Langzeit-Follow-up-Studie von Patienten, die Onasemnogen Abeparvovec-xioi erhalten
Eine Langzeit-Follow-up-Studie von Patienten in den klinischen Studien für spinale Muskelatrophie, die AVXS-101 erhielten
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.
Studienübersicht
Status
Status
Bedingungen
Bedingungen
Intervention / Behandlung
Intervention / Behandlung
Studientyp
Studientyp
Einschreibung (Tatsächlich)
Einschreibung
Phase
Phase
- Phase 3
Kontakte und Standorte
Studienorte
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New South Wales
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Randwick, New South Wales, Australien, 2145
- Sydney Children's Hospital
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Ghent, Belgien, 9000
- Universitair Ziekenhuis Gent
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Liège
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Liège, Liège, Belgien, B-4000
- Centre de Référence des Maladies Neuromusculaires
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Paris, Frankreich, 75012
- Hôpital Armand Trousseau
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Genova, Italien, 16147
- Instituto Gianninia Gaslini
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Milan, Italien, 20133
- Istituto Neurologico di Ricerca
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Milan, Italien, 20122
- Universita Degli Studi Di Milano
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Roma
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Roma, Roma, Italien, 00168
- Fondazione Policlinico Universitario Agostino Gemelli
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Tokyo, Japan, 162-8666
- Tokyo Women's Medical University Hospital
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Ontario
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Ottawa, Ontario, Kanada, K1H8L1
- Children's Hospital of Eastern Ontario Research Institute
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Taipei, Taiwan, 10048
- National Taiwan University Hospital
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California
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Palo Alto, California, Vereinigte Staaten, 94304
- Stanford University Medical Center
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Colorado
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Aurora, Colorado, Vereinigte Staaten, 80045
- Children's Hospital Colorado
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Illinois
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Chicago, Illinois, Vereinigte Staaten, 60611
- Ann Robert H. Lurie Children's Hospital of Chicago
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Maryland
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Baltimore, Maryland, Vereinigte Staaten, 21287
- John Hopkins Hospital - David M. Rubenstein Child Health Building
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Massachusetts
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Boston, Massachusetts, Vereinigte Staaten, 02114
- Massachusetts General Hospital
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Boston, Massachusetts, Vereinigte Staaten, 02115
- Boston Children's Hospital
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Michigan
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Grand Rapids, Michigan, Vereinigte Staaten, 49503
- Spectrum Health Hospitals Helen DeVos Children's Hospital
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Missouri
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St Louis, Missouri, Vereinigte Staaten, 63110
- Washington Unviersity School of Medicine in Saint Louis
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New York
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New York, New York, Vereinigte Staaten, 10032
- Columbia University Medical Center
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North Carolina
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Durham, North Carolina, Vereinigte Staaten, 27713
- Duke University
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Ohio
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Columbus, Ohio, Vereinigte Staaten, 43205
- Nationwide Children's Hospital
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Pennsylvania
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Philadelphia, Pennsylvania, Vereinigte Staaten, 19104
- Children's Hospital of Philadelphia
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Strasburg, Pennsylvania, Vereinigte Staaten, 17579
- Clinic for Special Children
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Texas
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Dallas, Texas, Vereinigte Staaten, 75235
- Children's Health Specialty Center Dallas Campus
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Utah
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Salt Lake City, Utah, Vereinigte Staaten, 84112
- University of Utah Health
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Virginia
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Norfolk, Virginia, Vereinigte Staaten, 23507
- Children's Hospital of The King's Daughters
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Richmond, Virginia, Vereinigte Staaten, 23298
- Virginia Commonwealth University
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Wisconsin
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Madison, Wisconsin, Vereinigte Staaten, 53792
- University of Wisconsin, Madison
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Newcastle upon Tyne, Vereinigtes Königreich, NE1 4LP
- The Newcastle Upon Tyne Hospitals NHS Foundation Trust
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London
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London, London, Vereinigtes Königreich, WC1N 3JH
- Great Ormond Street Hospital for Children NHS Foundation Trust
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Teilnahmekriterien
Zulassungskriterien
Zulassungskriterien
Studienberechtigtes Alter
- Kind
- Erwachsene
- Älterer Erwachsener
Akzeptiert gesunde Freiwillige
Studienberechtigte Geschlechter
Beschreibung
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
Studienplan
Wie ist die Studie aufgebaut?
Designdetails
- Hauptzweck: Behandlung
- Zuteilung: N / A
- Interventionsmodell: Einzelgruppenzuweisung
- Maskierung: Keine (Offenes Etikett)
Anzahl der Arme
Waffen und Interventionen
Teilnehmergruppe / ArmTeilnehmergruppe / Arm |
Intervention / BehandlungIntervention / Behandlung |
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Sonstiges: 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.
Andere Namen:
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Was misst die Studie?
Primäre Ergebnismessungen
Primäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
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Änderung von der Grundlinie in Höhenmessungen
Zeitfenster: Bis zu 5 Jahre
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Bis zu 5 Jahre
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Änderung von der Grundlinie in Gewichtsmessungen
Zeitfenster: Bis zu 5 Jahre
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Bis zu 5 Jahre
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Anzahl der Teilnehmer mit gleichzeitiger Medikation insgesamt und nach Art der Medikation
Zeitfenster: Bis zu 5 Jahre
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Bis zu 5 Jahre
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Anzahl der Teilnehmer mit anderen SMA-Therapien insgesamt und nach Art der Medikamente
Zeitfenster: Klasse 6 bis Klasse 15
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Klasse 6 bis Klasse 15
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Number of Participants Who Reach Developmental Milestones
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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)
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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)
Zeitfenster: 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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Mitarbeiter und Ermittler
Sponsor
Sponsor
Ermittler
Ermittler
- Studienstuhl: Sitra Tauscher-Wisniewski, MD, Novartis Gene Therapies, Inc.
Publikationen und hilfreiche Links
Studienaufzeichnungsdaten
Haupttermine studieren
Studienbeginn (Tatsächlich)
Studienbeginn
Primärer Abschluss (Geschätzt)
Primärer Abschluss
Studienabschluss (Geschätzt)
Studienabschluss
Studienanmeldedaten
Zuerst eingereicht
Zuerst eingereicht
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst gepostet (Tatsächlich)
Zuerst gepostet
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
Letztes Update gepostet
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Zuletzt verifiziert
Zuletzt verifiziert
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Schlüsselwörter
Zusätzliche relevante MeSH-Bedingungen
- Erkrankungen des zentralen Nervensystems
- Erkrankungen des Nervensystems
- Neuromuskuläre Erkrankungen
- Genetische Krankheiten, angeboren
- Neurodegenerative Krankheiten
- Heredodegenerative Erkrankungen, Nervensystem
- Erkrankungen des Rückenmarks
- Motoneuron-Krankheit
- Muskelatrophie, Wirbelsäule
- Angeborene, erbliche und neonatale Krankheiten und Anomalien
- Spinale Muskelatrophien der Kindheit
- Zolgensma
Andere Studien-ID-Nummern
Andere Studien-ID-Nummern
- AVXS-101-LT-002
- 2019-002611-26 (EudraCT-Nummer)
- 205305 (Andere Kennung: JapicCTI)
- COAV101A12103 (Andere Kennung: Novartis Pharmaceuticals)
- 2019 (US NIH Stipendium/Vertrag)
Plan für individuelle Teilnehmerdaten (IPD)
Planen Sie, individuelle Teilnehmerdaten (IPD) zu teilen?
Beschreibung des IPD-Plans
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.
Arzneimittel- und Geräteinformationen, Studienunterlagen
Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt
Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt
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