An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1

Alnylam Pharmaceuticals20 enrolled

Overview

The purpose of this study is to evaluate the long-term safety and tolerability of lumasiran in participants with Primary Hyperoxaluria Type 1.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

PH1 Primary Hyperoxaluria RNAi Therapeutic siRNA AGT

Interventions

LumasiranDRUG

Multiple doses of lumasiran by SC injection

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Enrollment within 12 months of completion of Study ALN-GO1-001 * In the opinion of the investigator tolerated the study drug * If taking Vitamin B6 (pyridoxine), willing to remain on a stable regimen for the study duration * Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception * Willing to provide written informed consent and to comply with study requirements Exclusion Criteria: * Clinically significant health concerns (with the exception of PH1) * Clinically significant cardiovascular abnormality * Abnormal for AST/ALT and any other clinical safety laboratory result considered clinically significant * Requirement for chronic dialysis

Locations (9)

Clinical Trial Site

Bordeaux, France

Clinical Trial Site

Lyon, France

Clinical Trial Site

Paris, France

Clinical Trial Site

Bonn, Germany

Outcomes

Primary Outcomes

Number of Participants With at Least One Adverse Event (AE)

AE is any untoward medical occurrence in a participant or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment. Safety analysis set included all participants who received any amount of study drug.

Time frame: Baseline (Day -1) up to 54 months

Secondary Outcomes

Change From Baseline in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) at 54 Months

Oxalate produced by the liver is the key toxic metabolite that drives disease pathology in participants with primary hyperoxaluria type 1 (PH1). The risk of disease complications increase continuously as oxalate levels increase. 24-hour urinary oxalate (millimole \[mmol\]/ 24 hour \[h\]/1.73 meters squared \[m\^2\]) corrected for BSA at each visit per participant was calculated as follows: \[Urine oxalate concentration (micromole per liter \[umol/L\])/1000 (umol/mmol)\]\*\[24hour urine volume (mL)/1000 (mL/L)\]\* \[24 hours/actual collection hours\]\*1.73/(BSA). Baseline was the derived baseline value from the lumasiran treated period of Study ALN-GO1-001. A negative change from baseline indicated a favorable outcome. PD analysis set included all participants who received any amount of study drug and who had at least 1 post-dose urine sample for PD. Overall number of participants analyzed are the number of participants with data available for analysis.

Time frame: Baseline (Day -1) up to 54 months

Change From Baseline in 24-hour Urinary Oxalate:Creatinine Ratio at 54 Months

Baseline is the derived baseline value from the lumasiran treated period of Study ALN-GO1-001. A negative change from baseline indicates a favorable outcome. PD analysis set included all participants who received any amount of study drug and who had at least 1 post-dose urine sample for PD.

Time frame: Baseline (Day -1) up to 54 months

Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at 54 Months

Data from ClinicalTrials.gov

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