Krabbe 환자에서 FBX-101의 안전성과 유효성을 평가하기 위한 장기 추적 연구
FBX-101(AAVrh.10-hGALC) 투여를 포함하는 유전자 치료 임상 시험에서 크라베 환자의 안전성과 유효성을 평가하기 위한 장기 추적 연구
This is an observational study that will enroll any patients with Krabbe disease that have participated in prior interventional clinical trials involving the administration of FBX-101.
연구 개요
상세 설명
FBX-101-LTFU is a multicenter, non-interventional, Long-Term Follow-Up (LTFU) study of participants from prior interventional trials involving the administration of FBX-101. Eligible participants will undergo clinical evaluations at prespecified intervals for at least 3 years from the last visit in the prior clinical trial (up to 5 years post-FBX-101 treatment). Overall safety and additional signs of efficacy will be collected with a series of laboratory tests, diagnostic tests, and performance surveys. Additionally, children participating in interventional trials that are terminated early will be transferred to this LTFU study and will complete any pending visits from the interventional trial before starting the clinical evaluations included in this protocol.
연구 유형
등록 (실제)
연락처 및 위치
연구 장소
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North Carolina
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Durham, North Carolina, 미국, 27705
- Duke University
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참여기준
자격 기준
공부할 수 있는 나이
- 어린이
- 성인
- 고령자
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
샘플링 방법
연구 인구
This is an observational long-term follow-up (LTFU) study of participants from prior interventional trials involving the administration of FBX-101.
설명
Inclusion Criteria:
- Participants that have completed a prior clinical trial involving the administration of FBX-101.
- Parent(s)/legal guardian(s) of participant willing and able to complete the informed consent process and comply with study procedures and visit schedule.
Exclusion Criteria:
•Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of FBX-101 during this study.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 관찰 모델: OTHER
- 시간 관점: PROSPECTIVE
코호트 및 개입
그룹/코호트 |
개입 / 치료 |
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Patients enrolled in the FBX-101-LTFU study
The participants will be followed for 36 months after they have concluded their participation in the interventional trial. They will complete 5 scheduled visits with assessments as specified in the schedule of assessments, to collect data for safety and additional signs of efficacy for FBX-101. Those patients enrolled from any other early terminated trial, will first complete pending evaluations from that trial. |
A replication-deficient adeno-associated virus gene transfer vector expressing the human galactocerebrosidase (hGALC) cDNA will be delivered one-time through a venous catheter inserted into a peripheral limb vein. |
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
기간 |
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FBX-101로 인한 심각한 유해 사례(SAE) 및 특별 관심 유해 사례(AESI)의 발생률로 평가된 장기 안전성
기간: 36개월
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36개월
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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가족 영향 모듈 및 다차원 피로 척도를 사용하는 소아 삶의 질(PedsQL) 일반 핵심 척도로 종단적으로 측정된 삶의 질 변화로 평가된 효능
기간: 36개월
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36개월
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특정 발달 이정표 설문지를 통해 종단적으로 측정된 언어 및 운동 기술과 같은 발달 이정표의 변화로 평가된 효율성
기간: 36개월
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36개월
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시각유발전위(VEP)에 의해 종방향으로 측정된 시각 기능의 변화로 평가된 효능
기간: 36개월
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36개월
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시력에 의해 종방향으로 측정된 시각 기능의 변화로 평가된 효능
기간: 36개월
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36개월
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전혈 공여자 키메라 현상의 변화로 평가한 효능
기간: 36개월
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36개월
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전혈 및 혈장 내 사이코신의 변화로 평가한 효능
기간: 36개월
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36개월
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행동 청력 측정법(BAUD)으로 종방향으로 측정한 청각 기능의 변화로 평가한 효능
기간: 36개월
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36개월
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신경 전도 속도(NCV) 평가에 의해 종방향으로 측정된 말초 신경 전도 속도의 변화로 평가된 효능
기간: 36개월
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36개월
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확산텐서영상(DTI)을 이용한 뇌자기공명영상(MRI)으로 종방향으로 측정한 부분이방성(FA)의 변화로 평가한 효능
기간: 36개월
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36개월
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혈장 및 뇌척수액(CSF)의 갈락토실세라미다제(GALC) 수준의 변화로 평가된 효능
기간: 36개월
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36개월
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Efficacy as assessed by change of gross motor function measured longitudinally by Peabody Developmental Motor Scale (PDMS-2)
기간: 36 months
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The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
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Efficacy as assessed by change of gross motor function measured longitudinally by Bruininks-Oseretsky Test of Motor Proficiency (BOT-2)
기간: 36 months
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The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
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Efficacy as assessed by change of gross motor function measured longitudinally by Gross Motor Function Measure 88 (GMFM-88)
기간: 36 months
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The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
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Efficacy as assessed by change of fine motor function measured longitudinally by Mullen Scales of Early Learning (MSEL)
기간: 36 months
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The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
|
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Efficacy as assessed by change of fine motor function measured longitudinally by Beery VMI Sixth Edition (VMI)
기간: 36 months
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The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
|
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Efficacy as assessed by change of fine motor function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)
기간: 36 months
|
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
|
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Efficacy as assessed by change of fine motor function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3)
기간: 36 months
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The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
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Efficacy as assessed by change of cognitive function measured longitudinally by Mullen Scales of Early Learning (MSEL)
기간: 36 months
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The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
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Efficacy as assessed by change of cognitive function measured longitudinally by the Differential Ability Scale II (DAS-II)
기간: 36 months
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The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
|
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Efficacy as assessed by change of cognitive function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)
기간: 36 months
|
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
|
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Efficacy as assessed by change of adaptive behaviour function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3).
기간: 36 months
|
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
|
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Efficacy as assessed by change of language function measured longitudinally by Mullen Scales of Early Learning (MSEL).
기간: 36 months
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The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
|
|
Efficacy as assessed by change of language function measured longitudinally by Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5)
기간: 36 months
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The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
|
|
Efficacy as assessed by change of language function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)
기간: 36 months
|
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
|
|
Efficacy as assessed by change of language function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3).
기간: 36 months
|
The site is providing raw data and sponsor is calculating derived scores according to specific manual. Higher values will mean better outcome. |
36 months
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Efficacy as assessed by change of hearing function measured longitudinally by Auditory Brainstem Responses (ABRs)
기간: 36 months
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Absolute values for Waves I, III and V in milliseconds will be recorded along interpretation and waveform morphology; and also Auditory Brainstem Responses (corrected). |
36 months
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공동 작업자 및 조사자
스폰서
간행물 및 유용한 링크
일반 간행물
- Escolar ML, Poe MD, Provenzale JM, Richards KC, Allison J, Wood S, Wenger DA, Pietryga D, Wall D, Champagne M, Morse R, Krivit W, Kurtzberg J. Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med. 2005 May 19;352(20):2069-81. doi: 10.1056/NEJMoa042604.
- Bascou N, DeRenzo A, Poe MD, Escolar ML. A prospective natural history study of Krabbe disease in a patient cohort with onset between 6 months and 3 years of life. Orphanet J Rare Dis. 2018 Aug 9;13(1):126. doi: 10.1186/s13023-018-0872-9.
- Beltran-Quintero ML, Bascou NA, Poe MD, Wenger DA, Saavedra-Matiz CA, Nichols MJ, Escolar ML. Early progression of Krabbe disease in patients with symptom onset between 0 and 5 months. Orphanet J Rare Dis. 2019 Feb 18;14(1):46. doi: 10.1186/s13023-019-1018-4.
- Yoon IC, Bascou NA, Poe MD, Szabolcs P, Escolar ML. Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease. Blood. 2021 Apr 1;137(13):1719-1730. doi: 10.1182/blood.2020005477.
- Wright MD, Poe MD, DeRenzo A, Haldal S, Escolar ML. Developmental outcomes of cord blood transplantation for Krabbe disease: A 15-year study. Neurology. 2017 Sep 26;89(13):1365-1372. doi: 10.1212/WNL.0000000000004418. Epub 2017 Aug 30.
- Gupta A, Poe MD, Styner MA, Panigrahy A, Escolar ML. Regional differences in fiber tractography predict neurodevelopmental outcomes in neonates with infantile Krabbe disease. Neuroimage Clin. 2014 Sep 26;7:792-8. doi: 10.1016/j.nicl.2014.09.014. eCollection 2015.
- Bradbury AM, Bagel J, Swain G, Miyadera K, Pesayco JP, Assenmacher CA, Brisson B, Hendricks I, Wang XH, Herbst Z, Pyne N, Odonnell P, Shelton GD, Gelb M, Hackett N, Szabolcs P, Vite CH, Escolar M. Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy. Mol Ther. 2024 Jan 3;32(1):44-58. doi: 10.1016/j.ymthe.2023.11.014. Epub 2023 Nov 11.
- Siddiqi ZA, Sanders DB, Massey JM. Peripheral neuropathy in Krabbe disease: effect of hematopoietic stem cell transplantation. Neurology. 2006 Jul 25;67(2):268-72. doi: 10.1212/01.wnl.0000230156.01228.33.
- Vander Lugt MT, Chen X, Escolar ML, Carella BA, Barnum JL, Windreich RM, Hill MJ, Poe M, Marsh RA, Stanczak H, Stenger EO, Szabolcs P. Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders. Blood Adv. 2020 Jul 14;4(13):3041-3052. doi: 10.1182/bloodadvances.2020001940.
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (실제)
연구 완료 (실제)
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
추가 관련 MeSH 약관
기타 연구 ID 번호
- PRO00120964
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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