Primary Hyperoxaluria Type 1
Conditions
Sponsors
Alnylam Pharmaceuticals, Inc
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Male and female subjects aged 18-64 years (or age of legal consent, whichever is older), inclusive (Part A) and 6-64 years, inclusive (Part B). 2. Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception. 3. Willing to provide written informed consent and to comply with study requirements. Additional Inclusion Criteria for Part B: 4. confirmation of PH1 disease. 5. 24-hour urinary oxalate excretion of >0.7 mmol/1.73m2/day. 6.Estimated GFR of >45 mL/min/1.73m2. 7. If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days .
Exclusion criteria
Exclusion criteria: 1. Any uncontrolled or serious disease, or any medical or surgical condition (with the exception of PH1 for patients in Part B) that may either interfere with participation in the clinical study, and/or put the subject at significant risk (according to the Investigator's judgment) if he/she participates in the clinical study. 2. Mental illness, alcoholism, drug abuse, or heavy smokers and users of nicotine 3. History of multiple drug allergies or intolerance to subcutaneous injection 4. Received an investigational agent within 3 months before the first dose of study drug or are in follow-up of another clinical study 5. Known history of allergic reaction to an oligonucleotide or GalNAc 6. History of intolerance to SC injection or relevant abdominal scarring 7. Women who are pregnant or breast feeding Part B only 8. Echocardiography (ECHO) assessment of normal left ventricular systolic function, defined as left ventricular ejection fraction the upper limit of normal (ULN) at screening
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary * The primary endpoint is the incidence of adverse events. Safety will also be evaluated through vital signs, electrocardiograms, clinical laboratory assessments, and physical examinations. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Endpoints for Part B o PK parameters including, but not limited to, Cmax, tmax, AUC, t*, fe/F, and CLR o Urinary oxalate excretion (oxalate concentration in 24-hour urine collection) o Urinary glycolate excretion (glycolate concentration in 24-hour urine collection) o Plasma glycolate concentration o Calculated creatinine clearance | — |
Countries
Netherlands
Outcome results
None listed