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Studie av DISC-0974 i deltakere med myelofibrose og anemi

14. september 2026 oppdatert av: Disc Medicine, Inc

En fase 1b/2a åpen studie for å evaluere sikkerheten, toleransen, farmakokinetikken og farmakodynamikken til DISC-0974 hos deltakere med myelofibrose og anemi

Denne fase 1b/2a åpne studien vil vurdere sikkerheten, tolerabiliteten, farmakokinetikken og farmakodynamikken til DISC-0974, samt kategorisere effektene på anemirespons hos personer med myelofibrose og anemi.

Studieoversikt

Status

Rekruttering

Forhold

Intervensjon / Behandling

Studietype

Intervensjonell

Registrering (Antatt)

150

Fase

  • Fase 2
  • Fase 1

Kontakter og plasseringer

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

Studiekontakt

Studiesteder

  • Australia
      • Nedlands, Australia, 6009
      • Saint Albans, Australia, 3021
        • Rekruttering
        • Western Health
        • Ta kontakt med:
        • Ta kontakt med:
        • Hovedetterforsker:
          • William Renwick, MBBS
      • Sydney, Australia, 2010
      • West Perth, Australia, 6005
        • Rekruttering
        • Perth Blood Institute
        • Ta kontakt med:
        • Hovedetterforsker:
          • Ross Baker, MBBS, BMedSc, FRACP, FACP
  • Forente stater
    • Arizona
      • Gilbert, Arizona, Forente stater, 85234
        • Rekruttering
        • Banner MD Anderson
        • Ta kontakt med:
        • Hovedetterforsker:
          • Mark Faber, DO
    • California
      • Duarte, California, Forente stater, 91010
        • Rekruttering
        • City of Hope - Duarte
        • Ta kontakt med:
        • Ta kontakt med:
        • Hovedetterforsker:
          • Idoroenyi Amanam, MD
      • Irvine, California, Forente stater, 92618
        • Rekruttering
        • City of Hope - Lennar
        • Hovedetterforsker:
          • Idoroenyi Amanam, MD
        • Ta kontakt med:
        • Ta kontakt med:
      • Los Angeles, California, Forente stater, 90095
      • San Francisco, California, Forente stater, 94143
        • Rekruttering
        • University of California, San Francisco
        • Hovedetterforsker:
          • Jerry Lee, MD, MS
        • Ta kontakt med:
        • Ta kontakt med:
    • Colorado
      • Aurora, Colorado, Forente stater, 80045
        • Rekruttering
        • University of Colorado Anschutz Medical Campus
        • Ta kontakt med:
        • Hovedetterforsker:
          • Brandon McMahon, MD
    • Florida
      • Jacksonville, Florida, Forente stater, 32224
        • Rekruttering
        • Mayo Clinic Jacksonville
        • Hovedetterforsker:
          • James Foran, MD
        • Ta kontakt med:
      • Miami, Florida, Forente stater, 33136
        • Rekruttering
        • Sylvester Cancer Center - U Miami
        • Ta kontakt med:
        • Ta kontakt med:
        • Hovedetterforsker:
          • Sangeetha Venugopal, MD, MS
      • Tampa, Florida, Forente stater, 33612
        • Rekruttering
        • Moffitt Cancer Center
        • Hovedetterforsker:
          • Andrew Kuykendall, MD
        • Ta kontakt med:
    • Georgia
      • Atlanta, Georgia, Forente stater, 30322
    • Michigan
      • Ann Arbor, Michigan, Forente stater, 48109
        • Rekruttering
        • University of Michigan
        • Ta kontakt med:
        • Hovedetterforsker:
          • Moshe Talpaz, MD
    • Minnesota
      • Rochester, Minnesota, Forente stater, 55905
        • Rekruttering
        • Mayo Clinic Rochester
        • Hovedetterforsker:
          • Naseema Gangat, MBBS
        • Ta kontakt med:
    • Missouri
      • St Louis, Missouri, Forente stater, 63110
        • Rekruttering
        • Washington University St.Louis
        • Ta kontakt med:
        • Hovedetterforsker:
          • Amy Zhou, MD
    • New Jersey
      • East Brunswick, New Jersey, Forente stater, 08816
    • New York
      • New York, New York, Forente stater, 10029
        • Rekruttering
        • Icahn School of Medicine at Mount Sinai
        • Hovedetterforsker:
          • John Mascarenhas, MD
        • Ta kontakt med:
        • Ta kontakt med:
      • New York, New York, Forente stater, 10021
        • Rekruttering
        • Memorial Sloan Kettering Cancer Center
        • Hovedetterforsker:
          • Prioty Islam, MD, MSc
        • Ta kontakt med:
        • Ta kontakt med:
      • New York, New York, Forente stater, 10467
        • Rekruttering
        • Montefiore
        • Hovedetterforsker:
          • Swati Goel, MD
        • Ta kontakt med:
        • Ta kontakt med:
    • North Carolina
      • Winston-Salem, North Carolina, Forente stater, 27157
        • Rekruttering
        • Atrium Health Wake Forest Baptist
        • Ta kontakt med:
        • Hovedetterforsker:
          • Anne Wofford, MD
    • Ohio
      • Canton, Ohio, Forente stater, 44718
        • Avsluttet
        • Gabrail Cancer Center Research
      • Cleveland, Ohio, Forente stater, 44195
        • Rekruttering
        • Cleveland Clinic
        • Hovedetterforsker:
          • Aaron Gerds, MD
        • Ta kontakt med:
        • Ta kontakt med:
      • Columbus, Ohio, Forente stater, 43201
        • Rekruttering
        • The Ohio State University
        • Hovedetterforsker:
          • Shivani Handa, MD
        • Ta kontakt med:
        • Ta kontakt med:
      • Maumee, Ohio, Forente stater, 43537
        • Rekruttering
        • Taylor Cancer Research Center
        • Hovedetterforsker:
          • John Nemunaitis, MD
        • Ta kontakt med:
        • Ta kontakt med:
    • Oregon
      • Portland, Oregon, Forente stater, 97239
        • Rekruttering
        • Oregon Health and Science University
        • Ta kontakt med:
        • Hovedetterforsker:
          • Ronan Swords, MD, PhD
    • Pennsylvania
      • Gettysburg, Pennsylvania, Forente stater, 17325
        • Tilbaketrukket
        • Sargon Research - Pennsylvania Cancer Specialists and Research Center
      • Philadelphia, Pennsylvania, Forente stater, 19104
    • Texas
      • Houston, Texas, Forente stater, 77030
        • Rekruttering
        • MD Anderson
        • Hovedetterforsker:
          • Prithviraj Bose, MD
        • Ta kontakt med:
    • Washington
      • Seattle, Washington, Forente stater, 98109
        • Rekruttering
        • University of Washington
        • Hovedetterforsker:
          • Anna Halpern, MD
        • Ta kontakt med:
    • Wisconsin
      • Milwaukee, Wisconsin, Forente stater, 53226
        • Rekruttering
        • Medical College of Wisconsin
        • Hovedetterforsker:
          • Laura Michaelis, MD
        • Ta kontakt med:

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

Inklusjonskriterier:

  1. Alder 18 år eller eldre på tidspunktet for signering av informert samtykke (ICF).
  2. For fase 1b: Dynamic International Prognostic Scoring System (DIPSS) score på 3 til 4 (middels-2 risiko) eller ≥ 5 (høyrisiko) primær MF, post-PV MF og/eller post-ET MF, som bekreftet i den siste lokale benmargsbiopsirapporten, ifølge Verdens helseorganisasjon (WHO) 2016-kriterier.
  3. Utvasking i minst 28 dager før screening av følgende behandlinger: androgener, erytropoietin, kladribin, immunmodulatorer (lenalidomid, thalidomid), interferon alfa-2a eller annen MF-rettet behandling. Systemiske kortikosteroider er tillatt for ikke-hematologiske tilstander hvis dosen er stabil eller reduseres i ≥ 28 dager før screening og mottar tilsvarende ≤ 10 mg prednison i de 28 dagene rett før screening.
  4. Anemi: For fase 1b: Hemoglobin (Hgb) < 10 g/dL ved ≥ 3 vurderinger over 84 dager før screening, uten RBC-transfusjon, eller Hgb < 10 g/dL og mottar RBC-transfusjoner periodisk, men oppfyller ikke kriteriene for TD-deltaker som definert for TD-kohorten. Grunnlinje Hgb-verdien for disse deltakerne er det laveste Hgb-nivået i løpet av de 84 dagene før screening, eller RBC-transfusjonsavhengighet, definert som en RBC-transfusjonsfrekvens på ≥ 6 enheter pakket RBC (PRBC) i løpet av de 84 dagene rett før screening. Det må ikke være noen sammenhengende 42-dagers periode uten RBC-transfusjon i 84-dagersperioden, og siste transfusjon må være innen 28 dager før screening. For fase 2a: RBC-transfusjonsavhengighet, definert som en RBC-transfusjonsfrekvens på ≥ 6 enheter PRBC i løpet av de 84 dagene rett før screening. Det må ikke være noen sammenhengende 42-dagers periode uten RBC-transfusjon i 84-dagersperioden, og siste transfusjon må være innen 28 dager før screening.
  5. Stabil dose av JAK-hemmer og/eller hydroksyurea, eller, hvis du tar annen behandling for MF, stabil i minst 4 måneder før screening.
  6. Eastern Cooperative Oncology Group (ECOG) ytelsespoeng ≤ 2.
  7. Infusjon av hematopoetisk stamcelletransplantasjon er ikke forventet innen 8 måneder etter screening.
  8. Jernkonsentrasjon i leveren ved MR < 7 mg/g tørrvekt.
  9. Serumferritin ≥ 30 μg/L ved screening.
  10. Blodplateantall ≥ 25 000/μL og < 1 000 000/μL; nøytrofiler ≥ 1000/μL; og totalt antall hvite blodlegemer (WBC) < 50 000/μL ved screening.
  11. Estimert glomerulær filtrasjonshastighet (eGFR) ≥ 30 ml/min/1,73 m2 av Chronic Kidney Disease-Epidemiology Collaboration (CKD-EPI) formelen.
  12. Aspartataminotransferase (AST) og alanintransaminase (ALT) < 3,0 x øvre normalgrense (ULN) ved screening.
  13. Direkte bilirubin < 2x ULN ved screening. Høyere nivåer er akseptable hvis disse kan tilskrives ineffektiv erytropoese av etterforskeren.

Ekskluderingskriterier:

Medisinsk historie:

  1. Arvelig hemokromatose
  2. Hemoglobinopati eller iboende RBC-defekt assosiert med anemi
  3. Splenektomi
  4. Hematopoetisk celletransplantasjon
  5. Nåværende anemi fra jernmangel, vitamin B12 eller folatmangel, infeksjon eller blødning
  6. Aktiv immun-mediert hemolytisk anemi
  7. Symptomatisk blødning, ikke relatert til kirurgi, i et kritisk område eller organ og/eller blødning som forårsaker en reduksjon i Hgb på ≥ 2 g/dL eller fører til transfusjon av ≥ 2 enheter RBC i løpet av de 6 månedene før screening
  8. Større operasjon innen 8 uker før screening eller ufullstendig restitusjon fra tidligere operasjoner

  9. Malignitet i løpet av de siste 3 årene, annet enn primær MF, post-ET eller post-PV MF. Følgende historikk eller samtidige forhold er tillatt:

    1. basal- eller plateepitelkarsinom
    2. karsinom in situ i livmorhalsen eller brystet
    3. histologisk funn av prostatakreft (T1a eller T1b ved bruk av tumor, noder, metastase [TNM] klinisk iscenesettelsessystem)
  10. Hjerneslag, dyp venetrombose eller lunge- eller arteriell emboli innen 6 måneder før screening
  11. Kjent allergisk reaksjon på ethvert hjelpestoff i studien, eller anafylaksi mot mat eller medikament
  12. En historie med dannelse av antistoff-antistoff
  13. Utilstrekkelig kontrollert hjertesykdom (New York Heart Association Classification 3 eller 4) og/eller kjent for å ha venstre ventrikkel ejeksjonsfraksjon < 35 %
  14. Aktiv hepatitt B eller C, eller humant immunsviktvirus (HIV) med påvisbar viral belastning

  15. Ukontrollert sopp-, bakterie- eller virusinfeksjon (pågående tegn/symptomer relatert til infeksjonen, uten bedring til tross for passende behandling)

    Behandlingshistorie:

  16. Samtidig eller planlagt behandling med momelotinib i løpet av studieperioden
  17. Jernkelatbehandling i de 3 månedene før screening

  18. Endring i antikoagulantbehandlingsregime innen 8 uker før screening

    Laboratorieekskluderinger:

  19. Myeloblaster i perifert blod ≥ 10 % av WBC-differensial ved siste evaluering før screening
  20. Positiv direkte antiglobulintest i forbindelse med et reaktivt RBC-eluat ved screening

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: Randomisert
  • Intervensjonsmodell: Sekvensiell tildeling
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Fase 1b: Doseeskalering
I fase 1b (dose-eskalering) delen av studien vil DISC-0974 administreres subkutant hver 4. uke.
Legemiddel: DISC-0974
DISC-0974 administreres subkutant.
Eksperimentell: Fase 2: Utvidelse
I fase 2-delen (utvidelse) av studien vil DISC-0974 administreres subkutant hver 4. uke.
Legemiddel: DISC-0974
DISC-0974 administreres subkutant.

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Safety and Tolerability of DISC-0974 (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Assessed by treatment-emergent adverse events
From Day 1 to the end of treatment on Day 169
Safety and Tolerability of DISC-0974 (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Assessed by vital signs through blood pressure (mmHg), heart rate (bpm), respiration rate (breaths/min), and temperature (°C)
From Day 1 to the end of treatment on Day 169
Safety and Tolerability of DISC-0974 (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Assessed by physical examinations
From Day 1 to the end of treatment on Day 169
Safety and Tolerability of DISC-0974 (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Assessed by electrocardiogram (ECG) parameters: heart rate, PR interval, QRS duration, QRS axis, QT interval, and QTcF interval)
From Day 1 to the end of treatment on Day 169
Safety and Tolerability of DISC-0974 assessed through blood testing (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
The blood testing will include a hematology panel, blood chemistry panel, serology/virology panel, biomarker assessments, PK assessments, and Anti-Drug Antibodies
From Day 1 to the end of treatment on Day 169
Safety and Tolerability of DISC-0974 assessed through urine testing (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
The urine testing will include a urinalysis
From Day 1 to the end of treatment on Day 169
Transfusion-dependent (TD) high cohort: transfusion independence (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Defined as the absence of packed red blood cell (PRBC) transfusions over any rolling 12-week interval during the treatment period with a minimum hemoglobin (Hgb) of 7 g/dL. Participants meeting this criterion will be considered to have a major response to treatment.
From Day 1 to the end of treatment on Day 169
TD low cohort: transfusion independence (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Defined as the absence of PRBC transfusions over any rolling 16-week interval during the treatment period with a minimum Hgb of 7 g/dL. Participants meeting this criterion will be considered to have a major response to treatment.
From Day 1 to the end of treatment on Day 169
Non-transfusion-dependent (nTD) cohort: anemia response (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Defined as the composite of the absence of transfusions over any rolling 12-week period and a concomitant mean Hgb increase of ≥1.5 g/dL over baseline. Participants meeting this criterion will be considered to have a major response to treatment.
From Day 1 to the end of treatment on Day 169

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Anemia response defined per IWG-MRT 2006 criteria (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Response in nTD participants is defined as ≥2.0 g/dL increase from baseline in Hgb levels. Response in TD participants requires absence of PRBC transfusions during any rolling 12-week period during the treatment period, capped by an Hgb level of ≥8.5 g/dL.
From Day 1 to the end of treatment on Day 169
TD high and TD low participants will be evaluated for absence of PRBC transfusions for any rolling 12-week interval during the treatment period (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
TD high participants will be evaluated for absence of PRBC transfusions with minimum Hgb of 7 g/dL during any rolling 12-week interval during the treatment period (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
TD low participants will be evaluated for absence of PRBC transfusions with minimum Hgb of 7 g/dL during any rolling 16-week interval during the treatment period (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
nTD participants will be evaluated for ≥1.5 g/dL increase from baseline Hgb levels during the treatment period (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
nTD participants will be evaluated for the composite of the absence of transfusions over any rolling 12-week period and a concomitant mean Hgb increase of ≥1.5 g/dL over baseline (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Incidence of TEAEs following repeated DISC-0974 SC doses in participants with myelodysplastic syndrome (MDS) or MDS/myeloproliferative neoplasm (MPN) without excess blasts (collectively referred to as MDS) and anemia (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Proportion of participants with treatment-emergent adverse events
From Day 1 to the end of treatment on Day 169
Incidence of clinically abnormal vital signs following repeated DISC-0974 SC doses in participants with myelodysplastic syndrome (MDS) or MDS/myeloproliferative neoplasm (MPN) without excess blasts and anemia (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Incidence of clinically abnormal physical exam following repeated DISC-0974 SC doses in participants with myelodysplastic syndrome (MDS) or MDS/myeloproliferative neoplasm (MPN) without excess blasts and anemia (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Incidence of clinically abnormal electrocardiograms (ECGs) following repeated DISC-0974 SC doses in participants with myelodysplastic syndrome (MDS) or MDS/myeloproliferative neoplasm (MPN) without excess blasts and anemia (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Incidence of abnormal laboratory test results following repeated DISC-0974 SC doses in participants with myelodysplastic syndrome (MDS) or MDS/myeloproliferative neoplasm (MPN) without excess blasts and anemia (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Pharmacokinetic data of DISC-0974 following repeated SC doses in participants with myelodysplastic syndrome (MDS) or MDS/myeloproliferative neoplasm (MPN) without excess blasts and anemia (Phase 1b only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Pharmacokinetic parameters include DISC-0974 pre-dose concentrations at different visits
From Day 1 to the end of treatment on Day 169
Proportion of participants achieving a mean Hgb increase ≥1 g/dL or ≥2 g/dL from baseline over any rolling 12-week period in absence of PRBC transfusions in each cohort (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
Participants who are nTD and achieve a mean Hgb increase ≥1 g/dL from baseline over any rolling 12-week period in the absence of transfusion will be considered to have a minor response to treatment.
From Day 1 to the end of treatment on Day 169
PD markers of mechanism engagement, including exploratory cohorts assessed through serum iron levels (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
PD markers of mechanism engagement, including exploratory cohorts assessed through TSAT levels (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
PD markers of mechanism engagement, including exploratory cohorts assessed through Ferritin levels (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
PD markers of mechanism engagement, including exploratory cohorts assessed through Transferrin levels (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
PD markers of mechanism engagement, including exploratory cohorts assessed through Serum-hepcidin-25 levels (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Hematologic Parameters assessed through Reticulocyte count (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Hematologic Parameters assessed through Hemoglobin levels (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Hematologic Parameters assessed through Reticulocyte Hemoglobin (CHr) (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Hematologic Parameters assessed through Red Blood Cell Count (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Rate of RBC transfusions per participant month during the treatment period for each cohort (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
The number of units of RBC transfusions per participant month during the treatment period for each cohort (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Transfusion-dependent cohorts will be evaluated for proportion of participants who reduce their transfusion requirement by ≥50%, as compared to baseline, over any rolling 12-week period during treatment. (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
Participants who are TD and achieve a reduction in transfusion requirement ≥50% as compared to baseline over any rolling 12-week period during treatment will be considered to have a minor response to treatment.
From Day 1 to the end of treatment on Day 169
nTD participants will be evaluated for longest duration of mean Hgb increase of ≥1.5 g/dL from baseline during the treatment period (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Mean change in Hgb over 12-week treatment periods will be evaluated for all cohorts (nTD, TD low, and TD high) (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Maximum duration of RBC-transfusion-independent response for TD participants (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Proportion of participants that require dose escalation in each cohort (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Proportion of participants that improve Functional Assessment of Cancer Therapy-Anemia (FACT-An) subscale by at least 3 points in each cohort during the treatment period (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Mean hemoglobin increase of ≥1.5 g/dL over any rolling 12-week interval and an increase in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue of 3 points by the end of study (EOS) for nTD participants (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
TD high cohort will be evaluated for absence of packed red blood cell (PRBC) transfusions a terminal 12-week interval during the treatment period with a minimum hemoglobin (Hgb) of 7 g/dL (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
TD low cohort: will be evaluated for the absence of PRBC transfusions a terminal 16-week interval during the treatment period with a minimum Hgb of 7 g/dL (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
From Day 1 to the end of treatment on Day 169
Non-transfusion-dependent (nTD) cohort will be evaluated for anemia response (Phase 1b and 2)
Tidsramme: From Day 1 to the end of treatment on Day 169
Defined as the composite of the absence of transfusions over any rolling 12-week period and a concomitant mean Hgb increase of ≥1.5 g/dL over baseline
From Day 1 to the end of treatment on Day 169
Safety and Tolerability of DISC-0974 (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Assessed by treatment-emergent adverse events
From Day 1 to the end of treatment on Day 169
Safety and Tolerability of DISC-0974 (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Assessed by vital signs through blood pressure (mmHg), heart rate (bpm), respiration rate (breaths/min), and temperature (°C)
From Day 1 to the end of treatment on Day 169
Safety and Tolerability of DISC-0974 (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Assessed by physical examinations
From Day 1 to the end of treatment on Day 169
Safety and Tolerability of DISC-0974 (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Assessed by electrocardiogram (ECG) parameters: heart rate, PR interval, QRS duration, QRS axis, QT interval, and QTcF interval)
From Day 1 to the end of treatment on Day 169
Safety and Tolerability of DISC-0974 assessed through blood testing (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
The blood testing will include a hematology panel, blood chemistry panel, serology/virology panel, biomarker assessments, PK assessments, and Anti-Drug Antibodies
From Day 1 to the end of treatment on Day 169
Safety and Tolerability of DISC-0974 assessed through urine testing (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
The urine testing will include a urinalysis
From Day 1 to the end of treatment on Day 169
Safety and tolerability of DISC-0974 following repeated SC doses in participants with MF receiving concomitant momelotinib or pacritinib therapy (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Assessed by treatment-emergent adverse events
From Day 1 to the end of treatment on Day 169
Safety and tolerability of DISC-0974 following repeated SC doses in participants with MF receiving concomitant momelotinib or pacritinib therapy (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Assessed by vital signs through blood pressure (mmHg), heart rate (bpm), respiration rate (breaths/min), and temperature (°C)
From Day 1 to the end of treatment on Day 169
Safety and tolerability of DISC-0974 following repeated SC doses in participants with MF receiving concomitant momelotinib or pacritinib therapy (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Assessed by physical examinations
From Day 1 to the end of treatment on Day 169
Safety and tolerability of DISC-0974 following repeated SC doses in participants with MF receiving concomitant momelotinib or pacritinib therapy (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
Assessed by electrocardiogram (ECG) parameters: heart rate, PR interval, QRS duration, QRS axis, QT interval, and QTcF interval)
From Day 1 to the end of treatment on Day 169
Safety and tolerability of DISC-0974 following repeated SC doses in participants with MF receiving concomitant momelotinib or pacritinib therapy assessed through blood testing (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
The blood testing will include a hematology panel, blood chemistry panel, serology/virology panel, biomarker assessments, PK assessments, and Anti-Drug Antibodies
From Day 1 to the end of treatment on Day 169
Safety and tolerability of DISC-0974 following repeated SC doses in participants with MF receiving concomitant momelotinib or pacritinib therapy assessed through urine testing (Phase 2 only)
Tidsramme: From Day 1 to the end of treatment on Day 169
The urine testing will include a urinalysis
From Day 1 to the end of treatment on Day 169

Andre resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Cmax (Phase 1b, 2, and Exploratory Cohorts)
Tidsramme: From Day 1 to the end of treatment on Day 169
Maximum drug concentration (observed). Will be determined from blood PK sampling if appropriate data are available
From Day 1 to the end of treatment on Day 169
Tmax (Phase 1b, 2, and Exploratory Cohorts)
Tidsramme: From Day 1 to the end of treatment on Day 169
Observed time of the maximum drug concentration. Will be determined from blood PK sampling if appropriate data are available
From Day 1 to the end of treatment on Day 169
AUC (Phase 1b, 2, and Exploratory Cohorts)
Tidsramme: From Day 0 to 29 days after the first dose
Area under the drug concentration-time curve calculated using linear trapezoidal summation from time zero to 29 days following the first dose. Will be determined from blood PK sampling if appropriate data are available.
From Day 0 to 29 days after the first dose

Samarbeidspartnere og etterforskere

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

Sponsor

Disc Medicine, Inc

Etterforskere

  • Studieleder: Will Savage, MD PhD, Disc Medicine

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)

6. juni 2022

Primær fullføring (Antatt)

1. mai 2027

Studiet fullført (Antatt)

1. juni 2027

Datoer for studieregistrering

Først innsendt

15. mars 2022

Først innsendt som oppfylte QC-kriteriene

1. april 2022

Først lagt ut (Faktiske)

11. april 2022

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

15. september 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

14. september 2026

Sist bekreftet

1. juni 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • DISC-0974-102

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

produkt produsert i og eksportert fra USA

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