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Langtidsoppfølgingsstudie av pasienter som får Onasemnogen Abeparvovec-xioi

21. september 2026 oppdatert av: Novartis Gene Therapies

En langsiktig oppfølgingsstudie av pasienter i kliniske studier for spinal muskelatrofi som mottar 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.

Studieoversikt

Status

Aktiv, ikke rekrutterende

Forhold

Intervensjon / Behandling

Studietype

Intervensjonell

Registrering (Faktiske)

85

Fase

  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Barn
  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

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

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Annen: 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 navn:
  • Zolgensma

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Endring fra grunnlinje i høydemålinger
Tidsramme: Inntil 5 år
Inntil 5 år
Endring fra baseline i vektmålinger
Tidsramme: Inntil 5 år
Inntil 5 år
Antall deltakere med samtidige medisiner totalt sett og etter type medisiner
Tidsramme: Inntil 5 år
Inntil 5 år
Antall deltakere med andre SMA-terapier totalt sett og etter type medisiner
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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Novartis Gene Therapies

Etterforskere

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

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Generelle publikasjoner

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

10. februar 2020

Primær fullføring (Antatt)

31. desember 2035

Studiet fullført (Antatt)

31. desember 2035

Datoer for studieregistrering

Først innsendt

31. juli 2019

Først innsendt som oppfylte QC-kriteriene

31. juli 2019

Først lagt ut (Faktiske)

1. august 2019

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

24. september 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

21. september 2026

Sist bekreftet

1. september 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

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

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (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.

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

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