Table of Contents
- Clinical trials overview
- Neurology and emergency seizure studies
- Cancer studies
- Pediatric and rare disease studies
- Psychiatry studies
- Women’s health study
- Main outcomes used in the trials
Clinical trials overview
The trial data show that Sodium Valproate is being studied in several different diseases, not just one condition.[1][2][3]
These studies include interventional trials, which means the researchers give a treatment and then measure what happens.[1]
The trial phases range from Phase 1 to Phase 4, so the research includes early safety work and later studies that look at treatment benefit in larger groups.[2][3]
Neurology and emergency seizure studies
Several trials focus on seizure-related conditions, including status epilepticus, which is a medical emergency involving ongoing or repeated seizures.[4][5]
In NCT05263674, the FAST TRIAL, researchers compare intravenous Sodium Valproate with other anti-seizure and sedation treatments in people with status epilepticus, and the main outcome is treatment failure after 24 hours.[5]
In NCT06549426, the TELSTAR-2 trial studies comatose cardiac arrest patients with status epilepticus on continuous EEG, and it asks whether stepwise anti-seizure treatment improves functional recovery at six months.[4]
The TELSTAR-2 primary outcome is the score on the extended Glasgow Outcome Scale (eGOS), which measures recovery after brain injury or severe illness.[4]
Cancer studies
One Phase 1 study, NCT06199557, looks at Sodium Valproate in patients with acute myeloid leukemia or high-risk myelodysplastic syndrome who are considered unfit for standard chemotherapy.[2]
This trial tests combinations with hydroxyurea or 6-mercaptopurine and focuses on safety and tolerability, including adverse events, dose-limiting toxicities, lab tests, and physical exams.[2]
It also measures early signs of clinical benefit and changes in Eastern Cooperative Oncology Group (ECOG) performance status, which is a score that shows how well a person can carry out daily activities.[2]
In 2024-516844-25-01, the REVOLUTION study tests whether adding valproic acid and Sodium Valproate to bevacizumab and oxaliplatin/fluoropyrimidine regimens can improve progression-free survival in people with RAS-mutated metastatic colorectal cancer.[6]
Pediatric and rare disease studies
The AUDIOWOLF study, NCT04940572, is a Phase 2 trial in people with Wolfram syndrome due to a monogenic mutation.[1]
Its main goal is to see whether daily Sodium Valproate can preserve hearing over three years, using audiometry and high-frequency hearing tests.[1]
Another Phase 2 study, 2022-502332-39-00, includes children with tuberous sclerosis complex under 4 months of age and measures neuropsychologic outcome at 24 months using the cognitive scale of the Bayley Scales of Infant and Toddler Development III (BSID-III).[7]
In the ependymoma program NCT02265770, Sodium Valproate appears in one treatment stratum for children unable to receive radiation therapy, where the study evaluates progression-free survival when valproate is added to the main chemotherapy strategy.[3]
Psychiatry studies
Two Phase 3 studies include people with mood and other psychiatric disorders.[8][9]
NCT05973786 studies a six-week intensified drug plan for bipolar depression in people who had a first-time treatment failure on first-line treatment, and it measures change in symptom severity using the Montgomery-Åsberg Depression Rating Scale (MADRS).[8]
NCT05603104 was a larger Phase 3 study in schizophrenia, schizoaffective disorder, schizophreniform disorder, major depressive disorder, and bipolar depression, but the trial status is withdrawn.[9]
That withdrawn study also measured symptom change, using the Positive and Negative Syndrome Scale for schizophrenia and MADRS for major depressive disorder and bipolar depression.[9]
Women’s health study
In 2025-523076-23-00, researchers are studying women of reproductive age with adenomyosis in a Phase 2 trial.[10]
The trial compares a plan that includes a single intralesional administration of valproic acid followed by oral dosing with placebo-based comparators, and it looks at pelvic pain and rescue medication use over 29 days.[10]
The primary endpoint uses change in AAPP, which is the trial’s pain measure, and it also records how much ibuprofen is needed as rescue medicine.[10]
Main outcomes used in the trials
The studies use different endpoints because they are asking different clinical questions.[1][2][3]
Functional recovery is used in the post-cardiac arrest trial and is measured by eGOS at six months.[4]
Safety and tolerability are central in the leukemia trial, where the team tracks adverse events, exams, and lab results.[2]
Hearing preservation is the key outcome in Wolfram syndrome, with testing over three years.[1]
Progression-free survival is used in the colorectal cancer and ependymoma studies to see how long the disease stays controlled.[6][3]
Symptom severity is measured in the bipolar depression study using MADRS, while the withdrawn psychiatric study used both MADRS and PANSS depending on the diagnosis.[8][9]
Neuropsychologic outcome is measured in young children with tuberous sclerosis complex using blinded testing at 24 months.[7]
