Phase 3
Completed N=39
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
Primary Hyperoxaluria Type 1 (PH1)
Source: ClinicalTrials.gov NCT03681184 ↗
Enrolled (actual)
39
Serious AEs
11.5%
Results posted
Jan 2021
Primary outcomePrimary: Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6 — -11.8; -65.4 percent change — p=<0.0001
◆ Published Evidence
Highly cited
503citations · ~101 / year
Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1.
Summary
The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).
Linked Publications (2)
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Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1.
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Final Results of the ILLUMINATE-A Phase 3 Clinical Trial of Lumasiran for Primary Hyperoxaluria 1.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6 |
-11.8; -65.4 | <0.0001 sig |
| SECONDARY Absolute Change in 24-hour Urinary Oxalate Corrected for BSA From Baseline to Month 6 |
-0.27; -1.24 | <0.0001 sig |
| SECONDARY Percent Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 |
-10.8; -62.5 | <0.0001 sig |
| SECONDARY Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below 1.5 x ULN at Month 6 |
0; 84.0 | <0.0001 sig |
| SECONDARY Percentage of Participants With 24-hour Urinary Oxalate Level Corrected for BSA at or Below ULN at Month 6 |
0; 52.0 | 0.0010 sig |
| SECONDARY Percentage Change in Plasma Oxalate From Baseline to Month 6 |
-0.3; -39.8 | <0.0001 sig |
| SECONDARY Absolute Change in Plasma Oxalate From Baseline to Month 6 |
1.3; -7.5 | <0.0001 sig |
| SECONDARY Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline to Week 2 and Months 1, 2, 3, 4, 5 and 6 |
-5; -4; -6; -2; -5; -2 | — |
| SECONDARY Absolute Change in 24-hour Urinary Oxalate Excretion Corrected for BSA From Baseline in the Extension Period |
-0.951; -1.086; -1.129 | — |
| SECONDARY Percentage Change in 24-hour Urinary Oxalate Excretion Corrected by BSA From Baseline in the Extension Period |
-55.57; -53.87; -53.98 | — |
| SECONDARY Percentage of Time That 24-hour Urinary Oxalate is at or Below 1.5 × ULN During Lumasiran Treatment |
89.44; 89.23 | — |
| SECONDARY Absolute Change in 24-hour Urinary Oxalate:Creatinine Ratio From Baseline in the Extension Period |
-0.145; -0.127; -0.138 | — |
| SECONDARY Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline in the Extension Period |
-12.860; -6.899; -2.892 | — |
| SECONDARY Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
9; 12; 25; 0; 1; 5 | — |
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT03681184) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.