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Español Current SCN2A Clinical Trials
Current clinical trials for SCN2A-related conditions
Active clinical trials
EMBOLD STUDY – Praxis Precision Medicines
Praxis Precision Medicines
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Study phase
2
Study drug
PRAX-562. Dose 0.5mg/kg/day
Drug type
Small molecule
Type of trial
DEE – SCN2A and SCN8A
Comparator
Placebo (given for 4 weeks)
Countries
United States
Key inclusion criteria
Aged 2–18 years
Diagnosed with SCN2A with seizures in first 3 months of life, or SCN8A and seizures in the first 6 months of life
Currently taking 1–4 anti-seizure medications at a stable dose
8 or more motor seizures in 4 weeks prior to screening, AND
8 or more motor seizures during the 28 day baseline observation period
Key exclusion criteria
Loss of function (LoF) mutations
2 or more episodes of status epilepticus requiring hospitalisation and intubation in the 6 months prior to screening
Study will assess
Safety
Frequency of motor seizures
Blood concentration of PRAX-562
Overall health
Change in seizure frequency
More information
EMBRACE STUDY – Praxis Precision Medicines
Praxis Precision Medicines
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Study phase
1/2
Study drug
PRAX-222
Drug type
Antisense oligonucleotide (ASO)
Type of trial
SCN2A-related DEE
Comparator
Placebo (given for 4 weeks)
Countries
United States
Key inclusion criteria
Aged 2–18 years
Diagnosed with SCN2A gain of function (GoF) mutation confirmed on genetic testing
Onset of seizures before 3 months of age
Weight of at least 10kg
Key exclusion criteria
Taking more than 2 sodium channel blocking anti-seizure medcations
Study will assess
Safety
Frequency of seizures
Proportion of participants with a treatment response
Overall health
Change in EEG
Change in sleep
Developmental milestones
More information
ClinicalTrials.gov
Clinical trials that have closed recruitment
PACIFIC STUDY – Longboard Pharmaceuticals
Longboard Pharmaceuticals
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Study phase
1/2
Study drug
LP352. Taken three times daily (by mouth) or via feeding tube
Drug type
Small molecule
Type of trial
Bucket (one treatment for multiple conditions)
Comparator
Placebo (80% chance of receiving LP352)
Countries
United States
Australia
Key inclusion criteria
Aged 12–65 years
Diagnosed with developmental and epileptic encephalopathy (DEE)
Currently taking 1–4 anti-seizure medications at a stable dose
4 or more motor seizures per month for 3 months
Have reliable caregiver or study partner
Key exclusion criteria
Using fenfluramine or lorcaserin
Cardiovascular (heart and blood vessels) or cerebrovascular (blood vessels in the brain) condition
Study will assess
Safety
Mental health
Overall health
Change in seizure frequency
How the body interacts with the treatment (pharmacokinetics)
More information