This study is researching an experimental drug called ALN-SOD (called "study drug"). This study is focused on people with Amyotrophic Lateral Sclerosis (ALS) caused by a change in a gene called the Superoxide Dismutase-1 (SOD1) gene. This type of ALS is known as "SOD1-ALS". This is the first time that ALN-SOD will be given to people. The aim of the study is to see how safe and tolerable the study drug is. The study is looking at several other research questions, including: * The effect the study drug has on specific biomarkers, which are substances in the blood or in the fluid that surrounds the brain and spinal cord, known as Cerebrospinal Fluid (CSF) * How much study drug is in the blood and in the CSF, at different times * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects) * What effects the study drug has on ALS symptoms
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
54
Administered per the protocol
Administered per the protocol
Administered per the protocol
Concord Repatriation General Hospital
Concord, New South Wales, Australia
RECRUITINGMacquarie University
Sydney, New South Wales, Australia
RECRUITINGSunshine Coast University Hospital
Birtinya, Queensland, Australia
RECRUITINGKU Leuven
Leuven, Vlaams-Brabant, Belgium
RECRUITINGUniversity of Alberta Hospital, Edmonton, Division of Neurology
Edmonton, Alberta, Canada
RECRUITINGUniversity Hospital - London Health Sciences Centre
London, Ontario, Canada
RECRUITINGSunnybrook Research Institute
Toronto, Ontario, Canada
RECRUITINGMontreal Neurological Institute and Hospital
Montreal, Quebec, Canada
RECRUITINGUniversitaetsklinikum Ulm AoR
Ulm, Baden-Wurttemberg, Germany
RECRUITINGHokkaido University Hospital
Sapporo, Hokkaido, Japan
RECRUITING...and 12 more locations
Incidence of Treatment-Emergent Adverse Event (TEAEs) in participants treated with ALN-SOD
Time frame: At week 4 and through week 228
Severity of TEAEs in participants treated with ALN-SOD
Time frame: At week 4 and through week 228
Concentration of Neurofilament Light chain (NfL) in plasma over time
Time frame: Up to approximately week 228
Change in concentration of NfL in plasma over time
Time frame: Up to approximately week 228
Concentration of SOD1 protein in Cerebrospinal Fluid (CSF) over time
Time frame: Up to approximately week 228
Change in concentration of SOD1 protein in CSF over time
Time frame: Up to approximately week 228
Concentration of NfL in CSF over time
Time frame: Up to approximately week 228
Change in concentration of NfL in CSF over time
Time frame: Up to approximately week 228
Change in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) over time
Time frame: Up to approximately week 228
Concentration of ALN-SOD in plasma over time
Time frame: Up to approximately week 228
Concentration of ALN-SOD in CSF over time
Time frame: Up to approximately week 228
Incidence of Anti-Drug Antibodies (ADAs) to ALN-SOD in serum over time
Time frame: Up to approximately week 228
Titer of ADAs to ALN-SOD in serum over time
Time frame: Up to approximately week 228
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