Trial Outcomes & Findings for Extension of BPS-MR-PAH-203 in Pulmonary Arterial Hypertension (PAH) Patients (NCT NCT00990314)
NCT ID: NCT00990314
Last Updated: 2019-09-16
Results Overview
A treatment-emergent adverse event (TEAE) is defined as an event not present prior to the initiation of the treatment or any event already present that worsens in either intensity or frequency following exposure to the treatment. AEs occurring more than 3 days after the last day study drug was taken in the study was not included in the statistical analyses or summaries (except for subjects with adverse events leading to study drug withdrawn). Only TEAEs that occurred during the treatment period of the BPS-MR-PAH-204 study were summarized. Any adverse event starting prior to the first dose of study drug was excluded from the summary analyses and only presented in the data listings. All efficacy results are descriptive; no statistical analysis was conducted
COMPLETED
PHASE2
31 participants
Up to 42 months
2019-09-16
Participant Flow
A Protocol Amendment was to include an optional arm investigating Beraprost Sodium Modified Release Tablets administered four times daily (QID), however, no participants were enrolled into this arm.
Participant milestones
| Measure |
Beraprost Sodium
Beraprost Sodium Modified Release Tablet, 60 micrograms(mcg), twice a day dosing
|
|---|---|
|
Overall Study
STARTED
|
31
|
|
Overall Study
COMPLETED
|
19
|
|
Overall Study
NOT COMPLETED
|
12
|
Reasons for withdrawal
| Measure |
Beraprost Sodium
Beraprost Sodium Modified Release Tablet, 60 micrograms(mcg), twice a day dosing
|
|---|---|
|
Overall Study
Death
|
2
|
|
Overall Study
Adverse Event
|
2
|
|
Overall Study
Withdrawal by Subject
|
3
|
|
Overall Study
Non-Compliance
|
1
|
|
Overall Study
Physician Decision
|
3
|
|
Overall Study
Lack of Efficacy
|
1
|
Baseline Characteristics
Baseline characteristics were defined as those recorded at the baseline visit of the lead-in study, BPS-MR-PAH-203.
Baseline characteristics by cohort
| Measure |
Beraprost Sodium
n=31 Participants
Beraprost Sodium Modified Release Tablet, 60 micrograms(mcg), twice a day dosing
|
|---|---|
|
Age, Continuous
|
46.5 years
STANDARD_DEVIATION 14.16 • n=5 Participants
|
|
Sex: Female, Male
Female
|
26 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
27 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
30 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Six-Minute Walk Distance
|
360.7 meters
STANDARD_DEVIATION 78.0 • n=5 Participants • Baseline characteristics were defined as those recorded at the baseline visit of the lead-in study, BPS-MR-PAH-203.
|
|
Borg Dyspnea Score
|
3.2 score
STANDARD_DEVIATION 1.97 • n=5 Participants • Baseline characteristics were defined as those recorded at the baseline visit of the lead-in study, BPS-MR-PAH-203.
|
PRIMARY outcome
Timeframe: Up to 42 monthsA treatment-emergent adverse event (TEAE) is defined as an event not present prior to the initiation of the treatment or any event already present that worsens in either intensity or frequency following exposure to the treatment. AEs occurring more than 3 days after the last day study drug was taken in the study was not included in the statistical analyses or summaries (except for subjects with adverse events leading to study drug withdrawn). Only TEAEs that occurred during the treatment period of the BPS-MR-PAH-204 study were summarized. Any adverse event starting prior to the first dose of study drug was excluded from the summary analyses and only presented in the data listings. All efficacy results are descriptive; no statistical analysis was conducted
Outcome measures
| Measure |
Beraprost Sodium
n=31 Participants
Beraprost Sodium Modified Release Tablet, 60 micrograms(mcg), twice a day dosing
|
|---|---|
|
Number of Subjects Reporting at Least One Treatment-Emergent Adverse Event (TEAE)
|
27 Participants
|
PRIMARY outcome
Timeframe: Up to 42 monthsA treatment-emergent adverse event (TEAE) is defined as an event not present prior to the initiation of the treatment or any event already present that worsens in either intensity or frequency following exposure to the treatment. AEs occurring more than 3 days after the last day study drug was taken in the study was not included in the statistical analyses or summaries (except for subjects with adverse events leading to study drug withdrawn). Only TEAEs that occurred during the treatment period of the BPS-MR-PAH-204 study were summarized. Any adverse event starting prior to the first dose of study drug was excluded from the summary analyses and only presented in the data listings. All efficacy results are descriptive; no statistical analysis was conducted.
Outcome measures
| Measure |
Beraprost Sodium
n=31 Participants
Beraprost Sodium Modified Release Tablet, 60 micrograms(mcg), twice a day dosing
|
|---|---|
|
Number of Reported Treatment-Emergent Adverse Events
|
230 TEAEs
|
SECONDARY outcome
Timeframe: Baseline and 42 monthsPopulation: Only participants with both a measurement at Baseline and at the End of Study visit are presented.
Area used for the Six Minute Walk Test (6MWT) was pre-measured at 30 meters in length. Rest periods were allowed if patient could no longer continue. If patient needed to rest, he/she could stand or sit and then begin again when rested but the clock continued to run. At the end of 6 minutes, the tester called "stop" while stopping the watch and then measured the distance walked. For purposes of the 6MWT, if patient was assessed at Baseline using oxygen therapy, all future 6MWT were conducted in the same manner. All efficacy results are descriptive; no statistical analysis was conducted.
Outcome measures
| Measure |
Beraprost Sodium
n=22 Participants
Beraprost Sodium Modified Release Tablet, 60 micrograms(mcg), twice a day dosing
|
|---|---|
|
Change in Six-Minute-Walk Distance (6MWD)
|
24.09 meters
Standard Deviation 81.01
|
SECONDARY outcome
Timeframe: Baseline and 42 monthsPopulation: Only participants with both a measurement at Baseline and at the End of Study visit are presented.
The modified 0-10 category-ratio Borg scale consists of an 11-point scale rating the maximum level of dyspnea experienced during the 6MWT. Scores range from 0 (for the best condition) and 10 (for the worst condition) with nonlinear spacing of verbal descriptors of severity corresponding to specific numbers. The participant chose the number or the verbal descriptor to reflect presumed ratio properties of sensation or symptom intensity. Baseline was defined as the last non-missing evaluation preceding the first dose of study drug in study BPS-MR-PAH-203. Only participants with both a measurement at baseline and at the given visit are presented. All efficacy results are descriptive; no statistical analysis was conducted.
Outcome measures
| Measure |
Beraprost Sodium
n=22 Participants
Beraprost Sodium Modified Release Tablet, 60 micrograms(mcg), twice a day dosing
|
|---|---|
|
Change in Borg Dyspnea Score
|
0.86 scores on a scale
Standard Deviation 1.89
|
SECONDARY outcome
Timeframe: Up to 42 monthsNumber of Participants that experienced Clinical Worsening in the opinion of the Investigator. Clinical Worsening was defined as any of these events following the Baseline visit: Death, Transplantation or atrial septostomy, Clinical deterioration as defined by: Hospitalization as a result of PAH symptoms or Initiation of any new PAH specific therapy (e.g. ERA, PDE-5 inhibitor, prostanoid). All efficacy results are descriptive; no statistical analysis was conducted.
Outcome measures
| Measure |
Beraprost Sodium
n=31 Participants
Beraprost Sodium Modified Release Tablet, 60 micrograms(mcg), twice a day dosing
|
|---|---|
|
Number of Participants That Experienced Clinical Worsening
Death
|
1 Participants
|
|
Number of Participants That Experienced Clinical Worsening
Hospitalization As A Result of PAH Symptoms
|
2 Participants
|
|
Number of Participants That Experienced Clinical Worsening
New PAH Therapies
|
4 Participants
|
|
Number of Participants That Experienced Clinical Worsening
Transplantation or atrial septostomy
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline and 42 monthsPopulation: Only participants with both a measurement at Baseline and at the End of Study visit are presented.
Change from Baseline in participant clinical status was recorded according to the World Health Organization (WHO) Functional Class. A change from lower to higher functional class (i.e. 'III to IV' or 'II to III') was considered as a deterioration. A change from higher to lower functional class (i.e. 'III to II' or 'II to I') was considered as an improvement. All efficacy results are descriptive; no statistical analysis was conducted.
Outcome measures
| Measure |
Beraprost Sodium
n=25 Participants
Beraprost Sodium Modified Release Tablet, 60 micrograms(mcg), twice a day dosing
|
|---|---|
|
Number of Participants With a Change in WHO Functional Class
Deteriorated: Change from Class II to Class III
|
5 Participants
|
|
Number of Participants With a Change in WHO Functional Class
Deteriorated: Change from Class III to Class IV
|
1 Participants
|
|
Number of Participants With a Change in WHO Functional Class
No Change in Class
|
10 Participants
|
|
Number of Participants With a Change in WHO Functional Class
Improved: Change from Class II to Class I
|
2 Participants
|
|
Number of Participants With a Change in WHO Functional Class
Improved: Change from Class III to Class II
|
7 Participants
|
Adverse Events
Beraprost Sodium
Serious adverse events
| Measure |
Beraprost Sodium
n=31 participants at risk
Beraprost Sodium Modified Release Tablet, 60 micrograms(mcg), twice a day dosing
|
|---|---|
|
Metabolism and nutrition disorders
Hyperkalaemia
|
3.2%
1/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
3.2%
1/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Respiratory, thoracic and mediastinal disorders
Worsening pulmonary arterial hypertension
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Cardiac disorders
Cardiac failure acute
|
3.2%
1/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Cardiac disorders
Right ventricular failure
|
3.2%
1/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
General disorders
Edema peripheral
|
3.2%
1/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Breast cancer
|
3.2%
1/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Cardiac disorders
Cardio-respiratory arrest
|
3.2%
1/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Cardiac disorders
Atrial flutter
|
3.2%
1/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Gastrointestinal disorders
Inguinal hernia
|
3.2%
1/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
General disorders
Chest pain
|
3.2%
1/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Gastrointestinal disorders
Enterocolitis
|
3.2%
1/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Infections and infestations
Varicella
|
3.2%
1/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Vascular disorders
Extremity necrosis
|
3.2%
1/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Infections and infestations
Bronchitis
|
3.2%
1/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
Other adverse events
| Measure |
Beraprost Sodium
n=31 participants at risk
Beraprost Sodium Modified Release Tablet, 60 micrograms(mcg), twice a day dosing
|
|---|---|
|
Nervous system disorders
Headache
|
32.3%
10/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Gastrointestinal disorders
Nausea
|
29.0%
9/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Gastrointestinal disorders
Diarrhoea
|
22.6%
7/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Nervous system disorders
Dizziness
|
16.1%
5/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
General disorders
Fatigue
|
16.1%
5/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Vascular disorders
Flushing
|
16.1%
5/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Cardiac disorders
Palpitations
|
12.9%
4/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
12.9%
4/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Infections and infestations
Bronchitis
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
9.7%
3/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Metabolism and nutrition disorders
Hypercholesterolaemia
|
9.7%
3/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Vascular disorders
Hypotension
|
9.7%
3/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Infections and infestations
Nasopharyngitis
|
9.7%
3/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Blood and lymphatic system disorders
Anaemia
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Investigations
Blood creatine phosphokinase increased
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Investigations
Carbon dioxide decreased
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
General disorders
Chest Pain
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Metabolism and nutrition disorders
Fluid overload
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Vascular disorders
Hot flush
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Investigations
Neutrophil count increased
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
General disorders
Oedema
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Skin and subcutaneous tissue disorders
Rash
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Nervous system disorders
Somnolence
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Nervous system disorders
Syncope
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Infections and infestations
Tooth abscess
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Investigations
Urine analysis abnormal
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Gastrointestinal disorders
Vomiting
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Investigations
White blood cell count increased
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
|
Investigations
Brain natriuretic peptide increased
|
6.5%
2/31 • From baseline to 30 days after study treatment discontinuation, up to 42 months.
|
Additional Information
Lung Biotechnology PBC Study Director
Lung Biotechnology PBC
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60