search
Back to results

Expanded 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
Alnylam Pharmaceuticals
About
Eligibility
Locations
Outcomes
Full info

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

undefined - undefined (Child, Adult, Older Adult)All Sexes

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

First Posted
October 10, 2019
Last Updated
June 16, 2023
Sponsor
Alnylam Pharmaceuticals
search

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
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

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

We'll reach out to this number within 24 hrs