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

Primary Hyperoxaluria Clinical Trial

— 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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03905694
• Other study ID #: ALN-GO1-004
• Secondary ID: 2018-004014-17
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: April 22, 2019
• Est. completion date: August 19, 2024

Study information

• Verified date: April 2024
• Source: Alnylam Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 18
• Est. completion date: August 19, 2024
• Est. primary completion date: June 29, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years to 5 Years

Eligibility

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

Related Conditions & MeSH terms

• Hyperoxaluria, Primary
• Primary Hyperoxaluria
• Primary Hyperoxaluria Type 1 (PH1)

Intervention

Drug:
• Lumasiran
Lumasiran will be administered by subcutaneous (SC) injection.

Locations

Country	Name	City	State
France	Clinical Trial Site	Lyon
France	Clinical Trial Site	Paris
Germany	Clinical Trial Site	Bonn
Israel	Clinical Trial Site	Haifa
Israel	Clinical Trial Site	Jerusalem
Israel	Clinical Trial Site	Nahariya
United Kingdom	Clinical Trial Site	London
United States	Clinical Trial Site	Houston	Texas
United States	Clinical Trial Site	Rochester	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Alnylam Pharmaceuticals

Countries where clinical trial is conducted

United States,  France,  Germany,  Israel,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Baseline to Month 6
Secondary	Percentage Change in Urinary Oxalate Excretion From Baseline to End of Study (Month 60)		Up to 60 months
Secondary	Absolute Change in Urinary Oxalate Excretion From Baseline		Up to 60 months
Secondary	Percentage of Time That Spot Urinary Oxalate:Creatinine Ratio = Near-normalization Threshold (=1.5 × ULN)		Up to 60 months
Secondary	Percentage of Participants With Urinary Oxalate Excretion = the Upper Limit of Normal (ULN) and = 1.5 x ULN		Up to 60 months
Secondary	Percentage Change in Plasma Oxalate From Baseline to End of Study (Month 60)		Up to 60 months
Secondary	Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60)		Up to 60 months
Secondary	Maximum Observed Plasma Concentration (Cmax) of Lumasiran		Up to 24 months
Secondary	Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran		Up to 24 months
Secondary	Elimination Half-life (t1/2beta) of Lumasiran		Up to 24 months
Secondary	Area Under the Concentration-time Curve (AUC) of Lumasiran		Up to 24 months
Secondary	Apparent Clearance (CL/F) of Lumasiran		Up to 24 months
Secondary	Apparent Volume of Distribution (V/F) of Lumasiran		Up to 24 months
Secondary	Change in Estimated Glomerular Filtration Rate (eGFR) From Baseline		Up to 60 months
Secondary	Frequency of Adverse Events (AEs)		Up to 60 months