VTx-002 for ALS
(ALS Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a new treatment called VTx-002 for individuals with Amyotrophic Lateral Sclerosis (ALS), a condition affecting nerve cells in the brain and spinal cord, leading to muscle weakness. Researchers are testing the safety and effects of different doses of VTx-002, administered as a single dose directly into the spinal fluid. They aim to assess the body's tolerance and any changes in disease markers. Individuals diagnosed with ALS within the last 18 months and experiencing muscle weakness might be suitable candidates for this trial. As a Phase 1 trial, this research focuses on understanding how VTx-002 works in people, offering participants the opportunity to be among the first to receive this new treatment.
Is there any evidence suggesting that VTx-002 is likely to be safe for humans?
Research has shown that VTx-002, a new treatment for ALS (Amyotrophic Lateral Sclerosis), has been safe in animal studies. Tests with monkeys demonstrated that the treatment was well-tolerated, meaning it didn't cause major side effects. This safety profile aligns with other treatments using the same method (AAV5, a type of gene therapy).
However, VTx-002 is now undergoing its first tests in humans. Researchers are closely monitoring participants' reactions to the treatment. The study includes two groups, each receiving a different dose to determine which is better tolerated. This approach helps identify the safest and most effective dose for future studies.
Participants can receive rescue medication, such as methylprednisolone, if they experience any side effects. This helps manage unwanted reactions. Overall, while the results are promising, more data from human trials are needed to fully understand the safety of VTx-002 for ALS.12345Why do researchers think this study treatment might be promising for ALS?
Most treatments for ALS focus on managing symptoms and might include medications like riluzole or edaravone, which aim to slow disease progression. But VTx-002 is different. It's a gene therapy administered directly into the cerebrospinal fluid via the intra cisterna magna route. This method allows targeted delivery to the central nervous system, potentially addressing the disease directly at its source. Researchers are excited because this innovative approach could offer a more direct and potentially more effective treatment option for ALS, moving beyond just symptom management.
What evidence suggests that VTx-002 might be an effective treatment for ALS?
Research shows that VTx-002 targets a protein called TDP-43, linked to ALS, a disease affecting nerve cells. In people with ALS, TDP-43 often forms clumps inside nerve cells, damaging them. VTx-002 aims to reduce these harmful clumps. Although human studies provide limited information, this method appears promising based on current ALS knowledge. Early animal studies suggest that reducing TDP-43 might slow the disease. This trial will test VTx-002 in two groups: one receiving a low dose and the other a high dose. VTx-002 could potentially help manage ALS by addressing one of its core issues.14678
Are You a Good Fit for This Trial?
This trial is for individuals with Amyotrophic Lateral Sclerosis (ALS). Participants will be involved in the study for up to 5 years and 5 weeks. Specific eligibility criteria are not provided, but typically include factors like disease stage, overall health status, and no conflicting medications or conditions.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a single intracisternal injection of VTx-002, with staggered dosing and safety monitoring
Follow-up
Participants are monitored for safety and effectiveness, with assessments and biomarker analysis
What Are the Treatments Tested in This Trial?
Interventions
- VTx-002
Trial Overview
The PIONEER-ALS study is testing VTx-002, a new treatment administered into the spinal canal. It's an open-label trial where everyone knows what treatment they're getting. There are two different doses being tested to see which one is safer and works better.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Gene Therapy: Vtx-002 3 participants will receive dose 2 administered intra cisterna magna. The participant dosing in group 2 will be staggered as it was in group 1 VTx-002 is a single dose therapy. Drug: Optional Rescue Medication - Methylprednisolone The study doctor may administer corticosteroids (methylprednisolone or prednisone) if a participant experiences immune reactions or other side effects.
Gene Therapy: Vtx-002 3 participants will receive dose 1 administered intra cisterna magna Dosing will be staggered at specific timepoints apart and with a safety monitoring committee review of health-related information in between each participant being dosed. VTx-002 is a single dose therapy Drug: Optional Rescue Medication: Methylprednisolone The study doctor may administer corticosteroids (methylprednisolone or prednisone) if a participant experiences immune reactions or other side effects.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Vector Y Therapeutics
Lead Sponsor
Citations
Study is to Assess the Safety and Tolerability of VTx-002 in ...
To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies. Change in Amyotrophic ...
FDA clears trial of 'vectorised' ALS antibody from VectorY
VTx-002 is not expected to be suitable for ALS patients whose disease is caused by SOD1 genetic mutations, who can be treated with Qalsody, as ...
3.
vectorytx.com
vectorytx.com/news/umc-utrecht-and-vectory-therapeutics-collaborate-in-als-biomarker-studyUMC Utrecht and VectorY Therapeutics collaborate in ALS ...
VectorY's lead program, VTx-002, is targeting TDP-43 for the treatment of ALS. ALS is a devastating condition that in the Western world has an ...
Amyotrophic lateral sclerosis: a neurodegenerative disorder ...
We review new insights into the pathophysiology of ALS which show promise for drug discovery. We describe advances in preclinical disease ...
FDA clears VectorY's PIONEER-ALS Phase I/II trial for ALS
VTx-002 is a vectorised antibody targeting transactive response DNA-binding protein 43 kDa (TDP-43) pathology in ALS. Go deeper with GlobalData.
VTx-002 - Disease modifying treatment for ALS
VTx-002 is a vectorized antibody that consists of a vector and the transgene that carries the instructions for a cell to make the therapeutic antibody.
VectorY Therapeutics Receives FDA Clearance of IND to ...
This IND clearance will make VTx-002 the first therapeutic in clinical development directed at the complete spectrum of TDP-43 pathology in ALS, ...
Vectory's VTX-002 gains IND clearance for ALS
In addition, VTX-002 has shown prevention of toxic aggregate formation, and transcriptomic restoration towards healthy motor neuron profiles.
Unbiased Results
We believe in providing patients with all the options.
Your Data Stays Your Data
We only share your information with the clinical trials you're trying to access.
Verified Trials Only
All of our trials are run by licensed doctors, researchers, and healthcare companies.