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BMN 110 · MOR-AUS
BMN 110 Phase 3B in Australian Patients
Ensayo de BIOMARIN PHARMACEUTICAL INC en Mucopolisacaridosis IVA (Sindrome de Morquio A).
- Fase
- Fase 3
- Estado
- Completado
- Participantes
- 13
- Centros
- 5
- Fin del objetivo primario
- jul 2016
Qué significa cada fase y cada estado.
Estudio de intervención, de un solo grupo, abierto. Comenzó en jul 2013.
Qué mide
Analisis de seguridad (The study period during which all non-serious AEs and SAEs will be reported begins after informed consent is obtained and through 30 days after the last study visit or 30 days after the last drug infusion, whichever comes first.) (del registro, en inglés)
Qué se prueba
El ensayo prueba BMN 110 en pacientes australianos con MPS IVA para evaluar su seguridad y tolerabilidad a una dosis de 2,0 mg/kg/semana.
Lo que dice el documentoThe objective of this Phase 3B open label study (110-502) will be to evaluate the safety and tolerability of 2.0 mg/kg/week (qw) of BMN 110 in Australian patients with MPS IVA. In addition, a number of secondary and tertiary efficacy endpoints will also be investigated.
Traducción automática (claude-sonnet-5). Lo comprobado es la cita: está literal en el documento y sus cifras cuadran con las de la frase. Todavía no la ha leído una persona.
Cómo lo describe el promotor
En el documentoEn inglés, del registroThere is currently no treatment for MPS IVA other than supportive care for the clinical manifestations of the disease. Enzyme replacement therapy (ERT) with BMN 110 to replace the deficient GALNS is a potential new treatment option for MPS IVA patients. BMN 110, containing recombinant human GALNS (rhGALNS) developed by BioMarin is expected to reduce the progressive, pathologic accumulation of KS, and improve signs and symptoms of the disease. The objective of this Phase 3B open label study (110-502) will be to evaluate the safety and tolerability of 2.0 mg/kg/week (qw) of BMN 110 in Australian patients with MPS IVA. In addition, a number of secondary and tertiary efficacy endpoints will also be investigated. The dose and regimen of BMN 110 have been selected on the basis of data from a Phase 1/2 clinical study with BMN 110, nonclinical and in vitro studies with BMN 110, and clinical and nonclinical data from other enzyme replacement therapies. Extension Phase is included per amendment dated 10Mar 2014: To provide patients enrolled in the Initial Phase access to BMN 110 until commercial product becomes available in Australia and continue to assess long-term safety
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Todos los de BMRN, en su ficha.
Ficha completa en ClinicalTrials.gov (NCT01966029), actualizada en sep 2019.