Alnylam Pharmaceuticals

Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1

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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EA Type
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 Type1

- Meet the urine oxalate excretion requirements

Exclusion Criteria:

- Clinically significant health concerns (with the exception of PH1)

- An estimated GFR of ≤ 45 mL/min/1.73m2 at screening

- Clinically significant abnormal laboratory results at screening

- 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 renal or liver transplant

- Women who are pregnant, planning a pregnancy, or breast-feeding or those of child bearing potential and not willing to use contraception

Eligibility Gender: 
Eligibility Age: 
Minimum: 6 Years

Alnylam EAP Hotline
(617) 715-0200

Tom Brown, PhD
Study Director
Alnylam Pharmaceuticals

Alnylam Pharmaceuticals
EA Number: 
Hyperoxaluria, Primary Hyperoxaluria, Kidney Diseases, Urologic Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Metabolic Diseases, RNA Therapeutic, siRNA
MeSH Terms: 
Hyperoxaluria, Primary
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