Study of Sotatercept in Newly Diagnosed Intermediate- and High-risk PAH Patients (HYPERION)
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|ClinicalTrials.gov Identifier: NCT04811092|
Recruitment Status : Recruiting
First Posted : March 23, 2021
Last Update Posted : August 12, 2021
|Condition or disease||Intervention/treatment||Phase|
|Pulmonary Arterial Hypertension||Drug: Sotatercept Other: Placebo||Phase 3|
Study A011-13 is Phase 3, randomized, double-blind, placebo-controlled study to evaluate sotatercept when added to background PAH therapy in newly diagnosed intermediate- or high risk PAH patients.
Participants enrolled in the study will have a diagnosis within 6 months of study screening of symptomatic PAH (WHO Group 1, classified as FC II or III) and presentation of idiopathic or heritable PAH, PAH associated with connective tissue diseases (CTD), drug- or toxin- induced PAH, post shunt correction PAH, or PAH presenting at least 1 year following the correction of congenital heart defects.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||662 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|Official Title:||A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate Sotatercept When Added to Background Pulmonary Arterial Hypertension (PAH) Therapy in Newly Diagnosed Intermediate- and High-risk PAH Patients|
|Actual Study Start Date :||July 28, 2021|
|Estimated Primary Completion Date :||June 2028|
|Estimated Study Completion Date :||August 2028|
Placebo Comparator: Placebo plus background PAH therapy
Administered subcutaneously (SC) every 21 days plus background PAH therapy
Experimental: Sotatercept plus background PAH therapy
Administered at a starting dose of 0.3 mg/kg, with a target dose of 0.7 mg/kg, subcutaneously (SC) every 21 days plus background PAH therapy
Sotatercept (ACE-011) is a recombinant fusion protein consisting of the extracellular domain of the human activin receptor type IIA linked to the Fc piece of human IgG1
Other Name: ACE-011
- Time to Clinical Worsening, defined as the first confirmed morbidity event or death. [ Time Frame: From screening to the first clinical worsening event, up to 56 months. ]Clinical worsening events are defined as all-cause death, non-planned PAH-related hospitalization of ≥ 24 hours in duration, atrial septostomy, lung transplant and deterioration in performance in 6-minute walk test from baseline combined with one of the following conditions: worsening of WHO functional class from baseline, signs/symptoms of increased right heart failure, addition of a background PAH therapy or change in the composition of PAH background therapy, including an increase in parenteral prostacyclin of ≥ 10%. All events will be adjudicated by a blinded, independent committee of clinical experts.
- Multicomponent improvement endpoint of 6-minute walk distance (6MWD), NT-proBNP and WHO functional class (FC). [ Time Frame: From initiation of treatment to Week 24 ]
Multicomponent improvement endpoint measured by the proportion of participants achieving all of the following at Week 24 relative to baseline
- Improvement in 6MWD
- Improvement or maintenance/achievement of NT-proBNP
- Improvement in WHO FC or maintenance of WHO FC II
- Proportion of participants who achieved a low Registry to Evaluate Early and Long Term PAH Disease Management (REVEAL) Lite 2 risk score. [ Time Frame: From initiation of treatment to Week 24 ]REVEAL Lite 2 risk score in each participant was measured at Week 24 versus baseline.
- Proportion of participants who maintain or achieve a low simplified French risk score (FC) [ Time Frame: From initiation of treatment to Week 24 ]Proportion of participants who maintain or achieve a low risk score at Week 24 versus baseline using the simplified French Risk score calculator was measured.
- Change from baseline in NT-proBNP levels. [ Time Frame: From initiation of treatment to Week 24 ]NT-proBNP was measured at baseline and Week 24.
- Proportion of participants who improve in WHO FC or maintain WHO FC II at 24 weeks from baseline. [ Time Frame: From initiation of treatment to Week 24 ]The severity of an individual's PAH symptoms was graded using WHO FC system at baseline and Week 24.
- Change in 6MWD. [ Time Frame: From initiation of treatment to Week 24 ]6-minute walk test is a clinical exercise test to assess the functional capacity. 6MWD at baseline and Week 24 were measured.
- Change in EuroQol - 5 dimensions scale 5 levels (EQ 5D 5L) index score. [ Time Frame: From initiation of treatment to Week 24 ]EQ 5D 5L index score measures health-related quality of life states in adults. The EQ 5D 5L questionnaire is designed for self-completion and captures information directly from the respondent. EQ 5D 5L index scores at baseline and Week 24 were measured.
- Change in Pulmonary Arterial Hypertension Symptoms and Impact (PAH-SYMPACT)® [ Time Frame: From initiation of treatment to Week 24 ]PAH SYMPACT is a self-rating questionnaire to assess symptoms and their physical and cognitive/emotional impact. The PAH-SYMPACT® questionnaire consists of consists of 16 symptom and 25 impact items. A higher score indicates worse symptoms. Responses in PAH-SYMPACT questionnaire at baseline and Week 24 were recorded.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04811092
|Contact: Clinical Trial Manager||617-649-9200||Clinicaltrials011@acceleronpharma.com|
|United States, Kansas|
|University of Kansas Medical Center||Recruiting|
|Kansas City, Kansas, United States, 66160|
|Contact: Luigi Boccardi 918-588-4022 firstname.lastname@example.org|
|Contact: Kimberly Cyan (918) 588 - 0046 email@example.com|
|Principal Investigator: Leslie A. Spikes, MD|
|United States, Michigan|
|Cardiovascular Center||Not yet recruiting|
|Ann Arbor, Michigan, United States, 48109|
|Principal Investigator: Victor Moles, MD|
|Study Director:||John Butler, MD||Acceleron Pharma Inc.|