A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 (ILLUMINATE-B)
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ClinicalTrials.gov Identifier: NCT03905694 |
Recruitment Status :
Active, not recruiting
First Posted : April 5, 2019
Results First Posted : July 19, 2021
Last Update Posted : January 18, 2023
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Primary Hyperoxaluria Primary Hyperoxaluria Type 1 (PH1) | Drug: Lumasiran | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 18 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 |
Actual Study Start Date : | April 22, 2019 |
Actual Primary Completion Date : | June 29, 2020 |
Estimated Study Completion Date : | August 19, 2024 |

Arm | Intervention/treatment |
---|---|
Experimental: Lumasiran
Lumasiran will be administered by subcutaneous (SC) injection.
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Drug: Lumasiran
Lumasiran will be administered by subcutaneous (SC) injection.
Other Name: ALN-GO1 |
- Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 [ Time Frame: Baseline to Month 6 ]Percent change in spot urinary oxalate:creatinine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome.
- Percentage Change in Urinary Oxalate Excretion From Baseline to End of Study (Month 60) [ Time Frame: Up to 60 months ]
- Absolute Change in Urinary Oxalate Excretion From Baseline [ Time Frame: Up to 60 months ]
- Percentage of Time That Spot Urinary Oxalate:Creatinine Ratio ≤ Near-normalization Threshold (≤1.5 × ULN) [ Time Frame: Up to 60 months ]
- Percentage of Participants With Urinary Oxalate Excretion ≤ the Upper Limit of Normal (ULN) and ≤ 1.5 x ULN [ Time Frame: Up to 60 months ]
- Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60) [ Time Frame: Up to 60 months ]
- Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60) [ Time Frame: Up to 60 months ]
- Maximum Observed Plasma Concentration (Cmax) of Lumasiran [ Time Frame: Up to 24 months ]
- Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran [ Time Frame: Up to 24 months ]
- Elimination Half-life (t1/2beta) of Lumasiran [ Time Frame: Up to 24 months ]
- Area Under the Concentration-time Curve (AUC) of Lumasiran [ Time Frame: Up to 24 months ]
- Apparent Clearance (CL/F) of Lumasiran [ Time Frame: Up to 24 months ]
- Apparent Volume of Distribution (V/F) of Lumasiran [ Time Frame: Up to 24 months ]
- Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline [ Time Frame: Up to 60 months ]
- Frequency of Adverse Events (AEs) [ Time Frame: Up to 60 months ]

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Ages Eligible for Study: | 0 Years to 5 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Has genetic confirmation of primary hyperoxaluria type 1 (PH1)
- Meets urinary oxalate excretion requirements
- If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days
Exclusion Criteria:
- If <12 months old at screening, has an abnormally high serum creatinine
- If ≥12 months old at screening, has an estimated glomerular filtration rate (GFR) of ≤45 mL/min/1.73m^2
- Clinical evidence of systemic oxalosis
- History of kidney or liver transplant

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03905694
United States, Minnesota | |
Clinical Trial Site | |
Rochester, Minnesota, United States, 55905 | |
United States, Texas | |
Clinical Trial Site | |
Houston, Texas, United States, 77030 | |
France | |
Clinical Trial Site | |
Lyon, France | |
Clinical Trial Site | |
Paris, France | |
Germany | |
Clinical Trial Site | |
Bonn, Germany | |
Israel | |
Clinical Trial Site | |
Haifa, Israel | |
Clinical Trial Site | |
Jerusalem, Israel | |
Clinical Trial Site | |
Nahariya, Israel | |
United Kingdom | |
Clinical Trial Site | |
London, United Kingdom |
Study Director: | Medical Director | Alnylam Pharmaceuticals |
Documents provided by Alnylam Pharmaceuticals:
Responsible Party: | Alnylam Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT03905694 |
Other Study ID Numbers: |
ALN-GO1-004 2018-004014-17 ( EudraCT Number ) |
First Posted: | April 5, 2019 Key Record Dates |
Results First Posted: | July 19, 2021 |
Last Update Posted: | January 18, 2023 |
Last Verified: | January 2023 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
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