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: 

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.

Available
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: 
All
Eligibility Age: 
Minimum: 6 Years
Contacts: 

Alnylam EAP Hotline
(617) 715-0200
eap@alnylam.com

Tom Brown, PhD
Study Director
Alnylam Pharmaceuticals

Alnylam Pharmaceuticals
EA Number: 
Keywords: 
Hyperoxaluria, Primary Hyperoxaluria, Kidney Diseases, Urologic Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Metabolic Diseases, RNA Therapeutic, siRNA
MeSH Terms: 
Hyperoxaluria, Primary
Disclaimer:
Expanded Access program information for companies listed in the Expanded Access Company Directory is pulled daily from ClinicalTrials.gov, a resource provided by the National Institutes of Health in cooperation with the U.S. Food and Drug Administration. Click here for more information about ClinicalTrials.gov.