Polycythemia VeraPhase 2PVNCT05870475
Pegylated Interferon α-2b in Combination With Ruxolitinib for Treating Hydroxyurea-resistant/Intolerant PV
A Phase 2 study testing Ruxolitinib and Pegylated interferon α-2b in people with Polycythemia Vera, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
Study purpose: To compare the efficacy and safety of pegylated interferon α-2b in combination with ruxolitinib versus pegylated interferon α-2b alone for treating hydroxyurea-resistant or hydroxyurea-intolerant polycythemia vera. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Polycythemia Vera
- What is being tested?
- Ruxolitinib and Pegylated interferon α-2b
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 94 participants
- Where
- 1 siteChina
- Study dates
- 30 Jun 2023to 31 May 2028
- 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
- ≥18 years old.
- Male or Female.
- Meets the diagnostic criteria for Polycythemia Vera according to WHO-2022.
- Resistant or intolerant to hydroxyurea (based on the 2013 European LeukemiaNet criteria).
- Have not previously received interferon preparations or ruxolitinib treatment, or the washout period between the last use of interferon preparations or ruxolitinib and the first use of the study drug should not be less than 4 weeks.
- Patients with indications for cytoreductive therapy.
- During screening, female hemoglobin (HGB) ≥10g/dL, male hemoglobin (HGB) ≥11g/dL; neutrophil count ≥1.5×109/L; platelet count ≥100×109/L.
- Voluntary written informed consent.
Who cannot join
- Symptomatic splenomegaly;
- Contraindications to interferon or ruxolitinib therapy;
- Severe or significant comorbidities that may affect the participant's ability to participate in the study, as determined by the investigator;
- History of major organ transplantation;
- Pregnant or breastfeeding women;
- History or current diagnosis of autoimmune thyroid disease (patients with controlled hypothyroidism on oral thyroid hormone replacement therapy may be included);
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- Documented evidence of any other autoimmune disease (such as active hepatitis, systemic lupus erythematosus, antiphospholipid antibody syndrome, or autoimmune arthritis);
- Clinically significant bacterial, fungal, mycobacterial, parasitic, or viral infection such as active hepatitis or HIV infection (patients with acute bacterial infections requiring antibiotic treatment should be deferred from screening/enrollment until completion of antibiotic treatment);
- Evidence of severe retinopathy or clinically significant ophthalmologic disease (due to diabetes or hypertension);
- Current clinically significant depression or history of depression, or any suicidal attempt or tendency during screening;
- Active bleeding or thrombotic complications;
- History of any malignant tumor within the past 5 years (except for stage 0 chronic lymphocytic leukemia [CLL], cured basal cell carcinoma, squamous cell carcinoma, and superficial melanoma);
- History of alcohol or substance abuse within the past year;
- Presence of blasts in the peripheral blood within the past 3 months;
- Use of any investigational drug or participation in any other clinical trial within 4 weeks prior to the first dose of the study drug, or failure to recover from any effects of previously administered study drugs;
- The investigator deems the presence of any concurrent condition that may jeopardize the safety of the participant or the compliance to the protocol.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Institute of Hematology & Blood Diseases Hospital
Tianjin
Lei Zhang, MD, 8602223909240, zhanglei1@ihcams.ac.cn
About this study
Official title: A Randomized Controlled Study Evaluating the Efficacy and Safety of Pegylated Interferon α-2b in Combination With Ruxolitinib vs. Pegylated Interferon α-2b Monotherapy for Treating Hydroxyurea-resistant/Intolerant Polycythemia Vera
Study purpose: To compare the efficacy and safety of pegylated interferon α-2b in combination with ruxolitinib versus pegylated interferon α-2b alone for treating hydroxyurea-resistant or hydroxyurea-intolerant polycythemia vera.
Read the detailed description
Study purpose: To compare the efficacy and safety of pegylated interferon α-2b in combination with ruxolitinib versus pegylated interferon α-2b alone for treating hydroxyurea-resistant or hydroxyurea-intolerant polycythemia vera.
The subjects will be randomly divided into two groups:
pegylated interferon alpha-2b combined with ruxolitinib group: pegylated interferon alpha-2b at a starting dose of 180ug will be administered subcutaneously once a week; ruxolitinib at a starting dose of 10mg will be administered orally twice daily.
pegylated interferon alpha-2b group: pegylated interferon alpha-2b at a starting dose of 180ug will be administered subcutaneously once a week.
If complete hematologic remission is not achieved after 12 weeks of treatment with pegylated interferon alpha-2b alone, the subject may be switched to the pegylated interferon alpha-2b combined with ruxolitinib group. If ruxolitinib is not tolerated, the subject may be switched to the pegylated interferon alpha-2b group alone.
- Conditions
- Polycythemia Vera
- Being tested
- Ruxolitinib, Pegylated interferon α-2b
- Medical subject headings
- Polycythemia Vera
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibDrug
Also called RUX
Ruxolitinib at a starting dose of 10mg, orally administered twice daily. If ruxolitinib is not tolerated, cross-over to the pegylated interferon α-2b alone group is allowed.
Pegylated interferon α-2bDrug
Also called PEG IFNα-2b
Starting dose of 180ug, subcutaneous injection once a week. If complete hematological remission is not achieved after 12 weeks of treatment with pegylated interferon α-2b alone, cross-over to the pegylated interferon α-2b plus ruxolitinib group is allowed.
| Study group | Role | What they receive |
|---|---|---|
| pegylated interferon α-2b in combination with ruxolitinib group | Experimental | Pegylated interferon α-2b in combination with ruxolitinib group: Pegylated interferon α-2b at a starting dose of 180ug, subcutaneous injection once a week; ruxolitinib at a starting dose of 10mg, orally administered twice daily. Receives: Drug: Ruxolitinib; Drug: Pegylated interferon α-2b |
| Pegylated interferon α-2b group | Active comparator | Pegylated interferon α-2b group: Starting dose of 180ug, subcutaneous injection once a week.
If complete hematological remission is not achieved after 12 weeks of treatment with pegylated interferon α-2b alone, cross-over to the pegylated interferon α-2b plus ruxolitinib group is allowed; if ruxolitinib is not tolerated, cross-over to the pegylated interferon α-2b alone group is allowed. Receives: Drug: Pegylated interferon α-2b |
What is being measured
The primary outcome is the main question the study is designed to answer.
The cumulative complete hematologic response (CHR) rate
The proportion of patients who can achieve CHR ( defined as hematocrit lower than 45% without phlebotomies; platelet count < 400×109/L, WBC count < 10×109/L for at least 12 weeks) among all patients.
Measured: From the start of study treatment (Week 0) up to the end of Week 24.
Secondary outcomes (15)
Cumulative CHR rates at Week 36.
Cumulative CHR rates at Week 36 will be compared between the two groups.
Measured: From the start of study treatment (Week 0) up to the end of Week 36.
Cumulative CHR rates at Week 52.
Cumulative CHR rates at Week 52 will be compared between the two groups.
Measured: From the start of study treatment (Week 0) up to the end of Week 52.
Time to CHR
The time of reaching CHR will be compared between the two groups.
Measured: From the start of study treatment (Week 0) up to the end of Week 52.
The CHR rates after crossover
The CHR rates within 52 weeks after crossover
Measured: From the start of study treatment (Week 0) up to the end of Week 52.
The rate of reduction in JAK2V617F, CALR, or MPL gene mutation burden.
The rate of reduction in JAK2V617F, CALR, or MPL gene mutation burden will be compared between the two groups.
Measured: From the start of study treatment (Week 0) up to the end of Week 52.
Impact of therapy on non-driver mutations
To compare the proportion of subjects that display change on key non-driver biomarkers of the disease-DNMT3A, ASXL1, TET2 or other mutations.
Measured: From the start of study treatment (Week 0) up to the end of Week 52.
Change of splenomegaly
All subjects with palpable splenomegaly at baseline will undergo ultrasound examination.
For subjects with palpable splenomegaly at baseline:
Improvement - no palpable splenomegaly during clinical treatment visits; No progress - ultrasound examination during clinical treatment visits shows an increase in spleen size of ≤ 25%; Progress - Ultrasound examination during clinical treatment visits shows an increase in spleen size of>25%.
Measured: From the start of study treatment (Week 0) up to the end of Week 52.]
Change of bone marrow pathology
Bone marrow histological remission defined as the presence of age-adjusted normocellularity and disappearance of trilinear hyperplasia, and absence of grade 1 reticulin fibrosis; no remission defined as the persistence of trilinear hyperplasia; progression defined as disease transformation into post-PV myelofibrosis, myelodysplastic syndrome or acute leukemia.
Measured: From the start of study treatment (Week 0) up to the end of Week 52
The incidence of major thrombotic events
To compare the incidence of major thrombotic events between the two groups.
Measured: From the start of study treatment (Week 0) up to the end of Week 52.
The incidence of major bleeding events
To compare the incidence of major bleeding events between the two groups.
Measured: From the start of study treatment (Week 0) up to the end of Week 52.
The incidence of progressing to acute leukemia
The incidence of progressing to acute leukemia will be compared between the two groups.
Measured: From the start of study treatment (Week 0) up to the end of Week 52.
Change in Myeloproliferative Neoplasm Symptom Assessment Form total symptom score
To compare the proportion of subjects that display change in Myeloproliferative Neoplasm Symptom Assessment Form total symptom score (0-100 scores, higher scores mean a worse outcome) between different treatment groups.
Measured: From the start of study treatment (Week 0) up to the end of Week 52.
Change of quality of life using QLQ-C30 V3.0 questionnaire.
The QLQ-C30 V3.0 questionnaire consists of 30 questions, with questions 1-28 scoring 1-4 and questions 29 and 30 scoring 1-7. Therefore, this questionnaire has a minimum score of 30 and a maximum score of 126, and the score is directly proportional to the quality of life.
Measured: From the start of study treatment (Week 0) up to the end of Week 52.
Change of microcirculation disturbance
The rate of patients with improvement in microcirculation disturbance (such as pruritus, headache, dizziness, chest tightness, erythematous limb pain and limb paresthesia) will be compared between the two groups
Measured: From the start of study treatment (Week 0) up to the end of Week 52.
Specific pre-defined toxicity
To compare incidence of specific pre-defined toxicity including fatigue, flu-like symptoms, dizziness, injection site necrosis, dyspnea, pain, depression, blurred Vision, insomnia, anorexia, weight Loss, weakness, pruritis, sweating, fever, decreased Libido, hot Flashes, flushing, infection,
Measured: From the start of study treatment (Week 0) up to the end of Week 52.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 May 2023
- Study start30 Jun 2023
- Primary completion31 Mar 2028 (estimated)
- Study completion31 May 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Polycythemia VeraStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- 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
- 94 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lei Zhang, MD, Principal investigator, Institute of Hematology & Blood Diseases Hospital, ChinaRongfeng Fu, Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05870475
- Sponsor's protocol ID
- IIT2023018
- Acronym
- PV
- Status last verified
- May 2024
- First submitted
- 10 Apr 2023
- Last updated
- 3 Jul 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Controlled Study Evaluating the Efficacy and Safety of Pegylated Interferon α-2b in Combination With Ruxolitinib vs. Pegylated Interferon α-2b Monotherapy for Treating Hydroxyurea-resistant/Intolerant Polycythemia Vera
- ClinicalTrials.gov ID
- NCT05870475
- Acronym / sponsor ID
- PV / IIT2023018
- Conditions
- Polycythemia Vera
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 94 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 30 Jun 2023 (actual)
- Primary completion
- 31 Mar 2028 (estimated)
- Study completion
- 31 May 2028 (estimated)
- First posted
- 23 May 2023 (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.
Study purpose: To compare the efficacy and safety of pegylated interferon α-2b in combination with ruxolitinib versus pegylated interferon α-2b alone for treating hydroxyurea-resistant or hydroxyurea-intolerant polycythemia vera.
Detailed description
Study purpose: To compare the efficacy and safety of pegylated interferon α-2b in combination with ruxolitinib versus pegylated interferon α-2b alone for treating hydroxyurea-resistant or hydroxyurea-intolerant polycythemia vera.
The subjects will be randomly divided into two groups:
pegylated interferon alpha-2b combined with ruxolitinib group: pegylated interferon alpha-2b at a starting dose of 180ug will be administered subcutaneously once a week; ruxolitinib at a starting dose of 10mg will be administered orally twice daily.
pegylated interferon alpha-2b group: pegylated interferon alpha-2b at a starting dose of 180ug will be administered subcutaneously once a week.
If complete hematologic remission is not achieved after 12 weeks of treatment with pegylated interferon alpha-2b alone, the subject may be switched to the pegylated interferon alpha-2b combined with ruxolitinib group. If ruxolitinib is not tolerated, the subject may be switched to the pegylated interferon alpha-2b group alone.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| pegylated interferon α-2b in combination with ruxolitinib group | Experimental | Drug: Ruxolitinib, Drug: Pegylated interferon α-2bPegylated interferon α-2b in combination with ruxolitinib group: Pegylated interferon α-2b at a starting dose of 180ug, subcutaneous injection once a week; ruxolitinib at a starting dose of 10mg, orally administered twice daily. |
| Pegylated interferon α-2b group | Active comparator | Drug: Pegylated interferon α-2bPegylated interferon α-2b group: Starting dose of 180ug, subcutaneous injection once a week. If complete hematological remission is not achieved after 12 weeks of treatment with pegylated interferon α-2b alone, cross-over to the pegylated interferon α-2b plus ruxolitinib group is allowed; if ruxolitinib is not tolerated, cross-over to the pegylated interferon α-2b alone group is allowed. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- ≥18 years old.
- Male or Female.
- Meets the diagnostic criteria for Polycythemia Vera according to WHO-2022.
- Resistant or intolerant to hydroxyurea (based on the 2013 European LeukemiaNet criteria).
- Have not previously received interferon preparations or ruxolitinib treatment, or the washout period between the last use of interferon preparations or ruxolitinib and the first use of the study drug should not be less than 4 weeks.
Key exclusion criteria (16 listed)
- Symptomatic splenomegaly;
- Contraindications to interferon or ruxolitinib therapy;
- Severe or significant comorbidities that may affect the participant's ability to participate in the study, as determined by the investigator;
- History of major organ transplantation;
- Pregnant or breastfeeding women;
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 |
|---|---|---|
| RuxolitinibAlso: RUXRuxolitinib at a starting dose of 10mg, orally administered twice daily. If ruxolitinib is not tolerated, cross-over to the pegylated interferon α-2b alone group is allowed. | Drug | pegylated interferon α-2b in combination with ruxolitinib group |
| Pegylated interferon α-2bAlso: PEG IFNα-2bStarting dose of 180ug, subcutaneous injection once a week. If complete hematological remission is not achieved after 12 weeks of treatment with pegylated interferon α-2b alone, cross-over to the pegylated interferon α-2b plus ruxolitinib group is allowed. | Drug | Pegylated interferon α-2b group, pegylated interferon α-2b in combination with ruxolitinib group |
Primary outcome
The cumulative complete hematologic response (CHR) rate
Time frame: From the start of study treatment (Week 0) up to the end of Week 24.
The proportion of patients who can achieve CHR ( defined as hematocrit lower than 45% without phlebotomies; platelet count < 400×109/L, WBC count < 10×109/L for at least 12 weeks) among all patients.
15 secondary outcomes
Cumulative CHR rates at Week 36.
Time frame: From the start of study treatment (Week 0) up to the end of Week 36.
Cumulative CHR rates at Week 52.
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Time to CHR
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
The CHR rates after crossover
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
The rate of reduction in JAK2V617F, CALR, or MPL gene mutation burden.
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Impact of therapy on non-driver mutations
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Change of splenomegaly
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.]
Change of bone marrow pathology
Time frame: From the start of study treatment (Week 0) up to the end of Week 52
The incidence of major thrombotic events
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
The incidence of major bleeding events
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
The incidence of progressing to acute leukemia
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Change in Myeloproliferative Neoplasm Symptom Assessment Form total symptom score
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Change of quality of life using QLQ-C30 V3.0 questionnaire.
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Change of microcirculation disturbance
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
Specific pre-defined toxicity
Time frame: From the start of study treatment (Week 0) up to the end of Week 52.
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 3 Jul 2024.
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RecruitingPhase 2/3
A Clinical Trial of Bomedemstat in Participants With Polycythemia Vera (MK-3543-025).
Polycythemia Vera
RecruitingPhase 2
Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis
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RecruitingPhase 2
A Study of Ropeg-Interferon in People With Polycythemia Vera
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Active, not recruitingPhase 2
A Phase 2, Open-Label Study of DISC-3405 in Participants With Polycythemia Vera (PV)
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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, MD principal investigator
Institute of Hematology & Blood Diseases Hospital, China
Rongfeng Fu principal investigator
Institute of Hematology & Blood Diseases Hospital, China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ruxolitinib and Pegylated interferon α-2b in people with Polycythemia Vera, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site 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 8 inclusion and 16 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 3 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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, MD (8602223909240, zhanglei1@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The cumulative complete hematologic response (CHR) rate (time frame: From the start of study treatment (Week 0) up to the end of Week 24.). 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 3 Jul 2024.