Dose finding study with the study medication PEG-Proline-Interferon alpha-2b for patients suffering from polycythemia vera.

Phase 1

• Conditions: Diagnosis for Polycythemia Vera as per the WHO or PVSG.; MedDRA version: 19.1Level: LLTClassification code 10036061Term: Polycythemia veraSystem Organ Class: 100000004864; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2010-018768-18-AT
• Lead Sponsor: AOP Orphan Pharmaceuticals AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-03-23
• Last Update Posted: 26 March 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 51

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

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.)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Investigation of maximum tolerated dose ;Secondary Objective: •Safety/Tolerability<br>•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 Outcome Measures

Name	Time	Method
Secondary end point(s): Determination of safety and tolerability of P1101 in patients with polycythaemia vera.<br>An exploratory analysis of efficacy and biomarker modulation will be also performed.;Timepoint(s) of evaluation of this end point: Throughout the whole study.<br><br>Efficacy: The first time after 12 weeks and then every 10 weeks thereafter.<br><br>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).