Fitusiran-profylakse hos mannlige pediatriske personer i alderen 1 til under 12 år med hemofili A eller B (ATLAS-PEDS)
ATLAS-PEDS: En åpen, multinasjonal studie av fitusiranprofylakse hos mannlige pediatriske personer i alderen 1 til under 12 år med hemofili A eller B
Primary Objective:
- To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B
Secondary Objectives:
- To characterize the safety and tolerability
- To determine fitusiran plasma concentrations at selected time points
Studieoversikt
Detaljert beskrivelse
The estimated total time on study is up to 256 weeks for participants who roll over into the extension study and up to 280 weeks for participants who do not roll over into the extension study (due to the requirement for up to an additional 6 months of follow-up for monitoring of AT levels).
Studietype
Registrering (Faktiske)
Fase
- Fase 2
- Fase 3
Kontakter og plasseringer
Studiesteder
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Ontario
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Hamilton, Ontario, Canada, L8N 3Z5
- Investigational Site Number : 1240001
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Ottawa, Ontario, Canada, K1H 8L1
- Investigational Site Number : 1240002
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California
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Los Angeles, California, Forente stater, 90027
- Children's Hospital Los Angeles Site Number : 8400002
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New Jersey
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Hackensack, New Jersey, Forente stater, 07601
- Hackensack University Medical Center- Site Number : 8400008
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Ohio
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Cleveland, Ohio, Forente stater, 44106
- University Hospitals of Cleveland- Site Number : 8400007
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Pennsylvania
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Hershey, Pennsylvania, Forente stater, 17033
- Penn State Milton S. Hershey Medical Center- Site Number : 8400006
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Bangalore, India, 560034
- Investigational Site Number : 3560006
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Mumbai, India, 400 022
- Investigational Site Number : 3560002
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Pune-411011, India, 411 011
- Investigational Site Number : 3560001
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Vellore, India, 632004
- Investigational Site Number : 3560004
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Florence, Italia, 50134
- Investigational Site Number : 3800002
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Milan, Italia, 20122
- Investigational Site Number : 3800001
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Madrid, Comunidad de
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Madrid, Madrid, Comunidad de, Spania, 28046
- Investigational Site Number : 7240002
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Adana, Tyrkia (Türkiye), 01130
- Investigational Site Number : 7920001
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Istanbul, Tyrkia (Türkiye), 34093
- Investigational Site Number : 7920002
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Izmir, Tyrkia (Türkiye), TR-35100
- Investigational Site Number : 7920003
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Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Beskrivelse
Inclusion Criteria:
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Male, aged 1 to <12 years at the time of enrollment.
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Severe hemophilia A or B (Factor VIII (FVIII) <1% or Factor IX (FIX) ≤2%)
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Participants must have inhibitory antibodies to FVIII or FIX and must meet one of the following Nijmegen-modified Bethesda assay results criteria:
- Inhibitor titer of ≥0.6 BU/mL at screening, OR
- Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, OR
- Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 1 inhibitor titer ≥0.6 BU/mL and a history of anamnestic response or severe allergic reaction (anaphylaxis or nephrotic syndrome)
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Adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol
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Weight requirements at the time of enrollment: 8 to <45 kg
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Willing and able to comply with the study requirements and to provide signed written informed consent obtained from parent(s)/legal guardian (hereinafter the "parent") and written or oral assent obtained from participant, per local and national requirements
Exclusion Criteria:
Participants are excluded from the study if any of the following criteria apply:
- Known co-existing bleeding disorders other than hemophilia A or B
- Antithrombin (AT) activity <60% at Screening
- Co-existing thrombophilic disorder
- Clinically significant liver disease
- Active Hepatitis C virus infection
- Acute or chronic Hepatitis B virus infection
- Acute Hepatitis A or hepatitis E infection
- HIV positive with a CD4 count of <400 cells/μL
- History of arterial or venous thromboembolism, unrelated to an indwelling venous access
- Inadequate renal function
- History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)
- Subjects with central or peripheral indwelling catheters, with history of venous access complications leading to hospitalization and/or systemic anticoagulation therapy.
- History of intolerance to subcutaneous (SC) injection(s)
- Use of emicizumab (Hemlibra®) within 6 months prior to screening
- Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
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Eksperimentell: Fitusiran
Participants will receive a selected dose of fitusiran at regular intervals, as per study protocol |
Pharmaceutical form:Solution for injection-Route of administration:Subcutaneous
Andre navn:
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Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
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Plasma antithrombin (AT) activity levels
Tidsramme: Day 1 to the AT analysis time point at the optimal therapeutic dose (approximately 256 weeks)
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Characterize the AT activity at the optimal therapeutic dose |
Day 1 to the AT analysis time point at the optimal therapeutic dose (approximately 256 weeks)
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Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
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Number of participants reported with adverse events
Tidsramme: Up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of AT follow up)
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Number of participants reported with treatment-emergent adverse events (TEAEs) |
Up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of AT follow up)
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Fitusiran plasma concentrations
Tidsramme: Day 1 and Day 85
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Plasma samples will be collected for measurement of plasma concentrations of fitusiran at specific time points post dose on Day 1 and pre dose on Day 85 |
Day 1 and Day 85
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Samarbeidspartnere og etterforskere
Sponsor
Etterforskere
- Studieleder: Clinical Sciences & Operations, Sanofi
Publikasjoner og nyttige lenker
Hjelpsomme linker
Studierekorddatoer
Studer hoveddatoer
Studiestart (Faktiske)
Primær fullføring (Faktiske)
Studiet fullført (Faktiske)
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
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- EFC15467
- 2019-000679-18 (EudraCT-nummer)
- U1111-1223-4368 (Registeridentifikator: ICTRP)
- 2024-512501-76 (Registeridentifikator: CTIS)
- 2019 (U.S. NIH-stipend/kontrakt)
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
IPD-planbeskrivelse
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Legemiddel- og utstyrsinformasjon, studiedokumenter
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