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VTx-002를 투여받는 ALS 환자에서의 안전성 및 내약성 평가 연구 (ALS)

2026년 11월 5일 업데이트: Vector Y Therapeutics

근위축성측삭경화증에 대한 새로운 실험적 요법의 1/2상 연구(Pioneer-ALS): VTx-002의 두 용량에 대한 안전성과 내약성을 평가하기 위한 개방형, 무대조군, 다기관 연구

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.

연구 개요

상태

모병

정황

개입 / 치료

상세 설명

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.

연구 유형

중재적

등록 (추정된)

36

단계

  • 2 단계
  • 1단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 연락처

  • 이름: Dr Olga Uspenskaya Chief medical Officer, VectorY Therapeutics, M.D; PhD
  • 전화번호: patients@vectorytx.com
  • 이메일: patients@vectorytx.com

연구 장소

  • 네덜란드
      • Utrecht, 네덜란드
        • 모병
        • UMC Utrecht
        • 연락하다:
        • 수석 연구원:
          • Prof Leonard Van Den Berg, Doctor of Medicine
  • 미국
    • Arizona
      • Phoenix, Arizona, 미국, 85013
        • 아직 모집하지 않음
        • St Joseph's Hospital and medical Center - Barrow Neurological Institute
        • 수석 연구원:
          • Dr Shafeeq Ladha, Doctor of Medicine
        • 연락하다:
    • California
      • San Diego, California, 미국, 92121
        • 모병
        • University of California San Diego Medical Center
        • 연락하다:
        • 수석 연구원:
          • Dr John Ravits, Doctor of Medicine
    • Florida
      • Jacksonville, Florida, 미국, 32224
        • 모병
        • Mayo Clinic in Florida
        • 연락하다:
        • 수석 연구원:
          • Dr Bjorn Oskarsson, Doctor of Medicine
    • Massachusetts
      • Boston, Massachusetts, 미국, 02114
        • 모병
        • Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital
        • 수석 연구원:
          • Dr Doreen Ho, Doctor of Medicine
        • 연락하다:
    • New York
      • New York, New York, 미국, 10032
        • 아직 모집하지 않음
        • The Eleanor & Lou Gehrig ALS Center
        • 수석 연구원:
          • Dr Neil Schneider, Doctor of Medicine
        • 연락하다:
    • Pennsylvania
      • Philadelphia, Pennsylvania, 미국, 19104
        • 모병
        • University of Pennsylvania
        • 수석 연구원:
          • Dr Colin Quinn, Doctor of Medicine
        • 연락하다:
  • 벨기에
      • Leuven, 벨기에
        • 모병
        • UZ Leuven
        • 연락하다:
        • 연락하다:
        • 수석 연구원:
          • Dr Philip Van Damme, Doctor of Medicine
  • 영국
      • Sheffield, 영국, S10 2JF
        • 모병
        • Royal Hallamshire Hospital
        • 연락하다:

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

18년 이상 (성인, 고령자)

건강한 자원 봉사자를 받아들입니다

아니

연구 대상 성별

모두

설명

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

-

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 무작위화되지 않음
  • 중재 모델: 순차적 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: 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
유전적: VTx-002

An investigational gene therapy targeting a specific protein.

의약품: Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone

To reduce the risk of reactions caused by the study treatment, steroid medicines will be given in advance.

다른 이름들:
  • 프레드니솔론
  • 프레드니손
실험적: 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
유전적: VTx-002

An investigational gene therapy targeting a specific protein.

의약품: Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone

To reduce the risk of reactions caused by the study treatment, steroid medicines will be given in advance.

다른 이름들:
  • 프레드니솔론
  • 프레드니손

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
기간: 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)
기간: 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)
기간: 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)
기간: 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)
기간: 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)
기간: 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)
기간: 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

2차 결과 측정

결과 측정
측정값 설명
기간
To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies.
기간: 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.
기간: 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.
기간: 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.
기간: 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.
기간: 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.
기간: 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.

기타 결과 측정

결과 측정
측정값 설명
기간
To assess changes in biomarkers
기간: Over 5 years

Analysis of blood samples to assess viral shedding
Over 5 years
To assess changes in muscle strength assessments from baseline.
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Vector Y Therapeutics

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2025년 12월 19일

기본 완료 (추정된)

2027년 10월 15일

연구 완료 (추정된)

2027년 10월 15일

연구 등록 날짜

최초 제출

2025년 11월 19일

QC 기준을 충족하는 최초 제출

2025년 12월 11일

처음 게시됨 (실제)

2025년 12월 17일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 11월 6일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 11월 5일

마지막으로 확인됨

2026년 9월 1일

추가 정보

이 연구와 관련된 용어

키워드

추가 관련 MeSH 약관

기타 연구 ID 번호

  • VTx-002-01-001
  • 2025-522697-37-00 (씨티스)
  • PIONEER-ALS (기타 식별자: VectoryTherapeutics)
  • 2025 (미국 NIH 보조금/계약)

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

아니요

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

구독하다