Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1 (NCT02706886) | Clinical Trial Compass
CompletedPhase 1/2
Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1
France, Germany, Israel52 participantsStarted 2016-03-08
Plain-language summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single-ascending doses (SAD) and multiple-ascending doses (MAD) of lumasiran in healthy adult volunteers and subjects with primary hyperoxaluria type 1 (PH1). In Part A, single ascending dose (SAD) part, healthy adults were dosed with lumasiran or placebo once. In Part B, multiple ascending doses (MAD) part, patients with primary hyperoxaluria type 1 (PH1) were dosed with lumasiran or placebo. All patients that initially received placebo received lumasiran after completing placebo dosing.
Who can participate
Age range6 Years – 64 Years
SexALL
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Inclusion Criteria for Parts A and B:
* Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception.
* Willing to provide written informed consent and to comply with study requirements.
Additional Inclusion Criteria for Part B:
* Confirmation of PH1 disease
* Meet 24 hour urine oxalate excretion requirements
* Estimated glomerular filtration rate (GFR) of \>45 mL/min/1.73m\^2
* If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days
Exclusion Criteria for Parts A and B:
* Clinically significant health concerns (with the exception of PH1 for patients in Part B)
* Clinically significant electrocardiogram (ECG) abnormalities
* Abnormal for aspartate aminotransferase (AST)/alanine aminotransferase (ALT) and any other clinical safety laboratory result considered clinically significant
* Received an investigational agent within 3 months before the first dose of study drug or are in follow-up of another clinical study
* Known history of allergic reaction to an oligonucleotide or N-acetylgalactosamine (GalNAc)
* History of intolerance to subcutaneous injection
What they're measuring
1
Number of Participants With Adverse Events (AEs)
Timeframe: Part A (SAD): Up to 405 days; Part B (MAD): Up to 546 days