Polycythemia Vera (PV)Phase 3NCT06002490
A Study to Evaluate P1101 in Japanese PV Patients
A Phase 3 study testing P1101, Low-dose aspirin and Phlebotomy in people with Polycythemia Vera (PV), run by PharmaEssentia Japan K.K. at 6 sites in Japan.
Trial summary
This is a Phase 3 single arm study to investigate efficacy and safety of P1101's rapid titration for adult Japanese patients with PV. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Polycythemia Vera (PV)
- What is being tested?
- P1101, Low-dose aspirin and Phlebotomy
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 6 sitesJapan
- Study dates
- 17 Oct 2023to 26 Jul 2024
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 patients ≥18 years old at the time of informed consent to participate in the study
- Patients diagnosed with PV according to the WHO 2008 or 2016 criteria
- Patients with PV who have the inadequate response to an existing therapy or whom the existing therapy is inappropriate to apply (see Appendix 6)
- Patients with adequate hepatic function at screening defined as total bilirubin ≤1.5 x upper limit of normal (ULN), international normalized ratio (INR) of prothrombin time (PT) ≤1.5 x ULN, albumin >3.5 g/dL, alanine aminotransferase (ALT) ≤2.0 x ULN, and aspartate aminotransferase (AST) ≤2.0 x ULN.
- Patients with hemoglobin (HGB) ≥10 g/dL in female and ≥11 g/dL in male at screening
- Patients with a neutrophil count ≥1.5 x 10^9/L at screening
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- Patients with serum creatinine ≤1.5 x ULN at screening
- Males, and females of childbearing potential, as well as all women <2 years after the onset of menopause, must agree to use an acceptable form of birth control until 14 days following the last dose of the study drug. Also, women must agree not to breastfeed during the study (see Appendix 4 for details)
- Written informed consent has been obtained from the patient or the patient's legally authorized representative, and the patient is capable of complying with the study requirements.
Who cannot join
- Patients with contraindications to or hypersensitivity to IFN-α
- Patients with previous use of IFN-α, or previous treatment with ruxolitinib
- Patients with comorbidity with severe or serious condition that, in the opinion of the Investigator, may impact the patient's participation in the study
- Patients with a history of major organ transplantation
- Pregnant or lactating females
- Patients with any other medical condition that, in the opinion of the Investigator, might impair the outcome of the study or compliance with the requirements of the protocol. These diseases include, but are not limited to, the following:
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- 6.1 Patients with a history or presence of autoimmune thyroid dysfunction (clinical symptoms of hyper- or hypo-thyroidism), except late stages cases on the oral thyroid substitution therapy, where potential exacerbation under interferon therapy will not constitute any further harm to the patient 6.2 Patients with any documented history of autoimmune disease (for example, hepatitis, idiopathic thrombocytopenic purpura [ITP], scleroderma, psoriasis, or autoimmune arthritis) 6.3 Patients with clinically relevant pulmonary infiltrates, infectious pneumonia or noninfectious pneumonia at screening, or a history of interstitial pulmonary disease.
- 6.4 Patients with active infection with systemic symptoms (for example, bacterial, fungal, hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV) infection at screening).
- 6.5 Patients with any evidence of severe retinal disease (e.g., cytomegalovirus [CMV] retinitis, macular degeneration) or clinically relevant ophthalmologic disease (due to diabetes mellitus or hypertension) based on the ophthalmological assessment by specialists.
- 6.6 Patients with uncontrolled depression in the opinion of the Investigator 6.7 Patients with a history of suicide attempts or at risk for suicide at screening 6.8 Patients with uncontrolled diabetes mellitus (baseline HbA1c >7%) 6.9 Patients with active thromboembolism or active abdominal bleeding due to PV 6.10 Patients with a history of malignancy within the last 5 years [excluding adequately treated non-melanoma skin cancers, post-prostatectomy prostate-specific antigen (PSA) failure, healed cervical intraepithelial neoplasia and ductal carcinoma in situ (DCIS), stage 1 endometrial carcinoma, or solid tumors (without myelopathy), including other lymphomas that have not had evidence of disease for at least 2 years after treatment].
- 6.11 Patients with a history of alcohol or drug abuse within the recent one year 6.12 Patients with a history or evidence of post-polycythemia vera myelofibrosis (post-PV MF), essential thrombocythemia, or MPN other than PV
- Patients who received any other study treatment or concomitant medication with other study drug within 4 weeks prior to the first dose of study drug in this study or not recovered from any prior exposure to any other study drug.
- Patients with symptomatic splenomegaly
- Patients with circulating blasts in the peripheral blood within the last 12 weeks
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
Japan6 sites
Mie University Hospital
Mie, 514-8507
Osaka University Hospital
Osaka, 565-0871
Kansai Medical University Hospital
Osaka, 573-1191
Juntendo University Hospital
Tokyo, 113-8431
Tokyo Medical University Hospital
Tokyo, 160-0023
University of Yamanashi Hospital
Yamanashi, 409-3898
About this study
Official title: A Phase III b, Single-arm, Multicenter, Optimal Dose Finding Study to Assess the Efficacy and Safety of P1101 in Japanese Patients With Polycythemia Vera (PV).
This is a Phase 3 single arm study to investigate efficacy and safety of P1101's rapid titration for adult Japanese patients with PV.
Read the detailed description
Eligible patients will be treated with P1101, starting at 250 μg. A starting dose of P1101 is 250 mcg, an intermediate dose is 350 mcg, and a target dose is 500 mcg. As such, subjects will not be exposed to below optimal doses within the first 4 weeks. The maximum recommended single dose is 500 μg injected every two weeks.
A primary efficacy endpoint is the rate of phlebotomy-free complete hematologic response (CHR) at Week 24, where CHR is defined as the proportion of patients who have achieved a CHR and have not required phlebotomy in the previous 12 weeks. A responder for the primary endpoint is defined as meeting all of the following criteria at Week 24: Hct (<45%), WBC (≤10 x 10^9/L), and PLT (≤400 x 10^9/L).
- Conditions
- Polycythemia Vera (PV)
- Being tested
- P1101, Low-dose aspirin, Phlebotomy
- Keywords
- Myeloproliferative Neoplasms
- Medical subject headings
- Polycythemia Vera, Myeloproliferative Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
P1101Drug
Also called Ropeginterferon alfa-2b
The subjects will be treated with P1101(ropeginterferon alfa-2b), starting at a dose of 250 micrograms. The dose of P1101 will be increased to 350 micrograms 2 weeks later and to 500 micrograms another 2 weeks later, and then P1101 will be administered at a fixed dose of 500 micrograms throughout the treatment period. Although the dose may be reduced to the prior dose for reasons related to the safety or tolerability, the increased dose should be preferably maintained throughout the treatment period.
The dose of P1101 will be increased or decreased appropriately depending on the pathological condition in the range up to 500 micrograms.
Low-dose aspirinDrug
Also called Low-doaw Acetylsalicylic acid
Low-dose aspirin (acetylsalicylic acid) (75-150 mg/day) will be given as background therapy during the 12 months of study treatment, unless contraindicated.
PhlebotomyProcedure or surgery
Phlebotomy is performed aiming at a hematocrit < 45%. When the hematocrit value is 45% or higher, phlebotomy is performed. The volume of phlebotomy per procedure should be 200 to 400 mL while monitoring the circulatory dynamics such as blood pressure and pulse. In the elderly and patients with cardiovascular disorders, a small volume (100-200 mL) should be considered to avoid rapid changes in hemodynamics.
| Study group | Role | What they receive |
|---|---|---|
| P1101 | Experimental | Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks. Receives: Drug: P1101; Drug: Low-dose aspirin; Procedure: Phlebotomy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of Subjects Who Achieved Durable Phlebotomy-free Complete Hematological Response (CHR) at Week 24
Measured: 24 weeks
Secondary outcomes (10)
Rate of achieving "phlebotomy-free complete hematologic response (CHR)" (the same criteria as for the primary efficacy endpoint) at both Week 12 and Week 24.
Measured: Week12, Week24
Time to achieve CHR
Measured: Up to Week24
Time to reach response maintenance dose (three consecutive doses of the same dose)
Measured: Up to Week24
Numbers of phlebotomy required and changes in numbers of phlebotomy required from baseline
Measured: Baseline, up to Week24
Time to first response in peripheral blood count (Hct, WBC, and PLT)
Measured: Up to Week24
Duration of response in peripheral blood count (Hct, WBC, and PLT)
Measured: Up to Week24
Improvement of symptoms assessed by MPN-SAF TSS at each visit
Rated on a scale of 0-10 on an 11-point scale, with 0 being no symptoms, 1 being the best, and 10 being the worst.
Measured: Up to Week24
Proportion of subjects without thrombotic or hemorrhagic events at Weeks 12 and 24
Measured: Week12, week24
Change from baseline to Week 24 in JAK2 V617F allele burden level
Measured: Baseline, week24
Collect and measure Pharmacokinetics (PK) at trough
Measured: Day1, Week2, Week4, Week6, Week8, Week10, Week12, Week14, Week16, Week18, Week20, Week22, Week24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2023
- Study start17 Oct 2023
- Primary completion26 Jul 2024
- Study completion26 Jul 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Polycythemia Vera (PV)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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of P1101 (ropeginterferon alfa-2b) once every 2 weeks
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- PharmaEssentia Japan K.K.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06002490
- Sponsor's protocol ID
- A23-301
- Status last verified
- Nov 2024
- First submitted
- 31 Jul 2023
- Last updated
- 8 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase III b, Single-arm, Multicenter, Optimal Dose Finding Study to Assess the Efficacy and Safety of P1101 in Japanese Patients With Polycythemia Vera (PV).
- ClinicalTrials.gov ID
- NCT06002490
- Acronym / sponsor ID
- A23-301
- Conditions
- Polycythemia Vera (PV)
- Keywords
- Myeloproliferative Neoplasms
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- PharmaEssentia Japan K.K.Industry (pharma, biotech, devices)
- Study start
- 17 Oct 2023 (actual)
- Primary completion
- 26 Jul 2024 (actual)
- Study completion
- 26 Jul 2024 (actual)
- First posted
- 21 Aug 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.
This is a Phase 3 single arm study to investigate efficacy and safety of P1101's rapid titration for adult Japanese patients with PV.
Detailed description
Eligible patients will be treated with P1101, starting at 250 μg. A starting dose of P1101 is 250 mcg, an intermediate dose is 350 mcg, and a target dose is 500 mcg. As such, subjects will not be exposed to below optimal doses within the first 4 weeks. The maximum recommended single dose is 500 μg injected every two weeks.
A primary efficacy endpoint is the rate of phlebotomy-free complete hematologic response (CHR) at Week 24, where CHR is defined as the proportion of patients who have achieved a CHR and have not required phlebotomy in the previous 12 weeks. A responder for the primary endpoint is defined as meeting all of the following criteria at Week 24: Hct (<45%), WBC (≤10 x 10^9/L), and PLT (≤400 x 10^9/L).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Conventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of P1101 (ropeginterferon alfa-2b) once every 2 weeks
| Arm | Type | Receives |
|---|---|---|
| P1101 | Experimental | Drug: P1101, Drug: Low-dose aspirin, Procedure: PhlebotomyConventional treatment based on phlebotomies, low-dose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated proline-interferon alpha-2b (P1101, ropeginterferon alfa-2b) once every 2 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Male or female patients ≥18 years old at the time of informed consent to participate in the study
- Patients diagnosed with PV according to the WHO 2008 or 2016 criteria
- Patients with PV who have the inadequate response to an existing therapy or whom the existing therapy is inappropriate to apply (see Appendix 6)
- Patients with adequate hepatic function at screening defined as total bilirubin ≤1.5 x upper limit of normal (ULN), international normalized ratio (INR) of prothrombin time (PT) ≤1.5 x ULN, albumin >3.5 g/dL, alanine aminotransferase (ALT) ≤2.0 x ULN, and aspartate aminotransferase (AST) ≤2.0 x ULN.
- Patients with hemoglobin (HGB) ≥10 g/dL in female and ≥11 g/dL in male at screening
Key exclusion criteria (14 listed)
- Patients with contraindications to or hypersensitivity to IFN-α
- Patients with previous use of IFN-α, or previous treatment with ruxolitinib
- Patients with comorbidity with severe or serious condition that, in the opinion of the Investigator, may impact the patient's participation in the study
- Patients with a history of major organ transplantation
- Pregnant or lactating females
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 |
|---|---|---|
| P1101Also: Ropeginterferon alfa-2bThe subjects will be treated with P1101(ropeginterferon alfa-2b), starting at a dose of 250 micrograms. The dose of P1101 will be increased to 350 micrograms 2 weeks later and to 500 micrograms another 2 weeks later, and then P1101 will be administered at a fixed dose of 500 micrograms throughout the treatment period. Although the dose may be reduced to the prior dose for reasons related to the safety or tolerability, the increased dose should be preferably maintained throughout the treatment period. The dose of P1101 will be increased or decreased appropriately depending on the pathological condition in the range up to 500 micrograms. | Drug | P1101 |
| Low-dose aspirinAlso: Low-doaw Acetylsalicylic acidLow-dose aspirin (acetylsalicylic acid) (75-150 mg/day) will be given as background therapy during the 12 months of study treatment, unless contraindicated. | Drug | P1101 |
| PhlebotomyPhlebotomy is performed aiming at a hematocrit < 45%. When the hematocrit value is 45% or higher, phlebotomy is performed. The volume of phlebotomy per procedure should be 200 to 400 mL while monitoring the circulatory dynamics such as blood pressure and pulse. In the elderly and patients with cardiovascular disorders, a small volume (100-200 mL) should be considered to avoid rapid changes in hemodynamics. | Procedure or surgery | P1101 |
Primary outcome
Proportion of Subjects Who Achieved Durable Phlebotomy-free Complete Hematological Response (CHR) at Week 24
Time frame: 24 weeks
10 secondary outcomes
Rate of achieving "phlebotomy-free complete hematologic response (CHR)" (the same criteria as for the primary efficacy endpoint) at both Week 12 and Week 24.
Time frame: Week12, Week24
Time to achieve CHR
Time frame: Up to Week24
Time to reach response maintenance dose (three consecutive doses of the same dose)
Time frame: Up to Week24
Numbers of phlebotomy required and changes in numbers of phlebotomy required from baseline
Time frame: Baseline, up to Week24
Time to first response in peripheral blood count (Hct, WBC, and PLT)
Time frame: Up to Week24
Duration of response in peripheral blood count (Hct, WBC, and PLT)
Time frame: Up to Week24
Improvement of symptoms assessed by MPN-SAF TSS at each visit
Time frame: Up to Week24
Proportion of subjects without thrombotic or hemorrhagic events at Weeks 12 and 24
Time frame: Week12, week24
Change from baseline to Week 24 in JAK2 V617F allele burden level
Time frame: Baseline, week24
Collect and measure Pharmacokinetics (PK) at trough
Time frame: Day1, Week2, Week4, Week6, Week8, Week10, Week12, Week14, Week16, Week18, Week20, Week22, Week24
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Nov 2024.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing P1101, Low-dose aspirin and Phlebotomy in people with Polycythemia Vera (PV), run by PharmaEssentia Japan K.K. at 6 sites in Japan.
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 9 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: finished, as of the sponsor's last update on 8 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Japan. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of Subjects Who Achieved Durable Phlebotomy-free Complete Hematological Response (CHR) at Week 24 (time frame: 24 weeks). 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 8 Nov 2024.