Trial Outcomes & Findings for Pilot Chart Review Study Of Sitaxentan Sodium (Thelin) In Patients With Pulmonary Arterial Hypertension (PAH) (NCT NCT01445873)

NCT ID: NCT01445873

Last Updated: 2020-09-01

Results Overview

Participants still receiving Thelin with evidence of receipt of another PAH-related therapy (eg. bosentan, sildenafil) not previously received during the study period. Mean time in months to therapy augmentation.

• Recruitment status: COMPLETED
• Target enrollment: 36 participants
• Primary outcome timeframe: Day 1 to Month 6
• Results posted on: 2020-09-01

Participant Flow

Data for this retrospective non-interventional study were abtracted from the medical records of subjects who met study entry criteria.

Patterns of treatment with Thelin including duration and daily dosages reflect treament participants received in clinical practice. Treatment was not specified by the protocol.

Participant milestones

Measure	Sitaxentan (Thelin) Duration and dose as prescribed in clinical practice.
Overall Study STARTED	36
Overall Study COMPLETED	36
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pilot Chart Review Study Of Sitaxentan Sodium (Thelin) In Patients With Pulmonary Arterial Hypertension (PAH)

Baseline characteristics by cohort

Measure	Sitaxentan (Thelin) n=36 Participants Duration and dose as prescribed in clinical practice.
Age, Customized 19 to 24 years	0 Participants n=5 Participants
Age, Customized 25 to 30 years	2 Participants n=5 Participants
Age, Customized 31 to 36 years	2 Participants n=5 Participants
Age, Customized 37 to 42 years	1 Participants n=5 Participants
Age, Customized 43 to 48 years	2 Participants n=5 Participants
Age, Customized 49 to 54 years	4 Participants n=5 Participants
Age, Customized 55 to 60 years	2 Participants n=5 Participants
Age, Customized 61 to 66 years	5 Participants n=5 Participants
Age, Customized 67 to 72 years	9 Participants n=5 Participants
Age, Customized 73 to 78 years	7 Participants n=5 Participants
Age, Customized 79 to 84 years	1 Participants n=5 Participants
Age, Customized 85 to 90 years	1 Participants n=5 Participants
Sex: Female, Male Female	29 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants

PRIMARY outcome

Timeframe: Day 1 to Month 6

Population: Full analysis set (FAS): participants with idiopathic pulmonary arterial hypertension (PAH) or PAH secondary to connective tissue disease, receipt of Thelin for treatment of PAH, 6 months of follow-up (except for death) after initial receipt (IR) of Thelin, and at least 1 clinic visit in medical record during 6-month period after IR of Thelin.

Participants still receiving Thelin with evidence of receipt of another PAH-related therapy (eg. bosentan, sildenafil) not previously received during the study period. Mean time in months to therapy augmentation.

Outcome measures

Measure	Sitaxentan (Thelin) n=36 Participants Duration and dose as prescribed in clinical practice.
Patterns of Pharmacotherapy With Sitaxentan Sodium (Thelin): Mean Time to Therapy Augmentation	2.2 months Standard Deviation 1.2

PRIMARY outcome

Timeframe: Day 1 to Month 6

Population: FAS

Participants with evidence of discontinuation of Thelin therapy and evidence of receipt of another PAH-related therapy not previously received during the study period.

Outcome measures

Measure	Sitaxentan (Thelin) n=36 Participants Duration and dose as prescribed in clinical practice.
Patterns of Pharmacotherapy With Sitaxentan Sodium (Thelin): Therapy Switching	0 participants

PRIMARY outcome

Timeframe: Day 1 to Month 6

Population: FAS

Participants were designated as having discontinued Thelin therapy if there was evidence in the medical records that treatment had been terminated.

Outcome measures

Measure	Sitaxentan (Thelin) n=36 Participants Duration and dose as prescribed in clinical practice.
Patterns of Pharmacotherapy With Sitaxentan Sodium (Thelin): Therapy Discontinuation	0 participants

PRIMARY outcome

Timeframe: Day 1 to Month 6

Population: FAS; participants without evidence of discontinuation of Thelin therapy were censored at the end of follow-up (at 6 months or death if prior to 6 months).

Duration of Thelin therapy based on time from index date (Day 1 of treatment) until the date of discontinuation of Thelin therapy.

Outcome measures

Measure	Sitaxentan (Thelin) n=36 Participants Duration and dose as prescribed in clinical practice.
Patterns of Pharmacotherapy With Sitaxentan Sodium (Thelin): Therapy Duration	6 months Standard Deviation 0

PRIMARY outcome

Timeframe: Day 1 to Month 6

Population: Not analyzed: duration of Thelin therapy as number of therapy days dispensed was not summarized; this measure was reported as duration in months only.

Duration of Thelin therapy from initial receipt until date of discontinuation of thelin therapy or the end of follow-up, whichever occurred first.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Day 1 to Month 6

Population: FAS

Daily dosage of Thelin based on information in the medical record for the baseline visit and all follow-up clinic visits.

Outcome measures

Measure	Sitaxentan (Thelin) n=36 Participants Duration and dose as prescribed in clinical practice.
Patterns of Pharmacotherapy With Sitaxentan Sodium (Thelin): Daily Dosage	100.0 mg Interval 100.0 to 100.0

SECONDARY outcome

Timeframe: Day 1 to Month 6

Population: FAS

Use of PAH-related medications other than Thelin described by class of agent received.

Outcome measures

Measure	Sitaxentan (Thelin) n=36 Participants Duration and dose as prescribed in clinical practice.
Use of Other Pulmonary Arterial Hypertension (PAH)-Related Medications Anticoagulants	83.3 percentage of participants
Use of Other Pulmonary Arterial Hypertension (PAH)-Related Medications Diuretics	88.9 percentage of participants
Use of Other Pulmonary Arterial Hypertension (PAH)-Related Medications Calcium channel blockers	22.2 percentage of participants
Use of Other Pulmonary Arterial Hypertension (PAH)-Related Medications Digoxin	5.6 percentage of participants
Use of Other Pulmonary Arterial Hypertension (PAH)-Related Medications Supplemental oxygen	44.4 percentage of participants
Use of Other Pulmonary Arterial Hypertension (PAH)-Related Medications Prostanoids	27.8 percentage of participants
Use of Other Pulmonary Arterial Hypertension (PAH)-Related Medications Other endothelin receptor antagonists	0.0 percentage of participants
Use of Other Pulmonary Arterial Hypertension (PAH)-Related Medications Phosphodiesterase-5 inhibitors	47.2 percentage of participants

SECONDARY outcome

Timeframe: Baseline to Month 6

Population: FAS; N=number of participants with pre-index (prior to Thelin initiation) and ≥ 1 WHO value recorded during follow-up.

Class I: no limitation of usual (usl) physical activity (PA); PA does not increase(d) (incr) dyspnea (dys), fatigue (ftg), chest pain (CP), or syncope (syn); Class II: mild limitation of usl PA; no discomfort at rest, but normal PA causes incr dys, ftg, CP, or presyncope (presyn); Class III: marked limitation of PA; no discomfort at rest but < ordinary activity causes incr dys, ftg, CP, or presyn; Class IV: unable to perform any PA at rest; may have signs of right ventricular failure; sys and/or ftg at rest and symptoms are incr by almost any PA.

Outcome measures

Measure	Sitaxentan (Thelin) n=32 Participants Duration and dose as prescribed in clinical practice.
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) I to FC I	0 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) I to FC II	0 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) I to FC III	0 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) I to FC IV	0 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) II to FC I	0 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) II to FC II	1 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) II to FC III	2 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) II to FC IV	0 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) III to FC I	0 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) III to FC II	4 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) III to FC III	17 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) III to FC IV	3 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) IV to FC I	0 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) IV to FC II	0 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) IV to FC III	3 participants
Change From Baseline in World Health Organization (WHO) Functional Class of Pulmonary Hypertension Pre-index functional class (FC) IV to FC IV	2 participants

SECONDARY outcome

Timeframe: Baseline to Month 6

Population: FAS; N=number of participants with pre-index and ≥ 1 value recorded during follow-up period.

Difference between pre-index and follow-up value.

Outcome measures

Measure	Sitaxentan (Thelin) n=3 Participants Duration and dose as prescribed in clinical practice.
Change From Baseline in Mean Right Atrial Pressure	-1.3 mm Hg Interval -3.9 to 1.2

SECONDARY outcome

Timeframe: Baseline to Month 6

Population: Data not analyzed: no participants had pre-index and follow-up values reported for this outcome measure.

Difference between pre-index and follow-up value.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Month 6

Population: FAS; N=number of participants with pre-index and follow-up values.

Difference between pre-index and follow-up value.

Outcome measures

Measure	Sitaxentan (Thelin) n=3 Participants Duration and dose as prescribed in clinical practice.
Change From Baseline in Pulmonary Capillary Wedge Pressure	3.0 mm Hg Interval 0.1 to 5.9

SECONDARY outcome

Timeframe: Baseline to Month 6

Population: Data not analyzed: no participants had pre-index and follow-up values for this outcome measure.

Difference between pre-index and follow-up value.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to Month 6

Population: FAS; N=number of participants with pre-index and follow-up value.

Difference between pre-index and follow-up value.

Outcome measures

Measure	Sitaxentan (Thelin) n=3 Participants Duration and dose as prescribed in clinical practice.
Change From Baseline in Pulmonary Vascular Resistance	-214.3 Dyn/s/cm5 Interval -419.1 to -9.6

SECONDARY outcome

Timeframe: Baseline to Month 6

Population: FAS; N=number of participants with pre-index and follow-up value.

Difference between pre-index and follow-up value.

Outcome measures

Measure	Sitaxentan (Thelin) n=2 Participants Duration and dose as prescribed in clinical practice.
Change From Baseline in Cardiac Output	0.4 L/min Interval -1.2 to 1.9

SECONDARY outcome

Timeframe: Baseline to Month 6

Population: FAS; N=number of participants with pre-index and ≥ 1 follow-up value.

Difference between pre-index and follow-up value. Combined myocardial performance index calculated by adding isovolumic contraction time and isovolumic relaxation time and dividing the resulting sum by ejection time.

Outcome measures

Measure	Sitaxentan (Thelin) n=8 Participants Duration and dose as prescribed in clinical practice.
Change From Baseline in Tei Index	-0.1 ratio Interval -0.3 to 0.0

SECONDARY outcome

Timeframe: Baseline to Month 6

Population: FAS; N=number of participants with pre-index and ≥ 1 follow-up value.

Difference between pre-index and follow-up value.

Outcome measures

Measure	Sitaxentan (Thelin) n=19 Participants Duration and dose as prescribed in clinical practice.
Change From Baseline in Tricuspid Regurgitant Velocity	-0.3 m/sec Interval -0.5 to 0.0

SECONDARY outcome

Timeframe: Baseline to Month 6

Population: FAS; N=number of participants with pre-index and 1 value recorded during follow-up period.

6MWT was the distance that a participant could walk in 6 minutes. Participants were asked to perform the test at a pace that was comfortable to them, with as many breaks as they needed. Continuous pulse oximetry was conducted during the test for safety. Difference between pre-index and follow-up value.

Outcome measures

Measure	Sitaxentan (Thelin) n=30 Participants Duration and dose as prescribed in clinical practice.
Change From Baseline in the Total Distance Walked During 6 Minute Walk Test (6MWT)	28.5 meters Interval 4.7 to 52.3

SECONDARY outcome

Timeframe: Baseline to Month 6

Population: FAS; N=number of participants with ≥ 1 value recorded during follow-up period.

Borg dyspnoea scale is a 10-point scale where following scores stands for severity of dyspnoea: 0 (no breathlessness at all); 0.5 (very very slight [just noticeable]); 1 (very slight); 2 (slight breathlessness); 3 (moderate); 4 (some what severe); 5 (severe breathlessness); 7 (very severe breathlessness); 9 (very very severe [almost maximum] and 10 (maximum). Difference between pre-index and follow-up value.

Outcome measures

Measure	Sitaxentan (Thelin) n=27 Participants Duration and dose as prescribed in clinical practice.
Change From Baseline in Borg Dyspnoea Score	4.9 units on a scale Interval 4.0 to 5.7

SECONDARY outcome

Timeframe: Baseline to Month 6

Population: FAS; N=number of participants with ≥ 1 value recorded during follow-up period.

Difference between pre-index and follow-up value.

Outcome measures

Measure	Sitaxentan (Thelin) n=2 Participants Duration and dose as prescribed in clinical practice.
Change From Baseline in Percent of Predicted Peak VO2	3.5 percent Vo2 Interval 0.3 to 6.7

SECONDARY outcome

Timeframe: Day 1 to Month 6

Population: FAS

Occurrence of any of the following: death, unplanned PAH-related hospitalization, initiation of epoprostenol, arterial septostomy, lung or heart/lung transplantation, ≥15% decrease from baseline in 6 minute walk test, signs/symptoms of right sided heart failure, and/or worsening WHO functional class.

Outcome measures

Measure	Sitaxentan (Thelin) n=36 Participants Duration and dose as prescribed in clinical practice.
Pulmonary Arterial Hypertension (PAH) Severity and Functional Status: Time to Clinical Worsening	2.8 months Standard Deviation 1.6

SECONDARY outcome

Timeframe: Day 1 to Month 6

Population: FAS

Number of participants who died during the follow-up period.

Outcome measures

Measure	Sitaxentan (Thelin) n=36 Participants Duration and dose as prescribed in clinical practice.
Other Pulmonary Arterial Hypertension (PAH)-Related Outcomes: Mortality	0 participants

SECONDARY outcome

Timeframe: Day 1 to Month 6

Population: FAS

All hospitalizations during the follow-up period recorded in medical records.

Outcome measures

Measure	Sitaxentan (Thelin) n=36 Participants Duration and dose as prescribed in clinical practice.
Number of Hospitalizations	0.5 hospitalizations Interval 0.3 to 0.8

SECONDARY outcome

Timeframe: Day 1 to Month 6

Population: FAS; N=number of participants with hospitalizations during follow-up period.

Number of participants who received an lung transplant during hospitalization.

Outcome measures

Measure	Sitaxentan (Thelin) n=13 Participants Duration and dose as prescribed in clinical practice.
Other Pulmonary Arterial Hypertension (PAH)-Related Outcomes: Lung Transplantation	0 participants

SECONDARY outcome

Timeframe: Day 1 to Month 6

Population: FAS; N=number of participants with hospitalizations during follow-up period.

Number of participants who received an heart/lung transplant during hospitalization.

Outcome measures

Measure	Sitaxentan (Thelin) n=13 Participants Duration and dose as prescribed in clinical practice.
Other Pulmonary Arterial Hypertension (PAH)-Related Outcomes: Heart/Lung Transplantation	0 participants

SECONDARY outcome

Timeframe: Day 1 to Month 6

Population: FAS; N=number of participants with hospitalizations during follow-up period.

Number of participants who received an atrial septostomy (balloon or blade) during hospitalization.

Outcome measures

Measure	Sitaxentan (Thelin) n=13 Participants Duration and dose as prescribed in clinical practice.
Other Pulmonary Arterial Hypertension (PAH)-Related Outcomes: Atrial Septostomy	0 participants

Adverse Events

Sitaxentan (Thelin)

Serious events: 0 serious events

Other events: 27 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Sitaxentan (Thelin) n=36 participants at risk Duration and dose as prescribed in clinical practice.
General disorders Headache	8.3% 3/36 Pre-specified AEs of interest reported in medical records. Other events = emergency hospital admission, worsening PAH, unexplained anemia, alopecia, diarrhea, fatigue, obstipation, loss of appetite, gastric pain, numerous defecation, INH out of range, cardial decompensation, joint pain, pre-syncope, infection of IV line with need of explanation.
General disorders Peripheral edema	41.7% 15/36 Pre-specified AEs of interest reported in medical records. Other events = emergency hospital admission, worsening PAH, unexplained anemia, alopecia, diarrhea, fatigue, obstipation, loss of appetite, gastric pain, numerous defecation, INH out of range, cardial decompensation, joint pain, pre-syncope, infection of IV line with need of explanation.
General disorders Nausea	8.3% 3/36 Pre-specified AEs of interest reported in medical records. Other events = emergency hospital admission, worsening PAH, unexplained anemia, alopecia, diarrhea, fatigue, obstipation, loss of appetite, gastric pain, numerous defecation, INH out of range, cardial decompensation, joint pain, pre-syncope, infection of IV line with need of explanation.
General disorders Dizziness	33.3% 12/36 Pre-specified AEs of interest reported in medical records. Other events = emergency hospital admission, worsening PAH, unexplained anemia, alopecia, diarrhea, fatigue, obstipation, loss of appetite, gastric pain, numerous defecation, INH out of range, cardial decompensation, joint pain, pre-syncope, infection of IV line with need of explanation.
General disorders Nasal congestion	0.00% 0/36 Pre-specified AEs of interest reported in medical records. Other events = emergency hospital admission, worsening PAH, unexplained anemia, alopecia, diarrhea, fatigue, obstipation, loss of appetite, gastric pain, numerous defecation, INH out of range, cardial decompensation, joint pain, pre-syncope, infection of IV line with need of explanation.
General disorders Bleeding	13.9% 5/36 Pre-specified AEs of interest reported in medical records. Other events = emergency hospital admission, worsening PAH, unexplained anemia, alopecia, diarrhea, fatigue, obstipation, loss of appetite, gastric pain, numerous defecation, INH out of range, cardial decompensation, joint pain, pre-syncope, infection of IV line with need of explanation.
General disorders Other events	58.3% 21/36 Pre-specified AEs of interest reported in medical records. Other events = emergency hospital admission, worsening PAH, unexplained anemia, alopecia, diarrhea, fatigue, obstipation, loss of appetite, gastric pain, numerous defecation, INH out of range, cardial decompensation, joint pain, pre-syncope, infection of IV line with need of explanation.

Additional Information

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Results disclosure agreements

• Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
• Publication restrictions are in place

Restriction type: OTHER