Ensayos clínicos

Inclusion Criteria:

• At least 6 years of age at the time of signing the informed consent/assent.
• Documented diagnosis of PH3, confirmed by genotyping (historically available genotype information is acceptable for study eligibility).
• 24-hour Uox excretion ≥ 0.7 mmol (adjusted per 1.73 m^2 BSA in participants < 18 years of age) in both collections performed in the screening period.
• Less than 20% variation between the two 24-hour urinary creatinine measurements in the screening period. Individuals who do not achieve < 20% variation between the 2 screening values may undergo a second round of urine collection. An extra 7 calendar days may be added to the screening window for participants to complete a second round of urine collection. Should potential participants again fail to achieve the within < 20% variation, they will be excluded from participation.
• Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m^2 BSA, calculated using the CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) formula in participants aged ≥ 18 years (Levey & Stevens, 2010) or the multivariate formula by Schwartz in participants aged 6 to 17 years (Schwartz et al., 2012).
• History of at least one stone event within the last 12 months. Stone events are defined as any of the following:
  • Renal stone requiring medical intervention; e.g., outpatient procedures such as lithotripsy, or hospitalization or inpatient surgical intervention for confirmed stone-related pain and/or complications;
  • Stone passage with or without hematuria;
  • Renal colic requiring medication.
• Male or female
  • Male participants:
    • A male participant with a female partner of childbearing potential must agree to use contraception during the treatment period and for at least 12 weeks after the last dose of study intervention and refrain from donating sperm during this period.
  • Female participants:
    • A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least 1 of the following conditions applies:
    • Not a woman of childbearing potential (WOCBP);
    • A WOCBP who agrees to follow the contraceptive guidance for at least 12 weeks after the last dose of study intervention.
• Participant (and/or participant’s parent or legal guardian if participant is a minor [defined as patient < 18 years of age, or younger than the age of majority according to local regulations]) is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
• Adolescents (12 to < 18 years of age, or older than 12 years but younger than the age of majority according to local regulations) must be able to provide written assent for participation.
• For children younger than 12 years of age, assent will be based on local regulations.