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Langtidsopfølgningsundersøgelse af patienter, der får Onasemnogen Abeparvovec-xioi

21. september 2026 opdateret af: Novartis Gene Therapies

En langsigtet opfølgningsundersøgelse af patienter i de kliniske forsøg for spinal muskelatrofi, der modtager AVXS-101

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.

Studieoversigt

Status

Aktiv, ikke rekrutterende

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

85

Fase

  • Fase 3

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

  • Australien
    • New South Wales
      • Randwick, New South Wales, Australien, 2145
        • Sydney Children's Hospital
  • Belgien
      • Ghent, Belgien, 9000
        • Universitair Ziekenhuis Gent
    • Liège
      • Liège, Liège, Belgien, B-4000
        • Centre de Référence des Maladies Neuromusculaires
  • Canada
    • Ontario
      • Ottawa, Ontario, Canada, K1H8L1
        • Children's Hospital of Eastern Ontario Research Institute
  • Det Forenede Kongerige
      • Newcastle upon Tyne, Det Forenede Kongerige, NE1 4LP
        • The Newcastle Upon Tyne Hospitals NHS Foundation Trust
    • London
      • London, London, Det Forenede Kongerige, WC1N 3JH
        • Great Ormond Street Hospital for Children NHS Foundation Trust
  • Forenede Stater
    • California
      • Palo Alto, California, Forenede Stater, 94304
        • Stanford University Medical Center
    • Colorado
      • Aurora, Colorado, Forenede Stater, 80045
        • Children's Hospital Colorado
    • Illinois
      • Chicago, Illinois, Forenede Stater, 60611
        • Ann Robert H. Lurie Children's Hospital of Chicago
    • Maryland
      • Baltimore, Maryland, Forenede Stater, 21287
        • John Hopkins Hospital - David M. Rubenstein Child Health Building
    • Massachusetts
      • Boston, Massachusetts, Forenede Stater, 02114
        • Massachusetts General Hospital
      • Boston, Massachusetts, Forenede Stater, 02115
        • Boston Children's Hospital
    • Michigan
      • Grand Rapids, Michigan, Forenede Stater, 49503
        • Spectrum Health Hospitals Helen DeVos Children's Hospital
    • Missouri
      • St Louis, Missouri, Forenede Stater, 63110
        • Washington Unviersity School of Medicine in Saint Louis
    • New York
      • New York, New York, Forenede Stater, 10032
        • Columbia University Medical Center
    • North Carolina
      • Durham, North Carolina, Forenede Stater, 27713
        • Duke University
    • Ohio
      • Columbus, Ohio, Forenede Stater, 43205
        • Nationwide Children's Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, Forenede Stater, 19104
        • Children's Hospital of Philadelphia
      • Strasburg, Pennsylvania, Forenede Stater, 17579
        • Clinic for Special Children
    • Texas
      • Dallas, Texas, Forenede Stater, 75235
        • Children's Health Specialty Center Dallas Campus
    • Utah
      • Salt Lake City, Utah, Forenede Stater, 84112
        • University of Utah Health
    • Virginia
      • Norfolk, Virginia, Forenede Stater, 23507
        • Children's Hospital of The King's Daughters
      • Richmond, Virginia, Forenede Stater, 23298
        • Virginia Commonwealth University
    • Wisconsin
      • Madison, Wisconsin, Forenede Stater, 53792
        • University of Wisconsin, Madison
  • Frankrig
      • Paris, Frankrig, 75012
        • Hôpital Armand Trousseau
  • Italien
      • Genova, Italien, 16147
        • Instituto Gianninia Gaslini
      • Milan, Italien, 20133
        • Istituto Neurologico di Ricerca
      • Milan, Italien, 20122
        • Universita Degli Studi Di Milano
    • Roma
      • Roma, Roma, Italien, 00168
        • Fondazione Policlinico Universitario Agostino Gemelli
  • Japan
      • Tokyo, Japan, 162-8666
        • Tokyo Women's Medical University Hospital
  • Taiwan
      • Taipei, Taiwan, 10048
        • National Taiwan University Hospital

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Barn
  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

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

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Interventionel model: Enkelt gruppeopgave
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Andet: 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.
Biologisk: Onasemnogene Abeparvovec-xioi

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.

Andre navne:
  • Zolgensma

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Ændring fra baseline i højdemålinger
Tidsramme: Op til 5 år
Op til 5 år
Ændring fra baseline i vægtmålinger
Tidsramme: Op til 5 år
Op til 5 år
Antal deltagere med samtidig medicin samlet og efter type medicin
Tidsramme: Op til 5 år
Op til 5 år
Antal deltagere med andre SMA-terapier samlet og efter type medicin
Tidsramme: År 6 til år 15
År 6 til år 15
Number of Participants Who Reach Developmental Milestones
Tidsramme: Up to 5 years

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
Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
Tidsramme: Up to 5 years

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
Number of Participants Who Experience a Clinically Significant Change From Baseline in Pulmonary Assessment Results and Require Ventilatory Support
Tidsramme: Up to 15 years

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
Number of Participants Who Experience Swallowing Dysfunction and Require Nutritional Support
Tidsramme: Up to 5 years

Assessed via the swallowing function questionnaire, formed of 4 yes/ no questions and 1 body weight question.
Up to 5 years
Number of Participants Who Experience a Clinically Significant Change from Baseline in Physical Examination Findings
Tidsramme: Up to 5 years

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
Number of Participants Who Experience a Clinically Significant Change From Baseline in Vital Signs Measurements
Tidsramme: Up to 5 years

Vital sign measurements will include blood pressure, respiratory rate, pulse, axillary temperature, and pulse oximetry.
Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Clinical Laboratory Assessments
Tidsramme: Up to 5 years

Blood samples will be collected for hematology (including complete blood cell count) and chemistry.
Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Cardiac Assessments
Tidsramme: Up to 5 years

Cardiac assessments will include a 12-lead electrocardiogram, transthoracic echocardiogram and Troponin-I.
Up to 5 years
Number of Participants Who Experience a Clinically Significant Change From Baseline in Observational Phase Questionnaire Results
Tidsramme: Year 6 to Year 15

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
Number of Participants Who Experience at Least One Serious Adverse Event (SAE)
Tidsramme: Up to 15 years

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:

  • Fatal
  • Life-threatening
  • Results in persistent or significant disability/incapacity
  • Constitutes a congenital abnormality or birth defect
  • Requires in-patient hospitalization or prolongation of existing hospitalization
  • Is medically significant e.g. defined as an event that jeopardizes the participant or may require medical or surgical intervention to prevent one of the outcomes listed above
Up to 15 years
Number of Participants Who Experience at Least One Adverse Event of Special Interest (AESI)
Tidsramme: Up to 15 years

An AESI is defined as an AE occurring during any study phase that fulfills one of the following criteria:

  • Hepatotoxicity
  • Thrombotic microangiopathy
  • Cardiac adverse events
  • Dorsal root ganglia toxicity
  • New malignancies
  • New incidence of a neurologic disorder
  • New incidence of an autoimmune disorder
  • New incidence of hematologic disorder
Up to 15 years
Change From Baseline in Bayley Scales of Infant and Toddler Development
Tidsramme: Up to 42 months, 15 days of age

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
Change From Baseline in Revised Upper Limb Module (RULM) Score
Tidsramme: Up to 5 years

RULM score is based on a scale from 0 to 37 where lower scores reflect poorer upper limb functional ability.
Up to 5 years
Change From Baseline in Cogstate Computerized Cognitive Battery Performed in Age 48 Months and Older
Tidsramme: Up to 5 years

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
Change From Baseline in Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) Performed in All Participants 5 to 21 Years of Age
Tidsramme: Up to 5 years

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
Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND)
Tidsramme: Up to 5 years

ACEND score is based on a scale from 1 to 41 where higher scores represent a better caregiver experience
Up to 5 years

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Novartis Gene Therapies

Efterforskere

  • Studiestol: Sitra Tauscher-Wisniewski, MD, Novartis Gene Therapies, Inc.

Publikationer og nyttige links

Den person, der er ansvarlig for at indtaste oplysninger om undersøgelsen, leverer frivilligt disse publikationer. Disse kan handle om alt relateret til undersøgelsen.

Generelle publikationer

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

10. februar 2020

Primær færdiggørelse (Anslået)

31. december 2035

Studieafslutning (Anslået)

31. december 2035

Datoer for studieregistrering

Først indsendt

31. juli 2019

Først indsendt, der opfyldte QC-kriterier

31. juli 2019

Først opslået (Faktiske)

1. august 2019

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

24. september 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

21. september 2026

Sidst verificeret

1. september 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • AVXS-101-LT-002
  • 2019-002611-26 (EudraCT nummer)
  • 205305 (Anden identifikator: JapicCTI)
  • COAV101A12103 (Anden identifikator: Novartis Pharmaceuticals)
  • 2019 (U.S. NIH-bevilling/kontrakt)

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-planbeskrivelse

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.

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

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