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Tests treatment safety and results for Amyotrophic Lateral Sclerosis
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?
VTx-002 is an investigational medicine, given as an injectable medicine, being studied as a potential treatment for amyotrophic lateral sclerosis.
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
- PIONEER-ALS is a Phase 1/2, multicenter, open-label, ascending dose, uncontrolled, first-in-human study that will evaluate the safety, tolerability and effects on clinical and biomarker endpoints of intracisternal administration of Vtx-002 in participants with Amyotrophic Lateral Sclerosis (ALS). Two escalating dose (low dose and high dose) cohorts are planned.
- 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; clinically probable...
- 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\. Significant concomitant...
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\. Significant concomitant disease or condition within 6 months of Screening that could pose an...
The study team makes the final eligibility decision.
Where it's taking place
- Phoenix, Arizona, United States
- San Diego, California, United States
- Jacksonville, Florida, United States
- Miami, Florida, United States
- Boston, Massachusetts, United States
- New York, New York, United States
- Philadelphia, Pennsylvania, United States
- Leuven, Belgium
- Utrecht, Netherlands
- London, United Kingdom
- Sheffield, United Kingdom
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
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 - 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?
Study sites include Phoenix, Arizona, United States; San Diego, California, United States; Jacksonville, Florida, United States; Miami, Florida, United States; Boston, Massachusetts, United States; New York, New York, United States and 5 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.