SYMPHONYext: Clinical Study of Macitentan in Patients With PAH to Psychometrically Validate PAH-SYMPACT Instrument
Study Details
Study Description
Brief Summary
SYMPHONY Extension is an extension of AC-055-401, 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. The objective is to assess the long-term safety of macitentan in subjects with PAH beyond the treatment in the AC-055-401 study.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Macitentan Macitentan tablet, dose of 10 mg, once daily. |
Drug: Macitentan
Macitentan tablet, dose of 10 mg, once daily.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Incident Rate of Adverse Events (AEs). [From Visit 1 to Post-treatment safety follow-up visit (30 days after discontinuation of the study drug).]
Safety events are reported and documented as defined in study protocol from baseline to end of treatment period including safety follow-up visit.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Signed informed consent prior to any study-mandated procedure.
-
Patients with PAH who completed study AC-055-401
-
Women of childbearing potential must:
-
Have a negative urine pregnancy test at Visit 1 and agree to perform monthly serum pregnancy tests.
-
Agree to use two methods of contraception from Visit 1 until 1 month after study drug discontinuation.
Exclusion Criteria:
-
Patients who prematurely discontinued study drug in study AC-055-401
-
Females who are lactating or pregnant (positive Visit 1 pregnancy test) or plan to become pregnant during the study
-
Known hypersensitivity to macitentan or its excipients or drugs of the same class
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Kentuckiana Pulmonary Associates | Louisville | Kentucky | United States | |
| 2 | The Carl & Edyth Lindner Center for Research & Education at The Christ Hospital | Cincinnati | Ohio | United States |
Sponsors and Collaborators
- Actelion
Investigators
- Study Chair: Alain Romero, MD, Actelion Pharmaceuticals US, Inc
- Study Chair: Gary Palmer, MD, MBA, Actelion Pharmaceuticals US, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- AC-055-402
Study Results
Participant Flow
| Recruitment Details | All sites activated on the SYMPHONY AC-055-401 clinical trial were invited to participate in this open-label extension study. Due to meeting the study closure milestone, defined as the marketing of Opsumit on 18 OCT 2013, only two sites received activation. Recruitment occurred between Quarter 2 2013 and Quarter 3 2015. |
|---|---|
| Pre-assignment Detail | Subjects who completed the sixteen week treatment of macitentan on the SYMPHONY AC-055-401 clinical trial were eligible to enter this open-label extension study. |
| Arm/Group Title | Macitentan |
|---|---|
| Arm/Group Description | Macitentan tablet, dose of 10 mg, once daily. |
| Period Title: Baseline Extension Visit | |
| STARTED | 4 |
| COMPLETED | 4 |
| NOT COMPLETED | 0 |
| Period Title: Baseline Extension Visit | |
| STARTED | 4 |
| COMPLETED | 0 |
| NOT COMPLETED | 4 |
Baseline Characteristics
| Arm/Group Title | Macitentan |
|---|---|
| Arm/Group Description | Macitentan tablet, dose of 10 mg, once daily. |
| Overall Participants | 4 |
| Age (Count of Participants) | |
| <=18 years |
0
0%
|
| Between 18 and 65 years |
3
75%
|
| >=65 years |
1
25%
|
| Age (Years) [Mean (Full Range) ] | |
| Mean (Full Range) [Years] |
60.8
|
| Sex: Female, Male (Count of Participants) | |
| Female |
4
100%
|
| Male |
0
0%
|
| Ethnicity (NIH/OMB) (Count of Participants) | |
| Hispanic or Latino |
0
0%
|
| Not Hispanic or Latino |
4
100%
|
| Unknown or Not Reported |
0
0%
|
| Race (NIH/OMB) (Count of Participants) | |
| American Indian or Alaska Native |
0
0%
|
| Asian |
0
0%
|
| Native Hawaiian or Other Pacific Islander |
0
0%
|
| Black or African American |
1
25%
|
| White |
3
75%
|
| More than one race |
0
0%
|
| Unknown or Not Reported |
0
0%
|
Outcome Measures
| Title | Incident Rate of Adverse Events (AEs). |
|---|---|
| Description | Safety events are reported and documented as defined in study protocol from baseline to end of treatment period including safety follow-up visit. |
| Time Frame | From Visit 1 to Post-treatment safety follow-up visit (30 days after discontinuation of the study drug). |
Outcome Measure Data
| Analysis Population Description |
|---|
| Four participants represent the Extension Analysis Set, who transitioned from completion of SYMPHONY AC-055-401 to this open-label extension clinical trial. |
| Arm/Group Title | Macitentan |
|---|---|
| Arm/Group Description | Macitentan tablet, dose of 10 mg, once daily. |
| Measure Participants | 4 |
| Participants with severe intensity AEs |
1
25%
|
| Participants with AEs |
3
75%
|
Adverse Events
| Time Frame | ||
|---|---|---|
| Adverse Event Reporting Description | ||
| Arm/Group Title | Macitentan | |
| Arm/Group Description | Macitentan tablet, dose of 10 mg, once daily. | |
All Cause Mortality |
||
| Macitentan | ||
| Affected / at Risk (%) | # Events | |
| Total | 0/4 (0%) | |
Serious Adverse Events |
||
| Macitentan | ||
| Affected / at Risk (%) | # Events | |
| Total | 1/4 (25%) | |
| Cardiac disorders | ||
| Pulmonary Arterial Hypertension Exacerbation | 1/4 (25%) | 1 |
| Pulmonary Hypertension Exacerbation | 1/4 (25%) | 1 |
| Right Heart Failure | 1/4 (25%) | 1 |
| Respiratory, thoracic and mediastinal disorders | ||
| Chronic Obstructive Pulmonary Disease Exacerbation | 1/4 (25%) | 1 |
Other (Not Including Serious) Adverse Events |
||
| Macitentan | ||
| Affected / at Risk (%) | # Events | |
| Total | 3/4 (75%) | |
| Cardiac disorders | ||
| Right Heart Failure | 1/4 (25%) | 1 |
| Ear and labyrinth disorders | ||
| Vertigo | 1/4 (25%) | 1 |
| Gastrointestinal disorders | ||
| Diarrhoea | 1/4 (25%) | 1 |
| Nausea | 1/4 (25%) | 1 |
| Vomiting | 1/4 (25%) | 1 |
| General disorders | ||
| Chest Pain | 1/4 (25%) | 1 |
| Metabolism and nutrition disorders | ||
| Hyperlipidaemia | 1/4 (25%) | 1 |
| Hypokalaemia | 1/4 (25%) | 1 |
| Type 2 Diabetes Mellitus | 1/4 (25%) | 1 |
| Hypoglycaemia | 1/4 (25%) | 1 |
| Musculoskeletal and connective tissue disorders | ||
| Pain In Jaw | 1/4 (25%) | 1 |
| Nervous system disorders | ||
| Headache | 1/4 (25%) | 1 |
| Renal and urinary disorders | ||
| Renal Impairment | 1/4 (25%) | 1 |
| Respiratory, thoracic and mediastinal disorders | ||
| Chronic Obstructive Pulmonary Disease Exacerbation | 1/4 (25%) | 1 |
| Pulmonary Arterial Hypertension Exacerbation | 1/4 (25%) | 1 |
| Pulmonary Hypertension Exacerbation | 1/4 (25%) | 1 |
| Epistaxis | 1/4 (25%) | 1 |
| Skin and subcutaneous tissue disorders | ||
| Hyperhidrosis | 1/4 (25%) | 1 |
| Vascular disorders | ||
| Flushing | 1/4 (25%) | 1 |
Limitations/Caveats
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
Any study-related publication written independently by investigators must be submitted to Actelion for review at least 30 days prior to submission for publication or presentation. Upon review, Actelion provide comments, and may also request alterations and/or deletions for the sole purpose of protecting its confidential information an/or patient rights. Neither the institution nor the investigator should permit publication during such a review period.
Results Point of Contact
| Name/Title | Scott Tsurutani |
|---|---|
| Organization | Actelion Pharmaceuticals US, Inc. |
| Phone | 6508086586 |
| scott.tsurutani@actelion.com |
- AC-055-402