A Long-Term Extension Study of OnabotulinumtoxinA (BOTOX®) for Urinary Incontinence Due to Neurogenic Detrusor Overactivity

Urinary Incontinence Clinical Trial

Official title:

Long-term Extension Study of BOTOX® in the Treatment of Urinary Incontinence Due to Neurogenic Detrusor Overactivity in Patients 5 to 17 Years of Age

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01852058
• Other study ID #: 191622-121
• Secondary ID: 2012-004898-30
• Status: Completed
• Phase: Phase 3
• Start date: January 11, 2014
• Est. completion date: October 3, 2019

Study information

• Verified date: April 2020
• Source: Allergan
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study will evaluate the long-term safety and efficacy of onabotulinumtoxinA (botulinum toxin Type A; BOTOX®) for the treatment of urinary incontinence due to neurogenic detrusor overactivity in participants who successfully completed Study 191622-120 (NCT01852045).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 95
• Est. completion date: October 3, 2019
• Est. primary completion date: November 22, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 17 Years

Eligibility

Inclusion Criteria:

• Successfully completed participation in Study 191622-120

• Aged = 5 years to = 17 years at the time of entry into Study 191622-120

• Regularly using clean intermittent catheterization to empty the bladder

Exclusion Criteria:

• Myasthenia gravis, Eaton-Lambert syndrome, or amyotrophic lateral sclerosis

• Current or planned use of a baclofen pump

• Current or planned use of an electrostimulation/neuromodulation device for urinary incontinence

• Use of an indwelling catheter for urinary incontinence instead of using clean intermittent catheterization to empty the bladder

• Previous or current use of botulinum toxin therapy of any serotype for any urological condition, or treatment with botulinum toxin of any serotype for any other condition since entering study 191622-120

Related Conditions & MeSH terms

• Enuresis
• Urinary Incontinence

Intervention

Biological:
• OnabotulinumtoxinA
OnabotulinumtoxinA injected into the detrusor wall. Treatments were administered as needed with a minimum of a 12-week interval between doses.

Locations

Country	Name	City	State
Belgium	UZ Antwerpen	Antwerpen
Belgium	Ghent University Hospital	Gent
Belgium	UZ Leuven	Leuven
Canada	McMaster University Medical Centre	Hamilton	Ontario
Canada	CHU Sainte Justine	Montreal	Quebec
Czechia	Fakultni nemocnice Hradec Kralove	Hradec Kralove
Czechia	Fakultni nemocnice Olomouc	Olomouc
France	Hopital Pellegrin - Enfants	Bordeaux
France	CHU de Limoges - Hôpital Mère et l'Enfant	Limoges
France	Hôpital Necker Enfants-Malades	Paris
Italy	Seconda Università di Napoli	Naples
Italy	Bambin Gesù- Ospedale Pediatrico	Rome
Poland	Copernicus Podmiot Leczniczy Sp. z o. o. Kliniczny Oddzial Chirurgii i Urologii Dzieci i Mlodziezy GUMed	Gdansk
Poland	Specjalistyczny Gabinet Lekarski	Poznan
Poland	Medical University of Wroclaw	Wroclaw
Turkey	University of Ankara	Ankara
United States	University of Alabama at Birmingham Division of Urology Research Office	Birmingham	Alabama
United States	Medical University of South Carolina	Charleston	South Carolina
United States	McKay Urology Carolinas Medical Center	Charlotte	North Carolina
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center Cincinnati Center for Clinical Research and Outpatient Clinic	Cincinnati	Ohio
United States	Duke University	Durham	North Carolina
United States	Riley Hospital for Children	Indianapolis	Indiana
United States	Cedars-Sinai Medical Center	Los Angeles	California
United States	Children's Hospital of Wisconsin Department of Pediatric Urology	Milwaukee	Wisconsin
United States	Oklahoma Children's Hospital	Oklahoma City	Oklahoma
United States	Children's Hospital of Orange County	Orange	California
United States	William Beaumont Hospital Research Institute	Royal Oak	Michigan
United States	St. Louis Children's Hospital Division of Urology	Saint Louis	Missouri
United States	Pediatric Urology Associates, PC	Tarrytown	New York

Sponsors (1)

Lead Sponsor	Collaborator
Allergan

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  Czechia,  France,  Italy,  Poland,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Study Baseline in the Daily Normalized Daytime Average Number of Urinary Incontinence Episodes in Treatment Cycle 1	Urinary incontinence was defined as involuntary loss of urine as recorded by the participant in a bladder diary during 2 consecutive days in the week prior to the study visit (normalized to a 12 hour daytime period). Daytime is defined as the time between waking up to start the day and going to bed to sleep for the night. The number of daily daytime incontinence episodes were averaged during the 2-day period. A negative change from Baseline indicates improvement. Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Study Baseline (Prior to Day 1 in Study 120) to 2 consecutive days in the week prior to Week 6 in Treatment Cycle 1
Primary	Change From Study Baseline in the Daily Normalized Daytime Average Number of Urinary Incontinence Episodes in Treatment Cycle 2	Urinary incontinence was defined as involuntary loss of urine as recorded by the participant in a bladder diary during 2 consecutive days in the week prior to the study visit (normalized to a 12 hour daytime period). Daytime is defined as the time between waking up to start the day and going to bed to sleep for the night. The number of daily daytime incontinence episodes were averaged during the 2-day period. A negative change from Baseline indicates improvement. Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Study Baseline (Prior to Day 1 in Study 120) to 2 consecutive days in the week prior to Week 6 in Treatment Cycle 2
Primary	Change From Study Baseline in the Daily Normalized Daytime Average Number of Urinary Incontinence Episodes in Treatment Cycle 3	Urinary incontinence was defined as involuntary loss of urine as recorded by the participant in a bladder diary during 2 consecutive days in the week prior to the study visit (normalized to a 12 hour daytime period). Daytime is defined as the time between waking up to start the day and going to bed to sleep for the night. The number of daily daytime incontinence episodes were averaged during the 2-day period. A negative change from Baseline indicates improvement. Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Study Baseline (Prior to Day 1 in Study 120) to 2 consecutive days in the week prior to Week 6 in Treatment Cycle 3
Secondary	Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Treatment Emergent Adverse Events (STEAEs)	An adverse event is any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with use of a medicinal (investigational) product, whether or not related to medicinal (investigational) product. A serious adverse event (SAE) is any AE that resulted in death, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, life threatening, a congenital anomaly/birth defect, or an important medical event. A TEAE or STEAE is defined as any new AE or worsening of an existing condition after initiation of treatment. Data are summarized under the respective treatments that participants received in the corresponding treatment cycles.	First injection on Day 1 in Study 120 through completion of Study 121 (Up to 108 weeks)
Secondary	Percentage of Participants With = 50%, = 75%, = 90%, and = 100% Reduction From Baseline in the Number of Normalized Daytime Urinary Incontinence Episodes in Treatment Cycle 1	Urinary incontinence was defined as involuntary loss of urine as recorded by the participant in a bladder diary during 2 consecutive days in the week prior to the study visit (normalized to a 12 hour daytime period). Daytime is defined as the time between waking up to start the day and going to bed to sleep for the night. The number of daily incontinence episodes were averaged during the 2-day period. Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Study Baseline (Prior to Day 1 in Study 120) to 2 consecutive days in the week prior to Week 6 in Treatment Cycle 1
Secondary	Percentage of Participants With = 50%, = 75%, = 90%, and = 100% Reduction From Baseline in the Number of Normalized Daytime Urinary Incontinence Episodes in Treatment Cycle 2	Urinary incontinence was defined as involuntary loss of urine as recorded by the participant in a bladder diary during 2 consecutive days in the week prior to the study visit (normalized to a 12 hour daytime period). Daytime is defined as the time between waking up to start the day and going to bed to sleep for the night. The number of daily incontinence episodes were averaged during the 2-day period. Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Study Baseline (Prior to Day 1 in Study 120) to 2 consecutive days in the week prior to Week 6 in Treatment Cycle 2
Secondary	Percentage of Participants With = 50%, = 75%, = 90%, and = 100% Reduction From Baseline in the Number of Normalized Daytime Urinary Incontinence Episodes in Treatment Cycle 3	Urinary incontinence was defined as involuntary loss of urine as recorded by the participant in a bladder diary during 2 consecutive days in the week prior to the study visit (normalized to a 12 hour daytime period). Daytime is defined as the time between waking up to start the day and going to bed to sleep for the night. The number of daily incontinence episodes were averaged during the 2-day period. Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Study Baseline (Prior to Day 1 in Study 120) to 2 consecutive days in the week prior to Week 6 in Treatment Cycle 3
Secondary	Change From Baseline in Average Urine Volume at First Morning Catheterization in Treatment Cycle 1	The change in urine volume at first morning catherization was recorded by the participant in a bladder diary in the 2 consecutive days during the week prior to the study visit. The daily values were averaged during the 2-day period. A positive change from Baseline indicates improvement. Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Baseline (Prior to Day 1 in Study 120) to 2 consecutive days in the week prior to Week 6 in Treatment Cycle 1
Secondary	Change From Baseline in Average Urine Volume at First Morning Catheterization in Treatment Cycle 2	The change in urine volume at first morning catherization was recorded by the participant in a bladder diary in the 2 consecutive days during the week prior to the study visit. The daily values were averaged during the 2-day period. A positive change from Baseline indicates improvement. Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Baseline (Prior to Day 1 in Study 120) to 2 consecutive days in the week prior to Week 6 in Treatment Cycle 2
Secondary	Change From Baseline in Average Urine Volume at First Morning Catheterization in Treatment Cycle 3	The change in urine volume at first morning catherization was recorded by the participant in a bladder diary in the 2 consecutive days during the week prior to the study visit. The daily values were averaged during the 2-day period. A positive change from Baseline indicates improvement. Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Baseline (Prior to Day 1 in Study 120) to 2 consecutive days in the week prior to Week 6 in Treatment Cycle 3
Secondary	Percentage of Participants With Night Time Urinary Incontinence in Treatment Cycle 1	Urinary incontinence was defined as involuntary loss of urine. Night time urinary incontinence was recorded by the participant on the bladder diary as a presence or absence of urinary leakage upon waking, for 2 consecutive days in the week prior to the week 6 visit. Night time was defined as the time between going to bed to sleep for the night and waking up to start the day. The percentage of participants with night time urinary incontinence is presented in categories 0, 1, and 2 nights. Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Baseline (Prior to Day 1 in Study 120) and 2 consecutive days in the week prior to Week 6 in Treatment Cycle 1
Secondary	Percentage of Participants With Night Time Urinary Incontinence in Treatment Cycle 2	Urinary incontinence was defined as involuntary loss of urine. Night time urinary incontinence was recorded by the participant on the bladder diary as a presence or absence of urinary leakage upon waking, for 2 consecutive days in the week prior to the week 6 visit. Night time was defined as the time between going to bed to sleep for the night and waking up to start the day. The percentage of participants with night time urinary incontinence is presented in categories 0, 1, and 2 nights. Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Baseline (Prior to Day 1 in Study 120) and 2 consecutive days in the week prior to Week 6 in Treatment Cycle 2
Secondary	Percentage of Participants With Night Time Urinary Incontinence in Treatment Cycle 3	Urinary incontinence was defined as involuntary loss of urine. Night time urinary incontinence was recorded by the participant on the bladder diary as a presence or absence of urinary leakage upon waking, for 2 consecutive days in the week prior to the week 6 visit. Night time was defined as the time between going to bed to sleep for the night and waking up to start the day. The percentage of participants with night time urinary incontinence is presented in categories 0, 1, and 2 nights. Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Baseline (Prior to Day 1 in Study 120) and 2 consecutive days in the week prior to Week 6 in Treatment Cycle 3
Secondary	Average Time to Participant's Request for Retreatment	Time to request for re-treatment is the time in weeks between last injection and request for next injection, regardless of fulfillment of the re-treatment criteria. Data are summarized under the respective treatments that participants received across entire study. Data is reported for only participants that had at least one request for retreatment while on a specified BOTOX dose.	First injection on Day 1 in Study 120 through to the date of completion of Study 121 (Up to 108 weeks)
Secondary	Average Time to Participant's Qualification for Retreatment	The criteria for qualification of retreatment included 1) Participant/parent/caregiver requests retreatment; 2) Participant has a total of at least 2 daytime urinary incontinence episodes over the 2-day bladder diary collection period; 3) At least 12 weeks has elapsed since treatment 1 and 4) Participant has not experienced a serious treatment-related adverse event at any time. Data are summarized under the respective treatments that participants received across entire study. Data is reported for only participants that had at least one request for retreatment while on a specified BOTOX dose.	First injection on Day 1 in Study 120 through to the date of completion of Study 121 (Up to 108 weeks)
Secondary	Percentage of Participants With Positive Response on Modified Treatment Benefit Scale (TBS) in Treatment Cycle 1	The Modified TBS is a single-item scale which assesses the participant's condition (urinary problems, urinary incontinence) on a 4-point scale where 1 = greatly improved; 2 = improved; 3 = not changed; and 4 = worsened. A participant was considered to have a positive treatment response if they responded to the TBS question as either "greatly improved" or "improved". Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Week 6 in Treatment Cycle 1
Secondary	Percentage of Participants With Positive Response on Modified Treatment Benefit Scale (TBS) in Treatment Cycle 2	The Modified TBS is a single-item scale which assesses the participant's condition (urinary problems, urinary incontinence) on a 4-point scale where 1 = greatly improved; 2 = improved; 3 = not changed; and 4 = worsened. A participant was considered to have a positive treatment response if they responded to the TBS question as either "greatly improved" or "improved". Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Week 6 in Treatment Cycle 2
Secondary	Percentage of Participants With Positive Response on Modified Treatment Benefit Scale (TBS) in Treatment Cycle 3	The Modified TBS is a single-item scale which assesses the participant's condition (urinary problems, urinary incontinence) on a 4-point scale where 1 = greatly improved; 2 = improved; 3 = not changed; and 4 = worsened. A participant was considered to have a positive treatment response if they responded to the TBS question as either "greatly improved" or "improved". Data are summarized under the respective treatments that participants received in the corresponding treatment cycle.	Week 6 in Treatment Cycle 3

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