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Nirogacestat i ovariegranulosacelletumorer

31. juli 2026 oppdatert av: SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany

En fase 2-studie av Nirogacestat hos pasienter med tilbakevendende ovariegranulosacellesvulster

Denne fase 2 kliniske studien vil studere effektiviteten av nirogacestat i ovariegranulosacelletumorer (OvGCTs). Nirogacestat er en gammasekretasehemmer (GSI) som antas å redusere veksten og aktiviteten til ovariegranulosa-svulster.

Studieoversikt

Status

Fullført

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

Ovarian granulosacelletumorer (OvGCTs) representerer 5-7 % av alle eggstokkreft (~1,5 til 2k nydiagnostiserte pasienter/år i USA) og er den vanligste undertypen av ovarie-kjønnsstrengsvulster (70%). Behandling av granulosacelletumorer med nirogacestat forventes å hemme Notch-indusert granulosacelleproliferasjon.

Dette er en multisenter, enarms, fase 2 åpen behandlingsstudie for å bestemme effektiviteten, sikkerheten, toleransen og farmakokinetikken til nirogacestat hos voksne deltakere med residiverende/refraktær OvGCT. Deltakerne vil fortsette behandlingen inntil sykdomsprogresjon som bestemt av responsevalueringskriterier i solide svulster (RECIST) v1.1 eller uakseptabel toksisitet.

Studietype

Intervensjonell

Registrering (Faktiske)

53

Fase

  • Fase 2

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 1Z2
        • Cross Cancer Institute
  • Forente stater
    • California
      • Los Angeles, California, Forente stater, 90033
        • USC/Norris Comprehensive Cancer Center
      • Los Angeles, California, Forente stater, 90095
        • UCLA-JCCC Dept. of OBGYN - Women's Health Clinical Research Unit
    • Florida
      • Orlando, Florida, Forente stater, 32806
        • Orlando Health Cancer Institute
      • Orlando, Florida, Forente stater, 32804
        • AdventHealth Orlando
      • Tampa, Florida, Forente stater, 33612
        • H. Lee Moffitt Cancer Center and Research Institute
    • Louisiana
      • Covington, Louisiana, Forente stater, 70433
        • Women's Cancer Care
    • Maryland
      • Baltimore, Maryland, Forente stater, 21204
        • Greater Baltimore Medical Center
    • Massachusetts
      • Boston, Massachusetts, Forente stater, 02215
        • Dana-Farber Cancer Institute
    • Missouri
      • St Louis, Missouri, Forente stater, 63141
        • David C. Pratt Cancer Center
    • New Mexico
      • Albuquerque, New Mexico, Forente stater, 87102
        • University of New Mexico Comprehensive Cancer Center
    • New York
      • New York, New York, Forente stater, 10032
        • Columbia University Medical Center
      • New York, New York, Forente stater, 10065
        • Memorial Sloan Kettering Cancer Institute
    • Ohio
      • Kettering, Ohio, Forente stater, 45429
        • Women's Cancer Center at Kettering
    • Oklahoma
      • Oklahoma City, Oklahoma, Forente stater, 73104
        • OU Health Stephenson Cancer Center
    • Washington
      • Seattle, Washington, Forente stater, 98109
        • UW/Fred Hutch Cancer Center
    • Wisconsin
      • Milwaukee, Wisconsin, Forente stater, 53226
        • Froedtert and Medical College of Wisconsin
  • Polen
      • Bialystok, Polen, 15-027
        • Maria Sklodowska-Curie Bialystok Oncology Center
      • Krakow, Polen, 30-348
        • Jagiellonian Innovation Centre Clinical Research Centre
      • Poznan, Polen, 60-569
        • University Teaching Hospital Poznan, Department of Oncological Gynaecology
      • Warsaw, Polen, 02-781
        • Maria Sklodowska-Curie National Institute of Oncology-National Research Institute, Clinic of Oncological Gynecology

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

Beskrivelse

Viktige inkluderingskriterier:

  • Har histologisk bekreftet tilbakevendende granulosacelletumor av voksen type i eggstokken før første dose studiebehandling
  • Ha dokumentert radiologisk bevis på tilbakefall etter minst én systemisk behandling som ikke er mottakelig for kirurgi eller stråling og har målbar sykdom i henhold til RECIST v1.1-kriterier
  • Ha tilstrekkelig benmarg-, nyre- og leverfunksjon som definert av laboratorieverdier for screeningbesøk

Nøkkelekskluderingskriterier:

  • Har tegn på tarmobstruksjon som krever foreldrenes ernæring, malabsorpsjonssyndrom eller eksisterende gastrointestinale tilstander som kan svekke absorpsjonen av nirogacestat
  • Har hatt en alvorlig kardio- eller tromboembolisk hendelse innen 6 måneder etter å ha signert informert samtykke
  • Har unormalt QT-intervall ved screening, eller har medfødt eller ervervet lang QT-syndrom eller en historie med ytterligere risikofaktorer for Torsades de Pointes
  • Har nåværende eller kronisk historie med leversykdom eller kjente lever- eller galleavvik
  • Har mottatt noen behandling for OvGCT inkludert, men ikke begrenset til, følgende innen 28 dager (eller 5 halveringstider, avhengig av hva som er lengst) før den første dosen av studiebehandlingen: antiangiogene terapi, hormonbehandling, kjemoterapi, immunterapi, målrettet terapi eller annen undersøkelsesbehandling

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: Nirogacestat Åpen-etikett

Nirogacestat 150 mg per munn, to ganger daglig

Nirogacestat tablett for oral inntak: Nirogacestat tablett
Legemiddel: Nirogacestat
nirogacestat oral tablett
Andre navn:
  • PF-03084014

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Objective Response Rate (ORR)
Tidsramme: 2.5 years
The percentage of participants with complete response (CR) + partial response (PR) assessed using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 criteria.
2.5 years

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Progression Free Survival at 6 Months (PFS-6)
Tidsramme: First day of cycle 7 (approximately 6 months after the first dose of study treatment). Each cycle is 28 days.
The number of participants without progression according to RECIST v1.1 or death at 6 months.
First day of cycle 7 (approximately 6 months after the first dose of study treatment). Each cycle is 28 days.
Overall Survival at Year 2
Tidsramme: 2 years after first dose of study treatment
The number of participants who have not died of any cause by Year 2.
2 years after first dose of study treatment
Duration of Response
Tidsramme: First day of every other cycle (each cycle is 28 days) for the first year, and then every 3 cycles thereafter until study completion (estimated to be an average of 2.5 years).
The time from response (Complete Response [CR] + Partial Response [PR] using RECIST v.1.) to disease progression and/or death, in participants with CR or PR.
First day of every other cycle (each cycle is 28 days) for the first year, and then every 3 cycles thereafter until study completion (estimated to be an average of 2.5 years).
Change From Baseline at Cycle 2 Day 1 in the Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI) Score.
Tidsramme: Baseline and at Cycle 2 Day 1 (cycle length is 28 days).
Participant reported ovarian cancer symptoms as measured by Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI). The FOSI is an 8-question evaluation scored on a Likert scale of 0-4 from not at all to very much. Metrics assessed include lack of energy, vomiting, pain, nausea, swelling in stomach area, concern condition will get worse, quality of life assessment and stomach cramps. Higher score means worse outcome. The responses to the statements except for statement 7 (I am content with the quality of my life right now) were reversed and the sum of non-missing responses is calculated. The FOSI score is the sum multiplied by 8, then divided by the number of responded statements. The FOSI score was missing if a participants had 5 or more missing values in the 8 individual symptom scores at a visit.
Baseline and at Cycle 2 Day 1 (cycle length is 28 days).
Change From Baseline at Cycle 3 Day 1 Treatment in the Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI) Score
Tidsramme: Baseline and at Cycle 3 Day 1 (cycle length is 28 days).
Participant reported ovarian cancer symptoms as measured by Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI). The FOSI is an 8-question evaluation scored on a Likert scale of 0-4 from not at all to very much. Metrics assessed include lack of energy, vomiting, pain, nausea, swelling in stomach area, concern condition will get worse, quality of life assessment and stomach cramps. Higher score means worse outcome. The responses to the statements except for statement 7 (I am content with the quality of my life right now) were reversed and the sum of non-missing responses is calculated. The FOSI score is the sum multiplied by 8, then divided by the number of responded statements. The FOSI score was missing if a participants had 5 or more missing values in the 8 individual symptom scores at a visit.
Baseline and at Cycle 3 Day 1 (cycle length is 28 days).
Change From Baseline at Cycle 4 Day 1 in the Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI) Score
Tidsramme: Baseline and at Cycle 4 Day 1 (cycle length is 28 days).
Participant reported ovarian cancer symptoms as measured by Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI). The FOSI is an 8-question evaluation scored on a Likert scale of 0-4 from not at all to very much. Metrics assessed include lack of energy, vomiting, pain, nausea, swelling in stomach area, concern condition will get worse, quality of life assessment and stomach cramps. Higher score means worse outcome. The responses to the statements except for statement 7 (I am content with the quality of my life right now) were reversed and the sum of non-missing responses is calculated. The FOSI score is the sum multiplied by 8, then divided by the number of responded statements. The FOSI score was missing if a participants had 5 or more missing values in the 8 individual symptom scores at a visit.
Baseline and at Cycle 4 Day 1 (cycle length is 28 days).
Change From Baseline at Cycle 5 Day 1 in the Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI) Score
Tidsramme: Baseline and at Cycle 5 Day 1 (cycle length is 28 days).
Participant reported ovarian cancer symptoms as measured by Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI). The FOSI is an 8-question evaluation scored on a Likert scale of 0-4 from not at all to very much. Metrics assessed include lack of energy, vomiting, pain, nausea, swelling in stomach area, concern condition will get worse, quality of life assessment and stomach cramps. Higher score means worse outcome. The responses to the statements except for statement 7 (I am content with the quality of my life right now) were reversed and the sum of non-missing responses is calculated. The FOSI score is the sum multiplied by 8, then divided by the number of responded statements. The FOSI score was missing if a participants had 5 or more missing values in the 8 individual symptom scores at a visit.
Baseline and at Cycle 5 Day 1 (cycle length is 28 days).
Change From Baseline at End of Treatment in the Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI) Score
Tidsramme: Baseline and at end of treatment (estimated to be an average of 5.5 months [minimum of 0 month and maximum of 33 months]).
Participant reported ovarian cancer symptoms as measured by Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI). The FOSI is an 8-question evaluation scored on a Likert scale of 0-4 from not at all to very much. Metrics assessed include lack of energy, vomiting, pain, nausea, swelling in stomach area, concern condition will get worse, quality of life assessment and stomach cramps. Higher score means worse outcome. The responses to the statements except for statement 7 (I am content with the quality of my life right now) were reversed and the sum of non-missing responses is calculated. The FOSI score is the sum multiplied by 8, then divided by the number of responded statements. The FOSI score was missing if a participants had 5 or more missing values in the 8 individual symptom scores at a visit.
Baseline and at end of treatment (estimated to be an average of 5.5 months [minimum of 0 month and maximum of 33 months]).
Change From Baseline at Safety Follow-up in the Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI) Score
Tidsramme: Baseline and at safety follow-up (up to a maximum of 34 months).
Participant reported ovarian cancer symptoms as measured by Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI). The FOSI is an 8-question evaluation scored on a Likert scale of 0-4 from not at all to very much. Metrics assessed include lack of energy, vomiting, pain, nausea, swelling in stomach area, concern condition will get worse, quality of life assessment and stomach cramps. Higher score means worse outcome. The responses to the statements except for statement 7 (I am content with the quality of my life right now) were reversed and the sum of non-missing responses is calculated. The FOSI score is the sum multiplied by 8, then divided by the number of responded statements. The FOSI score was missing if a participants had 5 or more missing values in the 8 individual symptom scores at a visit.
Baseline and at safety follow-up (up to a maximum of 34 months).

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany

Etterforskere

  • Studieleder: Medical Responsible, SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Generelle publikasjoner

Hjelpsomme linker

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 (Faktiske)

30. august 2022

Primær fullføring (Faktiske)

8. juli 2025

Studiet fullført (Faktiske)

14. juli 2025

Datoer for studieregistrering

Først innsendt

13. april 2022

Først innsendt som oppfylte QC-kriteriene

20. april 2022

Først lagt ut (Faktiske)

27. april 2022

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

25. august 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

31. juli 2026

Sist bekreftet

1. juli 2026

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • NIR-OGT-201
  • 2022-001816-25 (EudraCT-nummer)

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

JA

IPD-planbeskrivelse

SpringWorks Therapeutics er forpliktet til datatransparens og deling av data for videre forskning, samtidig som personvernet og konfidensialiteten til forskningsdeltakerne opprettholdes. Relevante data på pasientnivå fra fullførte kliniske studier vil bli gjort tilgjengelig av SpringWorks for kvalifiserte forskere ved godkjenning av rimelige forespørsler etter avidentifikasjon/anonymisering i henhold til gjeldende lov.

IPD-delingstidsramme

IPD from completed trials will be publicly available within 6 months after all of the following events:

  • Approval of a product/new indication by major authorities (FDA, EMA, PMDA if requested) with no pending submissions
  • Public results via primary manuscript or trial registry disclosure
  • Legal authority to share data
  • Privacy protections are in place If approval is not sought or development is globally discontinued, data will be publicly available within 18 months after global trial completion. Further information on how to request data can be found on our website bit.ly/IPD21

Tilgangskriterier for IPD-deling

Qualified researchers may propose access to IPD from sponsored trials via https://vivli.org/members/ourmembers/.

IPD-deling Støtteinformasjonstype

  • STUDY_PROTOCOL
  • SEVJE
  • ANALYTIC_CODE
  • CSR

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. .

Abonnere