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

Primary Purpose

Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Lumasiran
Sponsored by
Alnylam Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Hyperoxaluria focused on measuring PH1, Primary Hyperoxaluria, Hyperoxaluria, RNAi Therapeutic, siRNA, AGT

Eligibility Criteria

0 Years - 5 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Lumasiran

Arm Description

Lumasiran will be administered by subcutaneous (SC) injection.

Outcomes

Primary Outcome Measures

Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From 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

Percentage Change in Urinary Oxalate Excretion From Baseline to End of Study (Month 60)
Absolute Change in Urinary Oxalate Excretion From Baseline
Percentage of Time That Spot Urinary Oxalate:Creatinine Ratio ≤ Near-normalization Threshold (≤1.5 × ULN)
Percentage of Participants With Urinary Oxalate Excretion ≤ the Upper Limit of Normal (ULN) and ≤ 1.5 x ULN
Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60)
Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60)
Maximum Observed Plasma Concentration (Cmax) of Lumasiran
Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran
Elimination Half-life (t1/2beta) of Lumasiran
Area Under the Concentration-time Curve (AUC) of Lumasiran
Apparent Clearance (CL/F) of Lumasiran
Apparent Volume of Distribution (V/F) of Lumasiran
Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline
Frequency of Adverse Events (AEs)

Full Information

First Posted
April 1, 2019
Last Updated
October 20, 2023
Sponsor
Alnylam Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT03905694
Brief Title
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
Acronym
ILLUMINATE-B
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
April 22, 2019 (Actual)
Primary Completion Date
June 29, 2020 (Actual)
Study Completion Date
August 19, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alnylam Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)
Keywords
PH1, Primary Hyperoxaluria, Hyperoxaluria, RNAi Therapeutic, siRNA, AGT

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
18 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Lumasiran
Arm Type
Experimental
Arm Description
Lumasiran will be administered by subcutaneous (SC) injection.
Intervention Type
Drug
Intervention Name(s)
Lumasiran
Other Intervention Name(s)
ALN-GO1
Intervention Description
Lumasiran will be administered by subcutaneous (SC) injection.
Primary Outcome Measure Information:
Title
Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6
Description
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.
Time Frame
Baseline to Month 6
Secondary Outcome Measure Information:
Title
Percentage Change in Urinary Oxalate Excretion From Baseline to End of Study (Month 60)
Time Frame
Up to 60 months
Title
Absolute Change in Urinary Oxalate Excretion From Baseline
Time Frame
Up to 60 months
Title
Percentage of Time That Spot Urinary Oxalate:Creatinine Ratio ≤ Near-normalization Threshold (≤1.5 × ULN)
Time Frame
Up to 60 months
Title
Percentage of Participants With Urinary Oxalate Excretion ≤ the Upper Limit of Normal (ULN) and ≤ 1.5 x ULN
Time Frame
Up to 60 months
Title
Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60)
Time Frame
Up to 60 months
Title
Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60)
Time Frame
Up to 60 months
Title
Maximum Observed Plasma Concentration (Cmax) of Lumasiran
Time Frame
Up to 24 months
Title
Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran
Time Frame
Up to 24 months
Title
Elimination Half-life (t1/2beta) of Lumasiran
Time Frame
Up to 24 months
Title
Area Under the Concentration-time Curve (AUC) of Lumasiran
Time Frame
Up to 24 months
Title
Apparent Clearance (CL/F) of Lumasiran
Time Frame
Up to 24 months
Title
Apparent Volume of Distribution (V/F) of Lumasiran
Time Frame
Up to 24 months
Title
Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline
Time Frame
Up to 60 months
Title
Frequency of Adverse Events (AEs)
Time Frame
Up to 60 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
0 Years
Maximum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Alnylam Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Clinical Trial Site
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Clinical Trial Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Clinical Trial Site
City
Lyon
Country
France
Facility Name
Clinical Trial Site
City
Paris
Country
France
Facility Name
Clinical Trial Site
City
Bonn
Country
Germany
Facility Name
Clinical Trial Site
City
Haifa
Country
Israel
Facility Name
Clinical Trial Site
City
Jerusalem
Country
Israel
Facility Name
Clinical Trial Site
City
Nahariya
Country
Israel
Facility Name
Clinical Trial Site
City
London
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
35913563
Citation
Hayes W, Sas DJ, Magen D, Shasha-Lavsky H, Michael M, Sellier-Leclerc AL, Hogan J, Ngo T, Sweetser MT, Gansner JM, McGregor TL, Frishberg Y. Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial. Pediatr Nephrol. 2023 Apr;38(4):1075-1086. doi: 10.1007/s00467-022-05684-1. Epub 2022 Aug 1.
Results Reference
derived
PubMed Identifier
34906487
Citation
Sas DJ, Magen D, Hayes W, Shasha-Lavsky H, Michael M, Schulte I, Sellier-Leclerc AL, Lu J, Seddighzadeh A, Habtemariam B, McGregor TL, Fujita KP, Frishberg Y; ILLUMINATE-B Workgroup. Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children. Genet Med. 2022 Mar;24(3):654-662. doi: 10.1016/j.gim.2021.10.024. Epub 2021 Dec 8.
Results Reference
derived

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A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1

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