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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03350451
Other study ID # ALN-GO1-002
Secondary ID 2016-003134-24
Status Completed
Phase Phase 2
First received
Last updated
Start date April 4, 2018
Est. completion date February 7, 2023

Study information

Verified date March 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 long-term safety and tolerability of lumasiran in participants with Primary Hyperoxaluria Type 1.


Recruitment information / eligibility

Status Completed
Enrollment 20
Est. completion date February 7, 2023
Est. primary completion date February 7, 2023
Accepts healthy volunteers No
Gender All
Age group 6 Years and older
Eligibility Inclusion Criteria: - Enrollment within 12 months of completion of Study ALN-GO1-001 - In the opinion of the investigator tolerated the study drug - If taking Vitamin B6 (pyridoxine), willing to remain on a stable regimen for the study duration - Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception - Willing to provide written informed consent and to comply with study requirements Exclusion Criteria: - Clinically significant health concerns (with the exception of PH1) - Clinically significant cardiovascular abnormality - Abnormal for AST/ALT and any other clinical safety laboratory result considered clinically significant - Requirement for chronic dialysis

Study Design


Intervention

Drug:
Lumasiran
Multiple doses of lumasiran by SC injection

Locations

Country Name City State
France Clinical Trial Site Bordeaux
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
Netherlands Clinical Trial Site Amsterdam
United Kingdom Clinical Trial Site Birmingham
United Kingdom Clinical Trial Site London

Sponsors (1)

Lead Sponsor Collaborator
Alnylam Pharmaceuticals

Countries where clinical trial is conducted

France,  Germany,  Israel,  Netherlands,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of Participants With at Least One Adverse Event (AE) AE is any untoward medical occurrence in a participant or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment. Safety analysis set included all participants who received any amount of study drug. Baseline (Day -1) up to 54 months
Secondary Change From Baseline in 24-hour Urinary Oxalate Corrected for Body Surface Area (BSA) at 54 Months Oxalate produced by the liver is the key toxic metabolite that drives disease pathology in participants with primary hyperoxaluria type 1 (PH1). The risk of disease complications increase continuously as oxalate levels increase. 24-hour urinary oxalate (millimole [mmol]/ 24 hour [h]/1.73 meters squared [m^2]) corrected for BSA at each visit per participant was calculated as follows: [Urine oxalate concentration (micromole per liter [umol/L])/1000 (umol/mmol)]*[24hour urine volume (mL)/1000 (mL/L)]* [24 hours/actual collection hours]*1.73/(BSA). Baseline was the derived baseline value from the lumasiran treated period of Study ALN-GO1-001. A negative change from baseline indicated a favorable outcome. PD analysis set included all participants who received any amount of study drug and who had at least 1 post-dose urine sample for PD. Overall number of participants analyzed are the number of participants with data available for analysis. Baseline (Day -1) up to 54 months
Secondary Change From Baseline in 24-hour Urinary Oxalate:Creatinine Ratio at 54 Months Baseline is the derived baseline value from the lumasiran treated period of Study ALN-GO1-001. A negative change from baseline indicates a favorable outcome. PD analysis set included all participants who received any amount of study drug and who had at least 1 post-dose urine sample for PD. Baseline (Day -1) up to 54 months
Secondary Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at 54 Months Baseline was defined as the last measurement prior to the first dose of lumasiran in the ALN-GO1-001 study. eGFR was calculated based on the Modification of Diet in Renal Disease (MDRD) formula for participants >=18 years of age at enrollment and the Schwartz Bedside formula for participants <18 years of age at enrollment. eGFR based on MDRD formula was calculated as follows: eGFR (mL/min/1.73 m^2) = 175 × (serum creatinine {SCr} [µmol/deciliter(dL)]/88.4)-1.154 × (age)-0.203 × (0.742, if female), or × (1.212, if African American) and based on Schwartz formula: eGFR (mL/min/1.73m2) = (36.2 × height [cm])/ SCr (µmol /dL). Safety analysis set included all participants who received any amount of study drug. Baseline (Day -1) up to 54 months
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