Långtidsuppföljningsstudie för att utvärdera säkerhet och effekt av FBX-101 hos Krabbepatienter
En långtidsuppföljningsstudie för att utvärdera säkerhet och effekt för Krabbe-patienter från kliniska prövningar av genterapi som involverar administrering av FBX-101 (AAVrh.10-hGALC)
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.
Studieöversikt
Detaljerad beskrivning
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.
Studietyp
Inskrivning (Faktisk)
Kontakter och platser
Studieorter
-
-
North Carolina
-
Durham, North Carolina, Förenta staterna, 27705
- Duke University
-
-
Deltagandekriterier
Urvalskriterier
Åldrar som är berättigade till studier
- Barn
- Vuxen
- Äldre vuxen
Tar emot friska volontärer
Kön som är behöriga för studier
Testmetod
Studera befolkning
This is an observational long-term follow-up (LTFU) study of participants from prior interventional trials involving the administration of FBX-101.
Beskrivning
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.
Studieplan
Hur är studien utformad?
Designdetaljer
- Observationsmodeller: OTHER
- Tidsperspektiv: PROSPECTIVE
Kohorter och interventioner
Grupp / Kohort |
Intervention / Behandling |
|---|---|
|
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. |
Vad mäter studien?
Primära resultatmått
Resultatmått |
Tidsram |
|---|---|
|
Långsiktig säkerhet bedömd utifrån förekomsten av allvarliga biverkningar (SAE) och biverkningar av särskilt intresse (AESI) som tillskrivs FBX-101
Tidsram: 36 månader
|
36 månader
|
Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
Effekt som bedöms genom förändring av livskvalitet mätt longitudinellt med Pediatric Quality of Life (PedsQL) Generic Core Scales med Family Impact Module och Multidimensional Fatigue Scale
Tidsram: 36 månader
|
36 månader
|
|
|
Effekt som bedöms genom förändring av utvecklingsmilstolpar såsom språk och motorik, mätt i längdriktningen med ett specifikt utvecklingsmilstolpar enkät
Tidsram: 36 månader
|
36 månader
|
|
|
Effekt som bedöms genom förändring av visuell funktion mätt i längdriktningen med visuellt framkallade potentialer (VEP)
Tidsram: 36 månader
|
36 månader
|
|
|
Effekt som bedöms genom förändring av synfunktionen mätt i längdriktningen med synskärpa
Tidsram: 36 månader
|
36 månader
|
|
|
Effekt som bedöms genom förändring av helblodsgivarens chimerism
Tidsram: 36 månader
|
36 månader
|
|
|
Effekt bedömd genom förändring av psykosin i helblod och plasma
Tidsram: 36 månader
|
36 månader
|
|
|
Effekt bedömd genom förändring av hörselfunktion mätt i längdriktningen med beteendeaudiometri (BAUD)
Tidsram: 36 månader
|
36 månader
|
|
|
Effekt som bedöms genom förändring av perifer nervledningshastighet mätt i längdriktningen med nervledningshastighetsbedömningar (NCV)
Tidsram: 36 månader
|
36 månader
|
|
|
Effekt som bedöms genom förändring av fraktionerad anisotropi (FA) mätt i längdriktningen med magnetisk resonanstomografi (MRT) i hjärnan med diffusionstensoravbildning (DTI)
Tidsram: 36 månader
|
36 månader
|
|
|
Effekt bedömd genom förändring av galaktosylceramidas (GALC) nivåer i plasma och cerebrospinalvätska (CSF)
Tidsram: 36 månader
|
36 månader
|
|
|
Efficacy as assessed by change of gross motor function measured longitudinally by Peabody Developmental Motor Scale (PDMS-2)
Tidsram: 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 gross motor function measured longitudinally by Bruininks-Oseretsky Test of Motor Proficiency (BOT-2)
Tidsram: 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 gross motor function measured longitudinally by Gross Motor Function Measure 88 (GMFM-88)
Tidsram: 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 Mullen Scales of Early Learning (MSEL)
Tidsram: 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)
Tidsram: 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 Bayley Scales of Infant Development (Bayley-III)
Tidsram: 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 Vineland Adaptive Behavior Scale (Vineland 3)
Tidsram: 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)
Tidsram: 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 the Differential Ability Scale II (DAS-II)
Tidsram: 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)
Tidsram: 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).
Tidsram: 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).
Tidsram: 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)
Tidsram: 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)
Tidsram: 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).
Tidsram: 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)
Tidsram: 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
|
Samarbetspartners och utredare
Sponsor
Publikationer och användbara länkar
Allmänna publikationer
- 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.
Studieavstämningsdatum
Studera stora datum
Studiestart (Faktisk)
Primärt slutförande (Faktisk)
Avslutad studie (Faktisk)
Studieregistreringsdatum
Först inskickad
Först inskickad som uppfyllde QC-kriterierna
Första postat (Faktisk)
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
Senast verifierad
Mer information
Termer relaterade till denna studie
Ytterligare relevanta MeSH-villkor
- Hjärnsjukdomar
- Sjukdomar i centrala nervsystemet
- Sjukdomar i nervsystemet
- Metabolism, medfödda fel
- Genetiska sjukdomar, medfödda
- Metaboliska sjukdomar
- Demyeliniserande sjukdomar
- Lipidmetabolismstörningar
- Hjärnsjukdomar, metabola, medfödda
- Hjärnsjukdomar, metaboliska
- Ärftliga demyeliniserande sjukdomar i centrala nervsystemet
- Leukoencefalopati
- Lipidmetabolism, medfödda fel
- Lysosomala lagringssjukdomar, nervsystemet
- Sfingolipidoser
- Lipidoser
- Medfödda, ärftliga och neonatala sjukdomar och abnormiteter
- Närings- och metabola sjukdomar
- Lysosomala lagringssjukdomar
- Leukodystrofi, Globoidcell
Andra studie-ID-nummer
- PRO00120964
Plan för individuella deltagardata (IPD)
Planerar du att dela individuella deltagardata (IPD)?
Läkemedels- och apparatinformation, studiedokument
Studerar en amerikansk FDA-reglerad läkemedelsprodukt
Studerar en amerikansk FDA-reglerad produktprodukt
Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .