metabolic disorders Primary Hyperoxaluria Type 1
Conditions
Interventions
Lumasiran (ALN-GO1) is an investigational agent comprised of a synthetic, small
interfering RNA (siRNA) (drug substance ALN-65585) covalently linked to a
triantennary N-acetylgalactosamine (GalNAc)
Sponsors
Alnylam Pharmaceuticals, Inc.
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Age 6 years or older. 2. Documentation or confirmation of PH1 as determined by genetic analysis prior to randomization. 3. Mean 24-hour urinary oxalate excretion from the first 2 valid 24-hour urine collections is >=0.70 mmol/24h/1.73m2 4. 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. 5. 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
Exclusion criteria: 1. Medical history includes clinical evidence of extrarenal systemic oxalosis, as determined by the Investigator. 2. 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 1.5 (patients on oral anticoagulant [eg, warfarin] with an INR =18 years of age and the Schwartz Bedside Formula for patients 2 units/day is excluded during the study (unit: 1 glass of wine [approximately 125 mL] = 1 measure of spirits [approximately 1 fluid ounce] = * pint of beer [approximately 284 mL] 13. History of alcohol abuse, within the last 12 months before screening, in the opinion of the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percent change in 24-hour urinary oxalate excretion from baseline to Month 6 | — |
Secondary
| Measure | Time frame |
|---|---|
| - Absolute change in 24-hour urinary oxalate corrected for body surface area (BSA) from baseline to Month 6 - Change in 24-hour urinary oxalate:creatinine ratio (value/upper limit of normal [ULN]) from baseline to Month 6 - Proportion of patients with 24-hour urinary oxalate level at or below 1.5 x ULN at Month 6 - Proportion of patients with 24-hour urinary oxalate level at or below ULN at Month 6 - Percent change in plasma oxalate from baseline to Month 6 - Absolute change in plasma oxalate from baseline to Month 6 - Change in estimated glomerular filtration rate (eGFR) from baseline to Month 6 - Change from baseline (percent and absolute) in 24-hour urinary oxalate excretion, percentage of time that 24-hour urinary oxalate is | — |
Countries
Netherlands
Outcome results
None listed