Polycythemia VeraPhase 1/2PEGINVERANCT01193699
Safety Study of Pegylated Interferon Alpha 2b to Treat Polycythemia Vera
A Phase 1/2 study testing PEG-P-INF alpha-2b (P1101) in people with Polycythemia Vera, run by AOP Orphan Pharmaceuticals AG at 6 sites in Austria.
Trial summary
The purpose of this study is the identification of the maximum tolerated dose (MTD) of the investigational medicinal product. Moreover the safety and tolerability will be assessed and an exploratory analysis of efficacy and biomarker modulation will be performed. Read more about this study
- Who is it for?
- Ages 18 to 90 years
- Conditions the study is for
- Polycythemia Vera
- What is being tested?
- PEG-P-INF alpha-2b (P1101)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 6 sitesAustria
- Study dates
- Aug 2010to 25 Jan 2018
- Phase
- Phase 1/2
- 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 to 90 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Written informed consent obtained prior to any study specific screening activities and able to comply with this protocol.
- Patients age ≥18 years
- Confirmed diagnosis of PV according to either the WHO criteria (2008, appendix 6) or the PSVG (appendix 7) criteria plus JAK-2 positivity, including newly diagnosed, pre-treated and on cytoreductive therapy.
- Eastern Cooperative Oncology Group performance status ≤ 2
- If female of childbearing potential - have a negative urine pregnancy test result within 7 days prior to the scheduled first application of investigational product and agree to employ adequate birth control measures for the duration of the study.
Who cannot join
- Diagnosis of any other myeloproliferative disorder
- Any clinically significant illness or surgery within 4 weeks prior to dosing
- Systemic infections, e.g. hepatitis B, hepatitis C, or HIV at screening
- Uncontrolled hypertension (systolic > 150 mmHg and diastolic > 100 mmHg, or clinically significant (i.e. active) cardiovascular disease: CVA/stroke (≤ 3 months prior to enrolment), myocardial infarction (≤ 3 months prior to enrolment), significant coronary artery stenosis, unstable angina, New York Heart Association (NYHA) Class 2 or greater Congestive heart failure, or serious cardiac arrhythmia requiring medication.
- Previous treatment with Interferon for PV
- Concurrent treatment with cytoreductive agents other than Hydroxyurea and investigational agents of any type
Show all 16Show fewer
- History of malignant disease, including solid tumours and haematological malignancies (except basal cell and squamous cell carcinomas of the skin and carcinoma in situ of the cervix that have been completely excised and are considered cured) within the last 3 years
- History of severe allergic (like anaphylaxis) or hypersensitivity reactions (like angioedema), any known or suspected intolerance to the investigational product.
- Use of any investigational drug or participation in any investigational drug study within the last 4 weeks
- Clinically significant history or known presence of psychiatric disorders, including but not limited to depression, anxiety and sleep disorders
- Organ transplant, past or planned
- Inadequate liver function defined by serum (total) bilirubin > 2,5 x ULN and/ or AST and ALT > 2,5 x ULN
- Clinically significant ECG findings
- History of renal disease requiring haemodialysis or seizure disorder requiring anticonvulsant therapy
- Pregnant or lactating females (pregnancy test to be assessed within 7 days prior to study treatment start)
- Acute or chronic infections or autoimmune diseases (collagen diseases, polyarthritis, immune thrombocythemia, thyroiditis, psoriasis, lupus nephritis or any other autoimmune disorder).
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
Austria6 sites
Study site
Innsbruck, Tyrol, 6020
Study site
Salzburg, 5020
Study site
Vienna, 1090
Study site
Vienna, 1140
Study site
Vienna, 1220
Study site
Wels, Upper Austria, 4600
About this study
Official title: An Open-label, Prospective, Multicentre, Phase I/II Dose Escalation Study to Determine the Maximum Tolerated Dose and to Assess the Safety and Efficacy of P1101, PEG-Proline-Interferon Alpha-2b in Patients With Polycythaemia Vera
The purpose of this study is the identification of the maximum tolerated dose (MTD) of the investigational medicinal product. Moreover the safety and tolerability will be assessed and an exploratory analysis of efficacy and biomarker modulation will be performed.
- Conditions
- Polycythemia Vera
- Being tested
- PEG-P-INF alpha-2b (P1101)
- Medical subject headings
- Polycythemia Vera
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PEG-P-INF alpha-2b (P1101)Drug
µg (starting with 50 µg), subcutaneously, 2-weekly administration
| Study group | Role | What they receive |
|---|---|---|
| P1101 | Experimental | Not described Receives: Drug: PEG-P-INF alpha-2b (P1101) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose (MTD)
The definition of MTD is based on a 3+3 dose escalation design. MTD is defined as the next lower dose of that dose which was considered to be untolerated (observed DLT frequency at least 2 out of 3 in one cohort or at least 2 out of six patients in 2 cohorts).
Measured: The incidence of dose limiting toxicities (DLTs), which define the MTD are assessed continously until achievement of MTD.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Sep 2010 (estimated)
- Study startAug 2010
- Primary completion25 Jan 2018
- Study completion25 Jan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Polycythemia VeraStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AOP Orphan Pharmaceuticals AG
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Barbara Grohmann-Izay, MD, Study director, AOP Orphan Pharmaceuticals AG
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01193699
- Sponsor's protocol ID
- P11012010
- Acronym
- PEGINVERA
- Status last verified
- Jan 2018
- First submitted
- 1 Sep 2010
- Last updated
- 30 Jan 2018
- Results posted
- No
- Data monitoring committee
- No
Publications
Gisslinger H, Zagrijtschuk O, Buxhofer-Ausch V, Thaler J, Schloegl E, Gastl GA, Wolf D, Kralovics R, Gisslinger B, Strecker K, Egle A, Melchardt T, Burgstaller S, Willenbacher E, Schalling M, Them NC, Kadlecova P, Klade C, Greil R. Ropeginterferon alfa-2b, a novel IFNalpha-2b, induces high response rates with low toxicity in patients with polycythemia vera. Blood. 2015 Oct 8;126(15):1762-9. doi: 10.1182/blood-2015-04-637280. Epub 2015 Aug 10.
PubMed 26261238
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Prospective, Multicentre, Phase I/II Dose Escalation Study to Determine the Maximum Tolerated Dose and to Assess the Safety and Efficacy of P1101, PEG-Proline-Interferon Alpha-2b in Patients With Polycythaemia Vera
- ClinicalTrials.gov ID
- NCT01193699
- Acronym / sponsor ID
- PEGINVERA / P11012010
- Conditions
- Polycythemia Vera
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- AOP Orphan Pharmaceuticals AGIndustry (pharma, biotech, devices)
- Study start
- Aug 2010 (actual)
- Primary completion
- 25 Jan 2018 (actual)
- Study completion
- 25 Jan 2018 (actual)
- First posted
- 2 Sep 2010 (estimated)
- 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.
The purpose of this study is the identification of the maximum tolerated dose (MTD) of the investigational medicinal product. Moreover the safety and tolerability will be assessed and an exploratory analysis of efficacy and biomarker modulation will be performed.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| P1101 | Experimental | Drug: PEG-P-INF alpha-2b (P1101) |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Written informed consent obtained prior to any study specific screening activities and able to comply with this protocol.
- Patients age ≥18 years
- Confirmed diagnosis of PV according to either the WHO criteria (2008, appendix 6) or the PSVG (appendix 7) criteria plus JAK-2 positivity, including newly diagnosed, pre-treated and on cytoreductive therapy.
- Eastern Cooperative Oncology Group performance status ≤ 2
- If female of childbearing potential - have a negative urine pregnancy test result within 7 days prior to the scheduled first application of investigational product and agree to employ adequate birth control measures for the duration of the study.
Key exclusion criteria (16 listed)
- Diagnosis of any other myeloproliferative disorder
- Any clinically significant illness or surgery within 4 weeks prior to dosing
- Systemic infections, e.g. hepatitis B, hepatitis C, or HIV at screening
- Uncontrolled hypertension (systolic > 150 mmHg and diastolic > 100 mmHg, or clinically significant (i.e. active) cardiovascular disease: CVA/stroke (≤ 3 months prior to enrolment), myocardial infarction (≤ 3 months prior to enrolment), significant coronary artery stenosis, unstable angina, New York Heart Association (NYHA) Class 2 or greater Congestive heart failure, or serious cardiac arrhythmia requiring medication.
- Previous treatment with Interferon for PV
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 |
|---|---|---|
| PEG-P-INF alpha-2b (P1101)µg (starting with 50 µg), subcutaneously, 2-weekly administration | Drug | P1101 |
Primary outcome
Maximum tolerated dose (MTD)
Time frame: The incidence of dose limiting toxicities (DLTs), which define the MTD are assessed continously until achievement of MTD.
The definition of MTD is based on a 3+3 dose escalation design. MTD is defined as the next lower dose of that dose which was considered to be untolerated (observed DLT frequency at least 2 out of 3 in one cohort or at least 2 out of six patients in 2 cohorts).
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 30 Jan 2018.
- Derived from this study: PubMed 26261238Gisslinger H, Zagrijtschuk O, Buxhofer-Ausch V, Thaler J, Schloegl E, Gastl GA, Wolf D, Kralovics R, Gisslinger B, Strecker K, Egle A, Melchardt T, Burgstaller S, Willenbacher E, Schalling M, Them NC, Kadlecova P, Klade C, Greil R. Ropeginterferon alfa-2b, a novel IFNalpha-2b, induces high response rates with low toxicity in patients with polycythemia vera. Blood. 2015 Oct 8;126(15):1762-9. doi: 10.1182/blood-2015-04-637280. Epub 2015 Aug 10.
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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.
Barbara Grohmann-Izay, MD study director
AOP Orphan Pharmaceuticals AG
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing PEG-P-INF alpha-2b (P1101) in people with Polycythemia Vera, run by AOP Orphan Pharmaceuticals AG at 6 sites in Austria.
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 5 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: finished, as of the sponsor's last update on 30 Jan 2018. 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 Austria. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
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?
Maximum tolerated dose (MTD) (time frame: The incidence of dose limiting toxicities (DLTs), which define the MTD are assessed continously until achievement of MTD.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01193699, which also lists 1 linked publication.
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 30 Jan 2018.