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Undersøgelse af DISC-0974 i deltagere med myelofibrose og anæmi

16. juli 2026 opdateret af: Disc Medicine, Inc

Et fase 1b/2a åbent studie til evaluering af sikkerhed, tolerabilitet, farmakokinetik og farmakodynamik af DISC-0974 hos deltagere med myelofibrose og anæmi

Denne fase 1b/2a åbne undersøgelse vil vurdere sikkerheden, tolerabiliteten, farmakokinetikken og farmakodynamikken af ​​DISC-0974 samt kategorisere virkningerne på anæmisrespons hos personer med myelofibrose og anæmi.

Studieoversigt

Status

Rekruttering

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

150

Fase

  • Fase 2
  • Fase 1

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Studiesteder

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

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

18 år og ældre (Voksen, Ældre voksen)

Tager imod sunde frivillige

Ingen

Beskrivelse

Inklusionskriterier:

  1. Alder 18 år eller ældre på tidspunktet for underskrivelsen af ​​det informerede samtykke (ICF).
  2. For fase 1b: Dynamic International Prognostic Scoring System (DIPSS) score på 3 til 4 (mellem-2 risiko) eller ≥ 5 (højrisiko) primær MF, post-PV MF og/eller post-ET MF, som bekræftet i den seneste lokale knoglemarvsbiopsirapport ifølge Verdenssundhedsorganisationen (WHO) 2016-kriterier.
  3. Udvaskning i mindst 28 dage før screening af følgende behandlinger: androgener, erythropoietin, cladribin, immunmodulatorer (lenalidomid, thalidomid), interferon alfa-2a eller enhver anden MF-rettet behandling. Systemiske kortikosteroider er tilladt til ikke-hæmatologiske tilstande, hvis dosis er stabil eller faldende i ≥ 28 dage før screening og modtagelse svarende til ≤ 10 mg prednison i de 28 dage umiddelbart før screening.
  4. Anæmi: For fase 1b: Hæmoglobin (Hgb) < 10 g/dL ved ≥ 3 vurderinger over 84 dage før screening, uden RBC-transfusion, eller Hgb < 10 g/dL og modtager RBC-transfusioner periodisk, men opfylder ikke kriterierne for TD-deltager som defineret for TD-kohorten. Baseline Hgb-værdien for disse deltagere er det laveste Hgb-niveau i løbet af de 84 dage før screening, eller RBC-transfusionsafhængighed, defineret som en RBC-transfusionsfrekvens på ≥ 6 units packed RBC'er (PRBC) i løbet af de 84 dage umiddelbart før screening. Der må ikke være nogen sammenhængende 42-dages periode uden en RBC-transfusion i den 84-dages periode, og den sidste transfusion skal være inden for 28 dage før screening. For fase 2a: RBC-transfusionsafhængighed, defineret som en RBC-transfusionsfrekvens på ≥ 6 enheder PRBC over de 84 dage umiddelbart før screening. Der må ikke være nogen sammenhængende 42-dages periode uden en RBC-transfusion i den 84-dages periode, og den sidste transfusion skal være inden for 28 dage før screening.
  5. Stabil dosis af JAK-hæmmer og/eller hydroxyurinstof, eller, hvis der tages anden behandling for MF, stabil i mindst 4 måneder før screening.
  6. Eastern Cooperative Oncology Group (ECOG) præstationsscore ≤ 2.
  7. Infusion af hæmatopoietisk stamcelletransplantation forventes ikke inden for 8 måneder efter screening.
  8. Leverjernkoncentration ved MRI < 7 mg/g tørvægt.
  9. Serum ferritin ≥ 30 μg/L ved screening.
  10. Blodpladetal ≥ 25.000/μL og < 1.000.000/μL; neutrofiler ≥ 1.000/μL; og totalt antal hvide blodlegemer (WBC) < 50.000/μL ved screening.
  11. Estimeret glomerulær filtrationshastighed (eGFR) ≥ 30 ml/min/1,73 m2 ved formlen for kronisk nyresygdom-epidemiologisk samarbejde (CKD-EPI).
  12. Aspartataminotransferase (AST) og alanintransaminase (ALT) < 3,0 x øvre normalgrænse (ULN) ved screening.
  13. Direkte bilirubin < 2x ULN ved screening. Højere niveauer er acceptable, hvis disse af investigator kan tilskrives ineffektiv erytropoiese.

Ekskluderingskriterier:

Medicinsk historie:

  1. Arvelig hæmokromatose
  2. Hæmoglobinopati eller iboende RBC-defekt forbundet med anæmi
  3. Splenektomi
  4. Hæmatopoietisk celletransplantation
  5. Aktuel anæmi fra jernmangel, vitamin B12 eller folatmangel, infektion eller blødning
  6. Aktiv immunmedieret hæmolytisk anæmi
  7. Symptomatisk blødning, som ikke er relateret til operation, i et kritisk område eller organ og/eller blødning, der forårsager et fald i Hgb på ≥ 2 g/dL eller fører til transfusion af ≥ 2 enheder RBC i de 6 måneder før screening
  8. Større operation inden for 8 uger før screening eller ufuldstændig genopretning fra en tidligere operation

  9. Malignitet inden for de seneste 3 år, bortset fra primær MF, post-ET eller post-PV MF. Følgende historie eller samtidige forhold er tilladt:

    1. basal- eller planocellulært karcinom
    2. carcinom in situ af livmoderhalsen eller brystet
    3. histologisk fund af prostatacancer (T1a eller T1b ved hjælp af tumor, noder, metastase [TNM] klinisk iscenesættelsessystem)
  10. Slagtilfælde, dyb venetrombose eller lunge- eller arteriel emboli inden for 6 måneder før screening
  11. Kendt allergisk reaktion på et hvilket som helst hjælpestof i undersøgelseslægemidlet eller anafylaksi over for enhver fødevare eller medicin
  12. En historie med dannelse af antistof-antistof
  13. Utilstrækkeligt kontrolleret hjertesygdom (New York Heart Association Classification 3 eller 4) og/eller kendt for at have venstre ventrikulær ejektionsfraktion < 35 %
  14. Aktiv hepatitis B eller C eller human immundefektvirus (HIV) med påviselig viral belastning

  15. Ukontrolleret svampe-, bakterie- eller virusinfektion (vedvarende tegn/symptomer relateret til infektionen, uden bedring trods passende behandling)

    Behandlingshistorie:

  16. Samtidig eller planlagt behandling med momelotinib i undersøgelsesperioden
  17. Jernchelatbehandling i de 3 måneder før screening

  18. Ændring i antikoagulantbehandlingsregimen inden for 8 uger før screening

    Laboratorieudelukkelser:

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

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: Randomiseret
  • Interventionel model: Sekventiel tildeling
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Fase 1b: Dosiseskalering
I fase 1b-delen (dosis-eskalering) af studiet vil DISC-0974 blive administreret subkutant hver 4. uge.
Medicin: DISC-0974
DISC-0974 indgives subkutant.
Eksperimentel: Fase 2: Udvidelse
I fase 2 (udvidelses) delen af ​​studiet vil DISC-0974 blive administreret subkutant hver 4. uge.
Medicin: DISC-0974
DISC-0974 indgives subkutant.

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
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
Foranstaltningsbeskrivelse
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
Foranstaltningsbeskrivelse
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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

Disc Medicine, Inc

Efterforskere

  • Studieleder: Will Savage, MD PhD, Disc Medicine

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

6. juni 2022

Primær færdiggørelse (Anslået)

1. maj 2027

Studieafslutning (Anslået)

1. juni 2027

Datoer for studieregistrering

Først indsendt

15. marts 2022

Først indsendt, der opfyldte QC-kriterier

1. april 2022

Først opslået (Faktiske)

11. april 2022

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

20. juli 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

16. juli 2026

Sidst verificeret

1. juni 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • DISC-0974-102

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

INGEN

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

produkt fremstillet i og eksporteret fra U.S.A.

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

Kliniske forsøg med Myelodysplastiske syndromer

Kliniske forsøg med DISC-0974

Søg i lignende forsøg

Sponsorer og samarbejdspartnere

Medicinske tilstande

Narkotikainterventioner

CROs by country

CROs in Sweden

Betingelser

Sjældne sygdomme

Narkotikainterventioner

Kosttilskud

Sponsor / samarbejdspartnere

Placeringer

Abonner