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

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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.

Status
Approved for marketing
EA Type
Individual Patients
Treatment IND/Protocol
Conditions
Primary Hyperoxaluria
Interventions

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
Investigators

Medical Director, Study Director
Alnylam Pharmaceuticals

Sponsors / Collaborators
Alnylam Pharmaceuticals
NCT Number
NCT04125472
Keywords
Hyperoxaluria
Primary Hyperoxaluria
Kidney Diseases
Urologic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
RNA Therapeutic
siRNA
MeSH Terms
Hyperoxaluria, Primary
Lumasiran