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Lo studio clinico NCT04571970 per Mucopolisaccaridosi di tipo II (MPS II) è completato. Consulti la vista a schede del Radar degli Studi Clinici e gli strumenti di scoperta IA per tutti i dettagli. Oppure, ponga pure una domanda qui. | ||
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REGENXBIORGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) Fase I, Fase II 6 Terapia genica
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La sperimentazione clinica NCT04571970 è stata uno studio interventistico di Fase I Fase II condotto per esaminare il trattamento per Mucopolisaccaridosi di tipo II (MPS II), attualmente completato. Avviato il 11 marzo 2021, ha previsto l'arruolamento di 6 partecipanti. Sotto la guida di REGENXBIO, si è concluso il 23 maggio 2024. I dati più recenti da ClinicalTrials.gov sono stati aggiornati l'ultima volta il 28 gennaio 2025.
Sommario breve
RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a phase I/II study to determine whether RGX-121 is safe, well tolerated, and potentially effective in children five years of age and over who have severe MPS II.
Descrizione dettagliata
MPS II is a rare X-linked recessive genetic disease caused by mutations in the iduronate-2-sulfatase (IDS) gene. Enzyme replacement therapy (ERT) with recombinant idursulfase (ELAPRASE®) is the only approved product for the treatment of Hunter syndrome; however, ERT as currently administered does not cross the blood brain barrier and is therefore unable to address the unmet need in MPS II patients with CNS (neurocogn...Mostra di più
Titolo ufficiale
A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of RGX-121 in Children 5 Years of Age and Older With MPS II (Hunter Syndrome)
Patologie
Mucopolisaccaridosi di tipo II (MPS II)Altri ID dello studio
- RGX-121-1102
Numero NCT
Data di inizio (effettiva)
2021-03-11
Ultimo aggiornamento pubblicato
2025-01-28
Data di completamento (stimata)
2024-05-23
Arruolamento (previsto)
6
Tipo di studio
Interventistico
FASE
Fase I
Fase II
Fase II
Stato
Completato
Parole chiave
MPS II
gene therapy
Hunter
gene therapy
Hunter
Scopo principale
Trattamento
Allocazione
N.D.
Modello di intervento
A gruppo singolo
Mascheramento
Nessuno (studio in aperto)
Bracci / Interventi
| Gruppo/Braccio di partecipanti | Intervento/Trattamento |
|---|---|
SperimentaleSingle Arm 6.5 × 10\^10 GC/g brain mass of RGX-121 | RGX-121 Recombinant adeno-associated virus serotype 9 capsid containing human iduronate-2-sulfatase expression cassette |
Esito primario
Esito secondario
| Misure di esito | Descrizione della misura | Arco temporale |
|---|---|---|
Number of participants with treatment-related adverse events and serious adverse events | Number of participants with treatment-related adverse events and serious adverse events as assessed by CTCAE (Version 5.0) | 24 Weeks |
| Misure di esito | Descrizione della misura | Arco temporale |
|---|---|---|
Number of participants with treatment-related adverse events and serious adverse events | Number of participants with treatment-related adverse events and serious adverse events as assessed by CTCAE (Version 5.0) | 104 Weeks |
Biomarkers | Change from baseline in Glycosaminoglycan levels (ng/mL) | Baseline, Week 1, Week 2, Week 4, Week 12, Week 24, Week 38, Week 52, Week 64, Week 78, Week 104 |
Biomarkers | Change from baseline in iduronate-2-sulfatase activity | Baseline, Week 1, Week 2, Week 4, Week 12, Week 24, Week 38, Week 52, Week 64, Week 78, Week 104 |
Change in neurodevelopmental parameters | Change from baseline in neurodevelopmental parameters of cognitive function as measured by the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) | Baseline, Week 52, Week 104 |
Change in neurodevelopmental parameters | Change from baseline in neurodevelopmental parameters of cognitive function as measured by the Mullen Scales of Early Learning (MSEL) | Baseline, Week 52, Week 104 |
Change in neurodevelopmental parameters | Change from baseline in neurodevelopmental parameters as measured by the Vineland Adaptive Behavior Scales, 2nd Edition (VABS-II), Comprehensive Interview Form | Baseline, Week 24, Week 52, Week 78, Week 104 |
Criteri di eleggibilità
Età idonea
Bambino
Età minima
5 Years
Sessi idonei
Maschio
Meets any of the following criteria:
- Has a documented diagnosis of MPS II AND a neurocognitive testing score ≤ 1 ½ standard deviation (SD) from the test normative mean (BSID-III: 77 and MSEL Visual Reception: 35), OR
- Has a documented diagnosis of MPS II AND has a decline of ≥ 1 standard deviation on serial neurocognitive testing administered between 3 to 36 months apart (BSID-III Cognitive or MSEL Visual Reception), OR
- Has a relative clinically diagnosed with neuronopathic MPS II who has the same IDS mutation as the participant AND the participant in the opinion of a geneticist has inherited a neuronopathic form of MPS II, OR
- Has documented mutation(s) in IDS that in the opinion of a geneticist is known to result in a neuronopathic phenotype AND in the opinion of a clinician has a neuronopathic form of MPS II
- Has contraindications for intracisternal injection, intracerebroventricular injection, or lumbar puncture
- Has contraindications for immunosuppressive therapy
- Has any neurocognitive deficit not attributable to MPS II or diagnosis of a neuropsychiatric condition
- Has had prior treatment with an AAV-based gene therapy product
- If receiving ELAPRASE® via intrathecal (IT) administration, must agree to discontinue IT idursulfase for the duration of the study
- Has experienced a serious hypersensitivity reaction to intravenous (IV) ELAPRASE®
- Is currently failing to respond to idursulfase (ELAPRASE®) IV due to neutralizing anti-idursulfase antibodies
- Has received any investigational product within 30 days of Day 1 or 5 half-lives before signing of the ICF, whichever is longer
- Has a platelet count <100,000 per microliter (µL), absolute neutrophil count <1.0 × 103/µL, or aminotransferase (ALT) or aspartate aminotransferase (AST) >3 × upper limit of normal (ULN) or total bilirubin >1.5 × ULN at screening unless the participant has a previously known history of Gilbert's syndrome
REGENXBIONessun dato di contatto
2 Centri dello studio in 2 paesi
California
University of California San Francisco, Benioff Children's Hospital, Oakland, California, 94609, United States
Quebec
McGill University Heath Center, Montreal, Quebec, H4A 3J1, Canada