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Studio per valutare la sicurezza e la tollerabilità di VTx-002 nei partecipanti con SLA (ALS)

5 novembre 2026 aggiornato da: Vector Y Therapeutics

Studio di Fase 1/2 su un Nuovo Regime Sperimentale nella Sclerosi Laterale Amiotrofica (Pioneer-ALS): Uno Studio Multicentrico, Aperto e Non Controllato per Valutare la Sicurezza e la Tollerabilità di Due Dosi di VTx-002

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.

Panoramica dello studio

Stato

Reclutamento

Condizioni

Intervento / Trattamento

Descrizione dettagliata

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.

Tipo di studio

Interventistico

Iscrizione (Stimato)

36

Fase

  • Fase 2
  • Fase 1

Contatti e Sedi

Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.

Contatto studio

  • Nome: Dr Olga Uspenskaya Chief medical Officer, VectorY Therapeutics, M.D; PhD
  • Numero di telefono: patients@vectorytx.com
  • Email: patients@vectorytx.com

Luoghi di studio

      • Leuven, Belgio
        • Reclutamento
        • UZ Leuven
        • Contatto:
        • Contatto:
        • Investigatore principale:
          • Dr Philip Van Damme, Doctor of Medicine
      • Utrecht, Olanda
        • Reclutamento
        • UMC Utrecht
        • Contatto:
        • Investigatore principale:
          • Prof Leonard Van Den Berg, Doctor of Medicine
      • Sheffield, Regno Unito, S10 2JF
        • Reclutamento
        • Royal Hallamshire Hospital
        • Contatto:
    • Arizona
      • Phoenix, Arizona, Stati Uniti, 85013
        • Non ancora reclutamento
        • St Joseph's Hospital and medical Center - Barrow Neurological Institute
        • Investigatore principale:
          • Dr Shafeeq Ladha, Doctor of Medicine
        • Contatto:
    • California
      • San Diego, California, Stati Uniti, 92121
        • Reclutamento
        • University of California San Diego Medical Center
        • Contatto:
        • Investigatore principale:
          • Dr John Ravits, Doctor of Medicine
    • Florida
      • Jacksonville, Florida, Stati Uniti, 32224
        • Reclutamento
        • Mayo Clinic in Florida
        • Contatto:
        • Investigatore principale:
          • Dr Bjorn Oskarsson, Doctor of Medicine
    • Massachusetts
      • Boston, Massachusetts, Stati Uniti, 02114
        • Reclutamento
        • Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital
        • Investigatore principale:
          • Dr Doreen Ho, Doctor of Medicine
        • Contatto:
    • New York
      • New York, New York, Stati Uniti, 10032
        • Non ancora reclutamento
        • The Eleanor & Lou Gehrig ALS Center
        • Investigatore principale:
          • Dr Neil Schneider, Doctor of Medicine
        • Contatto:
    • Pennsylvania
      • Philadelphia, Pennsylvania, Stati Uniti, 19104
        • Reclutamento
        • University of Pennsylvania
        • Investigatore principale:
          • Dr Colin Quinn, Doctor of Medicine
        • Contatto:

Criteri di partecipazione

I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.

Criteri di ammissibilità

Età idonea allo studio

18 anni e precedenti (Adulto, Adulto più anziano)

Accetta volontari sani

No

Sessi ammissibili allo studio

Tutto

Descrizione

Key Inclusion Criteria:

  1. Capable of, and willing to, provide written informed consent and comply with study procedures, including visits to the study site and visit requirements
  2. Male or female ≥ 18 years of age
  3. Has a diagnosis of ALS according to the El Escorial criteria (Brooks, et al., 2000) (probable, laboratory results supported; clinically probable, clinically definite)
  4. Confirmed absence of ALS caused by FUS and SOD1 gene mutations confirmed by laboratory tests.
  5. A maximum of 18 months since first appearance of weakness (e.g., limb weakness, dysarthria, dysphagia, shortness of breath)
  6. Erect (seated) SVC % predicted ≥ 80% at Screening
  7. Treatment Research Initiative to Cure ALS (TRICALS) risk score between -2 and -6 at Screening
  8. 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 progression prevents the participant of giving consent (local legal rules will apply).
  9. 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
  10. 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
  11. Women of nonchildbearing potential must be post-menopausal or surgically sterile (e.g. hysterectomy, bilateral tubal ligation, ovaries removed)

Key Exclusion Criteria:

  1. 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
  2. Spinal, cervical, or brain MRI/MRA indicating clinically significant abnormality
  3. Presence of tracheostomy and feeding tube at Screening
  4. 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 unacceptable safety risk to the participant or interfere with the participant's ability to comply with study procedures, e.g. heart disease, uncontrolled diabetes, liver disease, autoimmune diseases needing strong immune-suppressing drugs, cancer, etc or a current psychiatric diagnosis.

6. Clinically significant abnormalities in laboratory test results at Screening for example poor liver or kidney function, abnormal clotting or infections such as Hepatitis or HIV

7. Use of blood thinners (e.g., warfarin, heparin, and novel oral anticoagulants) and being unable to safely stop them before certain study procedures.

8. Contraindications to imaging methods MRI, MRA, CT due to claustrophobia and/or intolerance to contrast agents.

9. Contraindications to general anaesthesia (GA) or deep sedation

10 Positive test for illegal drugs (except prescribed medications or permitted medicinal/recreational marijuana if used responsibly)

11. Generally frail or if the Investigator deems participation in the study would not be in the best interest of the participant or is likely to prohibit further participation during the study

Other protocol-defined inclusion/exclusion criteria may apply

-

Piano di studio

Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.

Come è strutturato lo studio?

Dettagli di progettazione

  • Scopo principale: Trattamento
  • Assegnazione: Non randomizzato
  • Modello interventistico: Assegnazione sequenziale
  • Mascheramento: Nessuno (etichetta aperta)

Armi e interventi

Gruppo di partecipanti / Arm
Intervento / Trattamento
Sperimentale: Gene Therapy Group 1: Dose 1 (Low Dose)

Gene Therapy: VTx-002 18 participants will receive dose 1 administered intra cisterna magna. Dosing of the first 3 participants will be staggered at specific timepoints apart and with a safety monitoring committee review of health-related information in between each participant being dosed.

VTx-002 is a single dose therapy

Drug: Preventative (Prophylactic) Medication: Methylprednisolone

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.

Altri nomi:
  • Prednisolone
  • Prednisone
Sperimentale: Gene Therapy Group 2: Dose 2 (High Dose)

Gene Therapy: VTx-002 18 participants will receive dose 2 administered intra cisterna magna.

The participant dosing in group 2 will be staggered as it was in group 1. VTx-002 is a single dose therapy. Drug: Preventative (Prophylactic) Medication - Methylprednisolone

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.

Altri nomi:
  • Prednisolone
  • Prednisone

Cosa sta misurando lo studio?

Misure di risultato primarie

Misura del risultato
Misura Descrizione
Lasso di tempo
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Lasso di tempo: Over 5 years

Assessed by reviewing the nature, incidence, severity, relatedness, seriousness and outcome of treatment emergent adverse events.

Over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Lasso di tempo: Over 5 years

Assessed by reviewing laboratory values

Over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Lasso di tempo: Over 5 years

Assessed by reviewing Magnetic Resonance Imaging (MRI) findings.

Over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Lasso di tempo: over 5 years

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.

over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Lasso di tempo: Over 5 years

Assessed by reviewing cellular responses by analyzing blood samples.

Over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Lasso di tempo: Over 5 years

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.

Over 5 years
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
Lasso di tempo: At month 6 and month 12

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
At month 6 and month 12

Misure di risultato secondarie

Misura del risultato
Misura Descrizione
Lasso di tempo
To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies.
Lasso di tempo: At month 6 and month 12

Changes certain blood values

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.
Lasso di tempo: over 12 months

Time to assisted permanent ventilation or death

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.
Lasso di tempo: Over 5 years

Immune responses assessed by analyzing blood samples

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.
Lasso di tempo: At month 6 and month 12

Change in Slow Vital Capacity

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.
Lasso di tempo: At month 6 and month 12

Changes in CSF values

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.
Lasso di tempo: 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.

Change in Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)

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.

Altre misure di risultato

Misura del risultato
Misura Descrizione
Lasso di tempo
To assess changes in biomarkers
Lasso di tempo: Over 5 years

Analysis of blood samples to assess viral shedding

Over 5 years
To assess changes in muscle strength assessments from baseline.
Lasso di tempo: Measured at month 6 and month 12

Change in muscle strength measured by Hand-held Dynamometry

Measured at month 6 and month 12
To assess disease severity and improvement
Lasso di tempo: over 12 months

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)

over 12 months
To assess immunogenicity of VTx002
Lasso di tempo: Over 5 years

Assessed by the review of the immunogenicity to the capsid and transgene by analyzing blood samples.

Over 5 years
To assess changes Health Related Quality of Life
Lasso di tempo: Measured at month 6 and month 12

Change in Health Related Quality of Life measured using the EQ5D-L questionnaire.

*The EQ5D-L is a self-completed questionnaire assessing five core dimensions (Mobility, Self-care, Usual Activities, Pain/Discomfort, Anxiety/Depression). It is scored by combining patient responses across the five dimensions and then converting to a single utility score whereby 1.0 indicates perfect health and 0 indicates death

Measured at month 6 and month 12
To assess changes Health Related Quality of Life
Lasso di tempo: Measured at month 6 and month 12

Change in Health Related Quality of Life measured using the ALSAQ-5 questionnaire.

*The ALSAQ-5 is a brief, 5-item questionnaire used to measure the impact of Amyotrophic Lateral Sclerosis on a patient's quality of life. Five key areas are rated for difficulties and converted to a total score from 0 (worst) to 100 (best)

Measured at month 6 and month 12

Collaboratori e investigatori

Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.

Sponsor

Studiare le date dei record

Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.

Studia le date principali

Inizio studio (Effettivo)

19 dicembre 2025

Completamento primario (Stimato)

15 ottobre 2027

Completamento dello studio (Stimato)

15 ottobre 2027

Date di iscrizione allo studio

Primo inviato

19 novembre 2025

Primo inviato che soddisfa i criteri di controllo qualità

11 dicembre 2025

Primo Inserito (Effettivo)

17 dicembre 2025

Aggiornamenti dei record di studio

Ultimo aggiornamento pubblicato (Effettivo)

6 novembre 2026

Ultimo aggiornamento inviato che soddisfa i criteri QC

5 novembre 2026

Ultimo verificato

1 settembre 2026

Maggiori informazioni

Termini relativi a questo studio

Parole chiave

Altri numeri di identificazione dello studio

  • VTx-002-01-001
  • 2025-522697-37-00 (Ctis)
  • PIONEER-ALS (Altro identificatore: VectoryTherapeutics)
  • 2025 (Sovvenzione/contratto NIH degli Stati Uniti)

Piano per i dati dei singoli partecipanti (IPD)

Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?

NO

Informazioni su farmaci e dispositivi, documenti di studio

Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti

Sì

Studia un dispositivo regolamentato dalla FDA degli Stati Uniti

No

Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .