Clinical Study of Macitentan in Patients With Pulmonary Arterial Hypertension to Psychometrically Validate the PAH-SYMPACT Instrument
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|ClinicalTrials.gov Identifier: NCT01841762|
Recruitment Status : Completed
First Posted : April 29, 2013
Results First Posted : October 31, 2018
Last Update Posted : October 31, 2018
SYMPHONY is prospective, multi-center, open-label, single-arm, Phase 3b psychometric validation study of the PAH-SYMPACT, a new quality of life questionnaire for patients with pulmonary arterial hypertension. Patients will be in the study for 5 1/2 months, 4 months of which they will receive macitentan, 10 mg, once daily.
The primary objectives are to demonstrate the final content validity of the PAH SYMPACT instrument, to demonstrate the psychometric characteristics of reliability and construct validity of the PAH-SYMPACT instrument, and to demonstrate the ability of the PAH SYMPACT instrument to detect change. The secondary objective is to assess the safety of macitentan in patients with pulmonary arterial hypertension. The exploratory objective is to explore the effects of macitentan on PAH symptoms and their impact (as measured by the PAH-SYMPACT) in patients with pulmonary arterial hypertension.
|Condition or disease||Intervention/treatment||Phase|
|Pulmonary Arterial Hypertension||Drug: Macitentan||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||284 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Multi-center, Open-label, Single-arm, Phase 3b Study of Macitentan in Patients With Pulmonary Arterial Hypertension to Psychometrically Validate the PAH-SYMPACT Instrument|
|Actual Study Start Date :||April 1, 2013|
|Actual Primary Completion Date :||November 1, 2015|
|Actual Study Completion Date :||November 1, 2015|
Macitentan tablet, dose of 10 mg, once daily
Macitentan tablet, dose of 10 mg, once daily
Other Name: Macitentan / ACT-064992
- Development and Refinement of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT) [ Time Frame: From Screening Visit (Day -14) to End of Treatment (EOT) Visit (Visit 4, Week 16) ]Content validity of the PAH-SYMPACT was assessed using item performance, exploratory and confirmatory factor analysis. The final item content and domain structure of PAH-SYMPACT was determined based on these analyses from the Steering Committee (expert clinicians) and findings from the qualitative research done with patients previously.
- Validation of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT), With Reliability Assessed Via Test-retest Reliability. [ Time Frame: From ePRO period 1 (Days -14 to -8) to ePRO period 2 (Days -7 to -1) in screening period. ]The reliability of the PAH-SYMPACT is assessed by test-retest reliability. Intra-class correlation coefficients (ICCs) assess test-retest reliability for the symptom and impact part scores as well as domains. ICCs equal to or greater than 0.70 are considered to demonstrate good test-retest reliability for total and domain scores.
- Validation of the Patient-reported Outcome Measure of Symptoms and Their Impact in PAH (the PAH-SYMPACT), Assessing Internal Consistency Reliability. [ Time Frame: From ePRO period 1 (Days -14 to -8) to ePRO period 2 (Days -7 to -1) in screening period. ]The reliability of the PAH-SYMPACT is assessed by internal consistency reliability. This was determined using Cronbach's alpha-a value on an internal level scale from 0 to 1.0 with higher scores indicating a more-reliable (precise) instrument.
- Number of Participants With Treatment-emergent Adverse Events, Serious Adverse Events, and Adverse Events Resulting in Patient Study Drug Discontinuation Between Time Periods, BL to End of Study Visit (EoS, Week 16+30 Days for Follow-up Safety Visits) [ Time Frame: From Day 1 (Baseline Visit) to End of Study visit (EoS). ]Safety events are reported and documented as defined in study protocol.
- Assessment of the Sensitivity to Detect Change in the Symptoms and Impacts Domain Scores of the PAH-SYMPACT From Baseline to Week 16. [ Time Frame: From Screening period (Days -7 to -1) to Week 16 (7-day period prior to Week 16 visit). ]Sensitivity to change is an aspect of construct validity and represents the instrument's ability to detect underlying change. Sensitivity to change was examined to compare the difference in mean score in each domain of the PAH-SYMPACT. The symptoms and impacts domains consisted of 11 items each reported on a 7-point Likert Scale (from 0=no symptom/with no difficulty at all/not at all to 6=very severe symptoms/very much/extremely/not able at all). An average symptoms domain score is determined based on the daily scores of the containing items. An average impacts domain score is determined based on the items in the domain.
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Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01841762
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|Study Chair:||Alain Romero, PharmD, PhD||Actelion Pharmaceuticals US, Inc|
|Study Chair:||Gary Palmer, MD, MBA||Actelion Pharmaceuticals US, Inc.|