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

March 18, 2023 updated by: Alnylam Pharmaceuticals

A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients With Primary Hyperoxaluria Type 1

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

20

Phase

  • Phase 2

Expanded Access

Approved for sale to the public. See expanded access record.

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Bordeaux, France
        • Clinical Trial Site
      • Lyon, France
        • Clinical Trial Site
      • Paris, France
        • Clinical Trial Site
  • Germany
      • Bonn, Germany
        • Clinical Trial Site
  • Israel
      • Haifa, Israel
        • Clinical Trial Site
      • Jerusalem, Israel
        • Clinical Trial Site
  • Netherlands
      • Amsterdam, Netherlands
        • Clinical Trial Site
  • United Kingdom
      • Birmingham, United Kingdom
        • Clinical Trial Site
      • London, United Kingdom
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Lumasiran (ALN-GO1)
Drug: Lumasiran
Multiple doses of lumasiran by subcutaneous (sc) injection
Other Names:
  • ALN-GO1

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of Adverse Events
Time Frame: Up to approximately 1600 days
Up to approximately 1600 days

Secondary Outcome Measures

Outcome Measure
Time Frame
Change in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) Over Time
Time Frame: Up to approximately 1600 days
Up to approximately 1600 days
Change in 24-hour Urinary Oxalate:Creatinine Ratio Over Time
Time Frame: Up to approximately 1600 days
Up to approximately 1600 days
Change in Estimated Glomerular Filtration Rate (eGFR) Over Time
Time Frame: Up to approximately 1600 days
Up to approximately 1600 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 4, 2018

Primary Completion (Actual)

February 7, 2023

Study Completion (Actual)

February 7, 2023

Study Registration Dates

First Submitted

November 17, 2017

First Submitted That Met QC Criteria

November 20, 2017

First Posted (Actual)

November 22, 2017

Study Record Updates

Last Update Posted (Actual)

March 21, 2023

Last Update Submitted That Met QC Criteria

March 18, 2023

Last Verified

January 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-GO1-002

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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