A Study To Find Out How Fesoterodine Works In Children Aged 6 To 17 Years With Bladder Overactivity Caused By A Neurological Condition
- Conditions
- Urinary Bladder, Neurogenic
- Interventions
- Registration Number
- NCT01557244
- Lead Sponsor
- Pfizer
- Brief Summary
The objective of the study is to find out if the medicine fesoterodine is a useful treatment in children with bladder muscle overactivity caused by a neurological condition. Children will be aged 6 to 17 years old. This is done by finding out how well it works, what the body does to fesoterodine, what side effects are experienced and the safety of fesoterodine. It will be compared with the medicine oxybutynin, which is already available for treating the condition.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 181
- Subjects aged 6 to 17 years old
- Subjects with stable neurological disease and neurogenic detrusor overactivity
- Subjects using clean intermittent catheterization may participate
- Concomitant medications which may increase the risk to subjects or confound study results
- Other medical conditions which may increase the risk to subjects or confound study results
- Contraindications to the use of fesoterodine or oxybutynin
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Fesoterodine BIC 4 mg Fesoterodine BIC 4 mg Fesoterodine BIC 4 mg for first week followed by 11 weeks at 8 mg in the efficacy period, followed by 12 weeks in safety extension period. Fesoterodine PR 4 mg Fesoterodine PR 4 mg Fesoterodine PR 4 mg for 12 weeks in active comparator period, followed by 12 weeks in safety extension period Fesoterodine PR 8 mg Fesoterodine PR 8 mg Fesoterodine 8 mg for first week followed by 11 weeks at 8 mg in active control period, followed by 12 weeks in safety extension period. Fesoterodine BIC 2 mg Fesoterodine BIC 2 mg Fesoterodine BIC 2 mg for 12 weeks in efficicay period, followed by 12 weeks in safety extension period. Oxybutynin Fesoterodine PR Oxybutynin Oxybutynin Oxybutynin Oxybutynin
- Primary Outcome Measures
Name Time Method Change From Baseline in Maximum Cystometric Bladder Capacity at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 Maximum cystometric bladder capacity (in milliliter) was defined as maximal tolerable cystometric capacity, until voiding or leaking begins or at a pressure of \>=40 centimeter (cm) water (H2O).
- Secondary Outcome Measures
Name Time Method Change From Baseline in Bladder Volume at First Involuntary Detrusor Contraction (IDC) at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 Bladder volume (in milliliter) at first IDC was measured using urodynamic testing.
Change From Baseline in Bladder Compliance at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 Bladder compliance was defined as change in bladder volume in milliliter (mL) divided by change in bladder pressure in cm H2O (during the same time when change in bladder volume was estimated).
Change From Baseline in Mean Number of Micturitions Per 24 Hours at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The mean number of micturitions per 24 hours were calculated as the total number of micturitions divided by the total number of diary days collected at the assessment time point. Number of diary days collected at the assessment time point = number of calendar days when the diary was completed on, even if it was not a full 24 hour period. This outcome measure was only calculated for participants with \>0 micturitions at Baseline.
Change From Baseline in Mean Number of Urgency Episodes Per 24 Hours at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The mean number of urgency episodes per 24 hours were calculated as the total number of urgency episodes divided by the total number of diary days collected at the assessment time point. Number of diary days collected at the assessment time point = number of calendar days when the diary was completed; even if it was not a full 24 hours period. Urgency episodes were defined as urgency marked as 'yes' in the diary. This outcome measure was only calculated for participants with \>0 urgency episodes at Baseline.
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs): Active Comparator Phase/Efficacy Phase Baseline up to Week 12 An AE was any untoward medical occurrence in a participant who received investigational product without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly; medically important events. A treatment emergent AE was defined as an event that emerged during the treatment period that was absent before treatment, or worsened during the treatment period relative to the pretreatment state. AEs included both all serious and non-serious adverse events.
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs): Safety Extension Phase Week 12 up to Week 26 (including 2 weeks of follow up after last dose) An AE was any untoward medical occurrence in a participant who received investigational product without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly; medically important events. A treatment emergent AE was defined as an event that emerged during the treatment period that was absent before treatment, or worsened during the treatment period relative to the pretreatment state. AEs included both all serious and non-serious adverse events.
Change From Baseline in Detrusor Pressure at Maximum Bladder Capacity at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 Detrusor pressure (in cm H2O) at maximum urinary bladder capacity was measured using urodynamic testing.
Change From Baseline in Mean Number of Micturitions or Catheterizations Per 24 Hours at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The mean number of micturitions or catheterizations combined per 24 hours were calculated as the total number of micturitions and catheterizations combined divided by the total number of diary days collected at the assessment point. Number of diary days collected at the assessment time point = number of calendar days when the diary was completed; even if it was not a full 24 hour (hrs) period. This outcome was evaluated in those participants who had micturitions or catheterizations \>0 at Baseline.
Change From Baseline in Mean Number of Incontinence Episodes Per 24 Hours at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The mean number of incontinence episodes per 24 hours were calculated as the total number of incontinence episodes divided by the total number of diary days collected at the assessment time point. Number of diary days collected at the assessment time point = number of calendar days when the diary was completed; even if it was not a full 24 hours period. This outcome measure was only calculated for participants with \>0 incontinence episodes at Baseline.
Apparent Oral Clearance (CL/F) of Fesoterodine Week 4, Day 1: pre-dose (when dose administered at clinic) or if dose taken at home up to 3 hours before coming to the clinic, sampling just after arrival at clinic, 5 hours post-dose, 8-10 hours post-dose (if participants remained at clinic) Clearance determines the rate at which a drug is metabolized or eliminated by normal biological processes. PK analysis was not done separately for each dose of fesoterodine in respective cohorts and were combined for PK analysis using PK modelling approach.
Volume of Distribution (Vd) of Fesoterodine Week 4, Day 1: pre-dose (when dose administered at clinic) or if dose taken at home up to 3 hours before coming to the clinic, sampling just after arrival at clinic, 5 hours post-dose, 8-10 hours post-dose (if participants remained at clinic) Volume of distribution is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. PK analysis was not done separately for each dose of fesoterodine in respective cohorts and were combined for PK analysis using PK modelling approach.
Number of Participants With Shift From Baseline at Week 12 in Involuntary Detrusor Contractions (IDC): Active Comparator Phase/Efficacy Phase Baseline, Week 12 In this outcome measure, shift data have been reported using 4 categories: (1) number of participants who did not have IDC at Baseline and at Week 12, (2) number of participants who did not have IDC at Baseline but had IDC at Week 12, (3) number of participants who had IDC at Baseline but no IDC at Week 12, and (4) number of participants who had IDC at Baseline and at Week 12.
Change From Baseline in Mean Number of Catheterizations Per 24 Hours at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The mean number of catheterizations per 24 hours were calculated as the total number of catheterizations divided by the total number of diary days collected at the assessment time point. Number of diary days collected at the assessment time point = number of calendar days when the diary was completed on; even if it was not a full 24 hours period. This outcome measure was only calculated for participants with \>0 catheterizations at Baseline.
Change From Baseline in Mean Volume Voided Per Micturition at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The mean voided volume per micturition was calculated as sum of voided volume divided by the total number of micturition episodes with a recorded voided volume greater than 0.
Change From Baseline in Mean Volume Voided Per Catheterization at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The mean volume per catheterization was calculated as sum of voided volume divided by the total number of catheterization, with a recorded voided volume greater than 0.
Number of Participants Meeting Pre-defined Criteria for Vital Signs Values From Baseline Through Week 12: Active Comparator/Efficacy Phase Baseline up to Week 12 Pre-defined criteria for vital signs: 1) a) systolic blood pressure (SBP) of \<90 millimeter of mercury (mmHg), b) change \>=30 mmHg increase, c) change \>=30 mmHg decrease; 2) a) diastolic blood pressure (DBP) of \<50 mmHg, b) change \>=20 mmHg increase, c) change \>=20 mmHg decrease; 3) a) pulse rate value of \<40 beats per minute (bpm), b) pulse rate value \>120 bpm.
Number of Participants Meeting Pre-defined Criteria for Vital Signs Values From Baseline Through Week 24: Safety Extension Phase Baseline up to Week 24 Pre-defined criteria for vital signs: 1) a) systolic blood pressure (SBP) of \<90 millimeter of mercury (mmHg), b) change \>=30 mmHg increase, c) change \>=30 mmHg decrease; 2) a) diastolic blood pressure (DBP) of \<50 mmHg, b) change \>=20 mmHg increase, c) change \>=20 mmHg decrease; 3) a) pulse rate value of \<40 beats per minute (bpm), b) pulse rate value \>120 bpm.
Number of Participants With Clinical Laboratory Abnormalities: Safety Extension Phase Week 12 up to Week 26 Hematology: hemoglobin, hematocrit, erythrocytes \<0.8\*lower limit of normal (LLN), platelets\<0.5\*LLN\>1.75\*upper limit of normal (ULN), leukocytes \<0.6\*LLN\>1.5\*ULN, lymphocytes, neutrophils, \<0.8\*LLN \>1.2\*ULN, basophils, eosinophils, monocytes monocytes/leukocytes \>1.2\*ULN. Clinical chemistry: bilirubin, direct, bilirubin \>1.5\*ULN, aspartate aminotransferase (AT), alanine AT, gamma glutamyl transferase, lactate dehydrogenase, alkaline phosphatase\>3.0\*ULN, protein, albumin, phosphate \<0.8\*LLN \>1.2\*ULN, blood urea nitrogen, creatinine \>1.3\*ULN, urate \>1.2\*ULN, sodium\<0.95\*LLN\>1.05\*ULN, potassium, chloride, calcium bicarbonate\<0.9\*LLN\>1.1\*ULN, glucose\<0.6\*LLN\>1.5\*ULN, creatine kinase \>2.0\*ULN. Urinalysis: specific gravity \<1.003\>1.030, pH \<4.5\>8, urine glucose, ketones, urine protein, urine hemoglobin, urine bilirubin, nitrite, \>=1, urine erythrocytes, urine leukocytes \>=20, epithelial cells \>=6, bacteria \>20.
Number of Participants With Clinically Relevant Changes in Physical Examination Findings From Baseline to Week 12: Active Comparator/Efficacy Phase Baseline up to Week 12 Physical examination included assessment of the general appearance and the skin, head, ears, eyes, nose, mouth, throat, respiratory, cardiovascular, gastrointestinal, musculoskeletal and neurological systems. Clinically relevant changes in physical findings were assessed by the investigator.
Change From Baseline in Mean Volume Voided Per Micturition or Catheterization at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The mean voided volume per micturition or catheterization was calculated as sum of voided volume divided by the total number of micturition or catheterization episodes with a recorded voided volume greater than 0. This outcome was evaluated in those participants who had micturitions or catheterizations \>0 at Baseline.
Change From Baseline in Visual Accommodation at Week 24: Safety Extension Phase Baseline, Week 24 The visual accommodation is the distance for each eye at which vision became blurred and was calculated as the mean of triplicate measurements. The participants focused on a single letter of the 20/40 line of an eye chart and chart was moved slowly towards the participant until letter was blurred. At this point, the distance from eye to letter was measured for each eye. In this outcome measure data have been reported for right and left eye separately.
Number of Participants With Clinically Significant Urinary Tract Infections (UTI): Safety Extension Phase Week 12 up to Week 26 Clinically significant UTI, counted as an adverse event was defined as: positive urine culture with a uropathogen (defined as \>=10\^5 CFU/mL) and the presence of symptoms, or pyuria (defined as \>50 WBC per hpf and the presence of symptoms, or positive urine culture with a uropathogen (defined as \>=10\^5 CFU/mL) with or without symptoms in a participants with a documented history of VUR.
Change From Baseline in Post-Void Residual (PVR) Volume at Weeks 4, 12: Active Comparator Phase/Efficacy Phase Baseline, Week 4, 12 Post-void residual volume measurement was measured by an ultrasound. PVR volume was only assessed for participants who did not perform clean intermittent catheterization or in any participants who had \>1 UTI during the study.
Number of Participants With Clinical Laboratory Abnormalities: Active Comparator/Efficacy Phase Week 1 up to Week 12 Hematology: hemoglobin, hematocrit, erythrocytes \<0.8\*lower limit of normal (LLN), platelets\<0.5\*LLN\>1.75\*upper limit of normal (ULN), leukocytes \<0.6\*LLN\>1.5\*ULN, lymphocytes, neutrophils, \<0.8\*LLN \>1.2\*ULN, basophils, eosinophils, monocytes monocytes/leukocytes \>1.2\*ULN. Clinical chemistry: bilirubin, direct, bilirubin \>1.5\*ULN, aspartate aminotransferase (AT), alanine AT, gamma glutamyl transferase, lactate dehydrogenase, alkaline phosphatase\>3.0\*ULN, protein, albumin, phosphate \<0.8\*LLN \>1.2\*ULN, blood urea nitrogen, creatinine \>1.3\*ULN, urate \>1.2\*ULN, sodium\<0.95\*LLN\>1.05\*ULN, potassium, chloride, calcium bicarbonate\<0.9\*LLN\>1.1\*ULN, glucose\<0.6\*LLN\>1.5\*ULN, creatine kinase \>2.0\*ULN. Urinalysis: specific gravity \<1.003\>1.030, pH \<4.5\>8, urine glucose, ketones, urine protein, urine hemoglobin, urine bilirubin, nitrite, \>=1, urine erythrocytes, urine leukocytes \>=20, epithelial cells \>=6, bacteria \>20.
Change From Baseline in Post-Void Residual Volume at Week 24: Safety Extension Phase Baseline, Week 24 Post-void residual volume measurement was measured by an ultrasound. PVR volume was only assessed for participants who did not perform clean intermittent catheterization or in any participants who had \>1 UTI during the study.
Absorption Rate Constant (Ka) of Fesoterodine Week 4, Day 1: pre-dose (when dose administered at clinic) or if dose taken at home up to 3 hours before coming to the clinic, sampling just after arrival at clinic, 5 hours post-dose, 8-10 hours post-dose (if participants remained at clinic) Absorption rate constant is used to determine rate at which drug is entering into body. Pharmacokinetic (PK) analysis was not done separately for each dose of fesoterodine in respective cohorts and were combined for PK analysis using PK modelling approach.
Change From Baseline in Number of Pegs Dropped Assessment of Grooved Pegboard Test, 10 Pegs Group at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The grooved pegboard test was a manipulative dexterity test that assessed psychomotor speed, fine motor control, and rapid-visual motor coordination. It consisted of a small board of 25 holes with randomly positioned slots. Pegs with a key along one side must be rotated to match the hole before they can be inserted. Participants were asked to insert 10 grooved pegs into the holes within the given time limit (up to 300 seconds). In this outcome measure number of pegs dropped while putting in the holes were measured. The task needs to be completed once for each hand; firstly, using the dominant hand followed by the non-dominant hand.
Change From Baseline in Number of Pegs Dropped Assessment of Grooved Pegboard Test, 25 Pegs Group at Week 24: Safety Extension Phase Baseline, Week 24 The grooved pegboard test was a manipulative dexterity test that assessed psychomotor speed, fine motor control, and rapid-visual motor coordination. It consisted of a small board of 25 holes with randomly positioned slots. Pegs with a key along one side must be rotated to match the hole before they can be inserted. Participants were asked to insert 25 grooved pegs into the holes within the given time limit (up to 300 seconds). In this outcome measure number of pegs dropped while putting in the holes were measured. The task needs to be completed once for each hand; firstly, using the dominant hand followed by the non-dominant hand.
Change From Baseline in Number of Pegs Placed Correctly Assessment of Grooved Pegboard Test, 10 Pegs Group at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The grooved pegboard test was a manipulative dexterity test that assessed psychomotor speed, fine motor control, and rapid-visual motor coordination. It consisted of a small board of 25 holes with randomly positioned slots. Pegs with a key along one side must be rotated to match the hole before they can be inserted. Participants were asked to insert 10 grooved pegs into the holes within the given time limit (up to 300 seconds). In this outcome measure number of pegs placed correctly while putting in the holes were measured. The task needs to be completed once for each hand; firstly, using the dominant hand followed by the non-dominant hand.
Change From Baseline in Number of Pegs Placed Correctly Assessment of Grooved Pegboard Test, 25 Pegs Group at Week 24: Safety Extension Phase Baseline, Week 24 The grooved pegboard test was a manipulative dexterity test that assessed psychomotor speed, fine motor control, and rapid-visual motor coordination. It consisted of a small board of 25 holes with randomly positioned slots. Pegs with a key along one side must be rotated to match the hole before they can be inserted. Participants were asked to insert 25 grooved pegs into the holes within the given time limit (up to 300 seconds). In this outcome measure number of pegs placed correctly while putting in the holes were measured. The task needs to be completed once for each hand; firstly, using the dominant hand followed by the non-dominant hand.
Change From Baseline in Visual Acuity at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 Visual acuity (VA) was assessed using the Snellen method, where logarithm of minimum angle of resolution (logMAR) units were derived from the Snellen ratios. Participants had to read letters from the chart at a distance of 20 feet/6 meter or 4 meter. VA/Snellen ratio = distance between the chart and participant, divided by the distance at which participant was able to see or read chart without impairment; expressed as decimal. logMAR = log10 (1/decimal VA). In this outcome measure data have been reported for right and left eye separately.
Change From Baseline in Visual Acuity at Week 24: Safety Extension Phase Baseline, Week 24 VA was assessed using the Snellen method, where logMAR units were derived from the Snellen ratios. Participants had to read letters from the chart at a distance of 20 feet/6 meter or 4 meter. VA/Snellen ratio = distance between the chart and participant, divided by the distance at which participant was able to see or read chart without impairment; expressed as decimal. logMAR = log10 (1/decimal VA). In this outcome measure data have been reported for right and left eye separately.
Change From Baseline in Child Behavior Checklist (CBCL) T Score (Derived Score) at Week 24: Safety Extension Phase Baseline, Week 24 CBCL: 120 items questionnaire answered by parent/caregiver of child to assess a child's behavioral, emotional problems. Scale for each item: 0= not true, 1= somewhat/sometimes true, 2= very true/often true. Out of 120 items, 103 were categorized into 8 domains; aggressive behavior, anxious/depressed, attention problems, rule-breaking behavior, social problems, somatic complaints, thought problems, withdrawn. Summary scores: Internalizing problems=anxious/depressed + withdrawn + somatic complaints; Externalizing problems=rule-breaking + aggressive behavior. Total problems=8 domains + other 17 items. Raw scores for each domain, summary and total problems=sum of scores of related items. Using Assessment Data Manager (ADM) tool raw scores transformed/derived into standard T-scores, range: each domain=50 to 100, internalizing problems=34 to 100, externalizing problems=33 to 100, total problems=24 to 100. Lower T-score for each 8 domains, 2 summary and total problems scores=better outcomes.
Change From Baseline in Time to Completion Assessment of Grooved Pegboard Test, 10 Pegs Group at Week 24: Safety Extension Phase Baseline, Week 24 The grooved pegboard test was a manipulative dexterity test that assessed psychomotor speed, fine motor control, and rapid-visual motor coordination. It consisted of a small board of 25 holes with randomly positioned slots. Pegs with a key along one side must be rotated to match the hole before they can be inserted. In this outcome measure participants were asked to insert 10 grooved pegs into the holes within the given time limit (up to 300 seconds). The task needs to be completed once for each hand; firstly, using the dominant hand followed by the non-dominant hand. Time taken to complete the test was inversely correlated to the cognitive ability.
Change From Baseline in Time to Completion Assessment of Grooved Pegboard Test, 25 Pegs Group at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The grooved pegboard test was a manipulative dexterity test that assessed psychomotor speed, fine motor control, and rapid-visual motor coordination. It consisted of a small board of 25 holes with randomly positioned slots. Pegs with a key along one side must be rotated to match the hole before they can be inserted. In this outcome measure participants were asked to insert 25 grooved pegs into the holes within the given time limit (up to 300 seconds). The task needs to be completed once for each hand; firstly, using the dominant hand followed by the non-dominant hand. Time taken to complete the test was inversely correlated to the cognitive ability.
Change From Baseline in Number of Pegs Placed Correctly Assessment of Grooved Pegboard Test, 10 Pegs Group at Week 24: Safety Extension Phase Baseline, Week 24 The grooved pegboard test was a manipulative dexterity test that assessed psychomotor speed, fine motor control, and rapid-visual motor coordination. It consisted of a small board of 25 holes with randomly positioned slots. Pegs with a key along one side must be rotated to match the hole before they can be inserted. Participants were asked to insert 10 grooved pegs into the holes within the given time limit (up to 300 seconds). In this outcome measure number of pegs placed correctly while putting in the holes were measured. The task needs to be completed once for each hand; firstly, using the dominant hand followed by the non-dominant hand.
Change From Baseline in Visual Accommodation at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The visual accommodation was the distance for each eye at which vision became blurred and was calculated as the mean of triplicate measurements. The participants focused on a single letter of the 20/40 line of an eye chart and chart was moved slowly towards the participant until letter was blurred. At this point, the distance from eye to letter was measured for each eye. In this outcome measure data have been reported for right and left eye separately.
Change From Baseline in Child Behavior Checklist Total Score (Raw Score) at Week 24: Safety Extension Phase Baseline, Week 24 CBCL: It consisted of 120 items on behavior and emotional problems. Parent/caregiver of child answered 120 items, each on scale: 0= not true, 1= somewhat/sometimes true, 2= very/often true. 103 items were classified in 8 domains: aggressive behavior: total score range (TSR)= 0 to 36, anxious/depressed: TSR= 0 to 26, attention problems: TSR= 0 to 20, rule-breaking behavior: TSR= 0 to 34, social problems: TSR= 0 to 22, somatic complaints: TSR= 0 to 22, thought problems: TSR= 0 to 30, withdrawn (TSR)= 0 to 16. Rule-breaking and aggressive behavior summarized to externalizing problems with a TSR= 0 to 70. Anxious/depressed, withdrawn, somatic complaints summarized to internalizing problems with a TSR= 0 to 64. All 103 items of 8 domains and other 17 remaining items were combined to give total problems TSR = 0 to 240. TSR for each domain, summary and total problems was sum of scores of related items respectively. Lower scores for each domain, summary and total problems= better outcomes.
Change From Baseline in Time to Completion Assessment of Grooved Pegboard Test, 25 Pegs Group at Week 24: Safety Extension Phase Baseline, Week 24 The grooved pegboard test was a manipulative dexterity test that assessed psychomotor speed, fine motor control, and rapid-visual motor coordination. It consisted of a small board of 25 holes with randomly positioned slots. Pegs with a key along one side must be rotated to match the hole before they can be inserted. In this outcome measure participants were asked to insert 25 grooved pegs into the holes within the given time limit (up to 300 seconds). The task needs to be completed once for each hand; firstly, using the dominant hand followed by the non-dominant hand. Time taken to complete the test was inversely correlated to the cognitive ability.
Change From Baseline in Number of Pegs Dropped Assessment of Grooved Pegboard Test, 25 Pegs Group at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The grooved pegboard test was a manipulative dexterity test that assessed psychomotor speed, fine motor control, and rapid-visual motor coordination. It consisted of a small board of 25 holes with randomly positioned slots. Pegs with a key along one side must be rotated to match the hole before they can be inserted. Participants were asked to insert 25 grooved pegs into the holes within the given time limit (up to 300 seconds). In this outcome measure number of pegs dropped while putting in the holes were measured. The task needs to be completed once for each hand; firstly, using the dominant hand followed by the non-dominant hand.
Change From Baseline in Child Behavior Checklist (CBCL) T Score (Derived Score) at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 CBCL: 120 items questionnaire answered by parent/caregiver of child to assess a child's behavioral, emotional problems. Scale for each item: 0= not true, 1= somewhat/sometimes true, 2= very true/often true. Out of 120 items, 103 were categorized into 8 domains; aggressive behavior, anxious/depressed, attention problems, rule-breaking behavior, social problems, somatic complaints, thought problems, withdrawn. Summary scores: Internalizing problems=anxious/depressed + withdrawn + somatic complaints; Externalizing problems=rule-breaking + aggressive behavior. Total problems=8 domains + other 17 items. Raw scores for each domain, summary and total problems=sum of scores of related items. Using Assessment Data Manager (ADM) tool raw scores transformed/derived into standard T-scores, range: each domain=50 to 100, internalizing problems=34 to 100, externalizing problems=33 to 100, total problems=24 to 100. Lower T-score for each 8 domains, 2 summary and total problems scores=better outcomes.
Change From Baseline in Child Behavior Checklist Total Score (Raw Score) at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 CBCL: It consisted of 120 items on behavior and emotional problems. Parent/caregiver of child answered 120 items, each on scale: 0= not true, 1= somewhat/sometimes true, 2= very/often true. 103 items were classified in 8 domains: aggressive behavior: total score range (TSR)= 0 to 36, anxious/depressed: TSR= 0 to 26, attention problems: TSR= 0 to 20, rule-breaking behavior: TSR= 0 to 34, social problems: TSR= 0 to 22, somatic complaints: TSR= 0 to 22, thought problems: TSR= 0 to 30, withdrawn (TSR)= 0 to 16. Rule-breaking and aggressive behavior summarized to externalizing problems with a TSR= 0 to 70. Anxious/depressed, withdrawn, somatic complaints summarized to internalizing problems with a TSR= 0 to 64. All 103 items of 8 domains and other 17 remaining items were combined to give total problems TSR = 0 to 240. TSR for each domain, summary and total problems was sum of scores of related items respectively. Lower scores for each domain, summary and total problems= better outcomes.
Change From Baseline in Time to Completion Assessment of Grooved Pegboard Test, 10 Pegs Group at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The grooved pegboard test was a manipulative dexterity test that assessed psychomotor speed, fine motor control, and rapid-visual motor coordination. It consisted of a small board of 25 holes with randomly positioned slots. Pegs with a key along one side must be rotated to match the hole before they can be inserted. In this outcome measure participants were asked to insert 10 grooved pegs into the holes within the given time limit (up to 300 seconds). The task needs to be completed once for each hand; firstly, using the dominant hand followed by the non-dominant hand. Time taken to complete the test was inversely correlated to the cognitive ability.
Change From Baseline in Number of Pegs Dropped Assessment of Grooved Pegboard Test, 10 Pegs Group at Week 24: Safety Extension Phase Baseline, Week 24 The grooved pegboard test was a manipulative dexterity test that assessed psychomotor speed, fine motor control, and rapid-visual motor coordination. It consisted of a small board of 25 holes with randomly positioned slots. Pegs with a key along one side must be rotated to match the hole before they can be inserted. Participants were asked to insert 10 grooved pegs into the holes within the given time limit (up to 300 seconds). In this outcome measure number of pegs dropped while putting in the holes were measured. The task needs to be completed once for each hand; firstly, using the dominant hand followed by the non-dominant hand.
Change From Baseline in Number of Pegs Placed Correctly Assessment of Grooved Pegboard Test, 25 Pegs Group at Week 12: Active Comparator Phase/Efficacy Phase Baseline, Week 12 The grooved pegboard test was a manipulative dexterity test that assessed psychomotor speed, fine motor control, and rapid-visual motor coordination. It consisted of a small board of 25 holes with randomly positioned slots. Pegs with a key along one side must be rotated to match the hole before they can be inserted. Participants were asked to insert 25 grooved pegs into the holes within the given time limit (up to 300 seconds). In this outcome measure number of pegs placed correctly while putting in the holes were measured. The task needs to be completed once for each hand; firstly, using the dominant hand followed by the non-dominant hand.
Number of Participants With Clinically Significant Urinary Tract Infections (UTI): Active Comparator/Efficacy Phase Week 1 up to Week 12 Clinically significant UTI, counted as an adverse event was defined as: positive urine culture with a uropathogen (defined as \>=10\^5 colony forming unit per milliliter \[CFU/mL\]) and the presence of symptoms, or pyuria (defined as \>50 white blood cells \[WBC\] per high-pass filter \[hpf\]) and the presence of symptoms, or positive urine culture with a uropathogen (defined as \>=10\^5 CFU/mL) with or without symptoms in a participant with a documented history of vesicoureteral reflux (VUR).
Number of Participants With Clinically Relevant Changes in Physical Examination Findings From Baseline to Week 24: Safety Extension Phase Baseline up to Week 24 Physical examination included assessment of the general appearance and the skin, head, ears, eyes, nose, mouth, throat, respiratory, cardiovascular, gastrointestinal, musculoskeletal and neurological systems. Clinically relevant changes in physical findings were assessed by the investigator.
Trial Locations
- Locations (83)
Urological Associates of Southern Arizona
🇺🇸Tucson, Arizona, United States
Childrens Hospital of Orange County
🇺🇸Orange, California, United States
CHOC Children's Urology Center
🇺🇸Orange, California, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Georgia Urology, P.A.
🇺🇸Atlanta, Georgia, United States
Judson L. Hawk Jr. M.D.
🇺🇸Atlanta, Georgia, United States
Loyola University Medical Center
🇺🇸Maywood, Illinois, United States
Loyola University Outpatient Center
🇺🇸Maywood, Illinois, United States
The Iowa Clinic
🇺🇸West Des Moines, Iowa, United States
UNC Chapel Hill Memorial Hospital
🇺🇸Chapel Hill, North Carolina, United States
Scroll for more (73 remaining)Urological Associates of Southern Arizona🇺🇸Tucson, Arizona, United States