A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients With Primary Hyperoxaluria Type 1

ALN-GO1-002

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

2018-04-04

2023-02-07

2023-02-07

Completed

Early phase I

20

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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 20, 'type': 'ACTUAL'}}

2024-04-25