- ICH GCP
- US Clinical Trials Registry
- Klinisk utprøving NCT07630714
A Study to Investigate Pharmacokinetics, Pharmacodynamics, and Safety of Subcutaneous Anifrolumab in Pediatric Participants 5 to < 18 Years of Age With Systemic Lupus Erythematosus
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
Studieoversikt
Status
Forhold
Intervensjon / Behandling
Detaljert beskrivelse
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.
Studietype
Registrering (Antatt)
Fase
- Fase 2
Kontakter og plasseringer
Studiekontakt
- Navn: AstraZeneca Clinical Study Information Center
- Telefonnummer: 1-877-240-9479
- E-post: information.center@astrazeneca.com
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Barn
Tar imot friske frivillige
Beskrivelse
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).
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: Ikke-randomisert
- Intervensjonsmodell: Sekvensiell tildeling
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: 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.
Andre navn:
|
|
Eksperimentell: 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.
Andre navn:
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Maximum observed serum (peak) concentration (Cmax)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: 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)
Tidsramme: At Week 12
|
To characterize PK (Ctrough,ss) of anifrolumab in pediatric participants with SLE following SC administration.
|
At Week 12
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Suppression of type I IFN 21-gene signature
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
|
Andre resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Number with participants with adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESIs)
Tidsramme: 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)
|
Samarbeidspartnere og etterforskere
Sponsor
Studierekorddatoer
Studer hoveddatoer
Studiestart (Antatt)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
- Bindevevssykdommer
- Autoimmune sykdommer
- Sykdommer i immunsystemet
- Infeksjoner
- Gram-positive bakterielle infeksjoner
- Bakterielle infeksjoner
- Bakterielle infeksjoner og mykoser
- Actinomycetales infeksjoner
- Mycobacterium infeksjoner
- Hud- og bindevevssykdommer
- Lupus erythematosus, systemisk
- Mycobacterium-infeksjoner, ikke-tuberkuløse
- Anifrolumab
Andre studie-ID-numre
- D3465C00008
- 2025-524578-41-00 (Ctis)
- EMA/PE/0000246211 (Annen identifikator: Pediatric investigation plan [PIP])
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
IPD-planbeskrivelse
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.
IPD-delingstidsramme
Tilgangskriterier for IPD-deling
IPD-deling Støtteinformasjonstype
- STUDY_PROTOCOL
- SEVJE
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Studerer et amerikansk FDA-regulert enhetsprodukt
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