Lumasiran
- Generic Name
- Lumasiran
- Brand Names
- Oxlumo
- Drug Type
- Biotech
- CAS Number
- 1834610-13-7
- Unique Ingredient Identifier
- RZT8C352O1
- Background
Lumasiran is a small interfering RNA used in the treatment of primary hyperoxaluria type 1 (PH1). This condition, caused by a deficiency in the enzyme alanine-glyoxylate aminotransferase, leads to an accumulation of oxalate, causing calcium crystal formation. These patients experience frequent kidney stones, nephrocalcinosis, and renal failure.
Oxlumo, producted by Alnylam Pharmaceuticals, represents the first approved treatment for PH1. Prior to this approval, therapy consisted of symptomatic treatment such as hyperhydration, inhibitors of crystallization, pyridoxine, and renal transplant.
Lumasiran was granted FDA approval on 23 November 2020.
- Indication
Lumasiran is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary and plasma oxalate levels in pediatric and adult patients.
- Associated Conditions
- Primary Hyperoxaluria Type 1 (PH1)
Lumasiran in Hyperoxalaemic Patients on Haemodialysis
- Conditions
- Chronic Kidney Disease Requiring Chronic DialysisCardiovascular Risk FactorCardiovascular DiseaseHyperoxalemiaHaemodialysis
- Interventions
- Drug: 0.9% Sodium Chloride (placebo)
- First Posted Date
- 2024-01-26
- Last Posted Date
- 2024-08-21
- Lead Sponsor
- Charite University, Berlin, Germany
- Target Recruit Count
- 50
- Registration Number
- NCT06225544
- Locations
- 🇩🇪
Charite Universtiätsmedizin, Berlin, Germany
A Study to Evaluate Lumasiran in Adults With Recurrent Calcium Oxalate Kidney Stone Disease and Elevated Urinary Oxalate Levels
- Conditions
- Recurrent Calcium Oxalate Kidney Stone DiseaseElevated Urinary Oxalate Levels
- Interventions
- Drug: Placebo
- First Posted Date
- 2021-12-17
- Last Posted Date
- 2024-05-22
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 2
- Registration Number
- NCT05161936
- Locations
- 🇬🇧
Clinical Trial Site, Leeds, United Kingdom
A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
- Conditions
- Primary HyperoxaluriaPrimary Hyperoxaluria Type 1
- Interventions
- First Posted Date
- 2019-11-05
- Last Posted Date
- 2025-05-20
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 21
- Registration Number
- NCT04152200
- Locations
- 🇦🇪
Clinical Trial Site, Dubai, United Arab Emirates
Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1
- Conditions
- Primary Hyperoxaluria
- First Posted Date
- 2019-10-14
- Last Posted Date
- 2024-02-16
- Lead Sponsor
- Alnylam Pharmaceuticals
- Registration Number
- NCT04125472
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
- Conditions
- Primary HyperoxaluriaPrimary Hyperoxaluria Type 1 (PH1)
- Interventions
- First Posted Date
- 2019-04-05
- Last Posted Date
- 2025-02-14
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 18
- Registration Number
- NCT03905694
- Locations
- 🇬🇧
Clinical Trial Site, London, United Kingdom
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
- Conditions
- Primary Hyperoxaluria Type 1 (PH1)
- Interventions
- Drug: Placebo
- First Posted Date
- 2018-09-21
- Last Posted Date
- 2024-08-12
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 39
- Registration Number
- NCT03681184
- Locations
- 🇬🇧
Clinical Trial Site, London, United Kingdom
An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1
- Conditions
- PH1AGTPrimary HyperoxaluriasiRNARNAi Therapeutic
- Interventions
- First Posted Date
- 2017-11-22
- Last Posted Date
- 2024-04-25
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 20
- Registration Number
- NCT03350451
- Locations
- 🇬🇧
Clinical Trial Site, London, United Kingdom
Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1
- Conditions
- Primary Hyperoxaluria Type 1 (PH1)
- Interventions
- Drug: Placebo
- First Posted Date
- 2016-03-11
- Last Posted Date
- 2020-01-30
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 52
- Registration Number
- NCT02706886
- Locations
- 🇬🇧
Clinical Trial Site, London, United Kingdom
Drug Development Updates
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