- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03681184
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 (ILLUMINATE-A)
February 13, 2024 updated by: Alnylam Pharmaceuticals
ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study With an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
39
Phase
- Phase 3
Expanded Access
Approved for sale to the public.
See expanded access record.
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Lyon, France
- Clinical Trial Site
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Paris, France
- Clinical Trial Site
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Bonn, Germany
- Clinical Trial Site
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Haifa, Israel
- Clinical Trial Site
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Jerusalem, Israel
- Clinical Trial Site
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Nahariya, Israel
- Clinical Trial Site
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Amsterdam, Netherlands
- Clinical Trial Site
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Bern, Switzerland
- Clinical Trial Site
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Dubai, United Arab Emirates
- Clinical Trial Site
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Birmingham, United Kingdom
- Clinical Trial Site
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London, United Kingdom, NW3 2QG
- Clinical Trial Site
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London, United Kingdom, WC1N 3JH
- Clinical Trial Site
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California
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San Diego, California, United States, 92120
- Clinical Trial Site
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Florida
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Jacksonville, Florida, United States, 32216
- Clinical Trial Site
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Minnesota
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Rochester, Minnesota, United States, 55905
- Clinical Trial Site
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New York
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New York, New York, United States, 10029
- Clinical Trial Site
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Ohio
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Cleveland, Ohio, United States, 44195
- Clinical Trial Site
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
6 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Willing to provide written informed consent or assent and to comply with study requirements
- Confirmation of PH1 disease
- Meet the 24 hour urine oxalate excretion requirements
- If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days
Exclusion Criteria:
- Clinically significant health concerns (with the exception of PH1) or clinical evidence of extrarenal systemic oxalosis
- Clinically significant abnormal laboratory results
- Known active or evidence of HIV or hepatitis B or C infection
- An estimated GFR of < 30 mL/min/1.73m^2 at screening
- Received an investigational agent within 30 days or 5 half-lives before the first dose of study drug or are in follow-up of another clinical study
- History of kidney or liver transplant
- Known history of multiple drug allergies or allergic reaction to an oligonucleotide or GalNAc
- History of intolerance to subcutaneous injection
- Women who are pregnant, planning a pregnancy, or breast-feeding or those of child bearing potential and not willing to use contraception
- History of alcohol abuse within the last 12 months, or unable or unwilling to limit alcohol consumption throughout the study
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Placebo Comparator: Placebo
Lumasiran-matching placebo (normal saline [0.9% NaCl]) was administered subcutaneously (SC) at Day 1 and Months 1, 2 and 3 during the 6-Month Double-blind (DB) Period, followed by lumasiran SC, 3.0 mg/kg, at Months 6, 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month Open-label Extension (OLE) period.
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Placebo by SC injection
Lumasiran by SC injection
Other Names:
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Experimental: Lumasiran
Lumasiran was administered SC, 3.0 mg/kg, at Day 1 and Months 1, 2 and 3 during the 6-Month DB Period, followed by lumasiran SC, 3.0 mg/kg at Month 6, and lumasiran-matching placebo SC at Months 7 and 8 during the 3-Month Blinded Treatment Extension Period, followed by lumasiran SC, 3.0 mg/kg, at Month 9 and then every three months during the 51-Month OLE period.
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Placebo by SC injection
Lumasiran by SC injection
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6
Time Frame: Baseline to Month 6
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Percent change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average percent change from baseline across Months 3 through 6.
Only valid urine samples without any non-protocol-related issues were included in the analysis.
A negative change from Baseline indicates a favorable outcome.
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Baseline to Month 6
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Absolute Change in 24-hour Urinary Oxalate Corrected for BSA From Baseline to Month 6
Time Frame: Baseline to Month 6
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Absolute change in 24-hour urinary oxalate excretion corrected for BSA was estimated by an average absolute change from baseline across Months 3 through 6.
Only valid urine samples without any non-protocol-related issues were included in the analysis.
A negative change from Baseline indicates a favorable outcome.
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Baseline to Month 6
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Percent Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
Time Frame: Baseline to Month 6
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Percent change in 24-hour urinary oxalate:creatine ratio was estimated by an average percent change from baseline across Months 3 through 6.
A negative change from Baseline indicates a favorable outcome.
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Baseline to Month 6
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Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below 1.5 x ULN at Month 6
Time Frame: Month 6
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The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m^2
for 24-hour urinary oxalate excretion corrected for BSA.
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Month 6
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Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below ULN at Month 6
Time Frame: Month 6
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The upper limit of normal (ULN) = 0.514 mmol/24hr/1.73m^2
for 24-hour urinary oxalate excretion corrected for BSA.
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Month 6
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Percentage Change in Plasma Oxalate From Baseline to Month 6
Time Frame: Baseline to Month 6
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Percent change in plasma oxalate (umol/L) was estimated by an average percent change from baseline across Months 3 through 6.
A negative change from Baseline indicates a favorable outcome.
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Baseline to Month 6
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Absolute Change in Plasma Oxalate From Baseline to Month 6
Time Frame: Baseline to Month 6
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Absolute change in plasma oxalate (umol/L) was estimated by an average percent change from baseline across Months 3 through 6.
A negative change from Baseline indicates a favorable outcome.
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Baseline to Month 6
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Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Week 2 and Months 1, 2, 3, 4, 5 and 6
Time Frame: Baseline, Week 2, Months 1, 2, 3, 4, 5 and 6
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eGFR is calculated from serum creatinine based on the Modification of Diet in Renal Disease formula for patients ≥18 years of age and the Schwartz Bedside Formula for patients <18 years of age at screening.
Change from baseline to Month 6 is reported.
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Baseline, Week 2, Months 1, 2, 3, 4, 5 and 6
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Absolute Change in 24-hour Urinary Oxalate Excretion From Baseline Over Time During the Extension Period
Time Frame: Up to 60 months
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Up to 60 months
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Percentage Change in 24-hour Urinary Oxalate Excretion From Baseline Over Time During the Extension Period
Time Frame: Up to 60 months
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Up to 60 months
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Percentage of Time That 24-hour Urinary Oxalate is at or Below 1.5 × ULN During the Extension Period
Time Frame: Up to 60 months
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Up to 60 months
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Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline Over Time During the Extension Period
Time Frame: Up to 60 months
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Up to 60 months
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Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline Over Time During the Extension Period
Time Frame: Up to 60 months
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Up to 60 months
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Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Rate of Renal Stone Events
Time Frame: 12-Month Period prior to Informed Consent, 6-Month DB Period
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A renal stone event is defined as a patient-reported event that includes ≥1 of the following: visit to healthcare provider because of a renal stone; medication for renal colic; stone passage; macroscopic hematuria due to a renal stone.
Lower rates indicate a favorable outcome.
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12-Month Period prior to Informed Consent, 6-Month DB Period
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Change From Baseline in Nephrocalcinosis as Assessed by Renal Ultrasound
Time Frame: Baseline, Month 6
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Renal ultrasound data were used to grade medullary nephrocalcinosis findings (range: 0 to 3), where a higher grade indicates greater severity.
Improving=if both sides improve, or one side improves and the other side has no change; No change=if both sides have no change; Worsening=if both sides worsen, or one side worsens and the other side has no change; Indeterminate=if one side improves and the other side worsens.
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Baseline, Month 6
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Medical Director, Alnylam Pharmaceuticals
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
November 27, 2018
Primary Completion (Actual)
November 5, 2019
Study Completion (Actual)
January 12, 2024
Study Registration Dates
First Submitted
September 19, 2018
First Submitted That Met QC Criteria
September 19, 2018
First Posted (Actual)
September 21, 2018
Study Record Updates
Last Update Posted (Actual)
February 16, 2024
Last Update Submitted That Met QC Criteria
February 13, 2024
Last Verified
February 1, 2024
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Metabolic Diseases
- Kidney Diseases
- Urologic Diseases
- Genetic Diseases, Inborn
- Carbohydrate Metabolism, Inborn Errors
- Metabolism, Inborn Errors
- Hyperoxaluria
- Female Urogenital Diseases
- Female Urogenital Diseases and Pregnancy Complications
- Urogenital Diseases
- Male Urogenital Diseases
- Hyperoxaluria, Primary
- Renal Agents
- Lumasiran
Other Study ID Numbers
- ALN-GO1-003
- 2018-001981-40 (EudraCT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Primary Hyperoxaluria Type 1 (PH1)
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Dicerna Pharmaceuticals, Inc.CompletedKidney Diseases | Urologic Diseases | Genetic Disease | Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 2 (PH2)Poland, United Kingdom, New Zealand, United States, Australia, Canada, France, Germany, Israel, Italy, Japan, Lebanon, Netherlands, Romania, Spain
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Alnylam PharmaceuticalsCompletedPrimary Hyperoxaluria Type 1 (PH1)France, United Kingdom, Netherlands, Israel, Germany
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Alnylam PharmaceuticalsActive, not recruitingPrimary Hyperoxaluria Type 1 (PH1) | Primary HyperoxaluriaUnited States, France, United Kingdom, Israel, Germany
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyAvailablePrimary Hyperoxaluria Type 1 (PH1)
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk companyEnrolling by invitationKidney Diseases | Urologic Diseases | Genetic Disease | Primary Hyperoxaluria Type 1 (PH1) | Primary Hyperoxaluria Type 2 (PH2) | Primary Hyperoxaluria Type 3 (PH3)United States, France, Germany, Japan, Lebanon, Spain, United Kingdom, Australia, Canada, Italy, Netherlands, Norway, Turkey
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Hospices Civils de LyonRecruitingPatients With PH1 Treated With Lumasiran in FranceFrance
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Ann & Robert H Lurie Children's Hospital of ChicagoMayo Clinic; National Institute of Diabetes and Digestive and Kidney Diseases...CompletedPrimary Hyperoxaluria Type 1
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Dicerna Pharmaceuticals, Inc.TerminatedPrimary Hyperoxaluria Type 1Netherlands, Germany
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Hospices Civils de LyonCompletedIdiopathic Hypercalciuria | Primary Hyperoxaluria Type 1France
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