Study of PN20 in Adult Patients With Primary Immune Thrombocytopenia (ITP)

A Phase 1b, Open-label, Single-arm, Dose-escalation Study of PN20 in Patients With Primary Immune Thrombocytopenia (ITP)

The main aim of this clinical trial is to assess the safety and tolerability of PN20 in adult patients with primary immune thrombocytopenia (ITP). The main questions it aims to answer are:

• Is PN20 safe in these patients?
• Could these patients potentially benefit from PN20 treatment?

Participants will

• Receive one subcutaneous injection of PN20 according to weight;
• Visit the clinic for assessment.

Study Overview

• Status: Recruiting
• Conditions: Primary Immune Thrombocytopenia (ITP)
• Intervention / Treatment: Drug: PN20 treatment

Detailed Description

This is a multicenter, open-label, single-arm, dose-escalation Phase1b study aimed at evaluating the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), immunogenicity, and preliminary efficacy of a single dose of PN20 in adult patients with primary immune thrombocytopenia (ITP).

The trial is a dose escalation study, including Screening (no more than 14 days), Treatment (single dose) and the post-treatment follow-up (4 weeks).

And the safety, tolerability, pharmacokinetics, pharmacodynamics, immunogenicity, and preliminary efficacy of PN20 in ITP will be assessed in this study.

• Study Type: Interventional
• Enrollment (Estimated): 12
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Jing Sun; Phone Number: 86-13983367811; Email: sunj@pegbiocq.com

Study Locations

• China
  • Guizhou
    • Guiyang, Guizhou, China, 550004
      • Recruiting
      • Affiliated Hospital of Guizhou Medical University
      • Contact: Jishi Wang
  • Jiangxi
    • Nanchang, Jiangxi, China, 330006
      • Recruiting
      • The Second Affiliated Hospital of Nanchang University
      • Contact: Li Yu
  • Shandong
    • Jining, Shandong, China, 272011
      • Recruiting
      • Jining First People's Hospital
      • Contact: Tongfeng Zhao
  • Tianjin Municipality
    • Tianjin, Tianjin Municipality, China, 301609
      • Recruiting
      • Hematology Hospital of Chinese Academy of Medical Sciences
      • Contact: Lei Zhang
  • Yunnan
    • Kunming, Yunnan, China, 650000
      • Recruiting
      • The Second Affiliated Hospital of Kunming Medical University
      • Contact: Zeping Zhou

Participation Criteria

Eligibility Criteria

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

Description

Key Inclusion Criteria:

1. Aged between 18 and 65 years (inclusive), male or female;
2. Diagnosed with primary immune thrombocytopenia (ITP) and have a disease course of more than 6 months;
3. Patients who have no response or relapsed after splenectomy. Or patients who have not been splenectomised and have completed at least 1 prior treatment for ITP (such as glucocorticoids, immunoglobulin) and have failed to respond or relapsed after treatment;
4. During screening period, the mean of two platelet counts must be < 30 × 10^9/L, with none >35 × 10^9/L;
5. Fully understand and are able to comply with the requirements of the protocol, voluntarily participate and sign the informed consent form.

Key Exclusion Criteria:

1. History of bone marrow stem cell disorder. Any abnormal bone marrow findings other than typical of ITP.
2. Any active malignancy. If prior history of cancer other than basal cell carcinoma or cervical carcinoma in situ, no treatment or active disease within 5 years prior to signing the ICF.
3. Diagnosed with arterial thrombotic disease (such as cerebral thrombosis, transient ischemic attack, myocardial infarction, peripheral arterial disease, etc.); Patients with a history or complications of venous thrombosis (such as deep vein thrombosis, pulmonary embolism); or patients who are using anticoagulants or antiplatelet drugs at the beginning of screening.
4. Subjects who have used romiplostim or its analogs in the past and have not responded.
5. Subjects who have used eltrombopag, haitubopag, avatubopag, recombinant human thrombopoietin (rhTPO), or other drugs with c-MPl stimulating effects within the 4 weeks prior to signing the ICF.
6. Received any anti-malignancy agents (e.g., cyclophosphamide, mercaptopurine, vincristine, vinblastine, interferon-alfa) within 8 weeks prior to signing the ICF.
7. Less than 4 weeks since end of any clinical trials about therapeutic drug or device prior to signing the ICF.
8. Laboratory abnormalities with clinical significance at screening visit.
9. In the opinions of investigators, the patients are not suitable for participation in this trial.

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: PN20 treatment group-single dose; A single dose of PN20 will be administered.	Drug: PN20 treatment; Three dose cohorts, single dose, subcutaneous injections; Other Names: thrombopoietin receptor agonist

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Event (AE)	The incidence of adverse events related to PN20.	Treatment and post-treatment follow-up period (28 days after a single dose)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The percentage of subjects who reach the target therapeutic platelet level after administration	It is defined as the proportion of subjects whose platelet count reaches at least 2 times the baseline platelet count within 28 days after administration and is within the range of 50×10^9/L to 450×10^9/L (inclusive).	Treatment and post-treatment follow-up period (28 days after a single dose)

Collaborators and Investigators

• Sponsor: Chongqing Peg-Bio Biopharm Co., Ltd.
• Investigators: Principal Investigator: Lei Zhang, Chinese Academy of Medical Sciences

Study record dates

Study Major Dates

• Study Start (Actual): March 7, 2025
• Primary Completion (Estimated): February 27, 2026
• Study Completion (Estimated): April 28, 2026

Study Registration Dates

• First Submitted: March 11, 2025
• First Submitted That Met QC Criteria: March 11, 2025
• First Posted (Actual): March 17, 2025

Study Record Updates

• Last Update Posted (Actual): March 2, 2026
• Last Update Submitted That Met QC Criteria: February 26, 2026
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Keywords: thrombopoietin receptor agonist; Primary Immune Thrombocytopenia (ITP); PN20
• 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; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Hemic and Lymphatic Diseases; Purpura, Thrombocytopenic, Idiopathic
• Other Study ID Numbers: CQPJ-PN20-ITP-Ib

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
• product manufactured in and exported from the U.S.: No