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
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 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
Countries
Belgium
France
French Polynesia
United States
Locations

Program Site
Pittsburgh, Pennsylvania, 15224

Available

Contacts

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

Tom Brown, PhD
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