PN20 for raising low platelets in chronic liver disease before elective procedures

A Phase Ib/II Clinical Study to Evaluate the Efficacy, Safety, Pharmacokinetic and Pharmacodynamic Characteristics of a Single Subcutaneous Injection of Polyethylene Glycolated Thrombopoietin Peptide (PN20) in the Treatment of Thrombocytopenia in Patients With Chronic Liver Disease Undergoing an Elective Procedure

This study tests whether a single injection of PN20 can safely raise platelet counts in adults with chronic liver disease who have low platelets and need an elective invasive procedure.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 54 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Chongqing Peg-Bio Biopharm Co., Ltd. (Industry-sponsored)
• Locations: 8 sites (Beijing, Beijing Municipality and 7 other locations)
• Trial ID: NCT07446491 on ClinicalTrials.gov

What this trial studies

This two-stage, single-dose study first uses an open-label, dose-escalation Phase Ib to identify safe dose levels and then a randomized, double-blind, placebo-controlled Phase II to test efficacy. Eligible adults with chronic liver disease and platelet counts below 50 × 10^9/L receive a single subcutaneous injection of PN20 or placebo by weight before a planned elective procedure and return for clinic visits to monitor outcomes. Phase Ib enrolls sequential cohorts at three ascending doses, while Phase II randomizes participants 1:1 (or 1:1:1 if two doses are carried forward) to PN20 or placebo using the dose(s) selected from Phase Ib. Key endpoints include change in platelet count, bleeding events around the procedure, safety/tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity.

Who should consider this trial

Good fit: Adults (≥18) with chronic liver disease–related thrombocytopenia (baseline platelets <50 × 10^9/L), Child-Pugh A or B, ECOG 0–1, and a planned elective invasive procedure within 7–15 days are ideal candidates.

Not a fit: Patients with more advanced liver disease (Child-Pugh C), platelet counts ≥50 × 10^9/L, those needing emergency surgery, or with contraindicated comorbidities or pregnancy are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, PN20 could temporarily raise platelet counts to lower bleeding risk around elective procedures and reduce the need for platelet transfusions.

How similar studies have performed: Other thrombopoietin-receptor agonists (for example, avatrombopag and lusutrombopag) have successfully increased platelets for procedures, so the general approach has clinical precedent, though PN20 itself is newly tested here.

Eligibility criteria

Inclusion Criteria:

* Age ≥ 18 years, Male or female
* Diagnosed with chronic liver disease-related thrombocytopenia (baseline platelet count \< 50 × 10⁹/L), Child-Pugh score A or B;
* Planned elective invasive procedure or minor surgery within Days 7-15 post-enrollment, excluding laparotomy, thoracotomy, craniotomy, open-heart surgery, organ resection, or partial organ resection (for tissue biopsy and other types of tissue resection, enrollment is permitted if the investigator considers the risk of bleeding and invasiveness to be comparable to or lower than that of the procedures listed in the surgical examples list);
* Eastern Cooperative Oncology Group (ECOG) performance status score of 0 or 1;
* Women of childbearing potential must have a negative pregnancy test
* Agree to use reliable contraception through 3 months post-study.
* Participants must understand the study requirements and provide written informed consent.

Exclusion Criteria:

* The following conditions or medical history:

  * Presence of other conditions that may lead to thrombocytopenia;
  * History of thrombotic or significant cardiovascular events;
  * Uncontrolled bleeding;
  * Advanced liver cancer;
  * Severe comorbidities;
* Currently using or with a history of the following treatments:

  * Recent major surgery or splenectomy;
  * Liver transplant;
  * Recent therapies affecting platelet counts;
* Significant lab abnormalities;
* Recent participation in other clinical trials;
* Known hypersensitivity to the investigational productstudy drug；
* Active substance abuse;
* Any condition deemed by the investigator to compromise safety or study integrity.

Where this trial is running

Beijing, Beijing Municipality and 7 other locations

• Capital Medical University Affiliated Beijing Ditan Hospital — Beijing, Beijing Municipality, China (Recruiting)
• The Second Affiliated Hospital of Chongqing Medical University — Chongqing, Chongqing Municipality, China (Recruiting)
• The Eighth Affiliated Hospital of Guangzhou Medical University — Guangzhou, Guangdong, China (Recruiting)
• Henan Provincial People's Hospital — Zhengzhou, Henan, China (Recruiting)
• Zhenjiang Third People's Hospital — Zhenjiang, Jiangsu, China (Recruiting)
• Jilin University First Hospital — Changchun, Jilin, China (Recruiting)
• Liaocheng People's Hospital — Chaozhou, Shandong, China (Recruiting)
• Qingdao Municipal Hospital — Qingdao, Shandong, China (Recruiting)

Study contacts

• Study coordinator: Jing Sun

Conditions Thrombocytopenia Associated With Chronic Liver Disease; PN20; Thrombocytopenia; Chronic Liver Disease