Phase 3 N=336 Randomized Treatment

Study to Evaluate Efficacy of LAI When Added to Multi-drug Regimen Compared to Multi-drug Regimen Alone

Mycobacterium Infections, Nontuberculous

Bottom Line

View on ClinicalTrials.gov: NCT02344004 ↗

Enrolled (actual)

336

Serious AEs

20.3%

Results posted

Nov 2018

Primary outcome: Primary: Number of Participants Achieving Culture Conversion by Month 6 in the Liposomal Amikacin for Inhalation (LAI) + Multi-drug Regimen (MDR) Arm Compared to the MDR Alone Arm — 65; 10; 159; 102 Participants — p=<0.0001

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: LAI (Liposomal Amikacin for Inhalation) 590 mg (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Insmed Incorporated
Primary completion: Jul 2018

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants Achieving Culture Conversion by Month 6 in the Liposomal Amikacin for Inhalation (LAI) + Multi-drug Regimen (MDR) Arm Compared to the MDR Alone Arm	65; 10; 159; 102	<0.0001 sig
PRIMARY Number of Participants Achieving Durable Culture Conversion Through 3 Months Off Treatment in the LAI + MDR Arm Compared to the MDR Arm Alone	36; 0; 188; 112	<0.0001 sig
SECONDARY Change From Baseline (Day 1) to Month 6 in the Six-Minute Walk Test (6MWT) Distance in the LAI + MDR Arm Compared to the MDR Alone Arm	424.2; 420.9; 420.6; 420.3; -3.6; -0.5	0.7804
SECONDARY Time to Culture Conversion by Month 6 in the LAI + MDR Arm Compared to the MDR Alone Arm	2.13; NA; NA; NA	<0.0001 sig
SECONDARY Number of Participants Achieving Sustained Culture Conversion at the End of Treatment (EOT) in the LAI + MDR Arm Compared to the MDR Arm Alone	41; 3; 183; 109	—
SECONDARY Change in 6-Minute Walk Test (6MWT) Distance at EOT in the LAI Arm Compared to a Multi-drug Regimen Alone	—	—
SECONDARY Change From Baseline (Day 1) at Month 6 in the St. George's Respiratory Questionnaire (SGRQ) Total Score	36.555; 38.409; 38.715; 37.368; 2.009; -1.312	—

Summary

A study to evaluate the effectiveness of Liposomal Amikacin for Inhalation (LAI) 590 mg administered once daily (QD) when added to multi-drug regimen (MDR) in participants with Nontuberculous Mycobacterial (NTM) lung infection caused by Mycobacterium Avium Complex (MAC) that were refractory to treatment. Participants were randomized 2:1 to LAI 590 mg administered QD + MDR or MDR alone.

Eligibility Criteria

Inclusion Criteria

Be continually positive for MAC on sputum culture while adhering to a multi-drug treatment regimen for a minimum duration of 6 months which is either ongoing or was completed no more than 12 months before screening
Be diagnosed with MAC NTM lung infection with evidence of nodular bronchiectasis and/or fibrocavitary disease by chest CT
Be willing to adhere to multi-drug treatment regimen during the course of the study

Exclusion Criteria

Patients with cystic fibrosis
Positive pregnancy test or lactation at screening. All women of child bearing potential will be tested. Women not of childbearing potential are defined as postmenopausal (i.e., amenorrheic for at least 1 year), or surgically or naturally sterile.
Active pulmonary tuberculosis requiring treatment at screening
History of lung transplantation
Prior exposure to LAI (including clinical study).

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02344004). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.