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

• Register: ClinicalTrials.gov
• Last refreshed on: 29 April 2024
• Main ID: NCT03905694
• Date of registration: 01/04/2019
• Prospective Registration: Yes
• Primary sponsor: Alnylam Pharmaceuticals
• Public title: A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1 ILLUMINATE-B
• Scientific 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
• Date of first enrolment: April 22, 2019
• Target sample size: 18
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03905694
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

France, Germany, Israel, United Kingdom, United States

Contacts

• Name: Medical Director
• Affiliation: Alnylam Pharmaceuticals

Key inclusion & exclusion 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



Age minimum: 0 Years
Age maximum: 5 Years
Gender: All

Health Condition(s) or Problem(s) studied

Primary Hyperoxaluria

Primary Hyperoxaluria Type 1 (PH1)

Intervention(s)

Drug: Lumasiran

Primary Outcome(s)

Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 [Time Frame: Baseline to Month 6]

Secondary Outcome(s)

Apparent Clearance (CL/F) of Lumasiran [Time Frame: Up to 24 months]

Percentage Change in Urinary Oxalate Excretion From Baseline to End of Study (Month 60) [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]

Frequency of Adverse Events (AEs) [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]

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]

Time to Maximum Observed Plasma Concentration (Tmax) of Lumasiran [Time Frame: Up to 24 months]

Absolute Change in Plasma Oxalate From Baseline to End of Study (Month 60) [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 Urinary Oxalate Excretion From Baseline [Time Frame: Up to 60 months]

Maximum Observed Plasma Concentration (Cmax) 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]

Secondary ID(s)

2018-004014-17

ALN-GO1-004

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 19/07/2021
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03905694