BeDry

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Region Midtjylland

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Female Urogenital Diseases and Pregnancy Complications [C13], Diseases [C] - Male Urogenital Diseases [C12]

Trial duration

27 Jun 2024 → ongoing

Decision date (initial)

2024-04-19

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy

The primary objective is to evaluate the effect of solifenacin versus mirabegron versus combination therapy of solifenacin and mirabegron, in treatment of daytime urinary incontinence in children aged 5 to 14 years.

Secondary objectives 1

1. The secondary objective is to evaluate side effects, safety, and tolerability of solifenacin, mirabegron and combination therapy, as well as the effect of treatment on well-being and quality of life.

Conditions and MedDRA coding

Daytime urinary incontinence and overactive bladder

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. 1. The participants custody holder(s) must voluntarily sign and date an informed consent prior to initiation any study specific procedures.
2. Age 5 to 14 years (inclusive) at the time of signing the consent.
3. Overactive bladder as per ICCS criteria
4. At least 2 daytime urinary incontinens episodes per week
5. Inadequate effect of 4 weeks urotherapy (non-pharmacological treatment)
6. No previous treatment with solifenacin, mirabegron, intradetrusor botulinum toxin injections
7. No current constipation as per ROME IV criteria or fecal incontinence (laxative treatment is accepted)
8. Per investigator’s judgment, the participant can swallow or can learn to swallow study medication

Exclusion criteria 19

1. Inability of the patent(s) or parental custody holder(s) to understand the Danish written and oral information
2. Other significant ECG abnormalities
3. Known hypertension
4. ≤3 daily voiding, evaluated by 48-hour frequency-volume chart
5. Uroflowmetry suggestive of other pathology than OAB (staccato-shaped, interrupted-shaped, or plateau-shaped curve)
6. Post-void residual >50 ml after doublet voiding
7. Dipstick haematuria (≥2+ erythrocytes) or macroscopic haematuria
8. Pregnancy or breastfeeding
9. Ongoing constipation according to Rome IV-criteria which is intractable to medication or fecal incontinence
10. Inability to swallow study medication
11. Known or suspected hypersensitivity to study medication
12. Any contraindication to the use of the study medication
13. Known urogenital anatomical abnormalities affecting lower urinary tract function
14. Known kidney or bladder stones
15. Known diabetes insipidus (central or nephrogenic)
16. Ongoing symptomatic urinary tract infection (dysuria, fever, bacterial growth by urine culturing)
17. Recurrent urinary tract infection or ongoing prophylactic antibiotic treatment
18. Known QTc prolongation, QTc >460 ms, or risk of QTc prolongation (hypokalaemia, exercise-induced syncope, or familial long QT syndrome)
19. Female subjects of childbearing potential

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary outcome measure is treatment response as defined by non-response (<50% decrease in number of wet days) and response (50-100% decrease in number of wet days), assessed by a change from visit 2 and across the 12-week pharmacological treatment period. The number of wet days is assessed pr. 7 days by DryPie. The treatment response will be evaluated for solifenacin, mirabegron and combination of solifenacin and mirabegron.

Secondary endpoints 17

1. Change in number of wet days pr. 7 days assessed by Dry Pie, baseline to 18 weeks
2. Change in incontinence severity score pr. 7 days assessed by Dry Pie, baseline to 18 weeks
3. Change in urge severity quantified by Bower VAS Urgency, baseline to 18 weeks
4. Change in maximum volume voided (MVV), baseline to 18 weeks
5. Change in age standardized MVV (MVV as a percent of expected bladder capacity (EBC)), baseline to 18 weeks
6. Change in average voided volume (AVV), baseline to 18 weeks
7. Change in micturition frequency, baseline to 18 weeks
8. Change in fluid-intake, baseline to 18 weeks
9. Change in maximum flow rate (Qmax) assessed by uroflowmetry, baseline to 18 weeks
10. Change in Pediatric incontinence questionnaire total score, baseline to 18 weeks
11. Change in WHO-5 total score, baseline to 18 weeks
12. Adverse event (AE), serious adverse event (SAE) and Suspected Unexpected Serious Adverse Reaction (SUSAR) monitoring
13. Change in blood pressure and pulse, baseline to 18 weeks
14. Change in ultrasonic assessed post-void residual urine, baseline to 18 weeks
15. Identifications of ECG abnormalities by electrocardiogram
16. Identification of urinary tract infection by urine dipstick and verified by routine urine cultivation
17. Treatment response of solifenacin, mirabegron and combination of solifenacin and mirabegron, baseline across 18 week treatment period

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Region Midtjylland

Sponsor organisation

Region Midtjylland

Address

Hospitalsparken 15

City

Herning

Postcode

7400

Country

Denmark

Scientific contact point

Organisation

Region Midtjylland

Contact name

Ann-Kristine Mandøe Svendsen

Public contact point

Organisation

Region Midtjylland

Contact name

Ann-Kristine Mandøe Svendsen

Third parties 1

Locations

1 EU/EEA country · 5 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Application history

2 submissions — initial application plus substantial / non-substantial modifications