CompletedPhase 3

A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1

United States, Australia, Belgium21 participantsStarted 2020-01-21

Plain-language summary

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in patients with Advanced Primary Hyperoxaluria Type 1 (PH1).

Who can participate

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: * Has documented diagnosis of primary hyperoxaluria type 1 (PH1) * Estimated glomerular filtration rate (eGFR) ≤45 mL/min/1.73 m\^2 for patients ≥12 months of age (\<12 months of age, must have serum creatinine considered elevated for age) * Meets plasma oxalate level requirements * If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days * If on dialysis, may be on hemodialysis therapy only and must have been on a stable regimen for at least 4 weeks Exclusion Criteria: * Hemodialysis/peritoneal dialysis combination therapy or peritoneal dialysis alone * Diagnosis of conditions other than PH1 contributing to renal insufficiency * History of liver transplant * History of kidney transplant and currently receiving immunosuppressants

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

Cohort A: Percent Change in Plasma Oxalate From Baseline to Month 6

Timeframe: Baseline to Month 6

Cohort B: Percent Change in Pre-dialysis Plasma Oxalate From Baseline to Month 6

Timeframe: Baseline to Month 6

Trial details

NCT IDNCT04152200

SponsorAlnylam Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-05-18

Results submitted2022-05-18

View on ClinicalTrials.gov More Primary Hyperoxaluria Type 1 trials

Who can participate

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.