Amyotrophic Lateral Sclerosis study: VTx-002 vs Preventative
Official title Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
ClinicalTrials.gov ID: NCT07287397
What this study is testing
What is VTx-002?
An investigational gene therapy being studied as a potential treatment for Amyotrophic Lateral Sclerosis (ALS).
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study will look at how safe and well tolerated VTx‑002 is and whether it changes clinical signs or biomarkers in ALS patients.
- Who it's for
- Adults who have been diagnosed with amyotrophic lateral sclerosis (ALS).
- What's involved
- Participants will receive VTx‑002 in two dose levels, with visits for medical checks and tests to monitor safety and disease markers.
- Time commitment
- The study can last up to about 5 years and 5 weeks, with a screening period of up to 5 weeks before treatment starts.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Capable of, and willing to, provide written informed consent and comply with study procedures, including visits to the study site and visit...
- Male or female ≥ 18 years of age
- Has a diagnosis of ALS according to the El Escorial criteria (Brooks, et al., 2000) (probable, laboratory results supported
- Confirmed absence of ALS caused by FUS and SOD1 gene mutations confirmed by laboratory tests.
- A maximum of 18 months since first appearance of weakness (e.g., limb weakness, dysarthria, dysphagia, shortness of breath)
You likely can't join if
- Diagnosis of a significant CNS or peripheral nervous system disease other than ALS that may be a cause for the participant's ALS symptoms or may...
- Spinal, cervical, or brain MRI/MRA indicating clinically significant abnormality
- Presence of tracheostomy and feeding tube at Screening
- Contraindications to corticosteroid use (e.g. due to osteoporosis, uncontrolled blood pressure, diabetes or cholesterol). 5\.
See the full eligibility criteria
- Capable of, and willing to, provide written informed consent and comply with study procedures, including visits to the study site and visit requirements
- Male or female ≥ 18 years of age
- Has a diagnosis of ALS according to the El Escorial criteria (Brooks, et al., 2000) (probable, laboratory results supported; clinically probable, clinically definite)
- Confirmed absence of ALS caused by FUS and SOD1 gene mutations confirmed by laboratory tests.
- A maximum of 18 months since first appearance of weakness (e.g., limb weakness, dysarthria, dysphagia, shortness of breath)
- Erect (seated) SVC % predicted ≥ 80% at Screening
- Treatment Research Initiative to Cure ALS (TRICALS) risk score between -2 and -6 at Screening
- Has a reliable caregiver/partner/legal representative willing and able to support the participant in participation in the study and to give informed consent on behalf of the participant in the case that disease...
- Treatment with riluzole and/or edaravone is allowed if treatment was started and has remained at a stable dose for at least 2 weeks (riluzole) or one treatment cycle (edaravone) before the Screening visit
- Women of childbearing potential (WOCBP) and male participants with female partners who are WOCBP must agree to use highly effective contraception during and after the study. WOCBP cannot be pregnant or breastfeeding
- Women of nonchildbearing potential must be post-menopausal or surgically sterile (e.g. hysterectomy, bilateral tubal ligation, ovaries removed) Key
- Diagnosis of a significant CNS or peripheral nervous system disease other than ALS that may be a cause for the participant's ALS symptoms or may confound study objectives
- Spinal, cervical, or brain MRI/MRA indicating clinically significant abnormality
- Presence of tracheostomy and feeding tube at Screening
- Contraindications to corticosteroid use (e.g. due to osteoporosis, uncontrolled blood pressure, diabetes or cholesterol). 5\.
The study team makes the final eligibility decision.
Where it is enrolling
- San Diego, California, United States
- Jacksonville, Florida, United States
- Boston, Massachusetts, United States
- Philadelphia, Pennsylvania, United States
- Leuven, Belgium
- Utrecht, Netherlands
- Sheffield, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at San Diego, California, United States; Jacksonville, Florida, United States; Boston, Massachusetts, United States; Philadelphia, Pennsylvania, United States; Leuven, Belgium; Utrecht, Netherlands and 1 more location. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.