NCT04677543

Validation of Patient Reported Outcome Measures in Participants With Nontuberculous Mycobacterial Lung Infection Caused by Mycobacterium Avium Complex

A Randomized, Double-Blind, Placebo-Controlled, Active Comparator, Multicenter Study to Validate Patient-Reported Outcome Instruments in Adult Subjects With Newly Diagnosed Nontuberculous Mycobacterial (NTM) Lung Infection Caused by Mycobacterium Avium Complex (MAC)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 99 (actual)
• Sponsor: Insmed Incorporated · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to generate evidence demonstrating the domain specification (via modern psychometric methods), reliability, validity, and responsiveness (within-subject meaningful change) of the Patient-Reported Outcome (PRO) endpoints.

Detailed description

Participants will also complete the EXAcerbations of Chronic Pulmonary Disease Tool (EXACT), EXACT Respiratory Symptoms (EXACT-RS), St. George Respiratory Questionnaire (SGRQ), Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-Fatigue), Patient Global Impression of Severity - Respiratory (PGIS-Respiratory), and Patient Global Impression of Severity - Fatigue (PGIS-Fatigue) at baseline and throughout the study as anchors for the validation of the QoL-B and PROMIS F-SF 7a.

Conditions

• Mycobacterium Infections, Nontuberculous

Interventions

Type	Name	Description
DRUG	ALIS	Inhalation via nebulization over approximately 6 to 15 minutes.
DRUG	Azithromycin	Oral tablet
DRUG	Ethambutol	Oral tablet
DRUG	ELC	Inhalation via nebulization over approximately 6 to 15 minutes.

Timeline

• Start date: 2020-12-22
• Primary completion: 2023-05-09
• Completion: 2023-05-09
• First posted: 2020-12-21
• Last updated: 2024-06-28
• Results posted: 2024-06-28

Locations

62 sites across 12 countries: United States, Argentina, Australia, Austria, Denmark, Germany, Israel, Italy, New Zealand, South Korea, Spain, Taiwan

Regulatory

• FDA-regulated drug study