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Multi-target Drugs Sequential Combination Therapy in Adults Patients With Newly Diagnosed Primary Immune Thrombocytopenia

24 augustus 2026 bijgewerkt door: Institute of Hematology & Blood Diseases Hospital, China

Efficacy and Safety of Multi-Target Drugs Sequential Combination Therapy in Adults Patients With Newly Diagnosed Primary Immune Thrombocytopenia: A Prospective, Single-arm, Multicenter, Exploratory Trial

This prospective, single-arm, multicenter, exploratory study will enroll 38 adults patients with newly diagnosed primary immune thrombocytopenia (ITP). Patients will receive standard-dose corticosteroids plus a thrombopoietin receptor agonist (TPO-RA) as initial therapy During the core treatment phase (Weeks 1-12). Corticosteroids will be tapered and discontinued within 8 weeks, whereas TPO-RA treatment will continue for 12 weeks. For patients with treatment failure( defined as platelet count < 30 × 10^9/L or less than 2-fold increase of baseline platelet count or bleeding) ,a sequential multitarget combination strategy will be explored in subsequent treatment phases. Specifically, patients with treatment failure after 2 weeks of initial therapy (Weeks 3-12), will receive the ongoing TPO-RA in combination with either rituximab or an anti-CD38 monoclonal antibody（mAb） as sequential combination therapy.

During the core treatment follow-up phase (24 weeks), patients with treatment failure will enter the exploratory treatment phase (Weeks 13-36) .

Patients who received multi-target drug therapy during the core treatment period will switch to an alternative TPO-RA with cross-administered rituximab and anti-CD38 mAb, while those who did not will receive sequential rituximab or anti-CD38 mAb.

Finally, patients will enter the safety follow up period (4 weeks, weeks 37-40).

Studie Overzicht

Toestand

Werving

Conditie

Interventie / Behandeling

Gedetailleerde beschrijving

The study will be conducted in three phases.

Phase 1. Core treatment phase includes initial treatment and sequential combination therapy (12 weeks, weeks 1-12):

Patients will receive standard-dose methylprednisolone or prednisone in combination with a thrombopoietin receptor agonist (TPO-RA) as initial therapy.Corticosteroids will be tapered and discontinued within 8 weeks; tapering will begin immediately in patients who achieve complete response (CR) and will also be initiated after 4 weeks in patients who do not achieve response (R). TPO-RA therapy will continue for 12 weeks, with dose adjustments made according to the approved prescribing information. Patients with treatment failure( defined as platelet count < 30 × 10^9/L or less than 2-fold increase of baseline platelet count or bleeding) after 2 weeks of initial therapy (Weeks 3-12) will receive the ongoing TPO-RA in combination with either rituximab or an anti-CD38 monoclonal antibody as sequential combination therapy.

Phase 2. Core-treatment follow-up and exploratory treatment phase (24weeks, weeks 13-36):

During the core treatment follow-up phase, patients with treatment failure will enter the exploratory treatment phase. Patients who did not receive rituximab or anti-CD38 monoclonal antibody during the core treatment phase will receive a switched TPO-RA combined with either rituximab or anti-CD38 monoclonal antibody, whereas patients who received either rituximab or anti-CD38 monoclonal antibody during the core phase will receive a switched TPO-RA combined with cross-administered rituximab and anti-CD38 monoclonal antibody.TPO-RA therapy will also continue for 12 weeks.

Phase3. Safety follow-up phase (4weeks,weeks 37-40).

Rescue therapy includes, but is not limited to, intravenous immunoglobulin (IVIG), platelet transfusion, and vindesine. A switch to another thrombopoietin receptor agonist (TPO-RA) will be considered rescue therapy during the core treatment period. Administration of recombinant human thrombopoietin (rhTPO) will also be considered rescue therapy.

Studietype

Ingrijpend

Inschrijving (Geschat)

38

Fase

  • Fase 4

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Studie Contact Back-up

Studie Locaties

  • China
      • Tianjin, China, 300020
        • Werving
        • Ethics Committee of Hematology Hospital, Chinese Academy of Medical Sciences
        • Contact:

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

  • Volwassen
  • Oudere volwassene

Accepteert gezonde vrijwilligers

Nee

Beschrijving

Inclusion Criteria:

  1. Age ≥18 years.
  2. Confirmed newly diagnosed primary ITP with a platelet count <30 × 10^9/L.
  3. No prior ITP-related treatment before enrollment, except for standard-dose corticosteroids and/or IVIG for ≤5 days.
  4. Able to understand the study and provide signed informed consent.-

Exclusion Criteria:

  1. Use of corticosteroids or immunosuppressants for a non-ITP condition within 3 months.
  2. Contraindication to corticosteroid therapy.
  3. Arterial or venous thromboembolic event within 3 months.
  4. Pregnant or breastfeeding women.
  5. Current treatment with another investigational drug.

  6. Any other medical history or condition that, in the investigator's judgment, makes the participant unsuitable for the study.

    -

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Niet-gerandomiseerd
  • Interventioneel model: Sequentiële toewijzing
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: Initial therapy
standard-dose corticosteroids plus a thrombopoietin receptor agonist (TPO-RA)
Geneesmiddel: standard-dose methylprednisolone/prednisone.
Methylprednisolone 0.8-1mg/kg/day administered intravenously or orally; or prednisone 1 mg/kg/day, up to a maximum dose of 80 mg/day
Geneesmiddel: thrombopoietin receptor agonist(TPO-RA), including but not limited to hetrombopag or eltrombopag.
Dosing will follow the recommended dose in the prescribing information; in severe ITP, the initial dose will be selected according to BAT (best available therapy) principles.
Experimenteel: Sequential Combination Therapy
the ongoing TPO-RA in combination with either rituximab or an anti-CD38 monoclonal antibody（mAb）
Geneesmiddel: thrombopoietin receptor agonist(TPO-RA), including but not limited to hetrombopag or eltrombopag.
Dosing will follow the recommended dose in the prescribing information; in severe ITP, the initial dose will be selected according to BAT (best available therapy) principles.
Geneesmiddel: rituximab.
Administered as 375 mg/m² by intravenous infusion for a single dose, or 100 mg by intravenous infusion once weekly for a total of 4 doses.
Geneesmiddel: Daratumumab
Administered at 16 mg/kg by intravenous infusion once weekly for a total of 4-8 doses.
Experimenteel: Exploratory treatment
a switched TPO-RA combined with either rituximab or anti-CD38 monoclonal antibody
Geneesmiddel: thrombopoietin receptor agonist(TPO-RA), including but not limited to hetrombopag or eltrombopag.
Dosing will follow the recommended dose in the prescribing information; in severe ITP, the initial dose will be selected according to BAT (best available therapy) principles.
Geneesmiddel: rituximab.
Administered as 375 mg/m² by intravenous infusion for a single dose, or 100 mg by intravenous infusion once weekly for a total of 4 doses.
Geneesmiddel: Daratumumab
Administered at 16 mg/kg by intravenous infusion once weekly for a total of 4-8 doses.

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
sustained response rate
Tijdsspanne: between weeks 13 and 36
Defined as the proportion of patients who achieve a platelet count ≥30×10^9/L at least 6 out of 12 scheduled visits during the 24 weeks following the core treatment period (weeks 13-36), in the absence of rescue therapy
between weeks 13 and 36

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
time to response
Tijdsspanne: in 0-12 weeks
The time from treatment initiation to achieve a complete response(platelet count ≥100 × 109/L and absence of bleeding) or a partial response(platelet count ≥30 × 10^9/L and at least 2-fold increase of the baseline platelet count and absence of bleeding)
in 0-12 weeks
Early response.
Tijdsspanne: at 1 week
Proportion of patients achieving platelet count ≥30 × 10^9/L and at least doubling baseline at 1 week after treatment initiation.
at 1 week
Initial response
Tijdsspanne: at 1 month
Proportion of patients achieving platelet count ≥30 × 10^9/L and at least doubling baseline at 1 month after treatment initiation.
at 1 month
overall response(OR) rate at week 12
Tijdsspanne: at week 12
Proportion of patients achieving complete response (CR) plus response (R) at Week 12
at week 12
Proportion of patients requiring rescue therapy
Tijdsspanne: in 0-36 weeks
in 0-36 weeks
bleeding scores
Tijdsspanne: in 0-40 weeks
Assessed according to the WHO Bleeding Scale and the ITP Bleeding Scale
in 0-40 weeks
Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ)
Tijdsspanne: in 0-40 weeks
In all participants ,use ITP-PAQ to assess the HRQoL before and after treatment.
in 0-40 weeks
Functional Assessment of 36-Item Short Form Survey (SF-36) scale
Tijdsspanne: in 0-40 weeks
In all participants ,use SF-36 scale to assess the HRQoL before and after treatment.
in 0-40 weeks

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Onderzoekers

  • Studie directeur: Lei Zhang, Thrombosis and Haemostasis Diagnosis Treatment Center

Publicaties en nuttige links

De persoon die verantwoordelijk is voor het invoeren van informatie over het onderzoek stelt deze publicaties vrijwillig ter beschikking. Dit kan gaan over alles wat met het onderzoek te maken heeft.

Algemene publicaties

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

22 juni 2026

Primaire voltooiing (Geschat)

30 mei 2027

Studie voltooiing (Geschat)

30 juni 2027

Studieregistratiedata

Eerst ingediend

5 augustus 2026

Eerst ingediend dat voldeed aan de QC-criteria

24 augustus 2026

Eerst geplaatst (Werkelijk)

26 augustus 2026

Updates van studierecords

Laatste update geplaatst (Werkelijk)

26 augustus 2026

Laatste update ingediend die voldeed aan QC-criteria

24 augustus 2026

Laatst geverifieerd

1 juli 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • IIT2025106
  • Investigator-Initiated Trial (Ander subsidie-/financieringsnummer: Chinese Academy of Medical Sciences (CAMS) Innovation Fund for Medical Sciences)

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

NEE

Beschrijving IPD-plan

No plan to share IPD has been established at this time

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Nee

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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