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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

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:

  1. Male or female subjects ≥18 years of age at Screening.
  2. Subject must be able to provide informed consent.
  3. 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.
  4. Subject has at least one platelet count <30×10⁹/L at any time during the screening period or at the Baseline visit.
  5. Females of childbearing potential must have a negative pregnancy test at Screening and Baseline.
  6. Female subjects of childbearing potential who are sexually active and male subjects who are sexually active must agree to use effective methods of contraception.
  7. Subject is willing and able to comply with all aspects of the protocol.

Exclusion Criteria:

  1. Thrombocytopenia due to a known condition other than primary ITP (e.g., systemic lupus erythematosus, H. pylori infection, splenomegaly, chronic liver disease).
  2. Any history of arterial or venous thrombosis, including partial or complete thrombosis (history of superficial thrombophlebitis is not exclusionary).
  3. Subjects with known inherited thrombocytopenia (e.g., MYH-9 disorders).
  4. History of myelodysplastic syndrome (MDS) or other hematologic malignancies.
  5. Current history of significant cardiac arrhythmias or decompensated congestive heart failure.
  6. History of hepatitis B (HBV), hepatitis C (HCV), or human immunodeficiency virus (HIV).
  7. Previous use of eltrombopag, romiplostim, recombinant human TPO or other platelet-producing agents.
  8. Surgical resection of the spleen.
  9. Previous use of mycophenolate mofetil (MMF), rituximab (or other B-cell lymphocyte depleting agents), mercaptopurine (6-MP) or alkylating agents.
  10. Concurrent malignant disease, other than non-melanoma skin cancer or cervical cancer in-situ.
  11. Known allergy to avatrombopag or any of its excipients.
  12. Subject is unable to take oral medication or has a malabsorption syndrome or any other uncontrolled gastrointestinal condition.
  13. 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.
  14. Any clinically relevant abnormality which makes the subject unsuitable for participation in the study, in the opinion of the Investigator.
  15. 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:
  • Doptelet

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

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

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

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