Clinical Trials

Inclusion Criteria:

• Age 6 years or older.
• Documentation or confirmation of PH1 as determined by genetic analysis prior to randomization.
• Mean 24-hour urinary oxalate excretion from the first 2 valid 24-hour urine collections is ≥ 0.70 mmol/24h/1.73m2.
• If taking pyridoxine (vitamin B6) for the treatment of PH1, must have been on stable regimen for at least 90 days before randomization, and willing to remain on this stable regimen for 12 months from first study drug administration.
• Patient is able to understand and is willing and able to comply with the study requirements and to provide written informed consent. In the case of patients under the age of legal consent, the legal guardian(s) must provide informed consent and the patient should provide assent per local and national requirements.

Exclusion Criteria:

• Medical history includes clinical evidence of extrarenal systemic oxalosis, as determined by the Investigator.
• Has any of the following laboratory parameter assessments at screening:
  • Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 × upper limit of normal (ULN);
  • Total bilirubin > 1.5 x ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert’s syndrome are eligible if the total bilirubin is < 2 x ULN;
  • International normalized ratio (INR) > 1.5 (patients on oral anticoagulant [eg, warfarin] with an INR < 3.5 will be allowed).
• Has known active human immunodeficiency virus (HIV) infection; or evidence of current or chronic hepatitis C virus (HCV) or hepatitis B virus (HBV) infection.
• Estimated GFR of < 30 mL/min/1.73m2 at screening (calculation will be based on the Modification of Diet in Renal Disease [MDRD] formula for patients ≥ 18 years of age and the Schwartz Bedside Formula for patients < 18 years of age).
• Received an investigational agent within the last 30 days or 5 half-lives, whichever is longer, prior to the first dose of study drug, or are in follow-up of another clinical study prior to randomization.
• History of renal or liver transplant.
• Has other medical conditions or comorbidities, which in the opinion of the Investigator, would interfere with study compliance or data interpretation.
• History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc.
• History of intolerance to subcutaneous (SC) injection(s(.
• Is not willing to comply with the contraceptive requirements during the study period.
• Female patient is pregnant, planning a pregnancy, or breast-feeding.
• Unwilling or unable to limit alcohol consumption throughout the course of the study.   Alcohol intake of >2 units/day is excluded during the study (unit: 1 glass of wine [approximately 125 mL] = 1 measure of spirits [approximately 1 fluid ounce] = ½ pint of
• beer [approximately 284 mL].
• History of alcohol abuse, within the last 12 months before screening, in the opinion of the investigator.