Fitusiran-profylaxe bij mannelijke pediatrische proefpersonen van 1 tot jonger dan 12 jaar met hemofilie A of B (ATLAS-PEDS)
ATLAS-PEDS: een open-label, multinationale studie van Fitusiran-profylaxe bij mannelijke pediatrische proefpersonen van 1 tot jonger dan 12 jaar met hemofilie A of 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
Studie Overzicht
Gedetailleerde beschrijving
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
Inschrijving (Werkelijk)
Fase
- Fase 2
- Fase 3
Contacten en locaties
Studie Locaties
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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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Bangalore, Indië, 560034
- Investigational Site Number : 3560006
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Mumbai, Indië, 400 022
- Investigational Site Number : 3560002
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Pune-411011, Indië, 411 011
- Investigational Site Number : 3560001
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Vellore, Indië, 632004
- Investigational Site Number : 3560004
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Florence, Italië, 50134
- Investigational Site Number : 3800002
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Milan, Italië, 20122
- Investigational Site Number : 3800001
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Madrid, Comunidad de
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Madrid, Madrid, Comunidad de, Spanje, 28046
- Investigational Site Number : 7240002
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Adana, Turkije (Türkiye), 01130
- Investigational Site Number : 7920001
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Istanbul, Turkije (Türkiye), 34093
- Investigational Site Number : 7920002
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Izmir, Turkije (Türkiye), TR-35100
- Investigational Site Number : 7920003
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California
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Los Angeles, California, Verenigde Staten, 90027
- Children's Hospital Los Angeles Site Number : 8400002
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New Jersey
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Hackensack, New Jersey, Verenigde Staten, 07601
- Hackensack University Medical Center- Site Number : 8400008
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Ohio
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Cleveland, Ohio, Verenigde Staten, 44106
- University Hospitals of Cleveland- Site Number : 8400007
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Pennsylvania
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Hershey, Pennsylvania, Verenigde Staten, 17033
- Penn State Milton S. Hershey Medical Center- Site Number : 8400006
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Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
Accepteert gezonde vrijwilligers
Geslachten die in aanmerking komen voor studie
Beschrijving
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.
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
- Primair doel: Behandeling
- Toewijzing: NVT
- Interventioneel model: Opdracht voor een enkele groep
- Masker: Geen (open label)
Wapens en interventies
Deelnemersgroep / Arm |
Interventie / Behandeling |
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Experimenteel: 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
Andere namen:
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Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
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Plasma antithrombin (AT) activity levels
Tijdsspanne: 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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Secundaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
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Number of participants reported with adverse events
Tijdsspanne: 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
Tijdsspanne: 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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Medewerkers en onderzoekers
Sponsor
Onderzoekers
- Studie directeur: Clinical Sciences & Operations, Sanofi
Publicaties en nuttige links
Nuttige links
Studie record data
Bestudeer belangrijke data
Studie start (Werkelijk)
Primaire voltooiing (Werkelijk)
Studie voltooiing (Werkelijk)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Werkelijk)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- EFC15467
- 2019-000679-18 (EudraCT-nummer)
- U1111-1223-4368 (Register-ID: ICTRP)
- 2024-512501-76 (Register-ID: CTIS)
- 2019 (Subsidie/contract van de Amerikaanse NIH)
Plan Individuele Deelnemersgegevens (IPD)
Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?
Beschrijving IPD-plan
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
Informatie over medicijnen en apparaten, studiedocumenten
Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel
Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct
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