A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1 (NCT03681184) | Clinical Trial Compass
CompletedPhase 3
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
United States39 participantsStarted 2018-11-27
Plain-language 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).
Who can participate
Age range6 Years
SexALL
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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
What they're measuring
1
Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) From Baseline to Month 6