A Study of Romiplostim N01 as the First-line Treatment for Newly Diagnosed Adult Patients With ITP

NCT06658834 | Recruiting Phase 2 DMC

• 1 other identifier: IIT2024046
• Study Type: interventional
• Target: 129
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a multicenter interventional research on the first-line treatment of newly diagnosed adult patients with immune thrombocytopenia (ITP) using romiplostim N01 in combination with glucocorticoids. The primary endpoint of this study is to assess the efficacy of romiplostim N01 combined with glucocorticoids in untreated newly diagnosed adult ITP patients after 6 months of administration. The subjects will be divided into the experimental group and the control group for treatment. Experimental group: Dexamethasone (HD-DXM) 40mg/d × 4 days, one cycle. If there is no response on the 10th day, repeat once, administered either orally or intravenously. Simultaneously, romiplostim N01 is administered at an initial dose of 3µg/kg, by subcutaneous injection, once a week, for a maximum of 6 months. Control group: Dexamethasone (HD-DXM) 40mg/d × 4 days, one cycle. If there is no response on the 10th day, repeat once, administered either orally or intravenously.

Conditions

Primary Immune Thrombocytopenia

Keywords

ITP; Romiplostim; Continuous Remission

Outcome Measures

Primary Outcomes (1)

• The proportion of patients with continuous remission.

  Continuous remission is defined as the maintenance of the therapeutic effect of patients for at least 6 months since achieving remission, without the need for additional ITP-specific treatment.

  6 months

Secondary Outcomes (8)

• The total effective rate GR

  6 months

• The proportion of patients with the initial response (reaching the effective standard within one month of the start of treatment)

  one month of the start of treatment

• The proportion of patients reaching the effective standard 3 months after the start of treatment.

  3 months after the start of treatment

• The proportion of patients reaching the effective standard 6 months after the start

  6 months after the start

• The maximum consecutive weeks of platelet response

  6 months

Study Arms (2)

romiplostim combined with glucocorticoids

EXPERIMENTAL

Dexamethasone (HD-DXM) at a dose of 40mg/d for 4 days constitutes one cycle. If there is no response on the 10th day, repeat it once. The administration can be either oral or intravenous. Meanwhile, romiplostim N01 is administered with an initial dose of 3µg/kg by subcutaneous injection once a week for up to 6 months.

Drug: Dexamethasone Combined with romiplostim N01

glucocorticoids

ACTIVE COMPARATOR

Dexamethasone (HD-DXM) 40mg/d × 4 days, one cycle. If there is no response on the 10th day, repeat once, administered either orally or intravenously.

Drug: Dexamethasone monotherapy

Interventions

Dexamethasone Combined with romiplostim N01 DRUG

Dexamethasone (HD-DXM) at a dose of 40mg/d for 4 days constitutes one cycle. If there is no response on the 10th day, repeat it once. The administration can be either oral or intravenous. Meanwhile, romiplostim N01 is administered with an initial dose of 3µg/kg by subcutaneous injection once a week for up to 6 months.

romiplostim combined with glucocorticoids

Dexamethasone monotherapy DRUG

Dexamethasone (HD-DXM) 40mg/d × 4 days, one cycle. If there is no response on the 10th day, repeat once, administered either orally or intravenously.

glucocorticoids

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Sign the written informed consent form before enrollment;
• Age ranging from 18 to 75 years old;
• Be clinically diagnosed with primary immune thrombocytopenia for less than 3 months before randomization;
• Have not received splenectomy or at least one first-line ITP treatment or emergency treatment in the past;
• Have not received romiplostim treatment;
• ECOG PS score: 0 - 2;
• Platelet value \< 30×10\^9/L;
• The expected survival period at the screening is ≥ 12 weeks;
• For subjects of reproductive age, agree to take reliable contraceptive measures throughout the study period (including male or female condoms, contraceptive foams, contraceptive gels, contraceptive membranes, contraceptive ointments, contraceptive suppositories, abstinence, and intrauterine device placement, etc.); Female subjects who have undergone hysterectomy, bilateral salpingectomy, bilateral tubal ligation or menopause for more than 1 year, and male subjects who have undergone bilateral vasectomy or ligation are excluded;
• Voluntarily join this study, sign the informed consent form, and have good compliance.

You may not qualify if:

• Suffering from other hematopoietic system diseases except ITP, including but not limited to leukemia, thrombocytopenia caused by tumor treatment, myeloproliferative diseases, multiple myeloma and myelodysplastic syndrome, etc.;
• Having undergone splenectomy before the first administration;
• Having received ITP drug treatment (including emergency treatment) before the first administration;
• Having used drugs with c-Mpl (thrombopoietin receptor) stimulating effects within 4 weeks before the first administration;
• Having received hematopoietic growth factor preparations (such as granulocyte colony-stimulating factor, macrophage colony-stimulating factor, erythropoietin, interleukin-11, etc.) within 4 weeks before the first administration;
• Having received antibody drugs (such as rituximab, etc.) within 14 weeks before the first administration;
• Having received any Chinese herbal medicine or nutritional supplement (except vitamin supplements and mineral supplements) for the purpose of increasing platelets within 1 week before the first administration;
• Having been diagnosed with arterial thrombosis (such as cerebral thrombosis, transient ischemic attack or myocardial infarction), or having a history or complication of venous thrombosis (such as deep vein thrombosis, pulmonary embolism), or using anticoagulants or antiplatelet drugs at the beginning of screening;
• Having a history of severe cardiovascular diseases (such as grade III/IV congestive heart failure, arrhythmia or angina pectoris that increases the risk of thromboembolic events, unstable angina pectoris, having undergone coronary artery stent implantation, angioplasty or coronary artery bypass grafting);
• Secondary thrombocytopenia caused by autoimmune diseases such as antiphospholipid antibody syndrome, systemic lupus erythematosus, Hashimoto's thyroiditis, Even's syndrome and Sjogren's syndrome;
• Positive results for either human immunodeficiency virus antibody or syphilis antibody screening; positive hepatitis C antibody and HCV-RNA exceeding the upper limit of the study center's laboratory test; positive hepatitis B surface antigen and HBV-DNA exceeding the upper limit of the study center's laboratory test;
• Having participated in other clinical studies within 3 months before the first administration;
• Being pregnant or lactating, or having a pregnancy plan;
• Having fertility and being judged by the researcher as not fully adopting contraceptive measures;
• Having a history of severe drug allergic reactions or being known to be allergic to glucocorticoids or Nplate® (romiplostim) or the components of QL0911;

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead
• The Second Affiliated Hospital of Kunming Medical University: collaborator
• Weifang People's Hospital: collaborator
• Shenzhen Second People's Hospital: collaborator
• The Affiliated Hospital of Qingdao University: collaborator
• Changzhi Medical College: collaborator
• Affiliated Hospital of Nantong University: collaborator
• The First People's Hospital of Yunnan: collaborator
• The First Affiliated Hospital of Bengbu Medical University: collaborator
• Suining Central Hospital: collaborator
• The Second Affiliated Hospital of Dalian Medical University: collaborator
• North China University of Science and Technology: collaborator
• Guizhou Provincial People's Hospital: collaborator

Study Sites (1)

Chinese Academy of Medical Science and Blood Disease Hospital

Tianjin, Tianjin Municipality, 300000, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

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

Central Study Contacts

Lei Zhang, MD

CONTACT

+8613502118379; zhanglei1@ihcams.ac.cn

Yunfei Chen, MD

CONTACT

+8618502220788; chenyunfei@ihcams.ac.cn

Study Design

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

Study Record Dates

• First Submitted: September 19, 2024
• First Posted: October 26, 2024
• Study Start: June 1, 2024
• Primary Completion: June 30, 2025
• Study Completion (Estimated): August 30, 2026
• Last Updated: October 26, 2024

Record last verified: 2024-04

Locations

CN (1)