Study to Evaluate Arikace™ in CF Patients With Chronic Infection Due to Pseudomonas Aeruginosa

NCT01315691 | Withdrawn Phase 3 DMC

• 1 other identifier: TR02-109
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief 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. 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).

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis; Respiratory Infections; Pulmonary Cystic Fibrosis; Amikacin; Anti-bacterial agents

Outcome Measures

Primary Outcomes (1)

• Time to first protocol defined pulmonary exacerbation

  84 days

Secondary Outcomes (6)

• Relative change in FEV1 (liters) and FEV1 (% predicted)

  84 days

• Proportion of subjects experiencing protocol defined exacerbations

  84 days

• Time to first antipseudomonal antibiotic treatment for pulmonary exacerbation

  84 days

• Change in Pseudomonas aeruginosa and Burkholderia sp. density in sputum

  84 days

• Change in patient reported outcomes/symptoms

  84 days

Study Arms (2)

Arikace™

EXPERIMENTAL

Liposomal amikacin for inhalation

Drug: Liposomal amikacin for inhalation

Placebo

PLACEBO COMPARATOR

Placebo for liposomal amikacin for inhalation

Drug: Placebo for liposomal amikacin for inhalation

Interventions

Liposomal amikacin for inhalation DRUG

* 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.

Arikace™

Placebo for liposomal amikacin for inhalation DRUG

* 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.

Placebo

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Insmed Incorporated: lead

MeSH Terms

Conditions

Cystic Fibrosis; Respiratory Tract Infections

Interventions

Inhalation

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Infections

Intervention Hierarchy (Ancestors)

Respiratory Mechanics; Respiration; Respiratory Physiological Phenomena; Circulatory and Respiratory Physiological Phenomena

Study Officials

• Gina Eagle, MD

  Insmed Incorporated

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 14, 2011
• First Posted: March 15, 2011
• Last Updated: July 31, 2018

Record last verified: 2018-07