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A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients

maanantai 14. syyskuuta 2026 päivittänyt: Novartis Pharmaceuticals

Specified Use-Results Survey of Zolgensma Intrathecal Injection (Spinal Muscular Atrophy)

This study aims to assess the safety and effectiveness of onasemnogene abeparvovec intrathecal injection in spinal muscular atrophy (SMA) patients in clinical practice in Japan.

This is a non-interventional study and does not impose a therapy protocol, diagnostic/therapeutic procedure, or a visit schedule. Patients will be treated according to the Japan package insert. Data will be collected using a case registration form (CRF) completed by the investigator at each study site.

Tutkimuksen yleiskatsaus

Tila

Ei vielä rekrytointia

Ehdot

Spinaalinen lihasatrofia

Opintotyyppi

Havainnollistava

Ilmoittautuminen (Arvioitu)

Yhteystiedot ja paikat

Tässä osiossa on tutkimuksen suorittajien yhteystiedot ja tiedot siitä, missä tämä tutkimus suoritetaan.

Opiskeluyhteys

Nimi: Novartis Pharmaceuticals
Puhelinnumero: +41613241111
Sähköposti: novartis.email@novartis.com

Tutki yhteystietojen varmuuskopiointi

Nimi: Novartis Pharmaceuticals

Osallistumiskriteerit

Tutkijat etsivät ihmisiä, jotka sopivat tiettyyn kuvaukseen, jota kutsutaan kelpoisuuskriteereiksi. Joitakin esimerkkejä näistä kriteereistä ovat henkilön yleinen terveydentila tai aiemmat hoidot.

Kelpoisuusvaatimukset

Opintokelpoiset iät

Lapsi
Aikuinen
Vanhempi Aikuinen

Hyväksyy terveitä vapaaehtoisia

Näytteenottomenetelmä

Ei-todennäköisyysnäyte

Tutkimusväestö

SMA patients administered onasemnogene abeparvovec by intrathecal injection in Japan.

Kuvaus

Inclusion criteria:

• All patients treated with onasemnogene abeparvovec.

Exclusion criteria:

• Patients who have received onasemnogene abeparvovec for unapproved indications under the Clinical Trials Act or GCP (eg, investigator-initiated clinical trial).

Opintosuunnitelma

Tässä osiossa on tietoja tutkimussuunnitelmasta, mukaan lukien kuinka tutkimus on suunniteltu ja mitä tutkimuksella mitataan.

Miten tutkimus on suunniteltu?

Suunnittelun yksityiskohdat

Ryhmien/kohorttien lukumäärä

Kohortit ja interventiot

Ryhmä/Kohortti
Onasemnogene Abeparvovec Intrathecal Group SMA patients treated with onasemnogene abeparvovec intrathecal injection in Japan.

Mitä tutkimuksessa mitataan?

Ensisijaiset tulostoimenpiteet

Tulosmittaus	Toimenpiteen kuvaus	Aikaikkuna
Number of Patients With Serious Adverse Events and Adverse Drug Reactions (ADRs) Aikaikkuna: Up to approximately 4 years and 5 months		Up to approximately 4 years and 5 months
Incidence of Serious Adverse Events and ADRs Aikaikkuna: Up to approximately 4 years and 5 months	Incidence based on the person-year method will be calculated.	Up to approximately 4 years and 5 months
Incidence of Serious Adverse Events and ADRs Corresponding to the Safety Specifications Aikaikkuna: Up to approximately 4 years and 5 months	Incidence based on the person-year method will be calculated.	Up to approximately 4 years and 5 months
Number of Patients by Time to Onset of Serious Adverse Events and ADRs Aikaikkuna: Up to approximately 4 years and 5 months		Up to approximately 4 years and 5 months
Number of Patients by Time to Onset of Serious Adverse Events and ADRs Corresponding to the Safety Specifications Aikaikkuna: Up to approximately 4 years and 5 months		Up to approximately 4 years and 5 months
Number of Patients With Serious Adverse Events and ADRs Corresponding to the Safety Specifications Aikaikkuna: Up to approximately 4 years and 5 months	Safety specifications include hepatotoxicity, transient thrombocytopenia, thrombotic microangiopathy, cardiac adverse events, dorsal root ganglia toxicity/peripheral sensory neuropathy, tumorigenicity due to chromosomal integration, and long-term monitoring of gene therapy.	Up to approximately 4 years and 5 months

Toissijaiset tulostoimenpiteet

Tulosmittaus	Toimenpiteen kuvaus	Aikaikkuna
Change From Baseline in Hammersmith Functional Motor Scale-Expanded (HFMSE) Score Aikaikkuna: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months	The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability.	Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Change From Baseline in Revised Upper Limb Module (RULM) Score Aikaikkuna: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months	RULM is a tool used to assess upper limb function in SMA patients. It consists of 20 items in total. The first entry item classifies overall functional ability and is not scored. The remaining 19 tasks are scored with 1 task scored as can/cannot (1 is the highest score) and 18 tasks rated on a 3-point scale as follows: 0 - unable to perform the task 1 - able to perform the task with some modification 2 - able to perform the task with no difficulty The total RULM score ranges from 0 to 37 with higher scores indicating better upper limb function.	Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Change From Baseline in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score Aikaikkuna: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months	CHOP-INTEND is a tool used to measure the motor abilities in infants with SMA. It consists of 16 items scored on a scale from 0 (no response/unable to perform the movement) to 4 (complete response/able to perform the task). CHOP-Intend total score ranges from 0 to 64 with higher scores indicating higher motor function.	Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Change From Baseline in Clinical Global Impression-Severity (CGI-S) Score Aikaikkuna: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months	The CGI-S scale is a physician-rated measure used to assess the severity of a patient's illness. Severity of illness is assessed with consideration for the overall clinical experience of patients of the same age. The CGI-S scores range from: 0 = Not evaluated 1 = Normal, not at all ill 2 = Borderline normal and ill 3 = Mildly ill 4 = Moderately ill 5 = Markedly ill 6 = Severely ill 7 = Among the most extremely ill	Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Number of Patients Categorized by Clinical Global Impression-Improvement (CGI-I) Score Aikaikkuna: 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months	The CGI-I scale is a physician-rated measure used to assess a patient's overall clinical status. The patient's overall clinical status is scored on a 7-point scale of the extent of change relative to before treatment initiation as follows: 1 = Very much improved 2 = Much improved 3 = Minimally improved 4 = No change 5 = Minimally worse 6 = Much worse 7 = Very much worse Number of patients and their 95% confidence intervals (CIs) will be calculated for two categories: improved (CGI-I score: 1 to 3) and unchanged or worsened (CGI-I score: 4 to 7).	6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Number of Patients Achieving Each Developmental Milestone Aikaikkuna: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months	Developmental milestones include: Sitting without support Hands-and-knees crawling Standing with assistance Walking with assistance Standing alone Walking alone	Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months
Number of Patients Maintaining Each Developmental Milestone Aikaikkuna: Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months		Baseline, 6 months, 12 months, 18 months, 24 months, and every year thereafter, up to approximately 4 years and 5 months

Yhteistyökumppanit ja tutkijat

Täältä löydät tähän tutkimukseen osallistuvat ihmiset ja organisaatiot.

Sponsori

Novartis Pharmaceuticals

Tutkijat

Opintojohtaja: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Opintojen ennätyspäivät

Nämä päivämäärät seuraavat ClinicalTrials.gov-sivustolle lähetettyjen tutkimustietueiden ja yhteenvetojen edistymistä. National Library of Medicine (NLM) tarkistaa tutkimustiedot ja raportoidut tulokset varmistaakseen, että ne täyttävät tietyt laadunvalvontastandardit, ennen kuin ne julkaistaan julkisella verkkosivustolla.

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