume and administration time are the same as for Arikace™. * Placebo will be administered for 28 days. | Drug | Placebo |
Primary outcome
Time to first protocol defined pulmonary exacerbation
Time frame: 84 days
6 secondary outcomes
Relative change in FEV1 (liters) and FEV1 (% predicted)
Time frame: 84 days
Proportion of subjects experiencing protocol defined exacerbations
Time frame: 84 days
Time to first antipseudomonal antibiotic treatment for pulmonary exacerbation
Time frame: 84 days
Change in Pseudomonas aeruginosa and Burkholderia sp. density in sputum
Time frame: 84 days
Change in patient reported outcomes/symptoms
Time frame: 84 days
Evaluation of safety and tolerability
Time frame: 84 days
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2018.
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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
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Liposomal amikacin for inhalation in people with Cystic Fibrosis, run by Insmed Incorporated.
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 6 inclusion and 12 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: withdrawn, as of the sponsor's last update on 31 Jul 2018. Status can change between updates, so confirm with the study contact before making plans.
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 phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Time to first protocol defined pulmonary exacerbation (time frame: 84 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 31 Jul 2018.