Trial Outcomes & Findings for Tolerability, Safety and Efficacy of Flexibly Dosed Paliperidone ER in Patients With Schizophrenia (NCT NCT01724359)

NCT ID: NCT01724359

Last Updated: 2013-03-15

Results Overview

The PANSS is a 30-item scale designed to assess various symptoms of schizophrenia. The 30 symptoms are rated on a 7-point scale that ranges from 1 (absent) to 7 (extreme psychopathology). The PANSS total score consists of the sum of all 30 PANSS items and ranges from 30 to 210. Higher scores indicate worsening.

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 95 participants
• Primary outcome timeframe: Baseline, Week 26
• Results posted on: 2013-03-15

Participant Flow

Patients who received paliperidone extended-release (ER) at least once and provide at least 1 post baseline efficacy measurement will be included in the intent-to-treat (ITT) analysis set for efficacy. Patients who received paliperidone ER at least once and provide any post-baseline information will be included in the ITT analysis set for safety.

Participant milestones

Measure	Paliperidone Extended-release (ER) The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.
Overall Study STARTED	95
Overall Study COMPLETED	64
Overall Study NOT COMPLETED	31

Reasons for withdrawal

Measure	Paliperidone Extended-release (ER) The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.
Overall Study Adverse Event	10
Overall Study Death	1
Overall Study Other	20

Baseline Characteristics

Tolerability, Safety and Efficacy of Flexibly Dosed Paliperidone ER in Patients With Schizophrenia

Baseline characteristics by cohort

Measure	Paliperidone Extended-release (ER) n=95 Participants The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.
Age Continuous	35.86 years STANDARD_DEVIATION 12.28 • n=5 Participants
Sex: Female, Male Female	41 Participants n=5 Participants
Sex: Female, Male Male	54 Participants n=5 Participants
Region of Enrollment Argentina	37 participants n=5 Participants
Region of Enrollment Colombia	58 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 26

Population: All participants of the intent-to-treat analysis set for efficacy with evaluable data at each measurement time point.

The PANSS is a 30-item scale designed to assess various symptoms of schizophrenia. The 30 symptoms are rated on a 7-point scale that ranges from 1 (absent) to 7 (extreme psychopathology). The PANSS total score consists of the sum of all 30 PANSS items and ranges from 30 to 210. Higher scores indicate worsening.

Outcome measures

Measure	Paliperidone Extended-release (ER) n=70 Participants The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.	Patients at Week 26: Paliperidone Extended-release (ER) The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.
Change From Baseline in Total Positive and Negative Syndrome Scale (PANSS) Score Baseline (n= 70)	83.99 scores on a scale Standard Deviation 22.16	—
Change From Baseline in Total Positive and Negative Syndrome Scale (PANSS) Score Week 26 (n= 70)	53.70 scores on a scale Standard Deviation 18.82	—

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: All participants of the intent-to-treat analysis set for efficacy with evaluable data at each measurement time point.

The PANSS Positive Subscale assesses seven positive-symptoms of schizophrenia. Positive symptoms refer to an excess or distortion of normal functions. The symptoms are rated on a 7-point scale, with a range of 7 (absent) to 49 (extreme psychopathology).

Outcome measures

Measure	Paliperidone Extended-release (ER) n=70 Participants The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.	Patients at Week 26: Paliperidone Extended-release (ER) The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.
Change From Baseline in Total Positive and Negative Syndrome Scale (PANSS) - Positive Subscale Score Baseline (n= 70)	18.53 scores on a scale Standard Deviation 7.30	—
Change From Baseline in Total Positive and Negative Syndrome Scale (PANSS) - Positive Subscale Score Week 26 (n= 70)	11.16 scores on a scale Standard Deviation 4.95	—

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: All participants of the intent-to-treat analysis set for efficacy with evaluable data at each measurement time point.

The PANSS Negative Subscale assesses seven negative-symptoms of schizophrenia. Negative symptoms represent a diminution or loss of normal functions. The symptoms are rated on a 7-point scale, with a range of 7 (absent) to 49 (extreme psychopathology).

Outcome measures

Measure	Paliperidone Extended-release (ER) n=70 Participants The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.	Patients at Week 26: Paliperidone Extended-release (ER) The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.
Change From Baseline in Total Positive and Negative Syndrome Scale (PANSS) - Negative Subscale Score Baseline (n= 70)	23.69 scores on a scale Standard Deviation 6.42	—
Change From Baseline in Total Positive and Negative Syndrome Scale (PANSS) - Negative Subscale Score Week 26 (n= 70)	14.64 scores on a scale Standard Deviation 5.24	—

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: All participants of the intent-to-treat analysis set for efficacy with evaluable data at each measurement time point.

The PANSS General Psychopathology Subscale Score assesses 16 general psychopathology symptoms. The symptoms are rated on a 7-point scale, with a range of 16 (absent) to 112 (extreme psychopathology).

Outcome measures

Measure	Paliperidone Extended-release (ER) n=70 Participants The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.	Patients at Week 26: Paliperidone Extended-release (ER) The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.
Change From Baseline in Total Positive and Negative Syndrome Scale (PANSS) - General Psychopathology Subscale Score Baseline (n= 70)	41.77 scores on a scale Standard Deviation 11.39	—
Change From Baseline in Total Positive and Negative Syndrome Scale (PANSS) - General Psychopathology Subscale Score Week 26 (n= 70)	27.90 scores on a scale Standard Deviation 10.34	—

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: All participants of the intent-to-treat analysis set for efficacy with evaluable data at each measurement time point.

The CGI-S rating scale is a 7 point global assessment that measures the clinician's impression of the severity of illness exhibited by a patient. A rating of 1 is equivalent to "Normal, not at all ill" and a rating of 7 is equivalent to "Among the most extremely ill patients". Higher scores indicate worsening.

Outcome measures

Measure	Paliperidone Extended-release (ER) n=95 Participants The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.	Patients at Week 26: Paliperidone Extended-release (ER) n=83 Participants The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.
Clinical Global Impression-Severity (CGIS) Normal, not at all ill	2 participants	9 participants
Clinical Global Impression-Severity (CGIS) Borderline mentally ill	3 participants	21 participants
Clinical Global Impression-Severity (CGIS) Mildly ill	14 participants	26 participants
Clinical Global Impression-Severity (CGIS) Moderately ill	39 participants	20 participants
Clinical Global Impression-Severity (CGIS) Markedly ill	26 participants	5 participants
Clinical Global Impression-Severity (CGIS) Severely ill	10 participants	2 participants
Clinical Global Impression-Severity (CGIS) Among the most extremely ill	1 participants	0 participants

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: All participants of the intent-to-treat analysis set for efficacy with evaluable data at each measurement time point.

This PSP assesses the degree of a patient's dysfunction within 4 domains of behavior: socially useful activities, personal and social relationships, self-care, and disturbing and aggressive behavior. The score ranges from 1 to 100, divided into 10 equal intervals to rate the degree of difficulty (i, absent to vi, very severe) in each of the 4 domains. Based on the four domains there will be one total score. Patients with a score of 71 to 100 have a mild degree of difficulty; from 31 to 70, varying degrees of disability; =< 30, functioning so poorly as to require intensive supervision.

Outcome measures

Measure	Paliperidone Extended-release (ER) n=95 Participants The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.	Patients at Week 26: Paliperidone Extended-release (ER) The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.
Personal and Social Performance (PSP) Scale Baseline (n= 95)	52.06 scores on a scale Standard Deviation 15.27	—
Personal and Social Performance (PSP) Scale Week 26 (n= 78)	68.24 scores on a scale Standard Deviation 15.91	—

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: All participants of the intent-to-treat analysis set for efficacy with evaluable data at each measurement time point.

The SF-36 is designed to examine a person's perceived health status. The SF-36 includes one multi-item scale measuring eight health concepts: vitality, physical functioning, bodily pain, general health perceptions, physical role-, emotional role-, social role functioning, and mental health. Answers to each question are scored and summed to produce raw scale scores for each health concept which are then transformed to a 0 - 100 scale, a high score defining a more favorable health state. An aggregate summary measure is calculated by averaging the scores from the eight health concepts.

Outcome measures

Measure	Paliperidone Extended-release (ER) n=95 Participants The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.	Patients at Week 26: Paliperidone Extended-release (ER) The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.
Health Status as Measured by Self-rated Health Status Survey SF-36 Baseline (n= 95)	56.39 scores on a scale Standard Deviation 18.14	—
Health Status as Measured by Self-rated Health Status Survey SF-36 Week 26 (n= 86)	67.02 scores on a scale Standard Deviation 21.56	—

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: All participants of the intent-to-treat analysis set for efficacy with evaluable data at each measurement time point.

This self-administered scale rates the quality of sleep. Patients will indicate on an 11-point scale how well they have slept in the previous 7 days, from 0 (very badly) to 10 (very well).

Outcome measures

Measure	Paliperidone Extended-release (ER) n=71 Participants The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.	Patients at Week 26: Paliperidone Extended-release (ER) The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.
Sleep Evaluation Scale Baseline (n= 71)	6.80 scores on a scale Standard Deviation 2.69	—
Sleep Evaluation Scale Week 26 (n= 71)	7.66 scores on a scale Standard Deviation 2.52	—

SECONDARY outcome

Timeframe: Baseline, Week 26

Population: All participants of the intent-to-treat analysis set for efficacy with evaluable data at each measurement time point.

This self-administered scale rates the daytime drowsiness. Patients will indicate on an 11-point scale how often they have felt drowsy within the previous 7 days, from 0 (not at all) to 10 (all the time).

Outcome measures

Measure	Paliperidone Extended-release (ER) n=71 Participants The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.	Patients at Week 26: Paliperidone Extended-release (ER) The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.
Daytime Drowsiness Evaluation Scale Baseline (n= 71)	4.68 scores on a scale Standard Deviation 3.21	—
Daytime Drowsiness Evaluation Scale Week 26 (n= 71)	2.58 scores on a scale Standard Deviation 2.93	—

Adverse Events

Paliperidone Extended-release (ER)

Serious events: 7 serious events

Other events: 39 other events

Deaths: 0 deaths

Serious adverse events

Measure	Paliperidone Extended-release (ER) n=95 participants at risk The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.
Psychiatric disorders Psychotic episode	3.2% 3/95
General disorders Clinical deterioration	1.1% 1/95
Gastrointestinal disorders Appendicitis	1.1% 1/95
Psychiatric disorders Major depressive disorder	1.1% 1/95
Psychiatric disorders Worsening of the psychiatric condition with aggressive behavior	1.1% 1/95

Other adverse events

Measure	Paliperidone Extended-release (ER) n=95 participants at risk The recommended Paliperidone ER dose is 6 mg/day. Some patients may benefit from higher or lower doses, in the range of 3 to 12 mg/day. Paliperidone ER will be administered orally once daily.
Nervous system disorders Akathisia	23.2% 22/95
Psychiatric disorders Insomnia	25.3% 24/95
Nervous system disorders Parkinsonism	15.8% 15/95

Additional Information

GERENTE MEDICO REGIONAL

Jan-Cil Mexico

Phone: 52 55 5237 3410

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: LTE60