Estudio de fase 3, aleatorizado, doble ciego, controlado con placebo y multicéntrico para evaluar la eficacia y seguridad de ALXN1850 (fosfatasa alcalina recombinante) administrado por vía subcutánea en participantes adolescentes (12 a < 18 años de edad) y adultos con hipofosfatasia que no hayan sido tratados previamente con asfotasa alfa

Activo, no reclutaFase 3ClinicalTrials.gov

ID: NCT06079281Tipo: INTERVENTIONALInicio: 3 de ene de 2024Fin estimado: 29 de mar de 2028

Resumen

El objetivo primario de este estudio es evaluar la eficacia de ALXN1850 frente a placebo sobre los resultados funcionales en participantes adolescentes y adultos con HPP que no hayan recibido tratamiento previo con asfotasa alfa.

Elegibilidad

Edad mínima: 12 YearsEdad máxima: 130 YearsSexo: ALL

Criterios de inclusión

Diagnosis of HPP documented in the medical records
Must meet 1 of the following criteria:
Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a Clinical Laboratory Improvement Amendments (CLIA) or ISO 15189 certified laboratory (Section 8.7 )
Plasma PLP above the upper limit of normal (ULN) during the Screening Period (central or local laboratory results allowed per local regulations)
Must meet 1 of the following criteria without a probably cause other than HPP:
Serum ALP activity below the age- and sex-adjusted normal range during the screening period as measured by the Central Laboratory
Two documented serum ALP activity results, at least 15 days apart, below the age- and sex-adjusted local laboratory normal range during the 24 months before the Day 1 Visit. Note: Local laboratories need to be CLIA or ISO 15189 certified, or have other local equivalent laboratory certification with Alexion's approval.
Two separate 6MWTs at below 85% of the predicted distance (for age, sex, weight, and height) during the Screening Period without a probable cause other than HPP

Criterios de exclusión

History or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, neurological disorders, or any other disorders that are capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study intervention; or interfering with the interpretation of data as determined by the Investigator
Diagnosis of primary or secondary hyperparathyroidism
Hypoparathyroidism, unless secondary to HPP
Any new fracture within 12 weeks before Day 1 (excluding pseudofractures)
Planned surgical intervention which may impact the results of study assessments (in the opinion of the Investigator) during the Randomized Evaluation Period
History of allergy or hypersensitivity to any ingredient contained in ALXN1850 or the placebo comparator (Table 9)

Intervenciones

drug

ALXN1850

ALXN1850 will be administered via subcutaneous (SC) injection.

drug

Placebo

Placebo will be administered via SC injection.

Ubicaciones

Research Site

Ciudad de Buenos Aires, Argentina

Patrocinadores

PrincipalAlexion Pharmaceuticals, Inc.

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