safety and efficacy of ruxolitinib in anemic myelofibrosis patients

• Register: EUCTR
• Last refreshed on: 30 June 2019
• Main ID: EUCTR2016-003552-75-GR
• Date of registration: 30/11/2016
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: safety and efficacy of ruxolitinib in anemic myelofibrosis patients
• Scientific title: A multicenter phase II, open label, single arm study to evaluate the efficacy and safety of ruxolitinib in the treatment of anemic myelofibrosis patients - REALISE
• Date of first enrolment: 05/01/2017
• Target sample size: 50
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-003552-75
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no; Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Austria, Belgium, Bulgaria, Canada, Chile, Germany, Greece, Hungary, Italy, Japan, Russian Federation, Spain, Turkey

Contacts

• Name: Eleni Konstantinidou
• Affiliation: Novartis (Hellas) S.A.C.I.

• Name: Eleni Konstantinidou
• Affiliation: Novartis (Hellas) S.A.C.I.

Key inclusion & exclusion criteria

Inclusion criteria:
- Male or female patients aged = 18 years of age.
- Patients must be diagnosed with PMF, according to the 2016 revised International Standard Criteria (Arber et al, 2016), PPV MF or PET-MF (Barosi 2008), irrespective of JAK2 mutation status.
- Patients with palpable splenomegaly that is equal to or greater than 5 cm below the left costal margin.
- Patients with a hemoglobin less than 10 g/dL
- Patients with a history of transfusions must have a documented transfusion record in the previous 12 weeks to baseline.
- Patients with a peripheral blood blast percentage count of < 10%.

Additional inclusion criteria as per full protocol may apply.
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 30

Exclusion criteria:
- Patients with prior treatment with any JAK1 or JAK2 inhibitor.
- Patients with inadequate bone marrow reserve at baseline visit asdemonstrated by at least one of the following:
? ANC that is = 1,000/µL.
? Platelet count that is <50,000/µL without the assistance of growth factors, thrombopoietic factors or platelet transfusions.
? Hemoglobin count that is = 6.5 g/dL despite transfusions.
- Patients with severely impaired renal function
- Patients with inadequate liver function
- Patients being treated concurrently with a strong (potent) systemic inhibitor or inducer of CYP3A4 at the time of Screening
- Acute viral hepatitis or active chronic hepatitis B or C infection.
- History of progressive multifocal leuko-encephalopathy (PML)

Additional exclusion criteria as per full protocol may apply.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Cancer [C04]

treatment of anemic myelofibrosis patients
MedDRA version: 19.0 Level: LLT Classification code 10074692 Term: Post essential thrombocythaemia myelofibrosis System Organ Class: 100000004864
MedDRA version: 19.0 Level: LLT Classification code 10074691 Term: Post polycythaemia vera myelofibrosis System Organ Class: 100000004864

Intervention(s)

Trade Name: Jakavi
Product Name: ruxolitinib
Product Code: INC424
Pharmaceutical Form: Tablet
INN or Proposed INN: ruxolitinib
CAS Number: 1092939-17-7
Current Sponsor code: INC424
Other descriptive name: RUXOLITINIB PHOSPHATE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-

Primary Outcome(s)

Secondary Objective: - To evaluate safety
- To determine the spleen length response rate at Week 48
- To evaluate the effect of ruxolitinib on spleen length
- To evaluate the effect of ruxolitinib on symptoms
- To evaluate the effect of ruxolitinib on Patient Global Impression of Change (PGIC)
- To evaluate the effect of ruxolitinib on transfusion requirements

Primary end point(s): Proportion of patients achieving a 50% reduction in spleen length at Week 24

Timepoint(s) of evaluation of this end point: Week 24

Main Objective: To determine the spleen length response rate at Week 24

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: - Week 48
- Week 48
- Week 48
- Week 48
- Week 48
- Week 48

Secondary end point(s): - Frequency and severity of adverse events and serious adverse events and AEs leading to discontinuations.
- Proportion of patients achieving a 50% reduction in spleen length at week 48
- Percent change from baseline in spleen length over time
- Summary of the Modified MFSAF v2.0 and MF-7 over time
- PGIC at each visit where measured
- Summary of transfusions over time

Secondary ID(s)

2016-003552-75-HU

CINC424A2411

Source(s) of Monetary Support

Novartis Pharma Services AG

Ethics review

Status: Approved
Approval date:
Contact: