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A Phase 3 study of an Investigational Drug, Lumasiran (ALN-GO1) in patients with advanced primary hyperoxaluria Type 1
EU CTIS ID: 2023-503382-29-00
What this study is testing
Cohort A Evaluate the effect of lumasiran on plasma oxalate in patients who are not on dialysis therapy Cohort B Evaluate the effect of lumasiran on plasma oxalate levels in patients who are on dialysis therapy
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Cohort A and B: Have reached at least 37 weeks estimated gestational age (full-term infant) at consent (or assent)
- Documentation or confirmation of PH1 as determined by genetic analysis prior to initial dosing.
- Patients with eGFR ≤45 mL/min/1.73 m2 as calculated by the MDRD formula if ≥18 years or Schwartz Bedside Formula if ≥12 months to <18 years, or patients <12 months of age with serum creatinine that is considered elevated for age at consent.
- The mean of the three most recent screening plasma oxalate samples collected prior to Day 1 is ≥20 μmol/L. In Cohort B, these 3 collections may include the first pre-dialysis sample from each plasma oxalate profile.
- If taking therapeutic vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days before consent, and is able and willing to remain on this stable regimen until at least the Month 6 visit. Dose adjustments for interval weight gain are acceptable.
- Patient is willing and able to comply with the study requirements and to provide written informed consent. In the case of patients under the age of legal consent, the legal guardian(s) must provide informed consent and the patient should provide assent per local and national requirements.
You likely can't join if
- Has any of the following laboratory parameter assessments at Screening: a. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2× ULN for age b. Total bilirubin >1.5×ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert's syndrome are eligible if the total bilirubin is <2× ULN c. International normalized ratio (INR) >1.5 for patients not on anticoagulants. Patients on oral anticoagulants (eg, warfarin) with an INR <3.5 will be allowed. d. Hemoglobin <8.0 mg/dL
- Patients with a previous kidney transplant who are currently receiving immunosuppression to prevent transplant rejection.
- Patients maintained on a peritoneal dialysis regimen.
- Patients who in the opinion of the Investigator plan to start dialysis replacement therapy in the next 6 months.
- Any comorbidity or condition that, in the opinion of the Investigator, is anticipated to prevent participation in at least 12 months of the study
- Is not willing to comply with the contraceptive requirements during the study period, as described in study protocol.
See the full eligibility criteria
Who can join
- Cohort A and B: Have reached at least 37 weeks estimated gestational age (full-term infant) at consent (or assent)
- Documentation or confirmation of PH1 as determined by genetic analysis prior to initial dosing.
- Patients with eGFR ≤45 mL/min/1.73 m2 as calculated by the MDRD formula if ≥18 years or Schwartz Bedside Formula if ≥12 months to <18 years, or patients <12 months of age with serum creatinine that is considered elevated for age at consent.
- The mean of the three most recent screening plasma oxalate samples collected prior to Day 1 is ≥20 μmol/L. In Cohort B, these 3 collections may include the first pre-dialysis sample from each plasma oxalate profile.
- If taking therapeutic vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days before consent, and is able and willing to remain on this stable regimen until at least the Month 6 visit. Dose adjustments for interval weight gain are acceptable.
- Patient is willing and able to comply with the study requirements and to provide written informed consent. In the case of patients under the age of legal consent, the legal guardian(s) must provide informed consent and the patient should provide assent per local and national requirements.
- Cohort B Only 1: On a stable hemodialysis regimen for at least 4 weeks prior to Screening plasma oxalate assessment; able and willing to maintain this regimen through Month 6 visit, with changes to dialysis regimen permitted only when medically indicated.
What rules you out
- Has any of the following laboratory parameter assessments at Screening: a. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2× ULN for age b. Total bilirubin >1.5×ULN. Patients with elevated total bilirubin that is secondary to documented Gilbert's syndrome are eligible if the total bilirubin is <2× ULN c. International normalized ratio (INR) >1.5 for patients not on anticoagulants. Patients on oral anticoagulants (eg, warfarin) with an INR <3.5 will be allowed. d. Hemoglobin <8.0 mg/dL
- Patients with a previous kidney transplant who are currently receiving immunosuppression to prevent transplant rejection.
- Patients maintained on a peritoneal dialysis regimen.
- Patients who in the opinion of the Investigator plan to start dialysis replacement therapy in the next 6 months.
- Any comorbidity or condition that, in the opinion of the Investigator, is anticipated to prevent participation in at least 12 months of the study
- Is not willing to comply with the contraceptive requirements during the study period, as described in study protocol.
- Female patient is pregnant, planning a pregnancy, or breast feeding.
- Has known active human immunodeficiency virus (HIV) infection; or evidence of current or chronic hepatitis C virus (HCV) or hepatitis B (HBV) infection.
- Received an investigational agent within the last 30 days or 5 halflives, whichever is longer, prior to the first dose of study drug, or are in follow-up of another clinical study during Screening. Consultation with the Medical Monitor is required if treated with unapproved products prior to consent regardless of the time interval prior to the first dose of study drug.
- Known history of allergic reaction to an oligonucleotide or GalNAc.
- Diagnosis of conditions other than PH1 contributing to renal insufficiency such as glomerulonephritis, nephrotic syndrome, or lupus nephritis.
- Any condition or comorbidity, which in the opinion of the Investigator, would make the patient unsuitable for dosing or would interfere with study compliance, data interpretation, patient safety, and/or patient participation in the study. This includes significant active and poorly controlled (unstable) cardiovascular, neurologic, gastrointestinal, endocrine, renal or psychiatric disorders unrelated to PH1 identified by key laboratory abnormalities or medical history.
- Unwilling or unable to limit alcohol consumption throughout the course of the study. Alcohol intake of >2 units/day is excluded during the study (unit: 1 glass of wine [approximately 125 mL] = 1 measure of spirits [approximately 1 fluid ounce] = ½ pint of beer [approximately 284 mL].
- History of alcohol abuse, within the last 12 months before screening, in the opinion of the investigator.
- Patients with a previous liver transplant or for whom a liver transplant is anticipated in the next 6 months.
The study team makes the final eligibility decision.
Where it's taking place
- Australia
- United Arab Emirates
- Turkey
- Israel
- United Kingdom
- Lebanon
- United States
- Jordan
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Australia; United Arab Emirates; Turkey; Israel; United Kingdom; Lebanon and 2 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.