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

NCT04125472 | Unknown

• 1 other identifier: ALN-GO1-006
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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),

Conditions

Primary Hyperoxaluria

Keywords

Hyperoxaluria; Primary Hyperoxaluria; Kidney Diseases; Urologic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; RNA Therapeutic; siRNA

Interventions

Lumasiran DRUG

Lumasiran administered as a subcutaneous (SC) injection

Also known as: ALN-GO1

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Documented diagnosis of Primary Hyperoxaluria Type 1

You may not qualify if:

• 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

Sponsors & Collaborators

• Alnylam Pharmaceuticals: lead

Related Links

• Information on the Early Access Program for Lumasiran

MeSH Terms

Conditions

Hyperoxaluria, Primary; Hyperoxaluria; Kidney Diseases; Urologic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases

Interventions

lumasiran

Condition Hierarchy (Ancestors)

Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Carbohydrate Metabolism, Inborn Errors; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases

Study Officials

• Medical Director

  Alnylam Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 10, 2019
• First Posted: October 14, 2019
• Last Updated: February 16, 2024

Record last verified: 2024-02