About the ILLUMINATE-A PH1 Clinical Trial

ILLUMINATE-A is a Phase 3, “randomized, double-blind, placebo-controlled study.” What does that mean?

During the first 6 months of the study two-thirds of the participants will be randomly assigned to receive the investigational medication, lumasiran (ALN-GO1), and the remaining one-third will receive placebo (a substance with no active medicine). The groups are “randomized,” meaning they are assigned at random, and “double-blind” means neither the researchers nor the participants will know which group a participant has been assigned to until after placebo dosing is complete. Finally, although a placebo is used in the study (“placebo-controlled”), it’s important to note that all participants will receive the investigational medication starting at month 6 of the trial.

The ILLUMINATE-A study is designed to study the safety and effectiveness of lumasiran (ALN-GO1) in children and adults with Primary Hyperoxaluria Type 1 (PH1).

Clinical trials don’t have to be confusing.

Visit our “Understanding Clinical Research” page for definitions and explanations.

ILLUMINATE-A has completed enrollment. Please sign up to be notified about future Alnylam PH1 clinical studies.

Who is eligible for the ILLUMINATE-A clinical trial?

Patients ages 6 years and above with a PH1 diagnosis as confirmed by genetic testing may be eligible for this clinical trial.

What is the purpose of ILLUMINATE-A?

The ILLUMINATE-A study is investigating the safety and effectiveness of the investigational medicine lumasiran in reducing urinary oxalate excretion (the amount of oxalate found in the urine of PH1 patients). That means we’re studying how safe and effective lumasiran is at reducing oxalate overproduction, which often results in painful, recurring kidney stones in people with Primary Hyperoxaluria Type 1 (PH1).

What happens in ILLUMINATE-A?

The ILLUMINATE-A study will enroll approximately 30 participants for up to a 5-year study participation commitment. The study includes 2 parts:

  1. A 6 month placebo controlled treatment period
  2. A 4.5 year open-label* extension period where all participants may receive lumasiran
    *See our “About Clinical Trials” page for definitions of these and other study-related terms.

Who is not eligible?

The ILLUMINATE-A study is not for people with PH1 who:

  • Have systemic oxalosis (oxalate is in organs outside the kidney)
  • Are on dialysis
  • Have had a kidney or liver transplant
  • Are pregnant or planning to become pregnant during the study
  • Are unwilling to limit their alcohol use if directed by the study team

Other criteria may apply. Please contact the ILLUMINATE study team to determine your specific eligibility.

ILLUMINATE-A has completed enrollment. Please sign up to be notified about future Alnylam PH1 clinical studies.