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 range
6 Years – 64 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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