Back to resultsExpanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1
Primary Purpose
Primary Hyperoxaluria
Status
Approved for marketing
Phase
Locations
Belgium
Study Type
Expanded Access
Intervention
Lumasiran
Sponsored by
About this trial
This is an expanded access trial for Primary Hyperoxaluria focused on measuring Hyperoxaluria, Primary Hyperoxaluria, Kidney Diseases, Urologic Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Metabolic Diseases, RNA Therapeutic, siRNA
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
Sites / Locations
- Program Site
- Program Site
- Program Site
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT04125472
First Posted
October 10, 2019
Last Updated
June 16, 2023
Sponsor
Alnylam Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT04125472
Brief Title
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
Study Type
Expanded Access
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Approved for marketing
Study Start Date
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Primary Completion Date
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Study Completion Date
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3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alnylam Pharmaceuticals
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Hyperoxaluria
Keywords
Hyperoxaluria, Primary Hyperoxaluria, Kidney Diseases, Urologic Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Metabolic Diseases, RNA Therapeutic, siRNA
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Lumasiran
Other Intervention Name(s)
ALN-GO1
Intervention Description
Lumasiran administered as a subcutaneous (SC) injection
10. Eligibility
Sex
All
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Alnylam Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Program Site
City
Brussels
Country
Belgium
Facility Name
Program Site
City
Ghent
Country
Belgium
Facility Name
Program Site
City
Liège
Country
Belgium
12. IPD Sharing Statement
Links:
URL
https://www.alnylam.com/medical-professional-resources/early-access-program/lumasiran/
Description
Information on the Early Access Program for Lumasiran
Learn more about this trial
Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1
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