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An Extension Study of an Investigational Drug, 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 : Enrolling by invitation
First Posted : November 22, 2017
Last Update Posted : December 12, 2018
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 ALN-GO1 in patients with Primary Hyperoxaluria Type 1.

Condition or disease Intervention/treatment Phase
PH1 Primary Hyperoxaluria RNAi Therapeutic siRNA AGT Drug: ALN-GO1 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
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 : September 2021
Estimated Study Completion Date : September 2021

Resource links provided by the National Library of Medicine


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




Primary Outcome Measures :
  1. Proportion of subjects experiencing adverse events [ Time Frame: Up to 850 days ]

Secondary Outcome Measures :
  1. Change in urinary oxalate excretion over time [ Time Frame: Up to 834 days ]
  2. Change in estimated glomerular filtration rate (eGFR) over time [ Time Frame: through Day 834 ]
  3. Change in measured creatinine clearance (mCrCl) over time [ Time Frame: through Day 834 ]


Information from the National Library of Medicine

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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
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
Study Director: Tracy McGregor, MD, MSCI Alnylam Pharmaceuticals

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03350451     History of Changes
Other Study ID Numbers: ALN-GO1-002
First Posted: November 22, 2017    Key Record Dates
Last Update Posted: December 12, 2018
Last Verified: December 2018

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:
Hyperoxaluria, Primary
Hyperoxaluria
Kidney Diseases
Urologic Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases