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A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 (ILLUMINATE-B)

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: NCT03905694
Recruitment Status : Active, not recruiting
First Posted : April 5, 2019
Last Update Posted : February 5, 2021
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE April 1, 2019
First Posted Date  ICMJE April 5, 2019
Last Update Posted Date February 5, 2021
Actual Study Start Date  ICMJE April 22, 2019
Actual Primary Completion Date June 29, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 4, 2019)
Percentage Change in Urinary Oxalate Excretion from Baseline to Month 6 [ Time Frame: Up to 6 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 7, 2019)
  • Percentage Change in Urinary Oxalate Excretion from Baseline to End of Study (Month 60) [ Time Frame: Up to 60 months ]
  • Absolute Change in Urinary Oxalate Excretion from Baseline [ Time Frame: Up to 60 months ]
  • Percentage of Time that Spot Urinary Oxalate:Creatinine Ratio ≤ Near-normalization Threshold (≤1.5 × ULN) [ Time Frame: Up to 60 months ]
  • Percentage of Participants with Urinary Oxalate Excretion ≤ the Upper Limit of Normal (ULN) and ≤ 1.5 x ULN [ Time Frame: Up to 60 months ]
  • Percentage Change in Plasma Oxalate from Baseline to End of Study (Month 60) [ Time Frame: Up to 60 months ]
  • Absolute Change in Plasma Oxalate from Baseline to End of Study (Month 60) [ Time Frame: Up to 60 months ]
  • Maximum Observed Plasma Concentration (Cmax) of Lumasiran [ Time Frame: Up to 24 months ]
  • Time to Maximum Observed Plasma Concentration (tmax) of Lumasiran [ Time Frame: Up to 24 months ]
  • Elimination Half-life (t1/2beta) of Lumasiran [ Time Frame: Up to 24 months ]
  • Area Under the Concentration-time Curve (AUC) of Lumasiran [ Time Frame: Up to 24 months ]
  • Apparent Clearance (CL/F) of Lumasiran [ Time Frame: Up to 24 months ]
  • Apparent Volume of Distribution (V/F) of Lumasiran [ Time Frame: Up to 24 months ]
  • Change in Estimated Glomerular Filtration Rate (eGFR) from Baseline [ Time Frame: Up to 60 months ]
  • Frequency of Adverse Events (AEs) [ Time Frame: Up to 60 months ]
Original Secondary Outcome Measures  ICMJE
 (submitted: April 4, 2019)
  • Percentage Change in Urinary Oxalate Excretion from Baseline to End of Study (Month 60) [ Time Frame: Up to 60 months ]
  • Absolute Change in Urinary Oxalate Excretion from Baseline [ Time Frame: Up to 60 months ]
  • Percentage of Participants with Urinary Oxalate Excretion ≤ the Upper Limit of Normal (ULN) and ≤ 1.5 x ULN [ Time Frame: Up to 60 months ]
  • Maximum Observed Plasma Concentration (Cmax) of Lumasiran [ Time Frame: Up to 24 months ]
  • Time to Maximum Observed Plasma Concentration (tmax) of Lumasiran [ Time Frame: Up to 24 months ]
  • Elimination Half-life (t1/2beta) of Lumasiran [ Time Frame: Up to 24 months ]
  • Area Under the Concentration-time Curve (AUC) of Lumasiran [ Time Frame: Up to 24 months ]
  • Apparent Clearance (CL/F) of Lumasiran [ Time Frame: Up to 24 months ]
  • Apparent Volume of Distribution (V/F) of Lumasiran [ Time Frame: Up to 24 months ]
  • Change in Estimated Glomerular Filtration Rate (eGFR) from Baseline [ Time Frame: Up to 60 months ]
  • Frequency of Adverse Events (AEs) [ Time Frame: Up to 60 months ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
Official Title  ICMJE ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
Brief Summary The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Primary Hyperoxaluria
  • Primary Hyperoxaluria Type 1 (PH1)
Intervention  ICMJE Drug: Lumasiran
Lumasiran will be administered by subcutaneous (SC) injection.
Other Name: ALN-GO1
Study Arms  ICMJE Experimental: Lumasiran
Participants will receive lumasiran during the study.
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: February 14, 2020)
18
Original Estimated Enrollment  ICMJE
 (submitted: April 4, 2019)
8
Estimated Study Completion Date  ICMJE August 19, 2024
Actual Primary Completion Date June 29, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Has confirmation of primary hyperoxaluria type 1 (PH1)
  • Meets urinary oxalate excretion requirements
  • If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days

Exclusion Criteria:

  • Abnormal serum creatinine levels at screening for infants who are less than 1 year old
  • Does not have relatively preserved kidney function
  • Clinical evidence of systemic oxalosis
  • History of kidney or liver transplant
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 5 Years   (Child)
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,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03905694
Other Study ID Numbers  ICMJE ALN-GO1-004
2018-004014-17 ( EudraCT Number )
Has Data Monitoring Committee Not Provided
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 February 2021

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