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A Study to Investigate Pharmacokinetics, Pharmacodynamics, and Safety of Subcutaneous Anifrolumab in Pediatric Participants 5 to < 18 Years of Age With Systemic Lupus Erythematosus

2 de junio de 2026 actualizado por: AstraZeneca

A Multicenter, Open-Label, Phase II Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Subcutaneous Anifrolumab in Pediatric Participants 5 to < 18 Years of Age With Systemic Lupus Erythematosus

The purpose of this study is to characterize the pharmacokinetics (PK), pharmacodynamics (PD), and safety of subcutaneous (SC) anifrolumab in pediatric participants with moderate to severe systemic lupus erythematosus (SLE) while on background standard of care (SoC) therapy.

Descripción general del estudio

Estado

Aún no reclutando

Intervención / Tratamiento

Descripción detallada

This is an open-label, multicenter study.

The study includes -

  • Screening Period of up to 35 days
  • Period A (12-week, open-label treatment period)
  • Period B (a possible 12-week dosing regimen adjustment period, if required)
  • Treatment Extension Period (up-to-40-week, optional)
  • Period C (a 12-week safety follow-up period)

The study intervention (anifrolumab) will be administered subcutaneously using an accessorized pre-filled syringe (APFS) in 2 cohorts.

Tipo de estudio

Intervencionista

Inscripción (Estimado)

24

Fase

  • Fase 2

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

  • Niño

Acepta Voluntarios Saludables

No

Descripción

Inclusion Criteria:

  • Diagnosis of SLE.
  • Must be receiving at least one of the following SoC regimens for ≥ 4 weeks: oral glucocorticoids (≤1.0 mg/kg/day or ≤ 40 mg/day prednisone equivalent), antimalarials (hydroxychloroquine, chloroquine, or quinacrine), or a single permitted immunosuppressant (azathioprine, mycophenolate mofetil/mycophenolic acid, methotrexate, mizoribine, or tacrolimus) within specified dose limits.
  • Participant must have moderate to severe active SLE disease defined as Systemic lupus erythematosus disease activity index 2000 (SLEDAI-2K) ≥ 6 total points.
  • Body weight ≥ 15 kg.
  • Participants must agree to follow study specific contraception requirements as per local regulations.

Exclusion Criteria:

  • Known diagnosis of an IFN mediated autoinflammatory interferonopathy.
  • History of, or current diagnosis of, clinically significant non-SLE related vasculitides.
  • Active, severe SLE-driven renal disease with significant proteinuria.
  • Active severe or unstable neuropsychiatric SLE including but not limited to aseptic meningitis; cerebral vasculitis; myelopathy; demyelination syndromes (ascending, transverse, acute inflammatory demyelinating polyradiculopathy); acute confusional state; impaired level of consciousness; psychosis; acute stroke or stroke syndrome; cranial neuropathy; status epilepticus; cerebellar ataxia; and mononeuritis multiplex.
  • In participants ≥ 11 years of age, a history or evidence of suicidal ideation (severity of 4 [active: method and intent, but no plan] or 5 [active: method, intent, and plan]) within the past 6 months; or any suicidal behavior within the past 12 months or recurrent suicidal behavior in the lifetime of the participant based on an assessment with the Columbia suicide severity rating scale (C-SSRS).
  • History of, or current diagnosis of, catastrophic antiphospholipid syndrome (APS).
  • History of recurrent or opportunistic infection requiring hospitalization and intravenous (IV) antibiotics.
  • Known history of a primary immunodeficiency, splenectomy, or any underlying condition that predisposes the participant to infection, or a positive result for human immunodeficiency virus (HIV) infection.
  • Active hepatitis B and C infection.
  • Any active or recent herpes zoster (HZ) infection that has not completely resolved within 12 weeks prior to study entry or that emerges between screening and Day 1.
  • Any history of severe or recurrent HZ, including non-cutaneous HZ, herpes encephalitis, ophthalmic herpes, or 2 or more prior HZ episodes.
  • Any cytomegalovirus (CMV) or Epstein Barr virus (EBV) infection that has not completely resolved.
  • History of cancer.
  • Prior receipt of anifrolumab.
  • Prior treatment with directly acting cytotoxic B-cell depleting therapeutics.
  • A known history of allergy or reaction to any component of the study intervention formulation or history of anaphylaxis to any human gamma globulin therapy, human proteins, or monoclonal antibodies (mAbs).

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: No aleatorizado
  • Modelo Intervencionista: Asignación Secuencial
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Cohort 1 (body weight > 40 kg)
Participants with body weight > 40 kg will receive anifrolumab as an injection in APFS.
Anifrolumab will be administered as a SC injection using an APFS.
Otros nombres:
  • MEDI-546
  • SAPHNELO™
Experimental: Cohort 2 (body weight ≥ 15 to ≤ 40 kg)
Participants with body weight ≥ 15 to ≤ 40 kg will receive anifrolumab as an injection in APFS.
Anifrolumab will be administered as a SC injection using an APFS.
Otros nombres:
  • MEDI-546
  • SAPHNELO™

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Maximum observed serum (peak) concentration (Cmax)
Periodo de tiempo: Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11
To characterize PK (Cmax) of anifrolumab in pediatric participants with SLE following SC administration.
Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11
Area under the serum concentration-time curve (AUC)
Periodo de tiempo: Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11
To characterize PK (AUC) of anifrolumab in pediatric participants with SLE following SC administration.
Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11
Time to maximum plasma concentration (tmax)
Periodo de tiempo: Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11
To characterize PK (tmax) of anifrolumab in pediatric participants with SLE following SC administration.
Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11
Apparent total body clearance of drug from plasma (CL/F)
Periodo de tiempo: Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11
To characterize PK (CL/F) of anifrolumab in pediatric participants with SLE following SC administration.
Cohort 1: From Day 1 to Day 8; Cohort 2: From Day 1 to Day 11
Trough drug concentration at steady state (Ctrough,ss)
Periodo de tiempo: At Week 12
To characterize PK (Ctrough,ss) of anifrolumab in pediatric participants with SLE following SC administration.
At Week 12

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Suppression of type I IFN 21-gene signature
Periodo de tiempo: At Week 12 and 52
To characterize PD of anifrolumab in pediatric participants with SLE following SC administration.
At Week 12 and 52
Change from baseline in anti-double-stranded deoxyribonucleic acid (dsDNA) antibodies
Periodo de tiempo: At Week 12 and 52
To characterize PD of anifrolumab in pediatric participants with SLE following SC administration.
At Week 12 and 52
Change from baseline in complement component 3 (C3) levels
Periodo de tiempo: At Week 12 and 52
To characterize PD of anifrolumab in pediatric participants with SLE following SC administration.
At Week 12 and 52
Change from baseline in complement component 4 (C4) levels
Periodo de tiempo: At Week 12 and 52
To characterize PD of anifrolumab in pediatric participants with SLE following SC administration.
At Week 12 and 52
Change from baseline in total hemolytic complement (CH50) levels
Periodo de tiempo: At Week 12 and 52
To characterize PD of anifrolumab in pediatric participants with SLE following SC administration.
At Week 12 and 52
Number and percentage of participants who develop anti drug antibody (ADA) against anifrolumab
Periodo de tiempo: From Day 1 to Week 52
To evaluate the immunogenicity of anifrolumab in pediatric participants with SLE following SC administration.
From Day 1 to Week 52

Otras medidas de resultado

Medida de resultado
Medida Descripción
Periodo de tiempo
Number with participants with adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESIs)
Periodo de tiempo: Up to follow-up visit (12 weeks post last dose of study intervention) (approximately 64 weeks)
To evaluate the safety and tolerability of anifrolumab in pediatric participants with SLE following SC administration.
Up to follow-up visit (12 weeks post last dose of study intervention) (approximately 64 weeks)

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Estimado)

28 de julio de 2026

Finalización primaria (Estimado)

3 de septiembre de 2030

Finalización del estudio (Estimado)

5 de septiembre de 2031

Fechas de registro del estudio

Enviado por primera vez

2 de junio de 2026

Primero enviado que cumplió con los criterios de control de calidad

2 de junio de 2026

Publicado por primera vez (Actual)

5 de junio de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

5 de junio de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

2 de junio de 2026

Última verificación

1 de mayo de 2026

Más información

Términos relacionados con este estudio

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

SÍ

Descripción del plan IPD

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment:

https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

Marco de tiempo para compartir IPD

AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Criterios de acceso compartido de IPD

When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.

Tipo de información de apoyo para compartir IPD

  • PROTOCOLO DE ESTUDIO
  • SAVIA

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

Sí

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

Sí

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

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