Study on Glucocorticoid Combined With Gamma Globulin and Ropristine N01 in Treatment of Initial Severe ITP

The goal of this clinical trial is to learn if romiplostim N01 works to treat severe primary immune thrombocytopenia in adults. It will also learn about the safety of romiplostim N01. The main question it aims to answer is:

TO evaluate the effectiveness of the combination treatment of glucocorticoids, gamma globulin and romiplostim N01 in patients with severe primary immune thrombocytopenia during the initial treatment period for one month.

Participants will:

Romiplostim N01, starting dose of 1 µg/kg, subcutaneous injection, given on the third day of IVIG, once a week, for up to one month.

IVIG, 400mg/kg/d, intravenous administration, continuous administration for 3-5 days.

Prednisone, 0.8mg/kg/d, taken orally or intravenously, for up to one month.

Study Overview

• Status: Recruiting
• Conditions: Primary Immune Thrombocytopenia
• Intervention / Treatment: Drug: corticosteroids combined with immunoglobulin and romiplostim

• Study Type: Interventional
• Enrollment (Estimated): 36
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Liang Tang; Phone Number: 13554153411; Email: lancet.tang@qq.com

Study Locations

• China
  • Hubei
    • Wuhan, Hubei, China, 430000
      • Recruiting
      • Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
      • Contact: Liang Tang; Phone Number: 13554153411; Email: lancet.tang@qq.com
      • Contact: Yu Hu, MD; Phone Number: 027-85726114; Email: dr_huyu387@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Participants must sign a written informed consent prior to enrollment;
2. Ages 18-75;
3. Platelet count <10×10^9/L with active bleeding or bleeding score ≥5;
4. No contraindications to the use of steroids, romiplostim, or IVIG, and willingness to receive these treatments;
5. No prior splenectomy or at least one first-line ITP treatment or emergency treatment;
6. No prior romiplostim treatment;
7. ECOG PS score: 0-2;
8. Female participants of childbearing potential must agree to use reliable contraception (including male or female condoms, contraceptive foam, contraceptive jelly, contraceptive patches, contraceptive cream, contraceptive suppositories, abstinence, and placement of intrauterine contraceptive devices) throughout the study; women who have had a hysterectomy, bilateral oophorectomy, bilateral salpingo-oophorectomy, or have been postmenopausal for more than 1 year, and men who have had bilateral vasectomy or vasectomy are excluded;
9. Voluntary participation in this study and good compliance.

Exclusion Criteria:

1. Individuals with the following hematological disorders other than ITP, including but not limited to leukemia, thrombocytopenia caused by cancer treatment, myeloproliferative diseases, multiple myeloma, and myelodysplastic syndrome;
2. Individuals with a history of thrombosis;
3. Pregnant or lactating individuals;
4. Individuals with severe cardiopulmonary insufficiency;
5. Individuals with severe or uncontrollable infections;
6. Individuals with viral infections;
7. Individuals who cannot comply due to psychological reasons;
8. Individuals who are deemed unsuitable for participation in the study by the investigator.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Corticosteroids combined with immunoglobulin and romiplostim; Romiplostim N01, starting dose of 1 µg/kg, subcutaneous injection, given on the third day of IVIG, once a week, for up to one month. IVIG, 400mg/kg/d, intravenous administration, continuous administration for 3-5 days. Prednisone, 0.8mg/kg/d, taken orally or intravenously, for up to one month.	Drug: corticosteroids combined with immunoglobulin and romiplostim; Romiplostim N01, starting dose of 1 µg/kg, subcutaneous injection, given on the third day of IVIG, once a week, for up to one month. IVIG, 400mg/kg/d, intravenous administration, continuous administration for 3-5 days. Prednisone, 0.8mg/kg/d, taken orally or intravenously, for up to one month.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The total effective rate of treatment for 30 days	GR = CR + R (Complete reaction CR, platelet count ≥ 100×10^9/L, and no bleeding symptoms; Effective R, platelet count between 30×10^9/L and 100×10^9/L, and at least double the baseline value, and no bleeding symptoms)	one months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Early (week) response rate R	Effective R, platelet count between 30×10^9/L and 100×10^9/L, and at least double the baseline value, and no bleeding symptoms	one months
Early Complete Response Rate (CR)	Complete reaction CR, platelet count ≥ 100×10^9/L, and no bleeding symptoms	one months
Time to Reaction	Time of Starting Treatment Until Platelet Count ≥ 30×10^9/L	one months
Adverse events during treatment	evaluated using Version 5.0 of the "Common Terminology Criteria for Adverse Events(NCI CTC AE)"	four months

Collaborators and Investigators

• Sponsor: Wuhan Union Hospital, China
• Collaborators: Qilu Hospital of Shandong University; The First Affiliated Hospital of Soochow University; Institute of Hematology & Blood Diseases Hospital, China

Study record dates

Study Major Dates

• Study Start (Actual): November 20, 2024
• Primary Completion (Estimated): September 20, 2025
• Study Completion (Estimated): December 20, 2025

Study Registration Dates

• First Submitted: November 11, 2024
• First Submitted That Met QC Criteria: November 11, 2024
• First Posted (Actual): November 13, 2024

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 19, 2025
• Last Verified: August 1, 2024

More Information

Terms related to this study

• Keywords: Romiplostim; severe ITP
• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Purpura, Thrombocytopenic, Idiopathic; Immunologic Factors; Physiological Effects of Drugs; Antibodies; Immunoglobulins; Immunoglobulins, Intravenous
• Other Study ID Numbers: 2024-0503

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No