search
Back to results

An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Patients With Primary Hyperoxaluria Type 1

Primary Purpose

PH1, Primary Hyperoxaluria, RNAi Therapeutic

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Lumasiran
Sponsored by
Alnylam Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for PH1 focused on measuring Hyperoxaluria, Primary, Hyperoxaluria, Kidney Diseases, Urologic Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Metabolic Diseases

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Enrollment within 12 months of completion of Study ALN-GO1-001
  • In the opinion of the investigator tolerated the study drug
  • If taking Vitamin B6 (pyridoxine), willing to remain on a stable regimen for the study duration
  • Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception
  • Willing to provide written informed consent and to comply with study requirements

Exclusion Criteria:

  • Clinically significant health concerns (with the exception of PH1)
  • Clinically significant cardiovascular abnormality
  • Abnormal for AST/ALT and any other clinical safety laboratory result considered clinically significant
  • Requirement for chronic dialysis

Sites / Locations

  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Lumasiran (ALN-GO1)

Arm Description

Outcomes

Primary Outcome Measures

Incidence of Adverse Events

Secondary Outcome Measures

Change in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) Over Time
Change in 24-hour Urinary Oxalate:Creatinine Ratio Over Time
Change in Estimated Glomerular Filtration Rate (eGFR) Over Time

Full Information

First Posted
November 17, 2017
Last Updated
March 18, 2023
Sponsor
Alnylam Pharmaceuticals
search

1. Study Identification

Unique Protocol Identification Number
NCT03350451
Brief Title
An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Patients With Primary Hyperoxaluria Type 1
Official Title
A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients With Primary Hyperoxaluria Type 1
Study Type
Interventional

2. Study Status

Record Verification Date
January 2023
Overall Recruitment Status
Completed
Study Start Date
April 4, 2018 (Actual)
Primary Completion Date
February 7, 2023 (Actual)
Study Completion Date
February 7, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alnylam Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
The purpose of this study is to evaluate the long-term safety and tolerability of lumasiran in patients with Primary Hyperoxaluria Type 1.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
PH1, Primary Hyperoxaluria, RNAi Therapeutic, siRNA, AGT
Keywords
Hyperoxaluria, Primary, Hyperoxaluria, Kidney Diseases, Urologic Diseases, Metabolism, Inborn Errors, Genetic Diseases, Inborn, Metabolic Diseases

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Lumasiran (ALN-GO1)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Lumasiran
Other Intervention Name(s)
ALN-GO1
Intervention Description
Multiple doses of lumasiran by subcutaneous (sc) injection
Primary Outcome Measure Information:
Title
Incidence of Adverse Events
Time Frame
Up to approximately 1600 days
Secondary Outcome Measure Information:
Title
Change in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) Over Time
Time Frame
Up to approximately 1600 days
Title
Change in 24-hour Urinary Oxalate:Creatinine Ratio Over Time
Time Frame
Up to approximately 1600 days
Title
Change in Estimated Glomerular Filtration Rate (eGFR) Over Time
Time Frame
Up to approximately 1600 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Enrollment within 12 months of completion of Study ALN-GO1-001 In the opinion of the investigator tolerated the study drug If taking Vitamin B6 (pyridoxine), willing to remain on a stable regimen for the study duration Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception Willing to provide written informed consent and to comply with study requirements Exclusion Criteria: Clinically significant health concerns (with the exception of PH1) Clinically significant cardiovascular abnormality Abnormal for AST/ALT and any other clinical safety laboratory result considered clinically significant Requirement for chronic dialysis
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
Clinical Trial Site
City
Bordeaux
Country
France
Facility Name
Clinical Trial Site
City
Lyon
Country
France
Facility Name
Clinical Trial Site
City
Paris
Country
France
Facility Name
Clinical Trial Site
City
Bonn
Country
Germany
Facility Name
Clinical Trial Site
City
Haifa
Country
Israel
Facility Name
Clinical Trial Site
City
Jerusalem
Country
Israel
Facility Name
Clinical Trial Site
City
Amsterdam
Country
Netherlands
Facility Name
Clinical Trial Site
City
Birmingham
Country
United Kingdom
Facility Name
Clinical Trial Site
City
London
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Patients With Primary Hyperoxaluria Type 1

We'll reach out to this number within 24 hrs