Primary Hyperoxaluria Clinical Trial
Official title:
A Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Administration of ALN-GO1 in Patients With Primary Hyperoxaluria Type 1
Verified date | March 2024 |
Source | Alnylam Pharmaceuticals |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The purpose of this study is to evaluate the long-term safety and tolerability of lumasiran in participants with Primary Hyperoxaluria Type 1.
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 |
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 |
Lead Sponsor | Collaborator |
---|---|
Alnylam Pharmaceuticals |
France, Germany, Israel, Netherlands, United Kingdom,
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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