Primary Hyperoxaluria Type 1
Conditions
Sponsors
Alnylam Pharmaceuticals, Inc
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Enrollment within 12 months of completion of Study ALN-GO1-001 and in the opinion of the investigator, tolerated the study drug 2. If taking vitamin B6 (pyridoxine), willing to remain on a stable regimen for the study duration 3. Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception from 14 days before first dose and throughout study participation until the completion of the follow-up period. 4. Willing and able to comply with the study requirements and to provide written informed consent and assent in the case of patients under the age of legal consent, per local and national requirements.
Exclusion criteria
Exclusion criteria: 1. Any uncontrolled or serious disease, or any medical or surgical condition (with the exception of PH1) that may either interfere with participation in the clinical study, and/or put the patient significant risk (according to the Investigator*s judgment) if he/she participates in the clinical study 2. An underlying known disease or surgical or medical condition (with the exception of PH1) that in the opinion of the investigator might interfere with the interpretation of the clinical study results 3. Requirement for chronic dialysis 4. Triplicate 12-lead ECG with clinically significant abnormalities at Baseline visit, at the discretion of the investigator 5. Echo assessment of abnormal left ventricular systolic function, defined as left ventricular ejection fraction
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary study outcome is the incidence of adverse events (AEs) | — |
Secondary
| Measure | Time frame |
|---|---|
| * Change in 24-hour urinary oxalate corrected for body surface area (BSA) over time * Change in 24-hour urinary oxalate: creatinine ratio over time * Change estimated glomerular filtration rate (eGFR) over time Exploratory * Describe the effects of ALN-GO1 on other safety parameters, including assessments of vital signs, electrocardiograms (ECGs), renal ultrasounds, clinical laboratory parameters, and physical examinations. * Assess ALN-GO1 plasma PK parameters including maximum plasma concentration (Cmax), time to maximum plasma concentration (tmax), elimination half-life (t1/2), area under the concentration-time curve (AUC), fraction of drug excreted in urine (Fe), apparent clearance (CL/F), and apparent volume of distribution (V/F). * Change from Baseline over the course of the study in the following PD parameters: - Urinary glycolate excretion - Plasma oxalate concentration - Plasma glycolate concentration - Spot urinary oxalate:creatinine ratio * Incidence of ADA. | — |
Countries
Netherlands
Outcome results
None listed