In short
VQ-101 is an investigational medicine being studied for Parkinson’s disease in people who carry a GBA1 gene mutation. A Phase 2 clinical trial is currently authorised in the EU to test whether VQ-101 can slow disease progression compared with placebo in 180 participants. The trial measures changes on the Movement Disorder Society – Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and monitors safety.
Key points
- VQ-101 is an investigational substance studied in GBA1-associated Parkinson’s disease.
- The only registered trial is a Phase 2 study with 180 participants.
- The trial status is Authorised in the EU Clinical Trials Information System.
- The main goal is to see whether VQ-101 slows disease progression measured by MDS-UPDRS.
- Safety is assessed through adverse events, laboratory tests, vital signs, ECGs, and the C-SSRS.
What is VQ-101?
VQ-101 is an investigational substance under study for GBA1-associated Parkinson’s disease (GBA-PD). The name VQ-101 is a development code used in the clinical trial registry; no trade name or marketing authorisation is recorded in the available data. The substance is given as a tablet for oral use. Because the trial is still in Phase 2, VQ-101 is not yet an authorised medicine.
How does VQ-101 work?
The available registry record does not describe the molecular target or mechanism of action of VQ-101. The trial is designed to evaluate whether VQ-101 can slow disease progression in people with GBA1-associated Parkinson’s disease, as measured by the Movement Disorder Society – Unified Parkinson’s Disease Rating Scale (MDS-UPDRS). No further details on how VQ-101 acts in the body are provided in the source data.
Which conditions is VQ-101 studied for?
- GBA1-associated Parkinson’s disease — a form of Parkinson’s disease linked to mutations in the GBA1 gene. The current trial focuses specifically on this genetic subgroup of Parkinson’s disease.
How is VQ-101 given?
In the registered trial, VQ-101 is administered as a tablet by oral use. The registry lists the dose as 0 mg, meaning the dose was not reported in the record. The trial also includes tablets matching VQ-101 as a placebo comparison.
What do the clinical trials show?
One clinical trial of VQ-101 is registered in the EU Clinical Trials Information System. It is a Phase 2, interventional study with a planned enrollment of 180 participants. The trial is currently authorised, meaning it has received regulatory approval to proceed but results are not yet available.
The primary goal is to evaluate the effect of VQ-101 versus placebo in slowing disease progression in participants with GBA1-associated Parkinson’s disease. The main measure is the Movement Disorder Society – Unified Parkinson’s Disease Rating Scale (MDS-UPDRS). The trial also evaluates safety through the incidence of treatment-emergent adverse events, changes in clinical laboratory tests, vital signs, and electrocardiograms, and responses to the Columbia-Suicide Severity Rating Scale (C-SSRS).
Participants in the trial receive either VQ-101 tablets or matching placebo tablets. The study is designed to compare the two groups over time to see whether VQ-101 slows progression more than placebo.
What side effects are known?
The registry record does not report side effects from completed studies, because the trial is still ongoing and results are not yet published. The trial measures treatment-emergent adverse events by frequency, severity, and seriousness, but no specific side effects are listed in the available data.