PEGINVERA: Safety Study of Pegylated Interferon Alpha 2b to Treat Polycythemia Vera

Sponsor

AOP Orphan Pharmaceuticals AG (Industry)

Overall Status

Completed

CT.gov ID

NCT01193699

Collaborator

(none)

Enrollment

Locations

Arm

89.8

Actual Duration (Months)

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Polycythemia Vera	Drug: PEG-P-INF alpha-2b (P1101)	Phase 1/Phase 2

Study Design

Study Type:

Interventional

Anticipated Enrollment :

24 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Aug 1, 2010

Actual Primary Completion Date :

Jan 25, 2018

Actual Study Completion Date :

Jan 25, 2018

Arms and Interventions

Arm	Intervention/Treatment
Experimental: P1101	Drug: PEG-P-INF alpha-2b (P1101) µg (starting with 50 µg), subcutaneously, 2-weekly administration

Arm

Intervention/Treatment

Experimental: P1101

Drug: PEG-P-INF alpha-2b (P1101)

µg (starting with 50 µg), subcutaneously, 2-weekly administration

Outcome Measures

Primary Outcome Measures

Maximum tolerated dose (MTD) [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).

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 90 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

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.

Exclusion criteria:

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
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).

Contacts and Locations

Locations

	City	State	Country	Postal Code
1	Innsbruck	Tirol	Austria	6020
2	Wels	Upper Austria	Austria	4600
3	Salzburg		Austria	5020
4	Vienna		Austria	1090
5	Vienna		Austria	1140
6	Vienna		Austria	1220

Sponsors and Collaborators

AOP Orphan Pharmaceuticals AG

Investigators

Study Director: Barbara Grohmann-Izay, MD, AOP Orphan Pharmaceuticals AG

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

AOP Orphan Pharmaceuticals AG

ClinicalTrials.gov Identifier:

NCT01193699

Other Study ID Numbers:

P11012010

First Posted:

Sep 2, 2010

Last Update Posted:

Jan 30, 2018

Last Verified:

Jan 1, 2018

Additional relevant MeSH terms:

Polycythemia Vera

Polycythemia

Study Results

No Results Posted as of Jan 30, 2018