Early Oxybutinin Treatment for Boys With Posterior Urethral Valves

NCT04526353 | Unknown Phase 2

• 1 other identifier: CHUBX 2018/65
• Study Type: interventional
• Target: 50
• Locations: 0 countries
• Sites: N/A

Brief Summary

Boys with posterior urethral valves have bladder dysfunction of varying severity. Early treatment of these children with anticholinergics is recommended by some teams, although there have never been any clear studies on the subject. To our knowledge, no comparative study of the evolution of valve bladders with or without treatment has been carried out to date. Anticholinergic treatment, although it may be beneficial in patients with abnormal bladder function, such as the neurologic bladders ( in Spina Bifida) for example, may have side effects and may not be of benefit for this valve population. The evolution of the valves could be spontaneously favorable. This study would be the first randomized clinical trial of early therapeutic drug intervention in the posterior urethral valve population.

Conditions

Male Urogenital Diseases

Keywords

posterior urethral valves; urodynamic; anticholinergic

Outcome Measures

Primary Outcomes (1)

• success of treatment defined by the association of the three events

  composite endpoints where the success of treatment at 9 months after inclusion is defined by the association of the three following events: * Voiding pressure \<60 cmH2O AND * Bladder Volume ≥70% of theoretical value AND * for those without pop-off mechanisms, Bladder compliance \>10mL/cmH2O A failure of treatment will be defined as the absence of at least one of these events. In presence of a pressure pop-off mechanism, only voiding pressure and bladder volume will be analyzed.

  9 months after inclusion

Secondary Outcomes (14)

• Proportion of adverse events in each group

  through study completion, an average of 9 months

• Type of adverse events in each group

  through study completion, an average of 9 months

• Incidence of urinary tract infections in each group

  through study completion, an average of 9 months

• Compliance with treatment

  9 months after inclusion

• Sonographic changes

  9 months after inclusion

Study Arms (2)

Oxybutynin during 9 months.

EXPERIMENTAL

0.1mg / kg 2x / day from inclusion and for 9 months.

Drug: Oxybutynin 1 mg/ml Syrup

No oxybutynin

NO INTERVENTION

No treatment affecting bladder function

Interventions

Oxybutynin 1 mg/ml Syrup DRUG

The Investigational Medicinal Product of this study is Oxybutynin 1 mg/ml Syrup (see Annex 1 for the Monograph of PMS-Oxybutynin provided by ANSM under the ATU.). It will be administered at the dose of 0.1 mg/kg/twice a day to patients randomized to the study treatment group. The dose will be adapted to the child's weight to the nearest kilogram. The recommended dosage for older children with neurologic bladder is 0.3 to 0.4mg/kg/day, whilst the dosage we will be using is effectively 0.2mg/kg/day. This is because we are taking into account the absence or pharmacological studies of oxybutynin use in children \<1 year of age, as well as their specific liver metabolism. Furthermore, the dose of 0.1mg/kg twice daily is the dose used in children within the same age group in the study by Casey et al, 2012

Oxybutynin during 9 months.

Eligibility Criteria

• Age: 3 Months - 6 Months
• Gender Eligibility Details: Boys, aged 3 to 6 months, diagnosed with posterior urethral valves. Urethral valves (PUV) are the most common form of congenital urethral obstruction with an incidence ranging from 1/3,000 and 1/8,000 male births.
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Boys
• Aged 3 to 6 months
• Diagnosed with posterior urethral valves, and having undergone valve resection within the first 3 months of life
• Having undergone urodynamic studies between 10 weeks and 6 months of age andshowing 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)/
• Holders of parental authority affiliated to French national health insurance
• With informed consent signed by holders of parental authority

You may not qualify if:

• Boys with posterior urethral valves and normal urodynamics or no urodynamic assessment
• Boys in whom urodynamic assessment is not possible for medical or anatomical reasons
• Boys requiring dialysis before the age of 3 months
• Contra-indication to oxybutynin such as hypersensitivity to oxybutynin or 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

Sponsors & Collaborators

• University Hospital, Bordeaux: lead

Related Publications (1)

• Casey JT, Hagerty JA, Maizels M, Chaviano AH, Yerkes E, Lindgren BW, Kaplan WE, Meyer T, Cheng EY. Early administration of oxybutynin improves bladder function and clinical outcomes in newborns with posterior urethral valves. J Urol. 2012 Oct;188(4 Suppl):1516-20. doi: 10.1016/j.juro.2012.03.068. Epub 2012 Aug 19.

  PMID: 22910256 BACKGROUND

MeSH Terms

Conditions

Male Urogenital Diseases

Interventions

oxybutynin

Condition Hierarchy (Ancestors)

Urogenital Diseases

Study Officials

• Laurent L FOURCADE, MD

  University Hospital, Limoges

  PRINCIPAL INVESTIGATOR

• Alice A FAURE, MD

  APHM - Hôpital Timone Enfants

  PRINCIPAL INVESTIGATOR

• Thomas BLANC, MD

  APHP - Hôpital Necker Enfants Malades

  PRINCIPAL INVESTIGATOR

• Alaa A EL GHONEIMI, MD

  APHP- Hôpital Robert Debré

  PRINCIPAL INVESTIGATOR

• Alexis A ARNAUD, MD

  Rennes University Hospital

  PRINCIPAL INVESTIGATOR

• Ourdia O BOUALI, MD

  University Hospital, Toulouse

  PRINCIPAL INVESTIGATOR

• Jean-Baptiste JB MARRET, MD

  University Hospital, Caen

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Luke L HARPER, MD

CONTACT

05 56 79 56 17; luke.harper@chu-bordeaux.fr

Aurore A CAPELLI, PhD

CONTACT

05 57 82 08 77; aurore.capelli@chu-bordeaux.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Phase 2 non comparative randomized multicentre clinical trial with two parallel groups: * Oxybutynin (0.1 mg/kg twice daily) during 9 months. * No oxybutynin To determine whether the observed effects of oxybutynin are really due to the treatment we need to perform a randomized clinical trial. There is insufficient evidence in the literature about the natural evolution of the bladder in boys with PUV. Given the paucity of literature data, our objective is not to formally test the superiority of oxybutynin versus no drug but to detect and quantify the effect of oxybutynin. This is why our clinical is randomized but non-comparative.

Study Record Dates

• First Submitted: July 29, 2020
• First Posted: August 25, 2020
• Study Start: September 10, 2020
• Primary Completion: September 10, 2023
• Study Completion: September 10, 2023
• Last Updated: August 25, 2020

Record last verified: 2020-07

Data Sharing

• IPD Sharing: Will not share