In short
Clinical trials are studying Valproic Acid in different patient groups, including some cancers, Wolfram syndrome, and tuberous sclerosis complex. These studies are mainly checking whether Valproic Acid can improve outcomes, such as disease control, hearing, or neuropsychologic development. Most trials are phase 2 studies.
Key points
- Clinical trials of Valproic Acid in this data set are mostly Phase 2 studies. They focus on very different groups, including people with metastatic pancreatic cancer, metastatic colorectal cancer, Wolfram syndrome, and tuberous sclerosis complex. Several trials test Valproic Acid as part of a combination treatment, not by itself. The main goals include improving progression-free survival, preserving hearing, and supporting neuropsychologic development. These studies are authorised or completed, and each one uses its own specific patient group and outcome measure.
Clinical trial overview
These studies are testing Valproic Acid in different diseases, mostly in Phase 2 interventional trials.
Across the trials, Valproic Acid is often given with other treatments, and the researchers are comparing these combinations with standard treatment plans or other study arms.
Metastatic pancreatic cancer study
The VESPA trial is a randomized Phase 2 study in untreated patients with metastatic pancreatic ductal adenocarcinoma (mPDAC).
This study is testing whether Valproic Acid plus simvastatin, together with gemcitabine and nab-paclitaxel-based regimens, may improve first-line treatment results in mPDAC.
The main endpoint is progression-free survival (PFS), which means the time from randomization until the cancer gets worse or the patient dies from any cause, whichever happens first.
The trial is authorised and plans to enroll 170 people.
Wolfram syndrome study
The AUDIOWOLF study is a Phase 2, open-label study in patients with Wolfram syndrome caused by a monogenic mutation.
It is studying daily sodium valproate, with the goal of preserving hearing over three years.
The main endpoint is whether hearing does not drop by more than 5 dB at 8 kHz in the high frequency average, measured with hearing tests called audiometry and high frequency pure tone audiometry.
The study is completed and enrolled 23 patients.
Tuberous sclerosis complex study
The PROTECT study is a Phase 2 interventional trial in children with tuberous sclerosis complex (TSC) under 4 months of age.
This study includes sodium valproate and Valproic Acid among several treatment options being studied for early care in TSC.
The main endpoint is neuropsychologic outcome at 24 months of age, measured with blinded testing using the cognitive scale of the Bayley Scales of Infant and Toddler Development III (BSID-III), compared with standard of care alone.
The trial is authorised and plans to enroll 64 children.
Metastatic colorectal cancer studies
Two Phase 2 studies are testing Valproic Acid in metastatic colorectal cancer, but they focus on different tumor types and treatment settings.
The VICTORIA study is in pretreated patients with RAS/BRAF wild-type metastatic colorectal cancer and is testing Valproic Acid with panitumumab and irinotecan against standard treatment.
Its main outcomes include PFS at 16 weeks in Part 1 and PFS at 8 weeks in Part 2, both based on whether patients are alive and not progressed.
The trial is authorised and plans to enroll 130 patients.
The REVOLUTION study is in patients with RAS-mutated metastatic colorectal cancer and is testing Valproic Acid with bevacizumab and oxaliplatin/fluoropyrimidine regimens as first-line treatment.
The main endpoint is PFS, to see whether the combination can help patients stay free from disease progression longer than standard treatment alone.
The trial is authorised and plans to enroll 200 patients.
Main endpoints used in these trials
A primary outcome is the main result the researchers want to measure first.
In these studies, the endpoints include PFS, hearing preservation, and neuropsychologic testing, which show that the trials are looking at both cancer control and non-cancer outcomes.
- Progression-free survival (PFS) measures how long a patient lives before the disease gets worse.
- Audiometry and high frequency hearing tests measure whether hearing stays stable over time.
- Bayley Scales of Infant and Toddler Development III measure early cognitive development in young children.
Who these studies are for
These trials do not study one broad group of patients, but several very specific groups.
- Adults with untreated metastatic pancreatic cancer in the VESPA trial.
- Patients with Wolfram syndrome and hearing loss in the AUDIOWOLF study.
- Very young children with tuberous sclerosis complex in the PROTECT study.
- Patients with pretreated RAS/BRAF wild-type metastatic colorectal cancer in the VICTORIA study.
- Patients with RAS-mutated metastatic colorectal cancer in the REVOLUTION study.
Because each trial has a different disease, age group, and treatment plan, the study results cannot be treated as one single answer for all patients.