Prospective Randomized Clinical Trial of Early Oxybutinin Treatment for Boys with Posterior Urethral Valves - Pretipuv -

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Centre Hospitalier Universitaire De Bordeaux

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male

Therapeutic area

Diseases [C] - Male Urogenital Diseases [C12]

Trial duration

10 Sep 2020 → ongoing

Decision date (initial)

2024-10-01

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Ministry of Health (PHRC-N, 2018, 16-0408)

External identifiers

EU CT number

2024-515844-22-00

EudraCT number

2019-004417-13

ClinicalTrials.gov

NCT04526353

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy, Safety

To estimate the effect of oxybutynin on urodynamic evolution of bladder function (bladder compliance, bladder volume, peak voiding pressure) in boys after posterior valve ablation

Secondary objectives 6

1. To evaluate the safety pharmacokinetic of oxybutynin
2. To evaluate the safety profile of oxybutynin in boys with PUV
3. To estimate the incidence of urinary tract infection in boys with or without treatment
4. To estimate the compliance with oxybutynin treatment
5. To estimate Sonographic changes in both groups
6. To estimate the renal function (estimation of creatinine clearance)

Conditions and MedDRA coding

urethral posterior valve

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Boys aged 3 to 6 months
2. Diagnosed with posterior urethral valves, and having undergone valve resection within the first 3 months of life
3. Having undergone urodynamic studies between 10 weeks and 6 months of age and showing abnormal urodynamics, notably: high voiding pressure (>60cm H2O)/ small capacity bladder (<70% expected bladder volume)and for those without pop-off mechanisms, poor compliance (<10ml/cmH2O)/
4. Affiliated person or beneficiary of a social security scheme.
5. Free, informed and written consent signed by the participant and the investigator (at the latest on the day of inclusion and before any examination required by the research).

Exclusion criteria 5

1. Boys with posterior urethral valves and normal urodynamics or no urodynamic assessment
2. Boys requiring dialysis before the age of 3 months.
3. Boys who cannot undergo urodynamic testing for medical or anatomical reasons
4. Boys with severe decreased gastrointestinal motility conditions
5. Contra-indication to oxybutynin treatment such as hypersensitivity to oxybutynin ou any of the excipients, digestive obstruction, occlusive or sub-occlusive syndrome, megacolon, digestive stasis, intestinal atony, paralytic ileus, ulcerative colitis, Hemorrhagic rectocolitis, Crohn’s disease, Inflammatory bowel disease, Inflammatory organic colopathy, myasthenia, congenital glaucoma

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Composite endpoint where the success of treatment at 9 months after treatment inclusion is defined by the association of the three following events: - Bladder compliance >10mL/cmH2O AND - Voiding pressure <60 cmH2O AND - Bladder Volume ≥70% of theoretical value

Secondary endpoints 6

1. Proportion and type of adverse events in each group
2. Incidence of urinary tract infections in each group
3. Compliance with treatment will be evaluated through the proportion of oxybutynin treatment interruption
4. Sonographic changes will be expressed as a degree of hydronephrosis at 12-15 months of life (9 months after inclusion)
5. AUC, Cmax, Cmin and half-life of oxybutynin and desethyloxybutynin (active metabolite) in treated boys over treatment. Pharmacokinetic samples (6 points from Cmin to H+3h) at 2 weeks, 3 and 9 months after inclusion will be used to study and determine the pharmacokinetic parameters. In order to avoid having to take blood sample from the child several times, it is proposed to use a small venous catheter during the time of the pharmacokinetic samples.
6. Creatinine clearance in each group

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Centre Hospitalier Universitaire De Bordeaux

Sponsor organisation

Centre Hospitalier Universitaire De Bordeaux

Address

12 Rue Dubernat, Cs 91286 Cs 91286

City

Talence

Postcode

33400

Country

France

Scientific contact point

Organisation

Centre Hospitalier Universitaire De Bordeaux

Contact name

Luke HARPER

Public contact point

Organisation

Centre Hospitalier Universitaire De Bordeaux

Contact name

Luke HARPER

Locations

1 EU/EEA country · 10 investigational sites

By country

Investigational sites

Country notifications

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

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 14 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

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