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. The duration of the study will be a maximum of 5 years and 5 weeks (265 weeks) for each participant. The screening period may last up to 5 weeks to complete screening procedures.
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.
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).
Sequential
VTx-002
GeneticAn investigational gene therapy targeting a specific protein.
Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone
DrugTo reduce the risk of reactions caused by the study treatment, steroid medicines will be given in advance.
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Assessed by reviewing the nature, incidence, severity, relatedness, seriousness and outcome of treatment emergent adverse events.
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Assessed by reviewing laboratory values
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Assessed by reviewing Magnetic Resonance Imaging (MRI) findings.
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Assessed by review of Treatment Induced Peripheral Neuropathy Assessment Scale (TNAS) \*TNAS a brief participant-reported questionnaire used to measure the severity and progression of peripheral neuropathy. Each item is rated on a scale of 0 to 10, where 0 means no symptom and 10 means the symptom is as bad as it can possibly be.
To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies.
Changes certain blood values
To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies.
Time to assisted permanent ventilation or death
To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies.
Immune responses assessed by analyzing blood samples
To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies.
Change in Slow Vital Capacity
To assess changes in biomarkers
Analysis of blood samples to assess viral shedding
To assess changes in muscle strength assessments from baseline.
Change in muscle strength measured by Hand-held Dynamometry
To assess disease severity and improvement
Disease severity and improvement measured using the Clinical Global Impression (CGI) scales for Severity (CGI-S) and Improvement (CGI-I). The CGI scales each use a seven-point scale where 1 indicates less severe (CGI-S) or most improved (CGI-I) and 7 indicates more severe illness (CGI-S) or worsening of condition (CGI-I)
To assess immunogenicity of VTx002
Assessed by the review of the immunogenicity to the capsid and transgene by analyzing blood samples.
Click on the lead sponsor to view all of their trials.
