Alfuzosin Treatment in Children and Adolescents With Neurogenic Urinary Bladder Dysfunction (ALFACHIN)

Sanofi

Status and phase: Completed; Phase 3

Conditions: Neurogenic Urinary Bladder

Treatments: Drug: Placebo; Drug: Alfuzosin

Study type: Interventional

Funder types: Industry

Identifiers

NCT00549939

EFC5722

2004-002397-38 (EudraCT Number)

Details and patient eligibility

About

The primary objective of the study was to evaluate the efficacy of Alfuzosin in comparison to Placebo on the detrusor Leak Point Pressure (LPP) in children and adolescents 2-16 years of age with elevated detrusor LPP of neuropathic etiology and detrusor LPP ≥ 40 cm H2O.

Secondary objectives were:

• To investigate the safety and tolerability of two doses of Alfuzosin in comparison to Placebo in children and adolescents,

• To evaluate the effects of the two doses of Alfuzosin in comparison to Placebo on:

  • Detrusor compliance,
  • Urinary tract infection,

• To investigate the pharmacokinetics of Alfuzosin (population kinetics),

• To evaluate the 12-month long-term safety of Alfuzosin 0.1 mg/kg/day and 0.2 mg/kg/day.

The study consisted of 2 periods:

• a 12-week double blind treatment period where patients were to receive either Alfuzosin 0.1 mg/kg/day or Alfuzosin 0.2 mg/kg/day or placebo then,
• a 40-week open label extension treatment period where patients were to receive either Alfuzosin 0.1 mg/kg/day or Alfuzosin 0.2 mg/kg/day.

Full description

Patients who met the study entry criteria were randomized (2:1:2:1) to one of the 4 dosage groups (Alfuzosin 0.1 mg/kg/day, matching placebo 0.1 mg/kg/day, Alfuzosin 0.2 mg/mg/kg, matching placebo 0.2 mg/kg/day).

Patients received their treatment using either solution or tablet formulation depending on age as follows:

• Solution to children 2-7 years of age or, children and adolescents 8-16 years of age if they were unable to swallow tablets or they preferred to take the solution or if they had a body weight < 30kg. The daily dose was devided in 3 doses given at at breakfast, lunch and dinner.
• Tablet to children and adolescents 8-16 years of age who were able to swallow tablets and had a body weight ≥ 30kg. The daily dose was devided in 2 doses given at at breakfast and dinner.

Patients who have completed the 12-week double-blind phase were offered to continue in the 40-week open-label extension study.

• Patients receiving Alfuzosin continued with their dosing regimen.
• Patients receiving Placebo were switched to Alfuzosin with a dose corresponding to their randomization dose group.

All patients had a one-week follow-up period after last dose intake.

Enrollment: 172 patients

Sex: All

Ages: 2 to 16 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patient with elevated detrusor Leak Point Pressure (LPP) of neuropathic etiology and Detrusor LPP ≥ 40 cm H2O and < 100 cm H2O.

Exclusion criteria

• Urological surgery in the last 4 months prior to the study,
• Patients who have urethral dilatation in the last 3 months prior to the baseline urodynamic assessment,
• α-blocker therapy in the last 4 weeks prior to the baseline urodynamic assessment,
• Detrusor injections of botulinum toxin in the last 6 months,
• Urological diseases/conditions other than functional bladder obstruction of neuropathic etiology that can lead to upper urinary tract dilatation (e.g., bladder anomalies, ureterocele),
• History of intolerance to α-blocker therapy,
• Orthostatic hypotension,
• History of risk factors for Torsade de pointes (e.g., family history of Long QT Syndrome).

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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172 participants in 3 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

Matching placebo 0.1 mg/kg/day or 0.2 mg/kg/day

Treatment:

Drug: Placebo

Alfuzosin 0.1 mg/kg/day

Experimental group

Treatment:

Drug: Alfuzosin

Alfuzosin 0.2 mg/kg/day

Experimental group

Treatment:

Drug: Alfuzosin