NCT04137978

Study Evaluating Patients With Cystinuria

Open Label, Multicentre Study, Evaluating the Safety, Tolerability, Efficacy, Compliance and Acceptability of Alkalising Treatments at Long-term in Patients With Cystinuria

• Status: Withdrawn
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Advicenne Pharma · Industry
• Sex: All
• Age: 6 Months – 70 Years
• Healthy volunteers: Not accepted

Summary

This is an open label, multicentre study, evaluating the safety, tolerability, efficacy, compliance and acceptability of alkalising treatments at long-term in patients with cystinuria.

Detailed description

B14CS study is an extension study which follows the B12CS-B13CS study. Patients who have participated in B12CS-B13CS Study will have the possibility to switch to B14CS Study for a long-term treatment period (2 year-period). The B14CS Study is an open-label long-term study and including 2 types of cohorts. * ADV7103 Cohort: Cohort of patients who will have completed either B12CS or B13CS Study. Following an optional titration period (for B12CS Study subjects), all patients will be maintained at their ADV7103 optimal dose for a 2-year-period. * SoC Cohort: Cohort of patients with cystinuria, matching-pair for age category to the patients of ADV7103 Cohort, will receive their own alkalinising treatment (SoC) taken at the usual dose and frequency and will follow their usual first intention treatment (hydration and diet) for a 2-year-period. This cohort is designed in order to contextualize the safety, tolerability and efficacy of the long-term ADV7103 treatment. The B14CS Study will be composed of 3 periods (more detailed hereafter): * Lead-in Period: Period of 3 weeks, during which the treatment (ADV7103 or SoC) will be taken at the optimal dose as previously defined (Maintenance Phase). The Lead-in Period can include a Titration Phase, for ADV7103 B12CS Cohort only, in order to define the individual optimal dose of ADV7103 for the patients of this cohort; * Assessment Period: Period of 7 days to evaluate the effect of study products (ADV7103 and SoC) at the optimal dose; * Follow-up Period: Period of 23 months to evaluate the effects of the study products (ADV7103 and SoC) at long-term.

Conditions

• Cystinuria

Interventions

Type	Name	Description
DRUG	ADV7103	Patients receive ADV7103 twice a day at optimal dose.
DRUG	Standard of Care	Cohort of patients with cystinuria, matching-pair for age category to the patients of ADV7103 Cohort, will receive their own alkalinising treatment (SoC) taken at the usual dose and frequency and will follow their usual first intention treatment (hydration and diet) for a 2-year-period.

Timeline

• Start date: 2024-05-01
• Primary completion: 2025-06-01
• Completion: 2025-08-01
• First posted: 2019-10-24
• Last updated: 2024-03-12

Locations

13 sites across 2 countries: Belgium, France