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 andpediatric patients with Primary Hyperoxaluria Type 1 (PH1),

Detailed Description

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study. To learn more about this study, please have your doctor contact the study research
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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
Eligibility Age
Minimum: N/A ~ Maximum: N/A
Countries
Belgium
Netherlands

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
Lumasiran