Onasemnogene Abeparvovec-xioi를 투여받은 환자의 장기 추적 연구
AVXS-101을 투여받은 척수근위축 임상시험 환자의 장기추적 연구
This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.
연구 개요
연구 유형
등록 (실제)
단계
- 3단계
연락처 및 위치
연구 장소
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Taipei, 대만, 10048
- National Taiwan University Hospital
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California
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Palo Alto, California, 미국, 94304
- Stanford University Medical Center
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Colorado
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Aurora, Colorado, 미국, 80045
- Children's Hospital Colorado
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Illinois
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Chicago, Illinois, 미국, 60611
- Ann Robert H. Lurie Children's Hospital of Chicago
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Maryland
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Baltimore, Maryland, 미국, 21287
- John Hopkins Hospital - David M. Rubenstein Child Health Building
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Massachusetts
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Boston, Massachusetts, 미국, 02114
- Massachusetts General Hospital
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Boston, Massachusetts, 미국, 02115
- Boston Children's Hospital
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Michigan
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Grand Rapids, Michigan, 미국, 49503
- Spectrum Health Hospitals Helen DeVos Children's Hospital
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Missouri
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St Louis, Missouri, 미국, 63110
- Washington Unviersity School of Medicine in Saint Louis
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New York
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New York, New York, 미국, 10032
- Columbia University Medical Center
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North Carolina
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Durham, North Carolina, 미국, 27713
- Duke University
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Ohio
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Columbus, Ohio, 미국, 43205
- Nationwide Children's Hospital
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Pennsylvania
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Philadelphia, Pennsylvania, 미국, 19104
- Children's Hospital of Philadelphia
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Strasburg, Pennsylvania, 미국, 17579
- Clinic for Special Children
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Texas
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Dallas, Texas, 미국, 75235
- Children's Health Specialty Center Dallas Campus
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Utah
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Salt Lake City, Utah, 미국, 84112
- University of Utah Health
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Virginia
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Norfolk, Virginia, 미국, 23507
- Children's Hospital of The King's Daughters
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Richmond, Virginia, 미국, 23298
- Virginia Commonwealth University
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Wisconsin
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Madison, Wisconsin, 미국, 53792
- University of Wisconsin, Madison
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Ghent, 벨기에, 9000
- Universitair Ziekenhuis Gent
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Liège
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Liège, Liège, 벨기에, B-4000
- Centre de Référence des Maladies Neuromusculaires
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Newcastle upon Tyne, 영국, NE1 4LP
- The Newcastle Upon Tyne Hospitals NHS Foundation Trust
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London
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London, London, 영국, WC1N 3JH
- Great Ormond Street Hospital for Children NHS Foundation Trust
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Genova, 이탈리아, 16147
- Instituto Gianninia Gaslini
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Milan, 이탈리아, 20133
- Istituto Neurologico di Ricerca
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Milan, 이탈리아, 20122
- Universita Degli Studi Di Milano
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Roma
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Roma, Roma, 이탈리아, 00168
- Fondazione Policlinico Universitario Agostino Gemelli
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Tokyo, 일본, 162-8666
- Tokyo Women's Medical University Hospital
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Ontario
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Ottawa, Ontario, 캐나다, K1H8L1
- Children's Hospital of Eastern Ontario Research Institute
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Paris, 프랑스, 75012
- Hôpital Armand Trousseau
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New South Wales
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Randwick, New South Wales, 호주, 2145
- Sydney Children's Hospital
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참여기준
자격 기준
공부할 수 있는 나이
- 어린이
- 성인
- 고령자
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
Inclusion Criteria:
- Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement therapy in a Novartis Gene Therapies-sponsored clinical study
- Participant/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule
Exclusion Criteria:
- Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 해당 없음
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
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다른: Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec-xioi
Participants received treatment with IV onasemnogene abeparvovec-xioi in an onasemnogene abeparvovec-xioi or received treatment with IT onasemnogene abeparvovec-xioi in an onasemnogene. |
Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the cytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec-xioi administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.
다른 이름들:
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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높이 측정의 기준선에서 변경
기간: 최대 5년
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최대 5년
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체중 측정의 기준선에서 변경
기간: 최대 5년
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최대 5년
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전체적으로 병용 약물을 사용하는 참가자 수 및 약물 유형별
기간: 최대 5년
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최대 5년
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기타 SMA 요법 전체 및 약물 유형별 참여자 수
기간: 6학년 ~ 15학년
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6학년 ~ 15학년
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Number of Participants Who Reach Developmental Milestones
기간: Up to 5 years
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Assessed via the developmental milestone checklist, formed of 10 yes/no questions. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone. |
Up to 5 years
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Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
기간: Up to 5 years
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The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments rated from 0 (unable to perform functional task) to 2 (able to perform functional task unassisted). Higher scores on the total scale of 0-66 indicates higher levels of motor ability. |
Up to 5 years
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Pulmonary Assessment Results and Require Ventilatory Support
기간: Up to 15 years
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Participants will receive pulmonary assessments by a pulmonologist or appropriate clinician. Respiratory device data will be reviewed for participants receiving non-invasive ventilatory support. |
Up to 15 years
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Number of Participants Who Experience Swallowing Dysfunction and Require Nutritional Support
기간: Up to 5 years
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Assessed via the swallowing function questionnaire, formed of 4 yes/ no questions and 1 body weight question. |
Up to 5 years
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Number of Participants Who Experience a Clinically Significant Change from Baseline in Physical Examination Findings
기간: Up to 5 years
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The physical examination includes review of the following systems: head, ears, eyes, nose and throat, lungs/thorax, cardiovascular, abdomen, musculoskeletal, neurologic, dermatologic, lymphatic, and genitourinary. In addition, visual inspection of the spine, back, shoulders, and hips looking for spinal curvature and asymmetry will be carried out. Joints will be assessed for loss of mobility and contractures. |
Up to 5 years
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Vital Signs Measurements
기간: Up to 5 years
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Vital sign measurements will include blood pressure, respiratory rate, pulse, axillary temperature, and pulse oximetry. |
Up to 5 years
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Clinical Laboratory Assessments
기간: Up to 5 years
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Blood samples will be collected for hematology (including complete blood cell count) and chemistry. |
Up to 5 years
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Cardiac Assessments
기간: Up to 5 years
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Cardiac assessments will include a 12-lead electrocardiogram, transthoracic echocardiogram and Troponin-I. |
Up to 5 years
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Observational Phase Questionnaire Results
기간: Year 6 to Year 15
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The observational phase questionnaire includes 7 yes/no questions. Observation categories include: adverse events, hospitalizations, concomitant medications, ventilatory support and feeding support. |
Year 6 to Year 15
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Number of Participants Who Experience at Least One Serious Adverse Event (SAE)
기간: Up to 15 years
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An SAE is defined as any adverse event (appearance of [or worsening of any pre existing]) undesirable sign(s), symptom(s), or medical conditions(s) which meets any one of the following criteria:
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Up to 15 years
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Number of Participants Who Experience at Least One Adverse Event of Special Interest (AESI)
기간: Up to 15 years
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An AESI is defined as an AE occurring during any study phase that fulfills one of the following criteria:
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Up to 15 years
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Change From Baseline in Bayley Scales of Infant and Toddler Development
기간: Up to 42 months, 15 days of age
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Third Edition (Bayley-III) to be performed in all patients up to 42 months, 15 days of age. |
Up to 42 months, 15 days of age
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Change From Baseline in Revised Upper Limb Module (RULM) Score
기간: Up to 5 years
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RULM score is based on a scale from 0 to 37 where lower scores reflect poorer upper limb functional ability. |
Up to 5 years
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Change From Baseline in Cogstate Computerized Cognitive Battery Performed in Age 48 Months and Older
기간: Up to 5 years
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The Cogstate Computerized Cognitive Battery consists of the Identification Test (scored 0 (best) to 1.5708 (worst)), the International Shopping List Test (scored 0 (worst) to 999 (best)), the International Shopping List Test-Delayed Recall (scored 0 (worst) to 999 (best)), the One Card Learning Test (scored 0 (worst) to 1.5708 (best)), and the One Back Test (scored 0 (worst) to 1.5708 (best)). |
Up to 5 years
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Change From Baseline in Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) Performed in All Participants 5 to 21 Years of Age
기간: Up to 5 years
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The CELF-5 Following Directions and Sentence Repetition subtests use scoring that varies based on age, but will be administered to participants 5-21 years of age. The Following Directions subtest will be scored from 0-33 with higher score being more advanced and the Recalling Sentences subtest will be scored from 0-78 with higher score being more advanced. |
Up to 5 years
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Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND)
기간: Up to 5 years
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ACEND score is based on a scale from 1 to 41 where higher scores represent a better caregiver experience |
Up to 5 years
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공동 작업자 및 조사자
수사관
- 연구 의자: Sitra Tauscher-Wisniewski, MD, Novartis Gene Therapies, Inc.
간행물 및 유용한 링크
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (추정된)
연구 완료 (추정된)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
- AVXS-101-LT-002
- 2019-002611-26 (EudraCT 번호)
- 205305 (기타 식별자: JapicCTI)
- COAV101A12103 (기타 식별자: Novartis Pharmaceuticals)
- 2019 (미국 NIH 보조금/계약)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
IPD 계획 설명
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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