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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 : July 15, 2021
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE November 17, 2017
First Posted Date  ICMJE November 22, 2017
Last Update Posted Date July 15, 2021
Actual Study Start Date  ICMJE April 4, 2018
Estimated Primary Completion Date June 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 27, 2020)
Incidence of Adverse Events [ Time Frame: Up to approximately 1600 days ]
Original Primary Outcome Measures  ICMJE
 (submitted: November 20, 2017)
Proportion of subjects experiencing adverse events [ Time Frame: Up to 850 days ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 27, 2020)
  • Change in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) Over Time [ Time Frame: Up to approximately 1600 days ]
  • Change in 24-hour Urinary Oxalate:Creatinine Ratio Over Time [ Time Frame: Up to approximately 1600 days ]
  • Change in Estimated Glomerular Filtration Rate (eGFR) Over Time [ Time Frame: Up to approximately 1600 days ]
Original Secondary Outcome Measures  ICMJE
 (submitted: November 20, 2017)
  • Change in urinary oxalate excretion over time [ Time Frame: Up to 834 days ]
  • Change in Estimated Glomerular Filtration Rate (eGFR) Over Time [ Time Frame: through Day 834 ]
  • Change in measured creatinine clearance (mCrCl) over time [ Time Frame: through Day 834 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Patients With Primary Hyperoxaluria Type 1
Official Title  ICMJE A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients With Primary Hyperoxaluria Type 1
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • PH1
  • Primary Hyperoxaluria
  • RNAi Therapeutic
  • siRNA
  • AGT
Intervention  ICMJE Drug: Lumasiran
Multiple doses of lumasiran by subcutaneous (sc) injection
Other Name: ALN-GO1
Study Arms  ICMJE Experimental: Lumasiran (ALN-GO1)
Intervention: Drug: Lumasiran
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: January 8, 2019)
20
Original Estimated Enrollment  ICMJE
 (submitted: November 20, 2017)
24
Estimated Study Completion Date  ICMJE June 2023
Estimated Primary Completion Date June 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France,   Germany,   Israel,   Netherlands,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03350451
Other Study ID Numbers  ICMJE ALN-GO1-002
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Alnylam Pharmaceuticals
Study Sponsor  ICMJE Alnylam Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director Alnylam Pharmaceuticals
PRS Account Alnylam Pharmaceuticals
Verification Date July 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP