A Clinical Study to Investigate How Solifenacin Fluid is Taken up, How Long it Stays in the Body and How Effective and Safe it is in Treating Children Aged From 6 Months to Less Than 5 Years With Symptoms of Neurogenic Detrusor Overactivity (NDO)

October 20, 2024 updated by: Astellas Pharma Europe B.V.

A Phase 3, Open-Label, Baseline-controlled, Multi-center, Sequential Dose-Titration Study to Assess the Pharmacokinetics, Long-Term Efficacy and Safety of Solifenacin Succinate Suspension in Children From 6 Months to Less Than 5 Years of Age With Neurogenic Detrusor Overactivity

The purpose of this study was to evaluate long term efficacy and safety of treatment with solifenacin succinate (the study drug) in children with neurogenic detrusor overactivity after multiple dose administration.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

In some children with a severe form of spina bifida (which is the consequence of a spinal cord defect), the bladder muscle (detrusor) contracts strongly and without warning (also known as neurogenic detrusor overactivity) and the urethra (the passage connecting the bladder with outside) does not relax. Though these children cannot void, urine leakage can happen when the overactive contractions are strong, and/or the pressure in the bladder is so high that it overcomes the closed urethra (overflow at high filling bladder pressure). This high bladder pressure puts these children at risk for kidney damage and can decrease the quality of the bladder. Therapy was aimed to decrease the high filling bladder pressure and the overactive detrusor contractions.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
23

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Gent, Belgium, 9000
        • Site BE3203 Gent University Hospital
  • Korea, Republic of
      • Seoul, Korea, Republic of, 110744
        • Site KR8207 Seoul National University Hospital
      • Seoul, Korea, Republic of, 120752
        • Site KR8201 Severance Hospital
  • Philippines
      • Manila, Philippines, 1015
        • Site PH6301 Philippines Children's Medical Center
  • Poland
      • Gdansk, Poland, 81-211
        • Site PL4803 Uniwersyteckie Centrum Kliniczne
      • Warszawa, Poland, 04-730
        • Site PL4801 Pomnik-Centrum Zdrowia Dziecka
  • United Kingdom
      • Sheffield, United Kingdom, S10 2TN
        • Site GB4401 Sheffield Children's Hospital
  • United States
    • New York
      • Tarrytown, New York, United States, 10591
        • Site US1008 Pediatric Urology Associates, P.C.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

6 months to 4 years (Child)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Myelomeningocoele
  • Documented diagnosis of NDO, confirmed by urodynamics
  • DSD (detrusor sphincter dyssynergia)
  • Practicing clean intermittent catheterization (CIC)

Exclusion Criteria:

  • Know genitourinary condition (other than NDO) that may cause incontinence
  • Bladder augmentation surgery
  • Current faecal impaction
  • Electro-stimulation therapy within 2 weeks prior to visit
  • Subjects with the following gastro-intestinal problems: partial or complete obstructions, decreased motility like paralytic ileus, subjects at risk of gastric retention
  • Reflux grade 3 to 5
  • Current urinary tract infection

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Solifenacin succinate
Children aged 6 months to less than 5 years were treated with sequential titrated doses of solifenacin up to 12 weeks in the Titration period after which a fixed dose of solifenacin was given for at least 40 weeks in the Fixed-dose assessment period. Children received solifenacin once daily during these 2 periods.
Drug: Solifenacin succinate
Oral suspension
Other Names:
  • YM905
  • solifenacin
  • solifenacin succinate suspension

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change From Baseline to Week 24 in Maximum Cystometric Capacity (MCC)
Time Frame: Baseline and Week 24
MCC was the volume instilled into the bladder prior to leakage or end of bladder-filling (whichever was reached first), as assessed by urodynamics (procedure: the bladder was to be filled until voiding/leakage began, or until it was stopped because either the participant experienced pain or discomfort or 135% of expected bladder capacity [EBC] was reached for participants ≥ 2 years or of maximum catheterized volume [MCV] for participants aged 6 months to < 2 years; the participants' bladder was emptied via catheterization).
Baseline and Week 24

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change From Baseline to Weeks 3, 6, 9, 12 and 52 in MCC
Time Frame: Baseline and Weeks 3, 6, 9, 12, 52
MCC was the volume instilled into the bladder prior to leakage or end of bladder-filling (whichever was reached first) as assessed by urodynamics (procedure: the bladder was to be filled until voiding/leakage began, or until it was stopped because either the participant experienced pain or discomfort or 135% of EBC was reached for participants ≥ 2 years or of MCV for participants aged 6 months to < 2 years. The participants' bladder was emptied via catheterization).
Baseline and Weeks 3, 6, 9, 12, 52
Change From Baseline to Weeks 3, 6, 9, 12, 24 and 52 in Bladder Compliance
Time Frame: Baseline and Weeks 3, 6, 9, 12, 24, 52
Bladder compliance gives an indication of the elasticity of the bladder wall. Bladder compliance was calculated by dividing the change in volume during the filling of the bladder by the change in detrusor pressure during that change in bladder volume using urodynamic assessments. The values for bladder volume and detrusor pressure at the beginning and end of filling were taken and used.
Baseline and Weeks 3, 6, 9, 12, 24, 52
Change From Baseline to Weeks 3, 6, 9, 12, 24 and 52 in Detrusor Pressure at End of Bladder-Filling
Time Frame: Baseline and Weeks 3, 6, 9, 12, 24, 52
Detrusor pressure was expressed as bladder pressure minus intra-abdominal pressure as assessed by urodynamics.
Baseline and Weeks 3, 6, 9, 12, 24, 52
Change From Baseline to Weeks 3, 6, 9, 12, 24 and 52 in Detrusor Pressure 5 Minutes After End of Bladder-Filling
Time Frame: Baseline and Weeks 3, 6, 9, 12, 24, 52
Detrusor pressure was expressed as bladder pressure minus intra-abdominal pressure as assessed by urodynamics.
Baseline and Weeks 3, 6, 9, 12, 24, 52
Change From Baseline to Weeks 3, 6, 9, 12, 24 and 52 in Catheterized Volume 5 Minutes After End of Bladder-Filling
Time Frame: Baseline and Weeks 3, 6, 9, 12, 24, 52
Catheterized volume was measured when the bladder was emptied via catheterization 5 minutes after the end of bladder-filling.
Baseline and Weeks 3, 6, 9, 12, 24, 52
Change From Baseline to Weeks 3, 6, 9, 12, 24 and 52 in Bladder Volume Until First Detrusor Contraction (> 15 cmH2O)
Time Frame: Baseline and Weeks 3, 6, 9, 12, 24, 52
Bladder volume as assessed by urodynamics.
Baseline and Weeks 3, 6, 9, 12, 24, 52
Change From Baseline to Weeks 3, 6, 9, 12, 24 and 52 in Bladder Volume at 10 cmH20 Detrusor Pressure
Time Frame: Baseline and Weeks 3, 6, 9, 12, 24, 52
Bladder volume as assessed by urodynamics.
Baseline and Weeks 3, 6, 9, 12, 24, 52
Change From Baseline to Weeks 3, 6, 9, 12, 24 and 52 in Bladder Volume at 20 cmH20 Detrusor Pressure
Time Frame: Baseline and Weeks 3, 6, 9, 12, 24, 52
Bladder volume as assessed by urodynamics.
Baseline and Weeks 3, 6, 9, 12, 24, 52
Change From Baseline to Weeks 3, 6, 9, 12, 24 and 52 in Bladder Volume at 30 cmH20 Detrusor Pressure
Time Frame: Baseline and Weeks 3, 6, 9, 12, 24, 52
Bladder volume as assessed by urodynamics.
Baseline and Weeks 3, 6, 9, 12, 24, 52
Change From Baseline to Weeks 3, 6, 9, 12, 24 and 52 in Number of Overactive Detrusor Contractions (> 15 cmH2O) Until Leakage or End of Bladder-Filling
Time Frame: Baseline and Weeks 3, 6, 9, 12, 24, 52
Baseline and Weeks 3, 6, 9, 12, 24, 52
Change From Baseline to Weeks 3, 6, 9, 12, 24, 36 and 52 in Average Catheterized Volume Per Catheterization
Time Frame: Baseline and Weeks 3, 6, 9, 12, 24, 36, 52
The average catheterized volume per catheterization was calculated using all available (non-zero) catheterized volumes recorded over both of the 2 measuring days in the diary, whether or not these 2 days were concurrent. Catheterized volumes were recorded for 2 days in a participant diary prior to each visit. Four to six catheterizations were performed every day.
Baseline and Weeks 3, 6, 9, 12, 24, 36, 52
Change From Baseline to Weeks 3, 6, 9, 12, 24, 36 and 52 in Maximum Catheterized Volume (MCV)
Time Frame: Baseline and Weeks 3, 6, 9, 12, 24, 36, 52
The maximum catheterized volume per day was calculated using all available (non-zero) catheterized volumes recorded for the 2 measuring days in the diary, whether or not these 2 days were concurrent. The maximum value was calculated separately for each measuring day and the mean of these two values was used.
Baseline and Weeks 3, 6, 9, 12, 24, 36, 52
Change From Baseline to Weeks 3, 6, 9, 12, 24, 36 and 52 in Average First Morning Catheterized Volume
Time Frame: Baseline and Weeks 3, 6, 9, 12, 24, 36, 52
The first morning catheterized volume was the volume associated with the first morning catheterization. The average first morning catheterized volume was calculated as the average of the available first morning catheterized volumes recorded for the 2 measuring days in the diary, whether or not these 2 days are concurrent.
Baseline and Weeks 3, 6, 9, 12, 24, 36, 52
Change From Baseline to Weeks 3, 6, 9, 12, 24, 36 and 52 in Mean Number of Periods Between the Clean Intermittent Catheterizations (CICs) With Incontinence Per 24 Hours
Time Frame: Baseline and Weeks 3, 6, 9, 12, 24, 36, 52
Participants were required to have 4-6 CICs per day on a schedule fixed for the duration of the study. To calculate the number of periods between CICs with incontinence in a diary day, the diary day was divided into periods between CICs (i.e. inter-CIC periods). The hour period, rather than the exact time, of each CIC and incontinence episode was recorded in the diary. When an incontinence episode and a CIC were marked in the same hour period, the incontinence episode was counted as occurring prior to the CIC (when the bladder had not yet emptied), rather than after it (when the bladder had recently been emptied), i.e. the inter-CIC period ended with the hour in which the CIC was recorded. The mean number of periods between CICs with incontinence per 24 hours was the number of periods between CICs when incontinence occurred, divided by the total number of valid diary days.
Baseline and Weeks 3, 6, 9, 12, 24, 36, 52
Percentage of Days With Catheterizations for Each Hour of 24 Hour Day at Baseline
Time Frame: 3 days prior to Baseline visit
For each one hour period of the 24 hour day, the percentage of days with catheterization was assessed as the number of days with catheterization occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Baseline visit
Percentage of Days With Catheterizations for Each Hour of 24 Hour Day During Week 3
Time Frame: 3 days prior to Week 3 visit
For each one hour period of the 24 hour day, the percentage of days with catheterization was assessed as the number of days with catheterization occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Week 3 visit
Percentage of Days With Catheterizations for Each Hour of 24 Hour Day During Week 6
Time Frame: 3 days prior to Week 6 visit
For each one hour period of the 24 hour day, the percentage of days with catheterization was assessed as the number of days with catheterization occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Week 6 visit
Percentage of Days With Catheterizations for Each Hour of 24 Hour Day During Week 9
Time Frame: 3 days prior to Week 9 visit
For each one hour period of the 24 hour day, the percentage of days with catheterization was assessed as the number of days with catheterization occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Week 9 visit
Percentage of Days With Catheterizations for Each Hour of 24 Hour Day During Week 12
Time Frame: 3 days prior to Week 12 visit
For each one hour period of the 24 hour day, the percentage of days with catheterization was assessed as the number of days with catheterization occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Week 12 visit
Percentage of Days With Catheterizations for Each Hour of 24 Hour Day During Week 24
Time Frame: 3 days prior to Week 24 visit
For each one hour period of the 24 hour day, the percentage of days with catheterization was assessed as the number of days with catheterization occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Week 24 visit
Percentage of Days With Catheterizations for Each Hour of 24 Hour Day During Week 36
Time Frame: 3 days prior to Week 36 visit
For each one hour period of the 24 hour day, the percentage of days with catheterization was assessed as the number of days with catheterization occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Week 36 visit
Percentage of Days With Catheterizations for Each Hour of 24 Hour Day During Week 52
Time Frame: 3 days prior to Week 52 visit
For each one hour period of the 24 hour day, the percentage of days with catheterization was assessed as the number of days with catheterization occurring within the one hour period divided by the number of valid diary days over all subjects. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Week 52 visit
Percentage of Days With Incontinence for Each Hour of 24 Hour Day at Baseline
Time Frame: 3 days prior to Baseline visit
Incontinence was defined as leakage where a diaper is not used, or dampness where a diaper is used. For each one hour period of the 24 hour day, the percentage of days with incontinence was assessed as the number of days with incontinence occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Baseline visit
Percentage of Days With of Incontinence for Each Hour of 24 Hour Day During Week 3
Time Frame: 3 days prior to Week 3 visit
Incontinence was defined as leakage where a diaper is not used, or dampness where a diaper is used. For each one hour period of the 24 hour day, the percentage of days with incontinence was assessed as the number of days with incontinence occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Week 3 visit
Percentage of Days With Incontinence for Each Hour of 24 Hour Day During Week 6
Time Frame: 3 days prior to Week 6 visit
Incontinence was defined as leakage where a diaper is not used, or dampness where a diaper is used. For each one hour period of the 24 hour day, the percentage of days with incontinence was assessed as the number of days with incontinence occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Week 6 visit
Percentage of Days With Incontinence for Each Hour of 24 Hour Day During Week 9
Time Frame: 3 days prior to Week 9 visit
Incontinence was defined as leakage where a diaper is not used, or dampness where a diaper is used. For each one hour period of the 24 hour day, the percentage of days with incontinence was assessed as the number of days with incontinence occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Week 9 visit
Percentage of Days With Incontinence for Each Hour of 24 Hour Day During Week 12
Time Frame: 3 days prior to Week 12 visit
Incontinence was defined as leakage where a diaper is not used, or dampness where a diaper is used. For each one hour period of the 24 hour day, the percentage of days with incontinence was assessed as the number of days with incontinence occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Week 12 visit
Percentage of Days With Incontinence for Each Hour of 24 Hour Day During Week 24
Time Frame: 3 days prior to Week 24 visit
Incontinence was defined as leakage where a diaper is not used, or dampness where a diaper is used. For each one hour period of the 24 hour day, the percentage of days with incontinence was assessed as the number of days with incontinence occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Week 24 visit
Percentage of Days With Incontinence for Each Hour of 24 Hour Day During Week 36
Time Frame: 3 days prior to Week 36 visit
Incontinence was defined as leakage where a diaper is not used, or dampness where a diaper is used. For each one hour period of the 24 hour day, the percentage of days with incontinence was assessed as the number of days with incontinence occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Week 36 visit
Percentage of Days With Incontinence for Each Hour of 24 Hour Day During Week 52
Time Frame: 3 days prior to Week 52 visit
Incontinence was defined as leakage where a diaper is not used, or dampness where a diaper is used. For each one hour period of the 24 hour day, the percentage of days with incontinence was assessed as the number of days with incontinence occurring within the one hour period divided by the number of valid diary days over all participants. Each participant contributed to up to three days of valid diary data for each visit.
3 days prior to Week 52 visit
Change From Baseline to Week 24 and 52 in Infant and Toddler Quality of Life Short Form-47 Questionnaire (ITQoL SF-47) - Overall Health Score
Time Frame: Baseline and Weeks 24, 52
The ITQoL SF-47 consisted of 47 individual items and was filled in by the child's parent. The individual items are based on Likert scales with either 5 responses (coded as 1 through to 5) or 4 responses (coded as 1 through to 4). From subsets of these coded values, 11 scales are derived: overall health, physical activities, development, discomfort, moods and temperaments, perceptions of current past and future health and perception of changes. Each scale score ranges from 0 (worst possible) to 100 (best possible).
Baseline and Weeks 24, 52
Change From Baseline to Week 24 and 52 in ITQoL SF-47 - Physical Abilities Score
Time Frame: Baseline and Weeks 24, 52
The ITQoL SF-47 consisted of 47 individual items and was filled in by the child's parent. The individual items are based on Likert scales with either 5 responses (coded as 1 through to 5) or 4 responses (coded as 1 through to 4). From subsets of these coded values, 11 scales are derived: overall health, physical activities, development, discomfort, moods and temperaments, perceptions of current past and future health and perception of changes. Each scale score ranges from 0 (worst possible) to 100 (best possible).
Baseline and Weeks 24, 52
Change From Baseline to Week 24 and 52 in ITQoL SF-47 - Growth and Development Score
Time Frame: Baseline and Weeks 24, 52
The ITQoL SF-47 consisted of 47 individual items and was filled in by the child's parent. The individual items are based on Likert scales with either 5 responses (coded as 1 through to 5) or 4 responses (coded as 1 through to 4). From subsets of these coded values, 11 scales are derived: overall health, physical activities, development, discomfort, moods and temperaments, perceptions of current past and future health and perception of changes. Each scale score ranges from 0 (worst possible) to 100 (best possible).
Baseline and Weeks 24, 52
Change From Baseline to Week 24 and 52 in ITQoL SF-47 - Pain Score
Time Frame: Baseline and Weeks 24, 52
The ITQoL SF-47 consisted of 47 individual items and was filled in by the child's parent. The individual items are based on Likert scales with either 5 responses (coded as 1 through to 5) or 4 responses (coded as 1 through to 4). From subsets of these coded values, 11 scales are derived: overall health, physical activities, development, discomfort, moods and temperaments, perceptions of current past and future health and perception of changes. Each scale score ranges from 0 (worst possible) to 100 (best possible).
Baseline and Weeks 24, 52
Change From Baseline to Week 24 and 52 in ITQoL SF-47 - Temperament and Moods Score
Time Frame: Baseline and Weeks 24, 52
The ITQoL SF-47 consisted of 47 individual items and was filled in by the child's parent. The individual items are based on Likert scales with either 5 responses (coded as 1 through to 5) or 4 responses (coded as 1 through to 4). From subsets of these coded values, 11 scales are derived: overall health, physical activities, development, discomfort, moods and temperaments, perceptions of current past and future health and perception of changes. Each scale score ranges from 0 (worst possible) to 100 (best possible).
Baseline and Weeks 24, 52
Change From Baseline to Week 24 and 52 in ITQoL SF-47 - Behaviour Score
Time Frame: Baseline and Weeks 24, 52
The ITQoL SF-47 consisted of 47 individual items and was filled in by the child's parent. The individual items are based on Likert scales with either 5 responses (coded as 1 through to 5) or 4 responses (coded as 1 through to 4). From subsets of these coded values, 11 scales are derived: overall health, physical activities, development, discomfort, moods and temperaments, perceptions of current past and future health and perception of changes. Each scale score ranges from 0 (worst possible) to 100 (best possible).
Baseline and Weeks 24, 52
Change From Baseline to Week 24 and 52 in ITQoL SF-47 - General Health Score
Time Frame: Baseline and Weeks 24, 52
The ITQoL SF-47 consisted of 47 individual items and was filled in by the child's parent. The individual items are based on Likert scales with either 5 responses (coded as 1 through to 5) or 4 responses (coded as 1 through to 4). From subsets of these coded values, 11 scales are derived: overall health, physical activities, development, discomfort, moods and temperaments, perceptions of current past and future health and perception of changes. Each scale score ranges from 0 (worst possible) to 100 (best possible).
Baseline and Weeks 24, 52
Change From Baseline to Week 24 and 52 in ITQoL SF-47 - Change in Health Score
Time Frame: Baseline and Weeks 24, 52
The ITQoL SF-47 consisted of 47 individual items and was filled in by the child's parent. The individual items are based on Likert scales with either 5 responses (coded as 1 through to 5) or 4 responses (coded as 1 through to 4). From subsets of these coded values, 11 scales are derived: overall health, physical activities, development, discomfort, moods and temperaments, perceptions of current past and future health and perception of changes. Each scale score ranges from 0 (worst possible) to 100 (best possible).
Baseline and Weeks 24, 52
Change From Baseline to Week 24 and 52 in ITQoL SF-47 - Parent-Emotional Impact Score
Time Frame: Baseline and Weeks 24, 52
The ITQoL SF-47 consisted of 47 individual items and was filled in by the child's parent. The individual items are based on Likert scales with either 5 responses (coded as 1 through to 5) or 4 responses (coded as 1 through to 4). From subsets of these coded values, 11 scales are derived: overall health, physical activities, development, discomfort, moods and temperaments, perceptions of current past and future health and perception of changes. Each scale score ranges from 0 (worst possible) to 100 (best possible).
Baseline and Weeks 24, 52
Change From Baseline to Week 24 and 52 in ITQoL SF-47 - Parent-Time Impact Score
Time Frame: Baseline and Weeks 24, 52
The ITQoL SF-47 consisted of 47 individual items and was filled in by the child's parent. The individual items are based on Likert scales with either 5 responses (coded as 1 through to 5) or 4 responses (coded as 1 through to 4). From subsets of these coded values, 11 scales are derived: overall health, physical activities, development, discomfort, moods and temperaments, perceptions of current past and future health and perception of changes. Each scale score ranges from 0 (worst possible) to 100 (best possible).
Baseline and Weeks 24, 52
Change From Baseline to Week 24 and 52 in ITQoL SF-47 - Family Cohesion Impact Score
Time Frame: Baseline and Weeks 24, 52
The ITQoL SF-47 consisted of 47 individual items and was filled in by the child's parent. The individual items are based on Likert scales with either 5 responses (coded as 1 through to 5) or 4 responses (coded as 1 through to 4). From subsets of these coded values, 11 scales are derived: overall health, physical activities, development, discomfort, moods and temperaments, perceptions of current past and future health and perception of changes. Each scale score ranges from 0 (worst possible) to 100 (best possible).
Baseline and Weeks 24, 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Astellas Pharma Europe B.V.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director, Astellas Pharma Europe B.V.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 25, 2013

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 18, 2015

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 18, 2015

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 6, 2013

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 6, 2013

First Posted (Estimated)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 13, 2013

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 31, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 20, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
October 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 905-CL-074
  • 2012-003178-22 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Access to anonymized individual participant level data will not be provided for this trial. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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