A study to evaluate the long-term safety and efficacy of BOTOX® for the treatment of urinary incontinence in patients 5 to 17 years of age
- Conditions
- eurogenic Detrusor Overactivity due to Urinary IncontinenceMedDRA version: 20.1 Level: PT Classification code 10046543 Term: Urinary incontinence System Organ Class: 10038359 - Renal and urinary disordersTherapeutic area: Not possible to specify
- Registration Number
- EUCTR2012-004898-30-CZ
- Lead Sponsor
- Allergan Ltd.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 100
1. patient was aged = 5 years to = 17 years of age at the time of informed consent for the preceding study, 191622-120
2. patient has participated in Study 191622-120 and fulfilled that study’s exit criteria (completed the qualification for retreatment/exit visit or the week 48/exit visit if they never qualified for retreatment)
3. written informed consent has been obtained from the legally authorized representative and written minor assent has been obtained, in accordance with local laws and institutional review board (IRB)/independent ethics committee (IEC) requirements
4. written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable (eg, Written Authorization for Use and Release of Health and Research Study Information [United States sites] and written Data Protection consent [European sites])
5. patient must be regularly using CIC to empty the bladder (CIC can be performed by either the patient or the parent/caregiver)
6. negative urine pregnancy test for females who are postmenarche
7. patient is able to complete study requirements including completion of bladder diaries and HRQOL questionnaires (these can also be completed by the parent/caregiver), and is likely to attend study visits in the opinion of the investigator
Are the trial subjects under 18? yes
Number of subjects for this age range: 100
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. patient has experienced an adverse event in the preceding study, 191622-120, that may indicate an unacceptable safety risk for additional BOTOX treatments, in the investigator’s opinion
2. patient has experienced a treatment-related serious adverse event in the preceeding study, 191622-120
3. investigator deems that, based on the patient’s response to the treatment received in study 191622-120, a dose reduction would be warranted for a subsequent treatment
4. patient has a newly diagnosed uncontrolled systemic disease, cancer or malignant disease, or uncontrolled epilepsy
5. patient has any newly diagnosed medical condition that may put them at increased risk with exposure to BOTOX including myasthenia gravis, Eaton-Lambert syndrome, or amyotrophic lateral sclerosis
6. patient currently uses or plans to use a baclofen pump
7. patient currently uses or plans to use an implantable or nonimplantable electrostimulation/neuromodulation device for the treatment of NDO
8. patient currently uses or plans to use an indwelling catheter, rather
than CIC, for treatment of NDO. (NOTE: an indwelling catheter can be
used if needed overnight as
long as it is not used during the diary collection periods)
9. patient has known allergy or sensitivity to components of any botulinum toxin preparation (including the study medication preparation), anesthetics or antibiotics to be used during the study
10. patient cannot withhold any antiplatelet, anticoagulant therapy or medications with anticoagulant effects for 3 days prior to treatment. Note: some medications may need to be withheld for > 3 days, per clinical judgment of the investigator (see Section 4.5.2, Prohibited Medications/Treatments for additional details).
11. patient has had previous botulinum toxin therapy of any serotype for any urological condition (other than the study medication) or for any nonurological condition since entry into the preceding study, 191622-120
12. postmenarche female patients who are pregnant, nursing, or planning to become pregnant during the study (postmenarche female patients must also either be sexually abstinent or use another acceptable form of contraception – see Section 4.5.1.1)
13. current enrollment in an investigational drug or device study or participation in such a study within 30 days of entry into this study (or longer if local requirements specify) other than Study 191622-120
14. patient has a condition or is in a situation which in the investigator’s opinion may put the patient at significant risk, may confound the study results, or may interfere significantly with the patient’s participation in the study
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the long-term safety and efficacy of BOTOX for the treatment of urinary incontinence due to Neurogenic Detrusor Overactivity (NDO) in patients 5 to 17 years of age who have not been adequately managed with anticholinergic therapy.;Secondary Objective: Not applicable;<br> Primary end point(s): The key efficacy measure is the number of daytime urinary incontinence episodes recorded in the bladder diary.<br> <br> ;<br> Timepoint(s) of evaluation of this end point: The timepoint of main interest is week 6 after each BOTOX treatment.<br>
- Secondary Outcome Measures
Name Time Method <br> Secondary end point(s): The following efficacy variables are being assessed in this long-term extension study:<br> • change from baseline in daily average frequency of daytime urinary incontinence episodes<br> • change from baseline in average urine volume at first morning catheterization (mL)<br> • presence or absence of night time urinary incontinence<br> • change from baseline in PinQ scoredomains (‘Role Limitations’ and ‘Social Limitations’)<br> • proportion of patients with a positive treatment response on the Modified TBS (ie, rating<br> their condition greatly improved” or improved”)<br> ;<br> Timepoint(s) of evaluation of this end point: The timepoint of main interest is week 6 after each BOTOX treatment.<br> <br>