Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) - Extension Phase

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03905642
• Date of registration: 02/04/2019
• Prospective Registration: No
• Primary sponsor: Insmed Incorporated
• Public title: Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) - Extension Phase
• Scientific 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
• Date of first enrolment: January 8, 2009
• Target sample size: 49
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03905642
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Belgium, Hungary, Macedonia, The Former Yugoslav Republic of, North Macedonia, Poland, Serbia, Slovakia, Ukraine

Contacts

• Name: Gina Eagle, MD
• Affiliation: Insmed Incorporated

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Written informed consent obtained from the patient or designated legal guardian prior
to the performance of any study related procedures.

2. Male or female study subjects = 6 years of age or older.

3. 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.

4. 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.

5. 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.

6. FEV1 = 40% of predicted at Screening.

7. SaO2 = 90% at Screening while breathing room air.

8. Ability to comply with study medication use, study visits, and study procedures as
judged by the investigator.

9. Ability to produce 0.5 grams sputum or be willing to undergo an induction to produce
sputum for clinical evaluation.

10. 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:

1. Written informed consent obtained from the patient or designated legal guardian prior
to the performance of any study-related procedures in the extension period.

2. Patient meets all of the above listed inclusion criteria (1-10) of the main protocol.

Exclusion Criteria:

1. Administration of any investigational drug within 8 weeks prior to Screening.

2. Emergency room visit or hospitalization for CF or respiratory-related illness within
the 4 weeks prior to Screening.

3. History of alcohol, medication, or illicit drug abuse within the 1 year prior to
Screening.

4. History of lung transplantation.

5. 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).

6. Positive pregnancy test. All women of childbearing potential will be tested.

7. Use of any anti-pseudomonal antibiotics (IV antibiotics, all inhalation antibiotics,
oral fluoroquinolones) within the 28 days prior to Screening.

8. Initiation of chronic therapy (i.e. TOBI®, high-dose ibuprofen, rhDNase, macrolide
antibiotics) within the 28 days prior to Screening.

9. History of sputum or throat swab culture yielding Burkholderia cepacia within 2 years
of Screening.

10. History of mycobacterial and/or Aspergillus infection requiring treatment within 2
years prior to Screening, and/or history of allergic bronchopulmonary aspergillosis
(ABPA).

11. History of biliary cirrhosis with portal hypertension, or splenomegaly (refer to study
manual).

12. GGT, AST, or ALT = 3 times the upper limit of normal at Screening visit.

13. ANC = 1000 performed at Screening visit.

14. Serum creatinine > 1.5 times normal performed at Screening visit.

15. History of daily, continuous oxygen supplementation or requirement for more than 2
L/min at night.

16. 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:

1. Patient meets any criteria for exclusion as listed above in the main protocol.

2. Patient who met any criteria for study drug discontinuation in the main protocol
(TR02-105).



Age minimum: 6 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Arikayce™

Primary Outcome(s)

Adverse Event Profile of 560 mg Once Daily Dose of Arikayce™ Administered for Six Cycles Over Eighteen Months. [Time Frame: 18 Months]

Secondary Outcome(s)

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]

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]

Antipseudomonal Rescue Therapy - Time to Therapy [Time Frame: 18 Months]

Antipseudomonal Rescue Therapy - Duration of Therapy [Time Frame: 18 Months]

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]

Secondary ID(s)

TR02-105 Extension

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 14/05/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03905642