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

April 17, 2024 updated by: Alnylam Pharmaceuticals

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

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).

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

18

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Lyon, France
        • Clinical Trial Site
      • Paris, France
        • Clinical Trial Site
  • Germany
      • Bonn, Germany
        • Clinical Trial Site
  • Israel
      • Haifa, Israel
        • Clinical Trial Site
      • Jerusalem, Israel
        • Clinical Trial Site
      • Nahariya, Israel
        • Clinical Trial Site
  • United Kingdom
      • London, United Kingdom
        • Clinical Trial Site
  • United States
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Clinical Trial Site
    • Texas
      • Houston, Texas, United States, 77030
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 second to 5 years (Child)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Lumasiran
Lumasiran will be administered by subcutaneous (SC) injection.
Drug: Lumasiran
Lumasiran will be administered by subcutaneous (SC) injection.
Other Names:
  • ALN-GO1

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Baseline to Month 6

Secondary Outcome Measures

Outcome Measure
Time Frame
Percentage Change in Urinary Oxalate Excretion From Baseline to End of Study (Month 60)
Time Frame: Up to 60 months
Up to 60 months
Absolute Change in Urinary Oxalate Excretion From Baseline
Time Frame: Up to 60 months
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
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
Up to 60 months
Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60)
Time Frame: Up to 60 months
Up to 60 months
Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60)
Time Frame: Up to 60 months
Up to 60 months
Maximum Observed Plasma Concentration (Cmax) of Lumasiran
Time Frame: Up to 24 months
Up to 24 months
Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran
Time Frame: Up to 24 months
Up to 24 months
Elimination Half-life (t1/2beta) of Lumasiran
Time Frame: Up to 24 months
Up to 24 months
Area Under the Concentration-time Curve (AUC) of Lumasiran
Time Frame: Up to 24 months
Up to 24 months
Apparent Clearance (CL/F) of Lumasiran
Time Frame: Up to 24 months
Up to 24 months
Apparent Volume of Distribution (V/F) of Lumasiran
Time Frame: Up to 24 months
Up to 24 months
Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline
Time Frame: Up to 60 months
Up to 60 months
Frequency of Adverse Events (AEs)
Time Frame: Up to 60 months
Up to 60 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Investigators

  • Study Director: Medical Director, Alnylam Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 22, 2019

Primary Completion (Actual)

June 29, 2020

Study Completion (Estimated)

August 19, 2024

Study Registration Dates

First Submitted

April 1, 2019

First Submitted That Met QC Criteria

April 4, 2019

First Posted (Actual)

April 5, 2019

Study Record Updates

Last Update Posted (Actual)

April 22, 2024

Last Update Submitted That Met QC Criteria

April 17, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-GO1-004
  • 2018-004014-17 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Access to Anonymized individual participant data that support these results is made available 12 months after study completion and not less than 12 months after the product and indication have been approved in the US and/or the EU.

Data will be provided contingent upon the approval of a research proposal and the execution of a data sharing agreement. Requests for access to data can be submitted via the website www.vivli.org.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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