Cystinuria MedDRA version: 20.0 Level: PT Classification code 10011778 Term: Cystinuria System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: ADV7103
Pharmaceutical Form: Prolonged-release granules
INN or Proposed INN: potassium citrate monohydrate
CAS Number: 6100-05-6
Other descriptive name: potassium citrate, tripotassium c
Sponsors
Advicenne
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: ADV7103 Cohort : • Patient who has participated to and completed the previous B12CS Study or B13CS Study. SoC Cohort : • Patient who has a diagnosis of cystinuria based on medical diagnosis (at least one previous or current episode of calculus of cystine, and/or one previous or current episode of cystine crystalluria) or on genetic diagnosis (only for patients of Subset 4). • Patient treated with an alkalising treatment at a well-adapted dose (defined as a daily dose deemed by the investigator aiming to maintain overtime urinary pH value = 7.0 and/or compatible with an acceptable safety profile and/or patient’s constraints or compliance). • Patient who, when treated with a second line therapy (chelator agent), presents a disease status enabling interruption of the chelator agent during the course of the B10CS research. • Patient male or female, including child aged between 6 months and 17 years old and adult aged = 18 years old up to 70 years old. Are the trial subjects under 18? yes Number of subjects for this age range: 24 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 12 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: ADV7103 Cohort : • Patient who has not participated to B12CS Study or B13CS Study • Patient for whom any safety issue could contraindicate her/his participation to the extension study. Soc Cohort : • Patient treated with the second line therapy and who cannot stop cystine chelating agents (sulfhydryl compounds) during the B14CS study. • Patient who presents kalaemia > 5.0 mmol/L. • Patient who presents a moderate or severe renal impairment (estimated glomerular filtration rate (eGFR) < 45 mL/min/1.73 m2 according to Schwartz formula for the children and both MDRDs and CKD-EPI for adults).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and the tolerability of ADV7103 and standard of care (SoC) after a long-term treatment. ;Secondary Objective: • To evaluate the effect of ADV7103 and SoC on urine pH during a long-term treatment (proportion/number of responders with urine pH values in the first morning urines and in pre-dose urines = 7.0 and = 7.5) • To evaluate the efficacy of ADV7103 and SoC on cystine crystalluria (including the volume of crystals of cystine - Vcys) and other other cystinuric parameters during a long-term treatment • To evaluate the efficacy of ADV7103 and SoC on other urine parameters during a longterm treatment • To evaluate the efficacy of ADV7103 and SoC on cystine calculi during a long-term treatment • To evaluate the time for any potential adding of a second line therapy with cystine chelators. • To evaluate the paraclinical and biological safety of ADV7103 and SoC during a long-term treatment.;Primary end point(s): Number and proportion of patients who have experienced treatment-emergent adverse events during the course of the study.;Timepoint(s) of evaluation of this end point: all along the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Main secondary efficacy endpoint: Values of the urine pH with a glass electrode pH-meter;Timepoint(s) of evaluation of this end point: - at each site visit from Visit 1 to Visit 7, in the fresh first and second morning urines - between site visits, at home, at least twice a week (total 4 time-points per week), including 2 time-points per day, in the morning and in the evening at approximately 12h-interval and before the treatment administration, in the first fresh morning urines and in the fresh evening urines, respectively | — |
Countries
Belgium, France, Spain, United Kingdom
Contacts
Public ContactResponsible of Clinical studies
Advicenne
Outcome results
None listed