Evaluating PN20 for treating adults with primary immune thrombocytopenia

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

This study is testing a new treatment called PN20 to see if it can help adults with primary immune thrombocytopenia who haven't had success with other treatments.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Chongqing Peg-Bio Biopharm Co., Ltd. (Industry-sponsored)
• Drugs / interventions: cyclophosphamide
• Locations: 5 sites (Guiyang, Guizhou and 4 other locations)
• Trial ID: NCT06880081 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to assess the safety and tolerability of PN20, a thrombopoietin receptor agonist, in adult patients diagnosed with primary immune thrombocytopenia (ITP). It is a multicenter, open-label, single-arm, dose-escalation Phase 1b study where participants will receive a single subcutaneous injection of PN20 based on their weight. The study will monitor various parameters including pharmacokinetics, pharmacodynamics, immunogenicity, and preliminary efficacy over a treatment period followed by a four-week post-treatment follow-up. The trial seeks to determine if PN20 can provide a beneficial effect for patients who have not responded to previous treatments.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 65 with a diagnosis of primary immune thrombocytopenia and a history of treatment failure.

Not a fit: Patients with active malignancies or significant bone marrow disorders may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could offer a new therapeutic option for patients with primary immune thrombocytopenia who have limited treatment alternatives.

How similar studies have performed: While this approach is novel in the context of PN20, similar studies with thrombopoietin receptor agonists have shown promise in treating ITP.

Eligibility criteria

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.

Where this trial is running

Guiyang, Guizhou and 4 other locations

• Affiliated Hospital of Guizhou Medical University — Guiyang, Guizhou, China (Recruiting)
• The Second Affiliated Hospital of Nanchang University — Nanchang, Jiangxi, China (Recruiting)
• Jining First People's Hospital — Jining, Shandong, China (Recruiting)
• Hematology Hospital of Chinese Academy of Medical Sciences — Tianjin, Tianjin Municipality, China (Recruiting)
• The Second Affiliated Hospital of Kunming Medical University — Kunming, Yunnan, China (Recruiting)

Study contacts

• Principal investigator: Lei Zhang — Chinese Academy of Medical Sciences
• Study coordinator: Jing Sun

Conditions Primary Immune Thrombocytopenia; thrombopoietin receptor agonist; PN20