Official Title
Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1
Brief Summary

The purpose of this study is to provide expanded access to lumasiran for adults and pediatric patients with Primary Hyperoxaluria Type 1 (PH1),

Detailed Description

Choosing to participate in an expanded access program is an important personal decision. Talk

with your doctor and family members or friends about deciding to join a research study. To

learn more about this study, please have your doctor contact the study research staff using

the Contacts provided. For general information, see the link provided in More Information.

Approved for marketing
Individual Patients
Treatment IND/Protocol
Primary Hyperoxaluria

Drug: Lumasiran
Lumasiran administered as a subcutaneous (SC) injection
Other Name: ALN-GO1

Eligibility Criteria

Inclusion Criteria: - Documented diagnosis of Primary Hyperoxaluria Type 1

Exclusion Criteria: - Clinically significant health concerns (with the exception of PH1) - Received an investigational agent within 30 days before the first dose of lumasiran or are in follow-up of another clinical study - Previously or currently participating in lumasiran clinical study - History of liver transplant

Eligibility Gender
All
Countries
Belgium
Italy
Netherlands
Spain

Medical Director
Study Director
Alnylam Pharmaceuticals

Alnylam Pharmaceuticals
NCT Number
Keywords
Hyperoxaluria
Primary Hyperoxaluria
Kidney Diseases
Urologic Diseases
Metabolism
Inborn Errors
Genetic Diseases
Inborn
Metabolic Diseases
RNA Therapeutic
siRNA
MeSH Terms
Hyperoxaluria, Primary
Intervention Name
Lumasiran