WithdrawnPhase 2/3

Study Evaluating Patients With Cystinuria

Stopped: Unspecified business decision/strategic reason

Belgium, France0Started 2024-05-01

Plain-language 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.

Who can participate

Age range6 Months – 70 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Patient who has participated to and completed the previous B12CS Study or B13CS Study.
✓. Patient for whom the safety and tolerability of ADV7103 were satisfactory during B12CS Study or B13CS Study.
✓. Female patient of childbearing potential (defined by the Clinical Trial Facilitation Group (CTFG) as woman fertile, following menarche until becoming post-menopausal unless permanently sterile\*) using an acceptable effective birth control method\*\* and having a negative pregnancy test at the inclusion, or a woman postmenopausal\*\*\* or a woman surgically sterilized\*.
✓. Patient and/or parents or legal representative(s) who is(are) willing and able to participate in the study, to understand and to comply with study procedures for the entire length of the study.
✓. Patient or parents or legal representative(s) who has(have) provided a signed written informed consent.
✓. Patient of ≤17 Patient of ≤17 years of age for whom the assent has been collected or has been tried to be collected.
✓. Patient who is affiliated to a social health insurance system and/or in compliance with the recommendations of the national law in force relating to biomedical research.
✓. 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).

Exclusion criteria

✕. 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
✕. Patient that is receiving the second line therapy -- cystine chelating agents (sulfhydryl compounds).
✕. 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).
✕. Patient who presents - barring the study disease - any previous or concurrent medical condition or any laboratory or clinical findings or any other condition that in the opinion of the investigator would be negatively affected by the study product or that would affect the study product or that precludes his participation, e.g. uncontrolled diabetes mellitus, adrenal insufficiency, cardiac impairment, repeated infections, metabolic alkalosis, chronic diarrhoea.
✕. Female patient who is pregnant or breast-feeding.
✕. Patient who cannot stop potassium sparing diuretics (e.g. antagonists of aldosterone as such spironolactone, canrenoate and eplerenone, amiloride, triamterene), angiotensin converting enzyme inhibitors, angiotensin II receptor antagonists, tacrolimus, potassium desodic salts.

What they're measuring

Percentage of urinary pH values ≥ 7.0 during 24h on Day 7 (after ADV7103 treatment period)

Timeframe: 7 Days

Trial details

NCT IDNCT04137978

SponsorAdvicenne Pharma

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-06

View on ClinicalTrials.gov More Cystinuria trials