VTx-002를 투여받는 ALS 환자에서의 안전성 및 내약성 평가 연구 (ALS)
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
상태
정황
정황
개입 / 치료
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
연구 유형
등록 (추정된)
등록
단계
단계
- 2 단계
- 1단계
연락처 및 위치
연구 연락처
연구 연락처
- 이름: Dr Olga Uspenskaya Chief medical Officer, VectorY Therapeutics, M.D; PhD
- 전화번호: patients@vectorytx.com
- 이메일: patients@vectorytx.com
연구 장소
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Utrecht, 네덜란드
- 모병
- UMC Utrecht
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연락하다:
- Prof Leonard Van Den Berg, Doctor of Medicine
- 전화번호: +31 887555555
- 이메일: l.h.vandenberg@umcutrecht.nl
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수석 연구원:
- Prof Leonard Van Den Berg, Doctor of Medicine
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Arizona
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Phoenix, Arizona, 미국, 85013
- 아직 모집하지 않음
- St Joseph's Hospital and medical Center - Barrow Neurological Institute
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수석 연구원:
- Dr Shafeeq Ladha, Doctor of Medicine
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연락하다:
- Dr S Ladha, Doctor of Medicine
- 전화번호: (602) 406-6262
- 이메일: fulton.research@commonspirit.org
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California
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San Diego, California, 미국, 92121
- 모병
- University of California San Diego Medical Center
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연락하다:
- Dr John Ravits, Doctor of Medicine
- 전화번호: (858) 246-1154
- 이메일: jravits@health.ucsd.edu
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수석 연구원:
- Dr John Ravits, Doctor of Medicine
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Florida
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Jacksonville, Florida, 미국, 32224
- 모병
- Mayo Clinic in Florida
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연락하다:
- Dr Bjorn Oskarsson, Doctor of Medicine
- 전화번호: (904) 953-0407
- 이메일: oskarsson.bjorn@mayo.edu
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수석 연구원:
- Dr Bjorn Oskarsson, Doctor of Medicine
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Massachusetts
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Boston, Massachusetts, 미국, 02114
- 모병
- Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital
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수석 연구원:
- Dr Doreen Ho, Doctor of Medicine
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연락하다:
- Winifred Asigri Study Coordinator
- 전화번호: (617) 724-5659
- 이메일: pioneeralshealey@mgb.org
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New York
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New York, New York, 미국, 10032
- 아직 모집하지 않음
- The Eleanor & Lou Gehrig ALS Center
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수석 연구원:
- Dr Neil Schneider, Doctor of Medicine
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연락하다:
- The Eleanor & Lou Gehrig ALS Center
- 전화번호: (212) 305-6788
- 이메일: alsresearch@cumc.columbia.edu
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Pennsylvania
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Philadelphia, Pennsylvania, 미국, 19104
- 모병
- University of Pennsylvania
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수석 연구원:
- Dr Colin Quinn, Doctor of Medicine
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연락하다:
- Spriha Thapa Study Coordinator
- 전화번호: 267-610-8822
- 이메일: Spriha.Thapa@pennmedicine.upenn.edu
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Leuven, 벨기에
- 모병
- UZ Leuven
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연락하다:
- Evi Parente Study Coordinator
- 전화번호: + 32 1638668
- 이메일: evi.parente@uzleuven.be
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연락하다:
- Ann Dhondt Study Coordinator
- 전화번호: + 32 16347577
- 이메일: ann.dhondt@uzleuven.be
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수석 연구원:
- Dr Philip Van Damme, Doctor of Medicine
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Sheffield, 영국, S10 2JF
- 모병
- Royal Hallamshire Hospital
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연락하다:
- Dr Chris McDermott, Doctor of medicine
- 전화번호: +44 114 226 1049
- 이메일: c.j.mcdermott@sheffield.ac.uk
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참여기준
자격 기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Key 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
- Male or female ≥ 18 years of age
- Has a diagnosis of ALS according to the El Escorial criteria (Brooks, et al., 2000) (probable, laboratory results supported; clinically probable, clinically definite)
- Confirmed absence of ALS caused by FUS and SOD1 gene mutations confirmed by laboratory tests.
- A maximum of 18 months since first appearance of weakness (e.g., limb weakness, dysarthria, dysphagia, shortness of breath)
- Erect (seated) SVC % predicted ≥ 80% at Screening
- Treatment Research Initiative to Cure ALS (TRICALS) risk score between -2 and -6 at Screening
- 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).
- 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
- 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
- Women of nonchildbearing potential must be post-menopausal or surgically sterile (e.g. hysterectomy, bilateral tubal ligation, ovaries removed)
Key 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
- Spinal, cervical, or brain MRI/MRA indicating clinically significant abnormality
- Presence of tracheostomy and feeding tube at Screening
- 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
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공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위화되지 않음
- 중재 모델: 순차적 할당
- 마스킹: 없음(오픈 라벨)
팔의 수
무기와 개입
참가자 그룹 / 팔참가자 그룹 / 팔 |
개입 / 치료개입 / 치료 |
|---|---|
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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 |
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.
다른 이름들:
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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 |
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.
다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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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
|
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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
|
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The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)
기간: At month 6 and month 12
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Assessed by performing 4 formal interim analyses
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At month 6 and month 12
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2차 결과 측정
2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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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
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Changes certain blood values |
At month 6 and month 12
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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
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Time to assisted permanent ventilation or death |
over 12 months
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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
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Immune responses assessed by analyzing blood samples |
Over 5 years
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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
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Change in Slow Vital Capacity |
At month 6 and month 12
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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
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Changes in CSF values |
At month 6 and month 12
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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.
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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
기타 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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To assess changes in biomarkers
기간: Over 5 years
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Analysis of blood samples to assess viral shedding |
Over 5 years
|
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To assess changes in muscle strength assessments from baseline.
기간: Measured at month 6 and month 12
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Change in muscle strength measured by Hand-held Dynamometry |
Measured at month 6 and month 12
|
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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
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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
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To assess changes Health Related Quality of Life
기간: Measured at month 6 and month 12
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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
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
연구 시작
기본 완료 (추정된)
기본 완료
연구 완료 (추정된)
연구 완료
연구 등록 날짜
최초 제출
최초 제출
QC 기준을 충족하는 최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
처음 게시됨
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
마지막 업데이트 게시됨
QC 기준을 충족하는 마지막 업데이트 제출
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
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?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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