Table of contents
- Clinical trial overview
- Metastatic pancreatic cancer study
- Wolfram syndrome study
- Tuberous sclerosis complex study
- Metastatic colorectal cancer studies
- Main endpoints used in these trials
- Who these studies are for
Clinical trial overview
These studies are testing Valproic Acid in different diseases, mostly in Phase 2 interventional trials.[1][2][3][4][5]
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.[1][4][5]
Metastatic pancreatic cancer study
The VESPA trial is a randomized Phase 2 study in untreated patients with metastatic pancreatic ductal adenocarcinoma (mPDAC).[1]
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.[1]
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.[1]
The trial is authorised and plans to enroll 170 people.[1]
Wolfram syndrome study
The AUDIOWOLF study is a Phase 2, open-label study in patients with Wolfram syndrome caused by a monogenic mutation.[2]
It is studying daily sodium valproate, with the goal of preserving hearing over three years.[2]
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.[2]
The study is completed and enrolled 23 patients.[2]
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.[3]
This study includes sodium valproate and Valproic Acid among several treatment options being studied for early care in TSC.[3]
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.[3]
The trial is authorised and plans to enroll 64 children.[3]
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.[4][5]
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.[4]
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.[4]
The trial is authorised and plans to enroll 130 patients.[4]
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.[5]
The main endpoint is PFS, to see whether the combination can help patients stay free from disease progression longer than standard treatment alone.[5]
The trial is authorised and plans to enroll 200 patients.[5]
Main endpoints used in these trials
A primary outcome is the main result the researchers want to measure first.[1][2][3][4][5]
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.[1][2][3][4][5]
Progression-free survival (PFS) measures how long a patient lives before the disease gets worse.[1][4][5]
Audiometry and high frequency hearing tests measure whether hearing stays stable over time.[2]
Bayley Scales of Infant and Toddler Development III measure early cognitive development in young children.[3]
Who these studies are for
These trials do not study one broad group of patients, but several very specific groups.[1][2][3][4][5]
Adults with untreated metastatic pancreatic cancer in the VESPA trial.[1]
Patients with Wolfram syndrome and hearing loss in the AUDIOWOLF study.[2]
Very young children with tuberous sclerosis complex in the PROTECT study.[3]
Patients with pretreated RAS/BRAF wild-type metastatic colorectal cancer in the VICTORIA study.[4]
Patients with RAS-mutated metastatic colorectal cancer in the REVOLUTION study.[5]
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.[1][2][3][4][5]
