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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
Results First Posted : July 19, 2021
Last Update Posted : October 18, 2021
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Study Description
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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).

Condition or disease Intervention/treatment Phase
Primary Hyperoxaluria Primary Hyperoxaluria Type 1 (PH1) Drug: Lumasiran Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 18 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : April 22, 2019
Actual Primary Completion Date : June 29, 2020
Estimated Study Completion Date : August 19, 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Lumasiran
Lumasiran will be administered by subcutaneous (SC) injection.
 Drug: Lumasiran
Lumasiran will be administered by subcutaneous (SC) injection.
Other Name: ALN-GO1


Outcome Measures
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Primary Outcome Measures :
  1. Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 [ Time Frame: Baseline to Month 6 ]
    Percent change in spot urinary oxalate:creatinine ratio was estimated by an average percent change from baseline across Months 3 through 6. A negative change from Baseline indicates a favorable outcome.


Secondary Outcome Measures :
  1. Percentage Change in Urinary Oxalate Excretion From Baseline to End of Study (Month 60) [ Time Frame: Up to 60 months ]
  2. Absolute Change in Urinary Oxalate Excretion From Baseline [ Time Frame: Up to 60 months ]
  3. Percentage of Time That Spot Urinary Oxalate:Creatinine Ratio ≤ Near-normalization Threshold (≤1.5 × ULN) [ Time Frame: Up to 60 months ]
  4. Percentage of Participants With Urinary Oxalate Excretion ≤ the Upper Limit of Normal (ULN) and ≤ 1.5 x ULN [ Time Frame: Up to 60 months ]
  5. Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60) [ Time Frame: Up to 60 months ]
  6. Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60) [ Time Frame: Up to 60 months ]
  7. Maximum Observed Plasma Concentration (Cmax) of Lumasiran [ Time Frame: Up to 24 months ]
  8. Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran [ Time Frame: Up to 24 months ]
  9. Elimination Half-life (t1/2beta) of Lumasiran [ Time Frame: Up to 24 months ]
  10. Area Under the Concentration-time Curve (AUC) of Lumasiran [ Time Frame: Up to 24 months ]
  11. Apparent Clearance (CL/F) of Lumasiran [ Time Frame: Up to 24 months ]
  12. Apparent Volume of Distribution (V/F) of Lumasiran [ Time Frame: Up to 24 months ]
  13. Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline [ Time Frame: Up to 60 months ]
  14. Frequency of Adverse Events (AEs) [ Time Frame: Up to 60 months ]

Eligibility Criteria
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Ages Eligible for Study:   up to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Has genetic 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:

  • If <12 months old at screening, has an abnormally high serum creatinine
  • If ≥12 months old at screening, has an estimated glomerular filtration rate (GFR) of ≤45 mL/min/1.73m^2
  • Clinical evidence of systemic oxalosis
  • History of kidney or liver transplant
Contacts and Locations
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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): NCT03905694


Locations
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United States, Minnesota
Clinical Trial Site
Rochester, Minnesota, United States, 55905
United States, Texas
Clinical Trial Site
Houston, Texas, United States, 77030
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
Clinical Trial Site
Nahariya, Israel
United Kingdom
Clinical Trial Site
London, United Kingdom
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
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Study Director: Medical Director Alnylam Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Alnylam Pharmaceuticals:
Study Protocol  [PDF] May 4, 2020
Statistical Analysis Plan  [PDF] May 4, 2020

More Information
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Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03905694    
Other Study ID Numbers: ALN-GO1-004
2018-004014-17 ( EudraCT Number )
First Posted: April 5, 2019    Key Record Dates
Results First Posted: July 19, 2021
Last Update Posted: October 18, 2021
Last Verified: October 2021
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:
PH1
Primary Hyperoxaluria
Hyperoxaluria
 RNAi Therapeutic
siRNA
AGT
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