Essential ThrombocythemiaPhase 2NCT06552429
Peginterferon α-2b Injection for Hydroxyurea Resistant or Intolerant ET
A Phase 2 study testing Peginterferon α-2b injection in people with Essential Thrombocythemia, run by Xiamen Amoytop Biotech Co., Ltd. at 8 sites in China.
Trial summary
This is a multicenter, randomized, open-label Phase 2 clinical study. It is aimed to enroll 27 essential thrombocytopenia (ET) patients who are resistant to or intolerant of hydroxyurea(HU). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Essential Thrombocythemia
- What is being tested?
- Peginterferon α-2b injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 8 sitesChina
- Study dates
- 29 Aug 2024to Sep 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female subjects, aged greater or equal to 18 years old at screening;
- Subjects diagnosed as high-risk ET according to the World Health Organization (WHO) 2016 criteria：1) who is older than 60 years and JAK2V617F positive at screening, 2) or who previously suffered from disease-related thrombosis or hemorrhage;
- Subjects who have previously received HU for ET, and the time interval between the last HU dose and the first dose of the study drug should not be less than 7 days;
- Interferon treatment-naïve, and for those who have previously received interferon the the time interval between the last dose of interferon and randomization should not be less than 1 month;
- Patients with confirmed hydroxyurea resistance or intolerant, as at least one of the following criteria is met:
- Platelet count remain greater than 600×10^9 /L after at least 3 months of HU treatment at a dose ≥2g/d (dose ≥2.5 g/d if subject weight > 80 kg);
Show all 17Show fewer
- Platelet count greater than 400*10^9/L while white blood cell (WBC) count lower than 2.5*10^9/L, or platelet count greater than 400*10^9 /L while hemoglobin lower than 100 g/L at any dose of HU;
- Presence of HU-related toxicities at any dose of HU: e.g. ulcers in legs, or any unacceptable skin mucosal manifestations or fever;
- Platelet counts > 450*10^9/L at screening;
- Neutrophil count ≥1.0*10^9/L at screening;
- Haemoglobin ≥11 g/dL at screening for males and 10 g/dL for females at screening;
- There is no serious function damage in liver and kidney: total bilirubin ≤1.5 upper limit of normal (ULN), alanine aminotransferase≤2.0 ULN, aspartate aminotransferase≤2.0 ULN, prothrombin time is prolonged by less than 4 seconds, Creatinine clearance ≥50 mL/min (according to Cockcroft-Gault formula) at screening;
- Both male and female subjects must agree take an appropriate contraceptive method, including:
- Male subjects: must agree to use reliable contraception from inform consent until 6 months following the last dose of the study drug.
- Female subjects: Must meet at least one of the following conditions:
- Women without childbearing potential; ii) Women of childbearing potential: no pregnant or breastfeed, negative in blood pregnancy test within 4 days prior to the first dosing, and must agree to use reliable contraception from inform consent until 6 months following the last dose of the study drug;
- Subjects understand the objective, characteristic, method and possible adverse reactions of the study, voluntarily participate in this study, and sign informed consent.
Who cannot join
- History of any other myeloproliferative tumors, or evidence of the presence of any other myeloproliferative tumors;
- Contraindications or hypersensitivities to interferons of any of its excipients;
- Severe medical conditions or serious comorbidities that the investigators determined could jeopardize the safety or protocol adherence, e.g. New York Heart Association [NYHA] Class III-IV, congestive heart failure, symptomatic arrhythmias，pulmonary hypertension;
- History of major organ transplantation;
- Documented autoimmune disease or history of autoimmune disease at screening, e.g. medication un-controlled thyroid dysfunction, autoimmune hepatitis, idiopathic thrombocytopenic purpura, scleroderma, psoriasis, or any autoimmune arthritis;
- Clinically significant pulmonary infiltration, infectious pneumonia, and non-infectious pneumonia at screening that, in the investigator's opinion, would jeopardize the safety of the subject or their compliance with the protocol;
Show all 14Show fewer
- Infection with systemic clinical manifestations at screening, e.g., bacteria, fungi, human immunodeficiency virus, excluding hepatitis B and/or C;
- Evidence of severe retinopathy, e.g., cytomegalovirus retinitis, symptomatic macular degeneration, or clinically significant eye disease, e.g. due to diabetes mellitus or hypertension;
- Diagnosed clinically significant depression or a history of depression and, in the investigator's opinion, previous suicide attempts or at any risk of suicide at screening;
- Diagnosed clinically significant neurological disease or a history of clinically significant neurological disease, except for a history of stable cerebral thrombosis or cerebral hemorrhage;
- History of any malignancy within 5 years (except stage 0 chronic lymphocytic leukemia, basal cell carcinoma, squamous cell carcinoma, and superficial melanoma);
- A history of alcohol or drug abuse within 1 year;
- Have used any investigational drug within 4 weeks prior to first dose of investigational drug, or not recovered from the effects of prior investigational drug administration;
- Other situations that, in the investigator's opinion, not appropriate for inclusion.
Trial locations and contacts
8 sites in 1 country. Contact the study team or a site near you to ask about taking part.
8 sites in 1 country
China8 sites, 8 recruiting
- Recruiting
Peking Union Hospital, Chinese Academy of Medical Sciences
Beijing
Minghui Duan, 13621262462, mhduan@sina.com
- Recruiting
Peking University People's Hospital
Beijing
Hongxia Shi, 13661049635, hxshi55@sina.com
- Recruiting
Union Hospital affiliated to Fujian Medical University
Fujian
Yong Wu, 13365910911, wuyong9195@126.com
- Recruiting
Nanfang Hospital, Southern Medical University
Guangzhou
Na Xu, 18620698390, sprenaa@163.com
- Recruiting
Harbin First Hospital
Harbin
Tiejun Gong, 13836027737, arac@sina.com
- Recruiting
Henan Cancer Hospital
Henan
Hu Zhou, 13939068863, tigerzhoupumc@163.com
- Recruiting
Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai
Wen Wu, 13611770009, ww10649@rjh.com.cn
- Recruiting
The First Affiliated Hospital of Zhejiang University School of Medicine
Zhejiang
Hongyan Tong, 13958122357, hongyantong@aliyun.com
About this study
Official title: A Phase 2 Multicenter, Randomized, Open-label Study to Evaluate the Pharmacokinetic, Safety and Efficacy of Peginterferon Alfa-2b Injection in Subjects With Essential Thrombocythemia Who Are Resistant to or Intolerant of Hydroxyurea.
This is a multicenter, randomized, open-label Phase 2 clinical study. It is aimed to enroll 27 essential thrombocytopenia (ET) patients who are resistant to or intolerant of hydroxyurea(HU). Eligible patients will be randomized to receive either Peginterferon α-2b 135 mcg or Peginterferon α-2b 180 mcg at a ratio of 1:2, and all subjects will go through a target treatment period (Weeks 1 ~ Week 48), an extension treatment period (Weeks 49 ~ Week 96) and a follow-up period (Weeks 97 ~ Week 100). Pharmacokinetics, safety, efficacy will be evaluated.
- Conditions
- Essential Thrombocythemia
- Being tested
- Peginterferon α-2b injection
- Keywords
- Essential Thrombocythemia, Peginterferon α-2b, hydroxyurea resistant, hydroxyurea intolerant
- Medical subject headings
- Thrombocythemia, Essential
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Peginterferon α-2b injectionDrug
Peginterferon α-2b injection, 135 mcg, s.c., once a week, during the targeted treatment period (the first 48 week), peginterferon α-2b dose is depended on the patient's response and tolerability during the extension treatment (week 49 to week 96).
Peginterferon α-2b injectionDrug
Peginterferon α-2b injection, 180 mcg, s.c., once a week, during the targeted treatment period (the first 48 week), peginterferon α-2b dose is depended on the patient's response and tolerability during the extension treatment (week 49 to week 96).
| Study group | Role | What they receive |
|---|---|---|
| Peginterferon α-2b 135 mcg dose group | Experimental | Not described Receives: Drug: Peginterferon α-2b injection |
| Peginterferon α-2b 180 mcg dose group | Experimental | Not described Receives: Drug: Peginterferon α-2b injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum concentration (Cmax)
Measured: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Time to maximum concentration (Tmax)
Measured: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Area under the plasma concentration-time curve
Measured: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Apparent volume of distribution after oral administration (Vz/f)
Measured: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Apparent plasma clearance (CL/F)
Measured: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Plasma elimination half-life (t1/2)
Measured: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Relationship between exposure and the effect (desired-effectiveness or undesirable-toxicity) in a pharmacokinetic model and pharmacodynamic model.
Measured: up to 96 weeks.
Secondary outcomes (17)
Rate of complete hematological remission.
Measured: Week 24, 36, 48, 60, 72, 84, 96.
Platelet counts change from baseline.
Measured: Week 12, 24, 36, 48, 60, 72, 84, 96.
White blood cell counts change from baseline.
Measured: Week 12, 24, 36, 48, 60, 72, 84, 96.
Complete remission rate.
Measured: Week 24, 36, 48, 60, 72, 84, 96.
Time to complete remission from baseline.
Measured: Week 48, 96.
Duration of complete remission.
Measured: Week 48, 96.
Remission rate of bone marrow.
Measured: Week 48, 96.
Incidence of disease progression.
Measured: Week 48, 96.
Change of JAK2V617F mutations load from baseline.
JAK2V617F mutations were quantitatively detected using next-generation sequencing
Measured: Week 48, 96.
Change of CALR mutations load from baseline.
CALR mutations were quantitatively detected using next-generation sequencing
Measured: Week 48, 96.
Change of MPL mutations load from baseline.
MPL mutations were quantitatively detected using next-generation sequencing
Measured: Week 48, 96.
Change of MPN-SAF TSS scores from baseline
Measured: Week 12, 24, 36, 48, 60, 72, 84,96.
Change of spleen size from baseline
The maximum length of the spleen was measured by ultrasound.
Measured: Week 12, 24, 36, 48, 60, 72, 84, 96.
Rate of complete hematological remission maintenance in patients received dose-reduction extension therapy
Measured: Week 96.
Duration of complete hematological remission in patients received dose-reduction extension therapy
Measured: Week 96.
Change of 3-level Version of EuroQol Five Dimensions(EQ-5D-3L) scores from baseline.
Measured: Week 12, 24, 36, 48, 60, 72, 84, 96.
Incidence of thrombotic and bleeding events.
Measured: through study completion, an average of 2 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Aug 2024
- Study start29 Aug 2024
- Primary completion26 Oct 2025
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Essential ThrombocythemiaStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 27 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xiamen Amoytop Biotech 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
- NCT06552429
- Sponsor's protocol ID
- P2b-4-3-002
- Status last verified
- Nov 2025
- First submitted
- 27 Jul 2024
- Last updated
- 17 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Multicenter, Randomized, Open-label Study to Evaluate the Pharmacokinetic, Safety and Efficacy of Peginterferon Alfa-2b Injection in Subjects With Essential Thrombocythemia Who Are Resistant to or Intolerant of Hydroxyurea.
- ClinicalTrials.gov ID
- NCT06552429
- Acronym / sponsor ID
- P2b-4-3-002
- Conditions
- Essential Thrombocythemia
- Keywords
- Essential Thrombocythemia, Peginterferon α-2b, hydroxyurea resistant, hydroxyurea intolerant
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 27 participants (estimated)
- Lead sponsor
- Xiamen Amoytop Biotech Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 29 Aug 2024 (actual)
- Primary completion
- 26 Oct 2025 (actual)
- Study completion
- Sep 2027 (estimated)
- First posted
- 14 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multicenter, randomized, open-label Phase 2 clinical study. It is aimed to enroll 27 essential thrombocytopenia (ET) patients who are resistant to or intolerant of hydroxyurea(HU). Eligible patients will be randomized to receive either Peginterferon α-2b 135 mcg or Peginterferon α-2b 180 mcg at a ratio of 1:2, and all subjects will go through a target treatment period (Weeks 1 ~ Week 48), an extension treatment period (Weeks 49 ~ Week 96) and a follow-up period (Weeks 97 ~ Week 100). Pharmacokinetics, safety, efficacy will be evaluated.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Peginterferon α-2b 135 mcg dose group | Experimental | Drug: Peginterferon α-2b injection |
| Peginterferon α-2b 180 mcg dose group | Experimental | Drug: Peginterferon α-2b injection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Male or female subjects, aged greater or equal to 18 years old at screening;
- Subjects diagnosed as high-risk ET according to the World Health Organization (WHO) 2016 criteria：1) who is older than 60 years and JAK2V617F positive at screening, 2) or who previously suffered from disease-related thrombosis or hemorrhage;
- Subjects who have previously received HU for ET, and the time interval between the last HU dose and the first dose of the study drug should not be less than 7 days;
- Interferon treatment-naïve, and for those who have previously received interferon the the time interval between the last dose of interferon and randomization should not be less than 1 month;
- Patients with confirmed hydroxyurea resistance or intolerant, as at least one of the following criteria is met:
Key exclusion criteria (14 listed)
- History of any other myeloproliferative tumors, or evidence of the presence of any other myeloproliferative tumors;
- Contraindications or hypersensitivities to interferons of any of its excipients;
- Severe medical conditions or serious comorbidities that the investigators determined could jeopardize the safety or protocol adherence, e.g. New York Heart Association [NYHA] Class III-IV, congestive heart failure, symptomatic arrhythmias，pulmonary hypertension;
- History of major organ transplantation;
- Documented autoimmune disease or history of autoimmune disease at screening, e.g. medication un-controlled thyroid dysfunction, autoimmune hepatitis, idiopathic thrombocytopenic purpura, scleroderma, psoriasis, or any autoimmune arthritis;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Peginterferon α-2b injectionPeginterferon α-2b injection, 135 mcg, s.c., once a week, during the targeted treatment period (the first 48 week), peginterferon α-2b dose is depended on the patient's response and tolerability during the extension treatment (week 49 to week 96). | Drug | Peginterferon α-2b 135 mcg dose group |
| Peginterferon α-2b injectionPeginterferon α-2b injection, 180 mcg, s.c., once a week, during the targeted treatment period (the first 48 week), peginterferon α-2b dose is depended on the patient's response and tolerability during the extension treatment (week 49 to week 96). | Drug | Peginterferon α-2b 180 mcg dose group |
Primary outcome
Maximum concentration (Cmax)
Time frame: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Time to maximum concentration (Tmax)
Time frame: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Area under the plasma concentration-time curve
Time frame: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Apparent volume of distribution after oral administration (Vz/f)
Time frame: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Apparent plasma clearance (CL/F)
Time frame: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Plasma elimination half-life (t1/2)
Time frame: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.
Relationship between exposure and the effect (desired-effectiveness or undesirable-toxicity) in a pharmacokinetic model and pharmacodynamic model.
Time frame: up to 96 weeks.
17 secondary outcomes
Rate of complete hematological remission.
Time frame: Week 24, 36, 48, 60, 72, 84, 96.
Platelet counts change from baseline.
Time frame: Week 12, 24, 36, 48, 60, 72, 84, 96.
White blood cell counts change from baseline.
Time frame: Week 12, 24, 36, 48, 60, 72, 84, 96.
Complete remission rate.
Time frame: Week 24, 36, 48, 60, 72, 84, 96.
Time to complete remission from baseline.
Time frame: Week 48, 96.
Duration of complete remission.
Time frame: Week 48, 96.
Remission rate of bone marrow.
Time frame: Week 48, 96.
Incidence of disease progression.
Time frame: Week 48, 96.
Change of JAK2V617F mutations load from baseline.
Time frame: Week 48, 96.
Change of CALR mutations load from baseline.
Time frame: Week 48, 96.
Change of MPL mutations load from baseline.
Time frame: Week 48, 96.
Change of MPN-SAF TSS scores from baseline
Time frame: Week 12, 24, 36, 48, 60, 72, 84,96.
Change of spleen size from baseline
Time frame: Week 12, 24, 36, 48, 60, 72, 84, 96.
Rate of complete hematological remission maintenance in patients received dose-reduction extension therapy
Time frame: Week 96.
Duration of complete hematological remission in patients received dose-reduction extension therapy
Time frame: Week 96.
Change of 3-level Version of EuroQol Five Dimensions(EQ-5D-3L) scores from baseline.
Time frame: Week 12, 24, 36, 48, 60, 72, 84, 96.
Incidence of thrombotic and bleeding events.
Time frame: through study completion, an average of 2 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Nov 2025.
- Individual participant dataSharing planned: No
Related trials for Essential Thrombocythemia
RecruitingPhase 3
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)
Primary Myelofibrosis (PMF), Post-polycythemia Vera Myelofibrosis (PPV-MF)
Active, not recruitingObservational study
Infection Prophylaxis and Management in Treating Cytomegalovirus (CMV) Infection in Patients With Hematologic Malignancies Previously Treated With Donor Stem Cell Transplant
Hematopoietic/Lymphoid Cancer, Accelerated Phase Chronic Myelogenous Leukemia
RecruitingPhase 1/2
Study of DISC-0974 (RALLY-MF) in Participants With Myelofibrosis or Myelodysplastic Syndrome and Anemia
Myelofibrosis; Anemia, Anemia
Active, not recruitingPhase 2
Hematology, IMG-7289, LSD1 (Lysine-Specific Demethylase 1) Inhibitor, Essential Thrombocythemia (ET), Ph 2
Thrombocythemia, Essential
RecruitingPhase 1
Therapeutic RSK1 Targeting in Myeloid Malignancies
Myeloid Malignancy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Lei Zhang principal investigator
Chinese Academy of Medical Sciences
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Peginterferon α-2b injection in people with Essential Thrombocythemia, run by Xiamen Amoytop Biotech Co., Ltd. at 8 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lei Zhang (13502118379, zhanglei1@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum concentration (Cmax) (time frame: week1,4, 8, 12, 24, 36, 48, 60, 72, 84, 96.). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Nov 2025.