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Trial record 1 of 6 for:    ALN-GO1
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An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Patients With Primary Hyperoxaluria Type 1

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03350451
Recruitment Status : Active, not recruiting
First Posted : November 22, 2017
Last Update Posted : May 5, 2020
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
PH1 Primary Hyperoxaluria RNAi Therapeutic siRNA AGT Drug: Lumasiran Phase 2

Expanded Access : An investigational treatment associated with this study is available outside the clinical trial.   More info ...

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : November 20, 2017
Estimated Primary Completion Date : June 2023
Estimated Study Completion Date : June 2023

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Lumasiran (ALN-GO1) Drug: Lumasiran
Multiple doses of lumasiran by subcutaneous (sc) injection
Other Name: ALN-GO1




Primary Outcome Measures :
  1. Incidence of Adverse Events [ Time Frame: Up to approximately 1600 days ]

Secondary Outcome Measures :
  1. Change in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) Over Time [ Time Frame: Up to approximately 1600 days ]
  2. Change in 24-hour Urinary Oxalate:Creatinine Ratio Over Time [ Time Frame: Up to approximately 1600 days ]
  3. Change in Estimated Glomerular Filtration Rate (eGFR) Over Time [ Time Frame: Up to approximately 1600 days ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03350451


Locations
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France
Clinical Trial Site
Bordeaux, France
Clinical Trial Site
Lyon, France
Clinical Trial Site
Paris, France
Germany
Clinical Trial Site
Bonn, Germany
Israel
Clinical Trial Site
Haifa, Israel
Clinical Trial Site
Jerusalem, Israel
Netherlands
Clinical Trial Site
Amsterdam, Netherlands
United Kingdom
Clinical Trial Site
Birmingham, United Kingdom
Clinical Trial Site
London, United Kingdom
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Tracy McGregor, MD, MSCI Alnylam Pharmaceuticals
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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03350451    
Other Study ID Numbers: ALN-GO1-002
First Posted: November 22, 2017    Key Record Dates
Last Update Posted: May 5, 2020
Last Verified: May 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alnylam Pharmaceuticals:
Hyperoxaluria, Primary
Hyperoxaluria
Kidney Diseases
Urologic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Additional relevant MeSH terms:
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Hyperoxaluria, Primary
Hyperoxaluria
Kidney Diseases
Urologic Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases