Phase 3 N=39 Randomized Quadruple-blind Treatment

A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1

Primary Hyperoxaluria Type 1 (PH1)

Bottom Line

View on ClinicalTrials.gov: NCT03681184 ↗

Enrolled (actual)

Serious AEs

11.5%

Results posted

Jan 2021

Primary outcome: Primary: 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

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Placebo (Drug); Lumasiran (Drug)
Age: Pediatric, Adult, Older Adult · 6+ yrs
Sex: All
Sponsor: Alnylam Pharmaceuticals
Primary completion: Nov 2019

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	—

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).

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03681184). 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.