Klinisk effektivitet og sikkerhed i den virkelige verden af Vesemnogene Lantuparvovec til spinal muskelatrofi (SMA) i lav- og mellemindkomstlande (LMIC).
Klinisk effektivitet og sikkerhed i den virkelige verden af Vesemnogene Lantuparvovec til spinal muskelatrofi (SMA) i lande med lav og mellemhøj indkomst (LMIC).
The study objective is to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec for the treatment of SMA.
The specific objectives are:
- To determine clinical effectiveness of Vesemnogene lantuparvovec therapy for SMA as evaluated by developmental gross motor milestone and survival.
- To describe the safety profile of Vesemnogene therapy for SMA as evaluated by adverse events reporting and laboratory tests, and monitoring of Adverse events of special interest.
Studieoversigt
Status
Betingelser
Intervention / Behandling
Detaljeret beskrivelse
This is an observational study designed to determine the real-world safety and effectiveness of Vesemnogene lantuparvovec therapy for SMA. Potential patients with genetic diagnosis of SMA will be evaluated for eligibility to undergo available gene therapies. Following the administration of Vesemnogene therapy, patient will be monitored for toxicity and response to treatment. No subjects will be withdrawn from the study, and subjects could freely drop out from the study anytime, simply by not showing up.
Undersøgelsestype
Fase
- Fase 3
Kontakter og lokationer
Studiesteder
-
-
Indonesia
-
Jakarta, Indonesia, Indonesien, 14470
- Tzu chi hospital
-
-
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
Inclusion Criteria:
- Willing and able to give written informed consent for participation in the study.
- Genetic confirmation of SMA (biallelic deletion or mutation of SMN1).
- SMA clinical phenotype and condition, that in the opinion of the treating physician, treatment with Vesemnogene will likely be beneficial.
- Absence of contraindications for spinal tap procedure or administration of intrathecal therapy.
- Total AAV antibody titres < 1:20 as determined by ELISA assay.
- Normal liver function (AST/ALT < 3XULN, Bilirubin <3.0 mg/dL).
- Unable to access or failure to respond to currently available curative treatments for SMA.
Exclusion Criteria:
None
Studieplan
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 |
|---|---|
|
Eksperimentel: The selected dose inchildren ≥ 6 months of age
Administration the selected dose of Vesemnogene Lantuparvovec in children > 6 months of age |
Exploratory study evaluating the safety and efficacy of vesemnogene lantuparvovec in patients with SMA. |
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Numbers of participants with adverse events (AEs), serious adverse events (SAEs)
Tidsramme: Baseline up to 5 years old
|
Participants are monitored for safety from baseline up to the end of the follow-up period. |
Baseline up to 5 years old
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Change from baseline in developmental gross motor milestones achieved according to WHO criteria
Tidsramme: Baseline up to 5 years old
|
For patients with SMA, the percentage of participants who are able to preserve ambulatory function. |
Baseline up to 5 years old
|
|
Event-free Survival until the 5th year or the last follow-up
Tidsramme: Baseline up to 5 vears old
|
Event-free survival was defined as the number of participants who did not die, did not require permanent ventilation and did not withdraw from the study until the 5th year or the last follow-up. |
Baseline up to 5 vears old
|
Samarbejdspartnere og efterforskere
Sponsor
Datoer for undersøgelser
Studer store datoer
Studiestart (Anslået)
Primær færdiggørelse (Anslået)
Studieafslutning (Anslået)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- HR 25-02
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Studerer et amerikansk FDA-reguleret enhedsprodukt
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