Diagnosis for Polycythemia Vera as per the WHO or PVSG. MedDRA version: 19.1 Level: LLT Classification code 10036061 Term: Polycythemia vera System Organ Class: 100000004864
Conditions
Interventions
Product Name: PEG-P-IFN a-2b
Product Code: P1101
Pharmaceutical Form: Solution for injection
Other descriptive name: pegylated Proline-IFNa-2b
Concentration unit: µg microgram(s)
Concentration type:
Sponsors
AOP Orphan Pharmaceuticals AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Written informed consent obtained prior to any study specific screening activities and able to comply with this protocol 2. Patients age =18 years 3. Confirmed diagnosis of PV according to either the WHO criteria 2008 or the PSVG criteria plus JAK-2 positivity. 4. Eastern Cooperative Oncology Group performance status = 2 5. 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. Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 37 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 14
Exclusion criteria
Exclusion criteria: 1. Diagnosis of any other myeloproliferative disorder 2. Any clinically significant illness or surgery within 4 weeks prior to dosing 3. Systemic infections, e.g. hepatitis B, hepatitis C, or HIV at screening 4. 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. 5. Previous treatment with Interferon for PV 6. Concurrent treatment with other cytoreductive agents other than Hydroxyurea and investigational agents of any type 7. 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 8. History of severe allergic (like anaphylaxis) or hypersensitivity reactions (like angioedema), any known or suspected intolerance to the investigational product. 9. Use of any investigational drug or participation in an investigational drug study within the last 4 weeks 10. Clinically significant history or known presence of psychiatric disorders, including but not limited to depression, anxiety and sleep disorders 11. Organ transplant, past or planned 12. Inadequate liver function: Serum (total) bilirubin > 2,5 x ULN, AST and ALT > 2,5 x ULN 13. Clinically significant ECG findings 14. History of renal disease requiring haemodialysis or seizure disorder requiring anticonvulsant therapy 15. Pregnant or lactating females (pregnancy test to be assessed within 7 days prior to study treatment start) 16. Acute or chronic infections or autoimmune diseases (collagen diseases, polyarthritis, immune thrombocythemia, psoriasis, lupus etc, thyroiditis.)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Investigation of maximum tolerated dose ;Secondary Objective: •Safety/Tolerability •Efficacy;Primary end point(s): Identification of the maximum tolerated dose (MTD) of the investigational medicinal product. ;Timepoint(s) of evaluation of this end point: When MTD is achieved. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Determination of safety and tolerability of P1101 in patients with polycythaemia vera. An exploratory analysis of efficacy and biomarker modulation will be also performed.;Timepoint(s) of evaluation of this end point: Throughout the whole study. Efficacy: The first time after 12 weeks and then every 10 weeks thereafter. Biomarker: Every 2 weeks until MTD, then 5 times every 2 weeks followed by an optional intensive biomarker blood sampling schedule (6 times in the course of 2 weeks). | — |
Countries
Austria
Contacts
Public ContactSimone Pleifer
AOP Orphan Pharmaceuticals AG
Outcome results
None listed