Abstract: INFO15-SA
Evaluation of ADV7103 Efficacy and Safety in Cystinuria
Session Information
- Informational Posters - III
November 09, 2019 | Location: Exhibit Hall, Walter E. Washington Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Genetic Diseases of the Kidneys
- No subcategory defined
Authors
- Guittet, Catherine, Advicenne, Nïmes, France
- Granier, Luc andre, Advicenne, Nïmes, France
- Bertholet-Thomas, Aurélia, Centre de Référence des Maladies Rénales Rares, Bron Cedex, France
- Knebelmann, Bertrand, Necker Hopsital, Paris, France
Description
BACKGROUND: Beside hyperhydration, alkaline urine pH is a cornerstone of therapeutic management of cystinuria. The presence of cystine crystals in urine (crystalluria), and more specifically high volume of crystals (Vcys > 3000 µ3/mm3) was shown to be predictive of recurrence of cystine stones (Daudon et al, 2003).
CORAL 1 is a European cystinuria clinical program including 3 studies. The main Phase IIb/III multicenter, randomized, placebo-controlled, double-blind, 4 parallel arms, dose-ranging study (B12CS) evaluates the efficacy, safety, tolerability and acceptability of repeated doses of ADV7103, after 7 days of treatment, in 60 patients with cystinuria. ADV7103 is a fixed-dose combination of potassium citrate and potassium bicarbonate formulated as prolonged release-granules. B12CS is a superiority study evaluating the efficacy of ADV7103 at 3 different doses compared to placebo. The primary objective evaluates the effect of ADV7103 compared to placebo on the percentage of urinary pH values ≥ 7.0 during repeated measures 24h after a 7-day treatment. Secondary objectives evaluate the effect and dose-response relationship of ADV7103 on urinary pH, cystine crystalluria (including Vcys) and cystinuria parameters after a 7 day-treatment.
Inclusion criteria include age 6 years and above, a confirmed cystinuria diagnosis, a well-adapted alkalizing therapy and a disease status enabling interruption of a cystine binding drug. Exclusion criteria include patients who cannot stop chelating agents, hyperkalemia > 5.0 mmol/L and eGFR < 45 mL/min/1.73 m2.
Patients below 6 years of age or urine incontinent will receive ADV7103 in an open-manner, in an exploratory sub-study (B13CS).
The first patients have been enrolled in May 2019.
A follow up study (B14CS) over 2 years will enroll patients after they finished the B12CS-B13CS study.
For more information contact lag@advicenne.com
Funding
- Sponsor Advicenne SA