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

• Register: ClinicalTrials.gov
• Last refreshed on: 27 March 2023
• Main ID: NCT03350451
• Date of registration: 17/11/2017
• Prospective Registration: Yes
• Primary sponsor: Alnylam Pharmaceuticals
• Public title: An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Patients With Primary Hyperoxaluria Type 1
• Scientific title: A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients With Primary Hyperoxaluria Type 1
• Date of first enrolment: April 4, 2018
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03350451
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

France, Germany, Israel, Netherlands, United Kingdom

Contacts

• Name: Medical Director
• Affiliation: Alnylam Pharmaceuticals

Key inclusion & exclusion criteria

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



Age minimum: 6 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

AGT

siRNA

RNAi Therapeutic

PH1

Primary Hyperoxaluria

Intervention(s)

Drug: Lumasiran

Primary Outcome(s)

Incidence of Adverse Events [Time Frame: Up to approximately 1600 days]

Secondary Outcome(s)

Change in 24-hour Urinary Oxalate:Creatinine Ratio Over Time [Time Frame: Up to approximately 1600 days]

Change in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) Over Time [Time Frame: Up to approximately 1600 days]

Change in Estimated Glomerular Filtration Rate (eGFR) Over Time [Time Frame: Up to approximately 1600 days]

Secondary ID(s)

ALN-GO1-002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors