Lusutrombopag in the Treatment of Immune Thrombocytopenia (ITP)

NCT06287567 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: IIT2023078
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 1

Brief Summary

This exploratory study is to investigate the efficacy, safety and tolerability of Lusutrombopag in the treatment of primary immune thrombocytopenia in Chinese patients who have failed first-line therapy

Conditions

Idiopathic Thrombocytopenic Purpura; Immune Thrombocytopenia

Keywords

Lusutrombopag; Immune thrombocytopenia; Thrombopoietin receptor agonists

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants with a Platelet Response after 4 Weeks

  Platelet responses to lusutrombopag was evaluated using the platelet count defined as ≥50x10\^9 /L on study drug treatment after 4 weeks of dosing（on Day 29) or prematurely ≥250x10\^9 /L prior to Day 29.

  on Day 29 of Treatment

Secondary Outcomes (17)

• Percentage of Participants with a Platelet Response after 12 Weeks (Day 85)

  on Day 85 of treatment

• Percentage of Participants Who Achieved a Platelet Count of ≥50×10^9/L After 6 Weeks of Dosing

  on Day 43 of treatment

• Percentage of Participants Who at least once Achieved a Platelet Count of ≥50x10^9/L in Week 1 ,2,3,4 in the Core Study

  Baseline, Week 4

• Change in Platelet Count

  Baseline, 12 Weeks

• Percentage of Participants Who at least once Achieved a Platelet Count of≥50x10^9 /L during the Treatment

  Baseline, up to 12 Weeks

Study Arms (1)

Lusutrombopag

EXPERIMENTAL

Participants receive lusutrombopag 3 mg administered orally once a day for up to 4 weeks and titrated to a maximum dose of 6 mg during week 5 and week12 based on the platelet count (PLT)

Drug: Lusutrombopag Oral Tablet

Interventions

Lusutrombopag Oral Tablet DRUG

Participants receive lusutrombopag 3 mg administered orally once a day for up to 4 weeks and doses are adjusted based on platelet counts during week 5-12. If a subject's platelet count remains \< 50x10\^9 /L, the dose could have been increased up to a maximum dose of 6 mg（2 tablets).If a subject's platelet count reaches ≥ 250x10\^9 /L during the first 4 weeks, treatment is stopped and participants are allowed to enter into Titration Study in advance when platelet count drops to ≥100x10\^9 /L.

Also known as: MULPLETA®, S-888711

Lusutrombopag

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males and females ≥18 years of age
• Subjects\>60 years must have had a diagnostic bone marrow aspiration in the last 3 years or responded to treatment (platelet count ≥50 x 10\^9/L)
• Participants diagnosed with primary persistent/chronic ITP (greater than or equal to 3 months duration) and an average of two platelet count less than 30 x 10\^9/L. Conditions which may cause thrombocytopenia other than ITP should be ruled out, including but not limited to systemic lupus erythematosus (SLE)，aplastic anemia (AA), and myelodysplastic syndromes (MDS)
• Relapsed persistent or chronic ITP status, with or without prior splenectomy. Participants who previously received one or more ITP therapies
• Subjects receiving rescue therapy (including but not limited to corticosteroids, immunoglobulins and immunosuppressant) must have completed these therapies for at least 1 week or failed within 1 week prior to dosing on the first day (Visit 1)
• Subjects receiving stable dosages of cyclosporine A, mycophenolate mofetil, azathioprine, or danazol are allowed, but must be receiving a dose that has been stable for at least 4 weeks prior to dosing on the first day (Visit 1)
• Subjects receiving Chinese herbal medicine must be stopped 1 weeks prior to dosing on the first day (Visit 1), and Chinese herbal medicine is not allowed during the study
• Subjects receiving steroid therapy must be on a stable dose for at least 2 weeks prior to screening (same milligram amount ± 10%； and ≤20 mg of equivalent dose of prednisone)
• Two consecutive mean platelet count \< 30×10\^9/(a single platelet count of\< 35×10\^9/L is allowed） during screening if subjects were not receiving steroids, or two consecutive mean platelet count \< 50×10\^9/ if they were receiving steroids. Two Platelet counts must be measured at an interval of greater than 2 days and less than 14 days，and the second platelet count must be measured within 96 hours of day1（Visit 1）
• Prothrombin time (PT) and activated partial thromboplastin time (APTT) within 20% of the upper limit of normal (ULN) at Screening or PT did not exceed normal value by ±3s and APTT by ±10s. No other history of coagulation state except ITP.
• All subjects must agree to take progestin or barrier contraception: patients with potential fertility (excluding hysterectomy, bilateral salpingectomy, bilateral tubal ligation or postmenopausal women for more than one year; Men with bilateral vasectomy) must take effective contraceptive measures at least 2 weeks before taking the study drug for the first time, throughout the study and within 28 days after the end of the study (or early termination of the study); Women with potential fertility must have a negative pregnancy test during the screening period and on the 0th day of the trial.
• A signed and dated written consent obtained prior to the performance of Screening procedures

You may not qualify if:

• History of inherited or acquired, clinically important hemorrhagic clotting disorder
• Females who were pregnant or lactating, or receiving other hormone/chemical contraceptives
• Patients with potential fertility refused to take contraceptive methods
• Laboratory abnormalities
• Hemoglobin \<10.0 g/dL for men or women, not clearly related to ITP
• Absolute neutrophil count \< 1000/mm3
• Abnormal peripheral blood smear with evidence of fibrosis confirmed by bone marrow biopsy
• Total bilirubin \> 1.5 x ULN
• Alanine aminotransferase (ALT) \> 1.5 x ULN
• Aspartate aminotransferase (AST) \> 1.5 x ULN
• Creatinine \> 1.5 x ULN
• Human immunodeficiency virus positive
• Hepatitis A Immunoglobulin M（IgM) antibody positive, hepatitis B surface antigen positive and HBV DNA ≥1000IU/ml or hepatitis C antibody positive，with a history of acute hepatitis, cirrhosis, portal hypertension or chronic active hepatitis.
• Thyroid stimulating hormone (TSH) \> 1.5 x ULN; or
• Free thyroxine (T4) \> 1.5 x ULN

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, 300020, China

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

lusutrombopag

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Zhang Lei, MD

  Chinese Academy of Medical Science and Blood Disease Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 1, 2024
• First Posted: March 1, 2024
• Study Start: April 17, 2024
• Primary Completion: November 13, 2024
• Study Completion: March 6, 2025
• Last Updated: December 19, 2025

Record last verified: 2025-12

Locations

CN (1)