Lumasiran for Primary Hyperoxaluria
(ILLUMINATE-C Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial is testing a medication called lumasiran for patients with a severe genetic disorder called Advanced Primary Hyperoxaluria Type 1 (PH1). The medication works by lowering the amount of a harmful substance called oxalate that the liver produces, which can help protect the kidneys and other organs.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications. However, if you are taking Vitamin B6 or are on hemodialysis, you must have been on a stable regimen for a certain period before joining the trial.
How does the drug Lumasiran differ from other treatments for primary hyperoxaluria?
Lumasiran is unique because it uses RNA interference to target and reduce the production of oxalate in the liver, which is the root cause of primary hyperoxaluria type 1. Unlike traditional treatments that focus on managing symptoms, Lumasiran directly addresses the disease mechanism by silencing the gene responsible for oxalate production, offering a novel approach to prevent kidney and systemic complications.12345
What data supports the effectiveness of the drug Lumasiran for treating Primary Hyperoxaluria?
Lumasiran has been shown to reduce oxalate levels in the body, which helps prevent kidney damage in patients with Primary Hyperoxaluria Type 1. Studies, including the ILLUMINATE-C and ILLUMINATE-B trials, have demonstrated its effectiveness and safety in both adults and young children, leading to symptom improvement and preventing disease progression.15678
Who Is on the Research Team?
Medical Director
Principal Investigator
Alnylam Pharmaceuticals
Are You a Good Fit for This Trial?
This trial is for patients with Advanced Primary Hyperoxaluria Type 1 (PH1). Eligible participants include those on stable hemodialysis for at least 4 weeks, diagnosed with PH1, meeting specific plasma oxalate levels, and if taking Vitamin B6, must have been on a consistent dose for over 90 days. Patients should have an eGFR ≤45 mL/min/1.73 m^2 or elevated serum creatinine for their age if under 12 months.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive open-label lumasiran to evaluate efficacy, safety, pharmacokinetics, and pharmacodynamics
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term Follow-up
Participants' quality of life and systemic oxalosis are assessed up to 60 months
What Are the Treatments Tested in This Trial?
Interventions
- Lumasiran
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alnylam Pharmaceuticals
Lead Sponsor
Dr. Yvonne Greenstreet
Alnylam Pharmaceuticals
Chief Executive Officer since 2021
MD from the University of Leeds, MBA from INSEAD
Dr. Pushkal Garg
Alnylam Pharmaceuticals
Chief Medical Officer since 2016
MD from Columbia University