The study to assess the efficacy and safety of new Interferon alpha-2b formulation in patients with primary myelofibrosis

Phase 1

• Conditions: Primary Myelofibrosis (grade MF-0 and MF-1) according to the WHO criteria; MedDRA version: 17.0 Level: LLT Classification code 10053134 Term: Osteomyelofibrosis System Organ Class: 100000004864; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2014-001367-13-AT
• Lead Sponsor: Medizinische Universtät Wien, Universitätsklinik für Innere Medizin I

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-07-07
• Last Update Posted: 10 December 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 24

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 primary myelofibrosis (grade MF-0 and MF-1) according to the WHO criteria (2008). Including either
newly diagnosed Interferon naive patients or patients pre-treated with Pegasys® or PegIntron®, who had stable disease for
the previous 3 months.
4.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.
5.Eastern Cooperative Oncology Group performance status = 2.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4

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.History of malignant disease, including solid tumours (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) and haematological
malignancies within the last 3 years
6. History of severe allergic (like anaphylaxis) or hypersensitivity reactions (like angioedema), any known or suspected
intolerance to the investigational product
7. Use of any investigational drug or participation in any investigational drug study within the last 4 weeks
8.Clinically significant history or known presence of psychiatric disorders, including but not limited to depression, anxiety
and sleep disorders
9. HADS score equal to or above 11 on either or both of the subscales (Appendix 3)
10. Any risk of suicide at screening or previous suicide attempts
11. Organ transplant, past or planned
12. Inadequate liver function defined by serum (total) bilirubin > 2,5 x ULN and/or 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 in fertile women to be assessed within 7 days prior to study treatment start)
16.Acute or chronic infections or autoimmune diseases (collagen diseases, polyarthritis, immune thrombocythemia, thyroiditis,
psoriasis, lupus nephritis or any other autoimmune disorder)
17.Advanced primary Myelofibrosis (> Grade 2)
18.Clinically relevant pulmonary infiltrates, pneumonia, and pneumonitis at screening
19.Any significant morbidity or abnormality which may interfere with the study participation
20.History of active substance or alcohol abuse within the last year
21.Evidence of severe retinopathy (e.g. cytomegalovirus retinitis, macular degeneration) or clinically relevant ophthalmological
disorder (due to diabetes mellitus or hypertension)
22.Thyroid dysfunction not adequately controlled
23.Patients tested positively with TgAb and / or TPOAb at screening
24.History of uncontrolled severe seizure disorder
25.Leukocytopenia at the time of screening
26.Thrombocytopenia at the time of screening

Study & Design

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