Actively Recruiting
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Led by Vector Y Therapeutics · Updated on 2026-05-14
12
Participants Needed
11
Research Sites
95 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Able and willing to provide written consent and follow study visits and procedures
- Male or female aged 18 years or older
- Diagnosed with ALS according to El Escorial criteria (probable or definite)
- No ALS caused by FUS or SOD1 gene mutations
- Symptoms of weakness appeared within the last 18 months
- Seated vital capacity of at least 80% predicted at screening
- TRICALS risk score between -2 and -6 at screening
- Has a reliable caregiver or legal representative to support participation
- Stable treatment with riluzole or edaravone before screening allowed
- Women of childbearing potential and males with partners who are WOCBP must use effective contraception
- Women not of childbearing potential must be post-menopausal or surgically sterile
You will not qualify if you...
- Other significant nervous system disease besides ALS
- Abnormal spinal, cervical, or brain MRI/MRA findings
- Presence of tracheostomy or feeding tube
- Contraindications to corticosteroids like osteoporosis or uncontrolled blood pressure
- Serious illness within 6 months that risks safety or study compliance
- Abnormal lab tests indicating liver, kidney problems, infections like Hepatitis or HIV
- Use of blood thinners that cannot be stopped safely before procedures
- Contraindications to MRI, MRA, CT imaging due to claustrophobia or contrast intolerance
- Contraindications to general anesthesia or deep sedation
- Positive test for illegal drugs (except allowed medications or responsible marijuana use)
- Generally frail or deemed unsuitable for study participation by investigator
- Other protocol-defined inclusion or exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 11 locations
1
St Joseph's Hospital and medical Center - Barrow Neurological Institute
Phoenix, Arizona, United States, 85013
Not Yet Recruiting
2
University of California San Diego Medical Center
San Diego, California, United States, 92121
Actively Recruiting
3
Mayo Clinic in Florida
Jacksonville, Florida, United States, 32224
Actively Recruiting
4
University of Miami School of Science
Miami, Florida, United States, 33136
Not Yet Recruiting
5
Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
6
Herbert Irving Comprehensive Cancer Center
New York, New York, United States, 10032
Not Yet Recruiting
7
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Not Yet Recruiting
8
UZ Leuven
Leuven, Belgium
Not Yet Recruiting
9
UMC Utrecht
Utrecht, Netherlands
Not Yet Recruiting
10
Kings College Hospital
London, United Kingdom, SE5 9RS
Not Yet Recruiting
11
Royal Hallamshire Hospital
Sheffield, United Kingdom, S10 2JF
Not Yet Recruiting
Research Team
D
Dr Olga Uspenskaya Chief medical Officer, VectorY Therapeutics, M.D; PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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