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

NCT06880081 | Recruiting Phase 1

• 1 other identifier: CQPJ-PN20-ITP-Ib
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 5

Brief Summary

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.

Conditions

Primary Immune Thrombocytopenia (ITP)

Keywords

Primary Immune Thrombocytopenia (ITP); thrombopoietin receptor agonist; PN20

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• The percentage of subjects who reach the target therapeutic platelet level after administration

  Treatment and post-treatment follow-up period (28 days after a single dose)

Study Arms (1)

PN20 treatment group-single dose

EXPERIMENTAL

A single dose of PN20 will be administered.

Drug: PN20 treatment

Interventions

PN20 treatment DRUG

Three dose cohorts, single dose, subcutaneous injections

Also known as: thrombopoietin receptor agonist

PN20 treatment group-single dose

Eligibility Criteria

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

You may qualify if:

• Aged between 18 and 65 years (inclusive), male or female;
• Diagnosed with primary immune thrombocytopenia (ITP) and have a disease course of more than 6 months;
• 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;
• During screening period, the mean of two platelet counts must be \< 30 × 10\^9/L, with none \>35 × 10\^9/L;
• Fully understand and are able to comply with the requirements of the protocol, voluntarily participate and sign the informed consent form.

You may not qualify if:

• History of bone marrow stem cell disorder. Any abnormal bone marrow findings other than typical of ITP.
• 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.
• 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.
• Subjects who have used romiplostim or its analogs in the past and have not responded.
• 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.
• Received any anti-malignancy agents (e.g., cyclophosphamide, mercaptopurine, vincristine, vinblastine, interferon-alfa) within 8 weeks prior to signing the ICF.
• Less than 4 weeks since end of any clinical trials about therapeutic drug or device prior to signing the ICF.
• Laboratory abnormalities with clinical significance at screening visit.
• In the opinions of investigators, the patients are not suitable for participation in this trial.

Sponsors & Collaborators

• Chongqing Peg-Bio Biopharm Co., Ltd.: lead

Study Sites (5)

Affiliated Hospital of Guizhou Medical University

Guiyang, Guizhou, 550004, China

RECRUITING

The Second Affiliated Hospital of Nanchang University

Nanchang, Jiangxi, 330006, China

RECRUITING

Jining First People's Hospital

Jining, Shandong, 272011, China

RECRUITING

Hematology Hospital of Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 301609, China

RECRUITING

The Second Affiliated Hospital of Kunming Medical University

Kunming, Yunnan, 650000, 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

Study Officials

• Lei Zhang

  Chinese Academy of Medical Sciences

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jing Sun

CONTACT

86-13983367811; sunj@pegbiocq.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 11, 2025
• First Posted: March 17, 2025
• Study Start: March 7, 2025
• Primary Completion: February 27, 2026
• Study Completion: April 28, 2026
• Last Updated: March 2, 2026

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (5)