A Study to Evaluate Setanaxib in Patients With Alport Syndrome

Inclusion Criteria

• Male or female patients aged 12 to 50 years inclusive, at the time of informed consent/assent. For sites in the European Union: Male or female patients aged 18 to 50 years, inclusive, at the time of informed consent;
• Diagnosis of Alport syndrome by genetic testing (documented mutation in a gene associated with Alport syndrome [ie, COL4A3, COL4A4, or COL4A5]); Patients with a variant of uncertain significance should not be included in the study;

Note: Genetic test results are to be available prior to initiating other Screening procedures. In cases where genetic testing results are not available but the patient is likely to fulfill the other inclusion and exclusion criteria and has provided consent/assent, a sample for genetic testing should be processed locally and the Screening Period duration can be prolonged with the time it takes to receive the genetic test results. All other Screening assessments must be completed within 4 weeks (± 7 days) prior to randomization.

• Weight ≥40 kg;
• Willing and able to give informed consent (and assent, where applicable), in accordance with local age requirements, and to comply with the requirements of the study;
• Female patients of childbearing potential must use a highly effective method of contraception to prevent pregnancy for ≥4 weeks before randomization and must agree to continue strict contraception (as specified in 5c) up to 90 days after the last dose of investigational medicinal product (IMP);
• For the purposes of this study, women of childbearing potential (WOCBP) are defined as "fertile, following menarche and until becoming postmenopausal unless permanently sterile. Permanent sterilization methods include hysterectomy, bilateral salpingectomy, and bilateral oophorectomy";
• Postmenopausal state is defined as no menses for 12 months without an alternative medical cause. In female patients who are not using hormonal contraception or hormonal replacement therapy but with suspected menopause and less than 12 months of amenorrhea, a high follicle-stimulating hormone level in the postmenopausal range will be required at Screening to confirm a postmenopausal state; and
• Highly effective contraception is defined as methods that can achieve a failure rate of less than 1% per year when used consistently and correctly. These methods include the following:
• Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal);
• Progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable);
• Intrauterine device;
• Intrauterine hormone-releasing system;
• Bilateral tubal occlusion;
• Vasectomized partner; and
• Sexual abstinence (refraining from heterosexual intercourse during the entire period of risk associated with the study treatments, ie, up to 90 days after the last dose of IMP). The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical study and the preferred and usual lifestyle of the patient. Periodic abstinence (calendar, symptothermal, or post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhea method are not acceptable methods of contraception.
• Female patients of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Visit 3 (after randomization and before dosing);
• Male patients with female partners of childbearing potential must be willing to use a condom and require their partner to use a highly effective contraceptive method (as defined in the list in inclusion criterion 5c). Female condom and male condom should not be used together. This requirement begins at the time of informed consent/assent and ends 90 days after receiving the last dose of IMP;
• Male patients must be willing not to donate sperm and female study patients must be willing not to d