Langdurig vervolgonderzoek om de veiligheid en werkzaamheid van FBX-101 bij Krabbe-patiënten te evalueren
Een langetermijnvervolgonderzoek om de veiligheid en werkzaamheid van Krabbe-patiënten te evalueren op basis van klinische onderzoeken naar gentherapie waarbij de toediening van FBX-101 (AAVrh.10-hGALC) betrokken is
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.
Studie Overzicht
Gedetailleerde beschrijving
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.
Studietype
Inschrijving (Werkelijk)
Contacten en locaties
Studie Locaties
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North Carolina
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Durham, North Carolina, Verenigde Staten, 27705
- Duke University
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Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
- Kind
- Volwassen
- Oudere volwassene
Accepteert gezonde vrijwilligers
Geslachten die in aanmerking komen voor studie
Bemonsteringsmethode
Studie Bevolking
This is an observational long-term follow-up (LTFU) study of participants from prior interventional trials involving the administration of FBX-101.
Beschrijving
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.
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Observatiemodellen: OTHER
- Tijdsperspectieven: PROSPECTIVE
Cohorten en interventies
Groep / Cohort |
Interventie / Behandeling |
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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. |
Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Tijdsspanne |
|---|---|
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Veiligheid op lange termijn, zoals beoordeeld op basis van de incidentie van ernstige bijwerkingen (SAE's) en bijwerkingen van bijzonder belang (AESI's) die worden toegeschreven aan FBX-101
Tijdsspanne: 36 maanden
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36 maanden
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Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
|---|---|---|
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Werkzaamheid zoals beoordeeld aan de hand van de verandering in de kwaliteit van leven, longitudinaal gemeten aan de hand van de Pediatric Quality of Life (PedsQL) Generieke Kernschalen met Family Impact Module en Multidimensionale Vermoeidheidsschaal
Tijdsspanne: 36 maanden
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36 maanden
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Werkzaamheid zoals beoordeeld door verandering van ontwikkelingsmijlpalen zoals taal- en motorische vaardigheden, zoals longitudinaal gemeten door een specifieke vragenlijst over ontwikkelingsmijlpalen
Tijdsspanne: 36 maanden
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36 maanden
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Werkzaamheid zoals beoordeeld aan de hand van verandering van de visuele functie zoals longitudinaal gemeten aan de hand van visueel opgeroepen potentieel (VEP)
Tijdsspanne: 36 maanden
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36 maanden
|
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Werkzaamheid zoals beoordeeld aan de hand van verandering van de visuele functie, longitudinaal gemeten aan de hand van de gezichtsscherpte
Tijdsspanne: 36 maanden
|
36 maanden
|
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Werkzaamheid zoals beoordeeld door verandering van chimerisme van volbloeddonoren
Tijdsspanne: 36 maanden
|
36 maanden
|
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Werkzaamheid zoals beoordeeld door verandering van psychosine in volbloed en plasma
Tijdsspanne: 36 maanden
|
36 maanden
|
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Werkzaamheid zoals beoordeeld aan de hand van de verandering van de gehoorfunctie, longitudinaal gemeten met gedragsaudiometrie (BAUD)
Tijdsspanne: 36 maanden
|
36 maanden
|
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Werkzaamheid zoals beoordeeld aan de hand van de verandering van de perifere zenuwgeleidingssnelheid, longitudinaal gemeten door middel van Nerve Conduction Velocity (NCV)-beoordelingen
Tijdsspanne: 36 maanden
|
36 maanden
|
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Werkzaamheid zoals beoordeeld door verandering van fractionele anisotropie (FA) zoals longitudinaal gemeten door middel van Magnetic Resonance Imaging (MRI) van de hersenen met Diffusion Tensor Imaging (DTI)
Tijdsspanne: 36 maanden
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36 maanden
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Werkzaamheid zoals beoordeeld door verandering van galactosylceramidase (GALC)-spiegels in plasma en hersenvocht (CSF)
Tijdsspanne: 36 maanden
|
36 maanden
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Efficacy as assessed by change of gross motor function measured longitudinally by Peabody Developmental Motor Scale (PDMS-2)
Tijdsspanne: 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 gross motor function measured longitudinally by Bruininks-Oseretsky Test of Motor Proficiency (BOT-2)
Tijdsspanne: 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 gross motor function measured longitudinally by Gross Motor Function Measure 88 (GMFM-88)
Tijdsspanne: 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 Mullen Scales of Early Learning (MSEL)
Tijdsspanne: 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 fine motor function measured longitudinally by Beery VMI Sixth Edition (VMI)
Tijdsspanne: 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 Bayley Scales of Infant Development (Bayley-III)
Tijdsspanne: 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)
Tijdsspanne: 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 cognitive function measured longitudinally by Mullen Scales of Early Learning (MSEL)
Tijdsspanne: 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 cognitive function measured longitudinally by the Differential Ability Scale II (DAS-II)
Tijdsspanne: 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 cognitive function measured longitudinally by Bayley Scales of Infant Development (Bayley-III)
Tijdsspanne: 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 adaptive behaviour function measured longitudinally by Vineland Adaptive Behavior Scale (Vineland 3).
Tijdsspanne: 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 Mullen Scales of Early Learning (MSEL).
Tijdsspanne: 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 Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5)
Tijdsspanne: 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 Bayley Scales of Infant Development (Bayley-III)
Tijdsspanne: 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).
Tijdsspanne: 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 hearing function measured longitudinally by Auditory Brainstem Responses (ABRs)
Tijdsspanne: 36 months
|
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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Medewerkers en onderzoekers
Sponsor
Publicaties en nuttige links
Algemene publicaties
- 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.
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Studie start (Werkelijk)
Primaire voltooiing (Werkelijk)
Studie voltooiing (Werkelijk)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Werkelijk)
Updates van studierecords
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Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Aanvullende relevante MeSH-voorwaarden
- Hersenziekten
- Ziekten van het centrale zenuwstelsel
- Ziekten van het zenuwstelsel
- Metabolisme, aangeboren fouten
- Genetische ziekten, aangeboren
- Metabole ziekten
- Demyeliniserende ziekten
- Stoornissen in het metabolisme van lipiden
- Hersenziekten, metabolisch, aangeboren
- Hersenziekten, Metabool
- Erfelijke demyeliniserende ziekten van het centrale zenuwstelsel
- Leuko-encefalopathieën
- Lipidenmetabolisme, aangeboren fouten
- Lysosomale stapelingsziekten, zenuwstelsel
- Sfingolipidosen
- Lipidosen
- Aangeboren, erfelijke en neonatale ziekten en afwijkingen
- Voedings- en stofwisselingsziekten
- Lysosomale stapelingsziekten
- Leukodystrofie, bolvormige cel
Andere studie-ID-nummers
- PRO00120964
Plan Individuele Deelnemersgegevens (IPD)
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