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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
First received
Last updated
Start date April 22, 2019
Est. completion date August 19, 2024

Study information

Verified date May 2024
Source Alnylam Pharmaceuticals
Contact n/a
Is FDA regulated No
Health authority
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

Study Design


Related Conditions & MeSH terms


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
See also
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Completed NCT03116685 - A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria Phase 3
Recruiting NCT05107830 - Phenotyping of Primary Hyperoxaluria
Active, not recruiting NCT04152200 - A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 Phase 3
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Completed NCT03392896 - Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria Phase 1
Completed NCT02012985 - Study to Evaluate the Efficacy and Safety of Oxabact (OC5) in Patients With Primary Hyperoxaluria Phase 1/Phase 2
Completed NCT01037231 - Phase 2/3 Oxabact Study Phase 2/Phase 3
Completed NCT00589225 - Primary Hyperoxaluria Mutation Genotyping Phase 1
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