Polycythemia Vera (PV)Phase 3NCT04655092
Extension Study of P1101 After Completion of Phase 2 Study in PV Patients or Phase 3 Study in ET Patients
A Phase 3 study testing P1101 (Ropeginterferon alfa-2b) in people with Polycythemia Vera (PV), run by PharmaEssentia Japan K.K. at 6 sites in Japan.
Trial summary
This is a Phase 3 open-label, multicenter, single arm study designed to evaluate the efficacy and safety and tolerability of P1101 patient with PV or ET in long-term. Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Polycythemia Vera (PV)
- What is being tested?
- P1101 (Ropeginterferon alfa-2b)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 67 participants
- Where
- 6 sitesJapan
- Study dates
- 19 Jan 2021to 30 Jun 2026
- Phase
- Phase 3
- 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 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients who have completed the 52-week treatment duration in Study A19-201 and are considered by the investigator or sub investigator to be eligible for participation in this study
- Patients who have given written informed consent to participate in this study
Who cannot join
- Patients who are considered by the investigator or sub investigator to be ineligible for continued treatment with P1101
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
Japan6 sites, 6 recruiting
- Recruiting
Juntendo University Hospital
Bunkyo-ku, Tokyo, 113-8431
- Recruiting
University of Yamanashi Hospital
Chuo-shi, Yamanashi, 409-3898
- Recruiting
Tokyo Medical University Hospital
Shinjuku-ku, Tokyo, 160-0023
- Recruiting
Osaka University Hospital
Suita-shi, Osaka, 565-0871
- Recruiting
Ehime University Hospital
Toon-shi, Ehime, 791-0295
- Recruiting
Mie University Hospital
Tsu, Mie-ken
About this study
Official title: Extension Study of P1101 in Japanese Patients Who Have Completed Phase 2 Single Arm Study in Polycythemia Vera (PV) Patients (Study A19-201) or Phase 3 Study in Essential Thrombocythemia (ET) Patients (Study P1101 ET)
This is a Phase 3 open-label, multicenter, single arm study designed to evaluate the efficacy and safety and tolerability of P1101 patient with PV or ET in long-term.
Read the detailed description
The study is to evaluate the long-term safety and efficacy of P1101 in PV or ET patients who participated in Study A19-201 or Study P1101 ET. The subjects who have completed the 52-week P1101 treatment duration in Study A19-201 will start treatment with P1101 at the dose at Week 50. The subjects who have completed the follow-up/end-of-study visit in Study P1101 ET will start treatment with P1101 at the dose at Week 50. The subjects who were treated with anagrelide will start treatment with P1101 at a dose of 250 μg. The dose of P1101 during this study may be increased or decreased up to 500 μg depending on the condition.
Evaluation of safety will include assessing vital signs, clinical safety laboratory tests, physical examinations, ECG evaluation, heart ECHO, lung X-ray, ECOG performance status, ocular examination, and AEs.
Efficacy evaluations, safety assessments, and immunogenicity evaluations of P1101 will be performed.
Evaluation of efficacy will include clinical laboratory assessments, allelic burden measurements of CALR, JAK-2, and MPL, spleen size measurements, bone marrow sampling.
- Conditions
- Polycythemia Vera (PV)
- Being tested
- P1101 (Ropeginterferon alfa-2b)
- 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.
P1101 (Ropeginterferon alfa-2b)Biological
The subjects who have completed the 52-week treatment duration in Study A19-201 will be treated with P1101, starting at the dose at Week 50. The dose during this study may be increased or decreased up to 500 μg depending on the condition. This study will be continued as a post-marketing clinical study after acquisition of the marketing approval of P1101.
| Study group | Role | What they receive |
|---|---|---|
| P1101 (Ropeginterferon alfa-2b) | Experimental | Conventional treatment based on phlebotomies, lowdose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated prolineinterferon alpha-2b (P1101, Ropeginterferon alfa-2b) once every 2 weeks. Receives: Biological: P1101 (Ropeginterferon alfa-2b) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maintenance rate of phlebotomy-free complete hematologic response (CHR) every 52 weeks
CHR will be defined as follows.
- Hematocrit <45% phlebotomy-free (absence of phlebotomy during the previous 12 weeks)
- Platelet count ≤ 400 x 10^9/L
- WBC count ≤ 10 x 10^9/L
Measured: Through study completion, an average of 2 year
Secondary outcomes (8)
Changes in hematocrit every 52 weeks over time
Baseline is defined as Week 52 in Study A19-201
Measured: Through study completion, an average of 2 year
Changes in white blood cell every 52 weeks over time
Baseline is defined as Week 52 in Study A19-201
Measured: Through study completion, an average of 2 year
Changes in platelet count every 52 weeks over time
Baseline is defined as Week 52 in Study A19-201
Measured: Through study completion, an average of 2 year
Changes in red blood cell count every 52 weeks over time
Baseline is defined as Week 52 in Study A19-201
Measured: Through study completion, an average of 2 year
Changes in spleen size every 52 weeks over time
Baseline is defined as Week 52 in Study A19-201
Measured: Through study completion, an average of 2 year
Necessity of phlebotomy
Baseline is defined as Week 52 in Study A19-201
Measured: Through study completion, an average of 2 year
Proportion of subjects without thrombotic or hemorrhagic events
Baseline is defined as Week 52 in Study A19-201
Measured: Through study completion, an average of 2 year
Changes in JAK2 V617F mutant allelic burden value every 52 weeks over time
Baseline is defined as Week 52 in Study A19-201
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 posted7 Dec 2020
- Study start19 Jan 2021
- Primary completion30 Jun 2026 (estimated)
- Study completion30 Jun 2026 (estimated)
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
- 67 participants (estimated)
Sponsor and investigators
- Lead sponsor
- PharmaEssentia Japan K.K.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Keita Kirito, MD, Principal investigator, University of Yamanashi Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04655092
- Sponsor's protocol ID
- A20-201
- Status last verified
- Nov 2024
- First submitted
- 18 Nov 2020
- Last updated
- 8 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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
- Extension Study of P1101 in Japanese Patients Who Have Completed Phase 2 Single Arm Study in Polycythemia Vera (PV) Patients (Study A19-201) or Phase 3 Study in Essential Thrombocythemia (ET) Patients (Study P1101 ET)
- ClinicalTrials.gov ID
- NCT04655092
- Acronym / sponsor ID
- A20-201
- Conditions
- Polycythemia Vera (PV)
- Keywords
- Myeloproliferative Neoplasms
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 67 participants (estimated)
- Lead sponsor
- PharmaEssentia Japan K.K.Industry (pharma, biotech, devices)
- Study start
- 19 Jan 2021 (actual)
- Primary completion
- 30 Jun 2026 (estimated)
- Study completion
- 30 Jun 2026 (estimated)
- First posted
- 7 Dec 2020 (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 open-label, multicenter, single arm study designed to evaluate the efficacy and safety and tolerability of P1101 patient with PV or ET in long-term.
Detailed description
The study is to evaluate the long-term safety and efficacy of P1101 in PV or ET patients who participated in Study A19-201 or Study P1101 ET. The subjects who have completed the 52-week P1101 treatment duration in Study A19-201 will start treatment with P1101 at the dose at Week 50. The subjects who have completed the follow-up/end-of-study visit in Study P1101 ET will start treatment with P1101 at the dose at Week 50. The subjects who were treated with anagrelide will start treatment with P1101 at a dose of 250 μg. The dose of P1101 during this study may be increased or decreased up to 500 μg depending on the condition.
Evaluation of safety will include assessing vital signs, clinical safety laboratory tests, physical examinations, ECG evaluation, heart ECHO, lung X-ray, ECOG performance status, ocular examination, and AEs.
Efficacy evaluations, safety assessments, and immunogenicity evaluations of P1101 will be performed.
Evaluation of efficacy will include clinical laboratory assessments, allelic burden measurements of CALR, JAK-2, and MPL, spleen size measurements, bone marrow sampling.
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 (Ropeginterferon alfa-2b) | Experimental | Biological: P1101 (Ropeginterferon alfa-2b)Conventional treatment based on phlebotomies, lowdose aspirin (acetylsalicylic acid, 75-150 mg/day) plus the subcutaneous administration of pegylated prolineinterferon alpha-2b (P1101, Ropeginterferon alfa-2b) once every 2 weeks. |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Patients who have completed the 52-week treatment duration in Study A19-201 and are considered by the investigator or sub investigator to be eligible for participation in this study
- Patients who have given written informed consent to participate in this study
Key exclusion criteria (1 listed)
- Patients who are considered by the investigator or sub investigator to be ineligible for continued treatment with P1101
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 |
|---|---|---|
| P1101 (Ropeginterferon alfa-2b)The subjects who have completed the 52-week treatment duration in Study A19-201 will be treated with P1101, starting at the dose at Week 50. The dose during this study may be increased or decreased up to 500 μg depending on the condition. This study will be continued as a post-marketing clinical study after acquisition of the marketing approval of P1101. | Biological | P1101 (Ropeginterferon alfa-2b) |
Primary outcome
Maintenance rate of phlebotomy-free complete hematologic response (CHR) every 52 weeks
Time frame: Through study completion, an average of 2 year
CHR will be defined as follows. * Hematocrit <45% phlebotomy-free (absence of phlebotomy during the previous 12 weeks) * Platelet count ≤ 400 x 10^9/L * WBC count ≤ 10 x 10^9/L
8 secondary outcomes
Changes in hematocrit every 52 weeks over time
Time frame: Through study completion, an average of 2 year
Changes in white blood cell every 52 weeks over time
Time frame: Through study completion, an average of 2 year
Changes in platelet count every 52 weeks over time
Time frame: Through study completion, an average of 2 year
Changes in red blood cell count every 52 weeks over time
Time frame: Through study completion, an average of 2 year
Changes in spleen size every 52 weeks over time
Time frame: Through study completion, an average of 2 year
Necessity of phlebotomy
Time frame: Through study completion, an average of 2 year
Proportion of subjects without thrombotic or hemorrhagic events
Time frame: Through study completion, an average of 2 year
Changes in JAK2 V617F mutant allelic burden value every 52 weeks over time
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 8 Nov 2024.
- Individual participant dataSharing planned: No
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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.
Keita Kirito, MD principal investigator
University of Yamanashi Hospital
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 (Ropeginterferon alfa-2b) 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 2 inclusion and 1 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 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?
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 Hiroaki Kawase (+81-3-6910-5103, hiroaki_kawase@pharmaessentia.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maintenance rate of phlebotomy-free complete hematologic response (CHR) every 52 weeks (time frame: Through study completion, an average of 2 year). 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.