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Studie av DISC-0974 i deltagare med myelofibros och anemi

14 september 2026 uppdaterad av: Disc Medicine, Inc

En öppen fas 1b/2a studie för att utvärdera säkerhet, tolerabilitet, farmakokinetik och farmakodynamik hos DISC-0974 hos deltagare med myelofibros och anemi

Denna öppna fas 1b/2a studie kommer att bedöma säkerheten, tolerabiliteten, farmakokinetiken och farmakodynamiken för DISC-0974 samt kategorisera effekterna på anemisvaret hos patienter med myelofibros och anemi.

Studieöversikt

Status

Rekrytering

Betingelser

Intervention / Behandling

Studietyp

Interventionell

Inskrivning (Beräknad)

150

Fas

  • Fas 2
  • Fas 1

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

Studieorter

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

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

18 år och äldre (Vuxen, Äldre vuxen)

Tar emot friska volontärer

Nej

Beskrivning

Inklusionskriterier:

  1. Ålder 18 år eller äldre vid tidpunkten för undertecknandet av det informerade samtycket (ICF).
  2. För fas 1b: Dynamic International Prognostic Scoring System (DIPSS)-poäng på 3 till 4 (mellanrisk 2) eller ≥ 5 (högrisk) primär MF, post-PV MF och/eller post-ET MF, som bekräftats i den senaste lokala benmärgsbiopsirapporten, enligt Världshälsoorganisationens (WHO) 2016 kriterier.
  3. Uttvättning i minst 28 dagar före Screening av följande behandlingar: androgener, erytropoietin, kladribin, immunmodulatorer (lenalidomid, talidomid), interferon alfa-2a eller någon annan MF-inriktad terapi. Systemiska kortikosteroider är tillåtna för icke-hematologiska tillstånd om dosen är stabil eller sjunkande i ≥ 28 dagar före screening och får motsvarande ≤ 10 mg prednison under de 28 dagarna omedelbart före screening.
  4. Anemi: För fas 1b: Hemoglobin (Hgb) < 10 g/dL vid ≥ 3 bedömningar under 84 dagar före screening, utan RBC-transfusion, eller Hgb < 10 g/dL och får RBC-transfusioner regelbundet men uppfyller inte kriterierna för TD-deltagare som definieras för TD-kohorten. Baslinje Hgb-värdet för dessa deltagare är den lägsta Hgb-nivån under de 84 dagarna före screening, eller RBC-transfusionsberoende, definierat som en RBC-transfusionsfrekvens på ≥ 6 enheter packade RBC (PRBC) under de 84 dagarna omedelbart före screening. Det får inte finnas någon på varandra följande 42-dagarsperiod utan en RBC-transfusion under 84-dagarsperioden, och den sista transfusionen måste ske inom 28 dagar före screening. För fas 2a: RBC-transfusionsberoende, definierat som en RBC-transfusionsfrekvens på ≥ 6 enheter PRBC under de 84 dagarna omedelbart före screening. Det får inte finnas någon på varandra följande 42-dagarsperiod utan en RBC-transfusion under 84-dagarsperioden, och den sista transfusionen måste ske inom 28 dagar före screening.
  5. Stabil dos av JAK-hämmare och/eller hydroxiurea, eller, om man tar någon annan behandling för MF, stabil i minst 4 månader före screening.
  6. Eastern Cooperative Oncology Group (ECOG) resultatpoäng ≤ 2.
  7. Infusion av hematopoetisk stamcellstransplantation förväntas inte inom 8 månader efter screening.
  8. Järnkoncentration i levern vid MRT < 7 mg/g torrvikt.
  9. Serumferritin ≥ 30 μg/L vid screening.
  10. Trombocytantal ≥ 25 000/μL och < 1 000 000/μL; neutrofiler ≥ 1 000/μL; och totalt antal vita blodkroppar (WBC) < 50 000/μL vid screening.
  11. Uppskattad glomerulär filtrationshastighet (eGFR) ≥ 30 ml/min/1,73 m2 enligt formeln Chronic Kidney Disease-Epidemiology Collaboration (CKD-EPI).
  12. Aspartataminotransferas (AST) och alanintransaminas (ALAT) < 3,0 x övre normalgräns (ULN) vid screening.
  13. Direkt bilirubin < 2x ULN vid screening. Högre nivåer är acceptabla om dessa av utredaren kan hänföras till ineffektiv erytropoes.

Exklusions kriterier:

Medicinsk historia:

  1. Ärftlig hemokromatos
  2. Hemoglobinopati eller inneboende RBC-defekt i samband med anemi
  3. Splenektomi
  4. Hematopoetisk celltransplantation
  5. Aktuell anemi från järnbrist, vitamin B12 eller folatbrist, infektion eller blödning
  6. Aktiv immunförmedlad hemolytisk anemi
  7. Symtomatisk blödning, utan samband med operation, i ett kritiskt område eller organ och/eller blödning som orsakar en minskning av Hgb på ≥ 2 g/dL eller leder till transfusion av ≥ 2 enheter RBC under de 6 månaderna före screening
  8. Stor operation inom 8 veckor före screening eller ofullständig återhämtning från någon tidigare operation

  9. Malignitet under de senaste 3 åren, annan än primär MF, post-ET eller post-PV MF. Följande historik eller samtidiga villkor är tillåtna:

    1. basal- eller skivepitelcancer
    2. karcinom in situ i livmoderhalsen eller bröstet
    3. histologiska fynd av prostatacancer (T1a eller T1b med tumör, noder, metastaser [TNM] kliniskt stadiesystem)
  10. Stroke, djup ventrombos eller lung- eller arteriell emboli inom 6 månader före screening
  11. Känd allergisk reaktion mot något studieläkemedelshjälpämne, eller anafylaxi mot något livsmedel eller läkemedel
  12. En historia av anti-läkemedelsantikroppsbildning
  13. Otillräckligt kontrollerad hjärtsjukdom (New York Heart Association Classification 3 eller 4) och/eller känd för att ha vänsterkammars ejektionsfraktion < 35 %
  14. Aktiv hepatit B eller C, eller humant immunbristvirus (HIV) med detekterbar virusmängd

  15. Okontrollerad svamp-, bakterie- eller virusinfektion (pågående tecken/symtom relaterade till infektionen, utan förbättring trots lämplig behandling)

    Behandlingshistorik:

  16. Samtidig eller planerad behandling med momelotinib under studieperioden
  17. Järnkelatbehandling under 3 månader före screening

  18. Ändring av antikoagulantiabehandlingsregimen inom 8 veckor före screening

    Laboratorieundantag:

  19. Myeloblaster i perifert blod ≥ 10 % av skillnaden mellan vita blodkroppar vid den senaste utvärderingen före screening
  20. Positivt direkt antiglobulintest i samband med ett reaktivt RBC-eluat vid screening

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: Randomiserad
  • Interventionsmodell: Sekventiell tilldelning
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: Fas 1b: Doseskalering
I fas 1b-delen (dosupptrappning) av studien kommer DISC-0974 att administreras subkutant var fjärde vecka.
Läkemedel: DISC-0974
DISC-0974 administreras subkutant.
Experimentell: Fas 2: Expansion
I fas 2 (expansion) delen av studien kommer DISC-0974 att administreras subkutant var fjärde vecka.
Läkemedel: DISC-0974
DISC-0974 administreras subkutant.

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Safety and Tolerability of DISC-0974 (Phase 1b only)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Anemia response defined per IWG-MRT 2006 criteria (Phase 1b only)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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

Andra resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Cmax (Phase 1b, 2, and Exploratory Cohorts)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Disc Medicine, Inc

Utredare

  • Studierektor: Will Savage, MD PhD, Disc Medicine

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

6 juni 2022

Primärt slutförande (Beräknad)

1 maj 2027

Avslutad studie (Beräknad)

1 juni 2027

Studieregistreringsdatum

Först inskickad

15 mars 2022

Först inskickad som uppfyllde QC-kriterierna

1 april 2022

Första postat (Faktisk)

11 april 2022

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

15 september 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

14 september 2026

Senast verifierad

1 juni 2026

Mer information

Termer relaterade till denna studie

Nyckelord

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • DISC-0974-102

Plan för individuella deltagardata (IPD)

Planerar du att dela individuella deltagardata (IPD)?

NEJ

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Ja

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

produkt tillverkad i och exporterad från U.S.A.

Nej

Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .

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