2025-522697-37-00
Recruiting
Phase 2
Phase 1/2 Investigation Of Novel Experimental Regimen in Amyotrophic Lateral Sclerosis (PIONEER-ALS): An Open-Label, Uncontrolled, Multicenter Study to Assess the Safety and Tolerability of Two Doses of VTx-002.
VectorY Therapeutics B.V.6 sites in 3 countries4 target enrollmentStarted: April 30, 2026Last updated:
ConditionsAmyotrophic Lateral Sclerosis
Overview
- Phase
- Phase 2
- Status
- Recruiting
- Sponsor
- VectorY Therapeutics B.V.
- Enrollment
- 4
- Locations
- 6
- Primary Endpoint
- Nature, incidence, severity, relatedness, seriousness, and outcome of treatment-emergent adverse events (TEAEs) including: o Laboratory values o Magnetic resonance imaging (MRI) findings o Treatment-induced Peripheral Neuropathy Assessment Scale (TNAS) o Assessment of cellular responses to both the vector and the transgene encoded protein o Columbia Suicide Severity Rating Scale (C-SSRS)
Overview
Brief Summary
To assess the safety and tolerability of increasing doses of a single administration of VTx-002
Eligibility Criteria
- Ages
- 18 years to 65+ years (65+ Years, 18-64 Years)
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Capable of, and willing to, provide written informed consent and comply with study procedures, including visits to the study site and visit requirements
- •Women of childbearing potential (WOCBP) and male participants with female partners who are WOCBP (based on gender assignation at birth) must agree to use highly effective (<1% failure rate) contraception for at least 30 days prior to the first dose of study medication, during the study, and for 90 days following end of study [EOS] including the long term follow-up. Male participants (based on gender assignation at birth) must refrain from sperm donation for the duration of the study and for 90 days after EOS including the long term follow-up; WOCBP (based on gender assignation at birth) must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline (Day 1)
- •Women of nonchildbearing potential must be either surgically sterile (hysterectomy, bilateral tubal ligation, salpingectomy, and/or bilateral oophorectomy at least 26 weeks before Screening) or postmenopausal, defined as spontaneous amenorrhea for at least 2 years, with follicle-stimulating hormone (FSH) level in the postmenopausal range at Screening based on the central laboratory's range
- •Men and WOCBP (i.e., ovulating, premenopausal, and not surgically sterile) must use a highly effective method of contraception consistently and correctly for the duration of the study including the long- term follow-up. Highly effective methods of contraception are those that, alone or in combination, result in a failure rate of less than 1% per year when used consistently and correctly (i.e., perfect use) o Acceptable forms of contraception for participating WOCBP include the following: ▪ Combined (estrogen and progestogen containing) oral, intravaginal, or transdermal hormonal contraception associated with inhibition of ovulation ▪ Oral, injectable, or implantable progestogen-only hormonal contraception associated with inhibition of ovulation ▪ Intrauterine device ▪ Intrauterine hormone-releasing system ▪ Bilateral tubal ligation or bilateral tubal occlusion (performed at least 3 months prior to Screening) ▪ Vasectomized partner (performed at least 3 months prior to Screening) ▪ Sexual abstinence (no sexual intercourse) o Acceptable forms of contraception for male participants include: ▪ Sexual abstinence (no sexual intercourse) ▪ History of vasectomy (performed at least 3 months prior to Screening) plus external condom ▪ Condom with spermicide used together with highly effective female contraceptive methods if the female partner(s) is of childbearing potential (see above for list of acceptable female contraceptive methods)
- •Women who are in exclusively same-sex relationships (as their preferred and usual lifestyle) are not required to use contraception; male participants in same sex relationships are also required to use an external condom to prevent exposure of their partner to the IMP
- •Men must agree to abstain from sperm donation for the duration of the study, including long-term follow-up and are required to use an external condom when engaging in any activity that allows for passage of ejaculate to another person during the main study period
- •Women must agree to abstain from egg donation for the duration of the study, including long-term follow-up
- •Women of childbearing potential cannot be pregnant or lactating/breastfeeding and must have a negative result for the serum pregnancy test (P-human chorionic gonadotropin [P-HCG]) at Screening
- •Must have pneumococcal pneumonia and shingles vaccination coverage within 10 years prior to Screening or consent to these vaccinations to be performed during the Screening period
- •Up to date with age and gender appropriate cancer screening as per local standard of care based on Principal Investigator's (PI) judgment
Exclusion Criteria
- •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
- •Any other abnormal Screening laboratory test result deemed clinically significant by the Investigator
- •Any type of prior gene or cell therapy
- •Immunizations (live vaccines) in the 4 weeks prior to Screening. Note: Pneumococcal and shingles vaccine administrations are allowed during the Screening period
- •Pregnant or breastfeeding
- •Use of blood thinners (e.g., warfarin, heparin, and novel oral anticoagulants) in the 2 weeks prior to Screening LP or (ICM) procedure, or the anticipated need to initiate blood thinners during the study. Antiplatelet therapies (e.g., prophylactic aspirin, clopidogrel) are acceptable if the participant is medically able to temporarily stop 48 hours to 7 days (depending on the antiplatelet medication used) prior to and at least 48 hours after intracisternal injection and LP
- •Contraindications or intolerance to imaging methods (MRI, MRA, computed tomography [CT]) inducing claustrophobia and/or intolerance to contrast agents used for MRI, MRA, or CT (including but not limited to gadolinium contrast agents)
- •Contraindications to general anesthesia (GA) or deep sedation
- •Positive urine test for drugs of abuse (including opiates, benzodiazepines, amphetamines, cocaine, barbiturates, and phencyclidine) without prescription and without a clear justification of the results (determined by the Investigator) at Screening and Day
- •Note: use of medical marijuana is permitted provided that the participant is on a stable regimen. It is also permitted if the participant resides in a state in which the recreational use of marijuana is legalized, so long as the participant does not meet the drug abuse criteria (as defined in the DSM-5)
Arms & Interventions
VTx-002 Diluent
Auxiliary
Intervention: VTx-002 Diluent (Drug)
VTx-002, VTx-002
Test
Intervention: VTx-002 (Drug)
Outcomes
Primary Outcomes
Nature, incidence, severity, relatedness, seriousness, and outcome of treatment-emergent adverse events (TEAEs) including: o Laboratory values o Magnetic resonance imaging (MRI) findings o Treatment-induced Peripheral Neuropathy Assessment Scale (TNAS) o Assessment of cellular responses to both the vector and the transgene encoded protein o Columbia Suicide Severity Rating Scale (C-SSRS)
Nature, incidence, severity, relatedness, seriousness, and outcome of treatment-emergent adverse events (TEAEs) including: o Laboratory values o Magnetic resonance imaging (MRI) findings o Treatment-induced Peripheral Neuropathy Assessment Scale (TNAS) o Assessment of cellular responses to both the vector and the transgene encoded protein o Columbia Suicide Severity Rating Scale (C-SSRS)
Secondary Outcomes
- Time to permanent assisted ventilation, or death over 12 months
- Change in revised ALS Functional Rating Scale (ALSFRS-R) over 6 and 12 months
- Change in slow vital capacity (SVC) over 6 and 12 months
- Survival
- Immunogenicity against adeno-associated virus (AAV) capsid
Investigators
CMO office
Scientific
VectorY Therapeutics B.V.
Study Sites (6)
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