21 Participants Needed

Lumasiran for Primary Hyperoxaluria

(ILLUMINATE-C Trial)

Recruiting at 15 trial locations
Age: Any Age
Sex: Any
Trial Phase: Phase 3
Sponsor: Alnylam Pharmaceuticals
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise

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?

MD

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

I have been diagnosed with primary hyperoxaluria type 1.
I have been on a stable hemodialysis regimen for at least 4 weeks.
My kidney function is low, with an eGFR of 45 or less.
See 2 more

Exclusion Criteria

I have kidney problems not caused by Primary Hyperoxaluria type 1.
I have had a kidney transplant and am on immunosuppressants.
I have had a liver transplant.
See 1 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive open-label lumasiran to evaluate efficacy, safety, pharmacokinetics, and pharmacodynamics

6 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Long-term Follow-up

Participants' quality of life and systemic oxalosis are assessed up to 60 months

54 months

What Are the Treatments Tested in This Trial?

Interventions

  • Lumasiran
Trial Overview The study tests Lumasiran's effectiveness and safety in treating PH1. It will also examine how the body processes the drug (pharmacokinetics) and its impact on the disease (pharmacodynamics).
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: LumasiranExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

Alnylam Pharmaceuticals

Lead Sponsor

Trials
81
Recruited
16,100+

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

Citations

Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial. [2023]
Lumasiran in the Management of Patients with Primary Hyperoxaluria Type 1: From Bench to Bedside. [2022]
Infantile Primary Hyperoxaluria Type 1 Treated With Lumasiran in Twin Males. [2022]
The effect of lumasiran therapy for primary hyperoxaluria type 1 in small infants. [2023]
Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial. [2023]
[Management of Primary Hyperoxaluria Type 1 in Italy]. [2021]
Lumasiran: First Approval. [2021]
Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1: A Placebo-Controlled Randomized Clinical Trial. [2023]
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