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.
All participants will receive a single injection of the study drug. During the first year of the study there will be 12 visits to the study center, including an overnight stay after dosing of at least 1 night. There will be a further 4 remote visits (telephone or video call). From Year 2-5 there will be 8 further visits. These will be every 6 months and will be either in-person at the study site or remote (telephone or video call) if needed or preferred. Throughout the 5-year observation period, there will be up to 20 study visits to complete follow-up tests and assessments and monitor the ongoing effects of the study drug.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
12
An investigational gene therapy targeting a specific protein.
To reduce the risk of reactions caused by the study treatment, steroid medicines will be given in advance.
St Joseph's Hospital and medical Center - Barrow Neurological Institute
Phoenix, Arizona, United States
NOT_YET_RECRUITINGUniversity of California San Diego Medical Center
San Diego, California, United States
RECRUITINGMayo Clinic in Florida
Jacksonville, Florida, United States
RECRUITINGUniversity of Miami School of Science
Miami, Florida, United States
NOT_YET_RECRUITINGSean M. Healey & AMG Center for ALS at Massachusetts General Hospital
Boston, Massachusetts, United States
RECRUITINGHerbert Irving Comprehensive Cancer Center
New York, New York, United States
NOT_YET_RECRUITINGUniversity of Pennsylvania
Philadelphia, Pennsylvania, United States
RECRUITINGUZ Leuven
Leuven, Belgium
RECRUITINGUMC Utrecht
Utrecht, Netherlands
RECRUITINGKings College Hospital
London, United Kingdom
NOT_YET_RECRUITING...and 1 more locations
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.
Time frame: Over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Assessed by reviewing laboratory values
Time frame: Over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Assessed by reviewing Magnetic Resonance Imaging (MRI) findings.
Time frame: Over 5 years
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.
Time frame: over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Assessed by reviewing cellular responses by analyzing blood samples.
Time frame: Over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Assessed by reviewing scores of the Columbia Suicide Severity Rating Scale (C-SSRS) \*C-SSRS is a widely used, evidence-based suicide risk assessment tool that helps identify whether someone is at risk for suicide and gauge the level of support needed. A low score is associated with a lower risk level in this brief questionnaire.
Time frame: Over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Assessed by performing 4 formal interim analyses 1. 6 months after Gene Therapy Group 1 receive the single dose treatment 2. 6 months after Gene Therapy Group 2 receive the single dose treatment 3. 12 months after Gene Therapy Group 1 receive the single dose treatment 4. 12 months after Gene Therapy Group 2 receive the single dose treatment
Time frame: At month 6 and month 12
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
Time frame: At month 6 and month 12
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
Time frame: over 12 months
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
Time frame: Over 5 years
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
Time frame: At month 6 and month 12
To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies.
Changes in CSF values
Time frame: At month 6 and month 12
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 Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
Time frame: At month 6 and month 12 *ALSFRS-R is a scale used to monitor disease progression. The 12 questions. Questions are scored from 0 to 4, with a maximum score of 48 indicating full function and a minimum score of 0 indicating significant impairment.
Dr Olga Uspenskaya Chief medical Officer, VectorY Therapeutics, M.D; PhD
CONTACT
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