Primary Immune Thrombocytopenia (ITP)Phase 1NCT06880081
Study of PN20 in Adult Patients With Primary Immune Thrombocytopenia (ITP)
A Phase 1 study testing PN20 treatment in people with Primary Immune Thrombocytopenia (ITP), run by Chongqing Peg-Bio Biopharm Co., Ltd. at 5 sites in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Primary Immune Thrombocytopenia (ITP)
- What is being tested?
- PN20 treatment
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 5 sitesChina
- Study dates
- 7 Mar 2025to 14 Feb 2026
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
China5 sites
Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou, 550004
Jining First People's Hospital
Jining, Shandong, 272011
The Second Affiliated Hospital of Kunming Medical University
Kunming, Yunnan, 650000
The Second Affiliated Hospital of Nanchang University
Nanchang, Jiangxi, 330006
Hematology Hospital of Chinese Academy of Medical Sciences
Tianjin, Tianjin Municipality, 301609
About this study
Official title: 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.
Read the 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.
- Conditions
- Primary Immune Thrombocytopenia (ITP)
- Being tested
- PN20 treatment
- Keywords
- Primary Immune Thrombocytopenia (ITP), thrombopoietin receptor agonist, PN20
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PN20 treatmentDrug
Also called thrombopoietin receptor agonist
Three dose cohorts, single dose, subcutaneous injections
| Study group | Role | What they receive |
|---|---|---|
| PN20 treatment group-single dose | Experimental | A single dose of PN20 will be administered. Receives: Drug: PN20 treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Event (AE)
The incidence of adverse events related to PN20.
Measured: 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
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).
Measured: Treatment and post-treatment follow-up period (28 days after a single dose)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2025
- Study start7 Mar 2025
- Primary completion14 Feb 2026
- Study completion14 Feb 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immune Thrombocytopenia (ITP)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Chongqing Peg-Bio Biopharm Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Lei Zhang, Principal investigator, Chinese Academy of Medical Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06880081
- Sponsor's protocol ID
- CQPJ-PN20-ITP-Ib
- Status last verified
- Oct 2026
- First submitted
- 11 Mar 2025
- Last updated
- 21 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No