- ICH GCP
- US Clinical Trials Registry
- Klinisk utprøving NCT05046327
Open-Label Study to Evaluate the Safety and Efficacy of Avatrombopag and Remission Rates in Adults With ITP of ≤6 Months
15. oktober 2021 oppdatert av: Sobi, Inc.
A Multicenter, Open-Label Study to Evaluate the Safety and Efficacy of Avatrombopag and Remission Rates in Adults With Immune Thrombocytopenia (ITP) of ≤6 Months Duration
This study will evaluate the safety and efficacy of avatrombopag in subjects with a confirmed diagnosis of primary ITP (≤6 months duration) over 26 weeks of treatment, and also evaluate the incidence of ITP remission.
Studieoversikt
Status
Tilbaketrukket
Forhold
Intervensjon / Behandling
Detaljert beskrivelse
This phase 3b, multi-center, open-label study will enroll approximately 75 adult subjects with a confirmed diagnoses of primary ITP (≤6 months duration) who have had a previous response to a first line treatment.
The study will consist of a 26-week treatment period to evaluate the safety and efficacy of avatrombopag.
Subjects with platelet counts ≥50×10⁹/L at Week 26 may enter a dose-tapering period in which the dose of avatrombopag will be decreased for up to 16 weeks until avatrombopag treatment is discontinued and the platelet count is maintained ≥50×10⁹/L.
Once avatrombopag treatment has been discontinued, the subjects will enter a remission evaluation period of up to 24 weeks to evaluate whether they have entered a state of remission.
Studietype
Intervensjonell
Fase
- Fase 3
Deltakelseskriterier
Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
18 år og eldre (Voksen, Eldre voksen)
Tar imot friske frivillige
Nei
Kjønn som er kvalifisert for studier
Alle
Beskrivelse
Inclusion Criteria:
- Male or female subjects ≥18 years of age at Screening.
- Subject must be able to provide informed consent.
- Subject has a confirmed diagnosis of primary ITP according to the International Consensus Report on the Investigation and Management of Primary ITP within the previous 6 months prior to Visit 1 and has had a previous response to a first line treatment (corticosteroids, IVIg, or anti-D), in the opinion of the Investigator.
- Subject has at least one platelet count <30×10⁹/L at any time during the screening period or at the Baseline visit.
- Females of childbearing potential must have a negative pregnancy test at Screening and Baseline.
- Female subjects of childbearing potential who are sexually active and male subjects who are sexually active must agree to use effective methods of contraception.
- Subject is willing and able to comply with all aspects of the protocol.
Exclusion Criteria:
- Thrombocytopenia due to a known condition other than primary ITP (e.g., systemic lupus erythematosus, H. pylori infection, splenomegaly, chronic liver disease).
- Any history of arterial or venous thrombosis, including partial or complete thrombosis (history of superficial thrombophlebitis is not exclusionary).
- Subjects with known inherited thrombocytopenia (e.g., MYH-9 disorders).
- History of myelodysplastic syndrome (MDS) or other hematologic malignancies.
- Current history of significant cardiac arrhythmias or decompensated congestive heart failure.
- History of hepatitis B (HBV), hepatitis C (HCV), or human immunodeficiency virus (HIV).
- Previous use of eltrombopag, romiplostim, recombinant human TPO or other platelet-producing agents.
- Surgical resection of the spleen.
- Previous use of mycophenolate mofetil (MMF), rituximab (or other B-cell lymphocyte depleting agents), mercaptopurine (6-MP) or alkylating agents.
- Concurrent malignant disease, other than non-melanoma skin cancer or cervical cancer in-situ.
- Known allergy to avatrombopag or any of its excipients.
- Subject is unable to take oral medication or has a malabsorption syndrome or any other uncontrolled gastrointestinal condition.
- Enrollment in another clinical study with any investigational drug or device within 30 days of Day 1/Visit 2 (or 5 half-lives, whichever is longer); however, participation in observational studies is permitted.
- Any clinically relevant abnormality which makes the subject unsuitable for participation in the study, in the opinion of the Investigator.
- Considered unable or unwilling to comply with the study protocol requirements.
Studieplan
Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
|
Eksperimentell: 20 mg Avatrombopag daily
Avatrombopag
|
Avatrombopag administered at a frequency to maintain a target platelet count between ≥50 and ≤150×10⁹/L
Andre navn:
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Cumulative Number of Weeks of Platelet Response
Tidsramme: 6 Months of Active Treatment
|
Cumulative number of weeks of platelet response in which the platelet count is ≥50×10⁹/L during 6 months of treatment in the absence of rescue therapy.
|
6 Months of Active Treatment
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Durable Platelet Response
Tidsramme: 8 Weeks of Treatment
|
Durable platelet response as defined by the incidence of subjects who have at least 6 out of 8 weekly platelet counts ≥50×10⁹/L during the last 8 weeks of treatment.
|
8 Weeks of Treatment
|
|
Incidence of ITP remission
Tidsramme: 24 Consecutive Weeks
|
Incidence of ITP remission as defined by platelet count ≥50×10⁹/L for 24 consecutive weeks with no ITP treatments (concomitant or rescue).
|
24 Consecutive Weeks
|
|
Incidence of subjects achieving a platelet count response
Tidsramme: 6 Months of Active Treatment
|
Incidence of subjects achieving a platelet count response (≥50×10⁹/L) during the active treatment period of the study.
|
6 Months of Active Treatment
|
Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
Studierekorddatoer
Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.
Studer hoveddatoer
Studiestart (Forventet)
14. oktober 2021
Primær fullføring (Forventet)
6. april 2024
Studiet fullført (Forventet)
19. februar 2025
Datoer for studieregistrering
Først innsendt
8. september 2021
Først innsendt som oppfylte QC-kriteriene
8. september 2021
Først lagt ut (Faktiske)
16. september 2021
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
22. oktober 2021
Siste oppdatering sendt inn som oppfylte QC-kriteriene
15. oktober 2021
Sist bekreftet
1. oktober 2021
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
- Patologiske prosesser
- Sykdommer i immunsystemet
- Autoimmune sykdommer
- Hematologiske sykdommer
- Blødning
- Hemoragiske lidelser
- Blodkoagulasjonsforstyrrelser
- Hudmanifestasjoner
- Blodplateforstyrrelser
- Trombotiske mikroangiopatier
- Purpura, trombocytopenisk
- Purpura
- Purpura, trombocytopenisk, idiopatisk
- Trombocytopeni
Andre studie-ID-numre
- AVA-ITP-306
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
Nei
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Ja
Studerer et amerikansk FDA-regulert enhetsprodukt
Nei
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