Safety and efficacy study of INC424 in patients with myelofibrosis

• Register: EUCTR
• Last refreshed on: 3 April 2017
• Main ID: EUCTR2010-024473-39-GR
• Date of registration: 07/07/2011
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: Safety and efficacy study of INC424 in patients with myelofibrosis
• Scientific title: An open-label, multicenter, expanded access study of INC424 for patients with primary myelofibrosis (PMF) of post polycythemia vera myelofibrosis (PPV MF) or post-essential thrombocythemia myelofibrosis (PET-MF)
• Date of first enrolment: 02/09/2011
• Target sample size: 950
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-024473-39
• 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): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Argentina, Austria, Belgium, Brazil, Canada, Colombia, Czech Republic, Germany, Greece, Hungary, Ireland, Italy, Korea, Republic of, Lebanon, Mexico, Poland, Portugal, Russian Federation, Saudi Arabia, Singapore, Slovakia, South Africa, Spain, Switzerland, Taiwan, Venezuela, Bolivarian Republic of

Key inclusion & exclusion criteria

Inclusion criteria:
1. patient must give written informed consent according to local guideliens prior to any screening procedure
2. patients must not be eligible for another ongoing INC424 clinical trial
3. male or female patients aged = 18 years of age
4. patients must be diagnosed with PMF, PPv mf or PET MF according to the WHO citeria 2008
5. PMF patients requiruing therapy must be classified as high risk or intermediate risk level 2 or intermediate risk level 1 with enlarged spleen
6. patients with intermediate-1 and splenomegaly must have a palpable spleen measuring 5 cm or greater from the costal margin to the point of greatest splenic protrusion

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 5
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 25

Exclusion criteria:
1. Patients eligible for hematopoietic stem cell transplantation
2. Patients with a history of malignancy in the past 3 years, except for treated early stage squamous or basal cell carcinoma in situ
3. patients undergoing treatment with hematopoietic growth factor receptor agonists, granulocyte colony stimulant factor at nay time within 2 weeks prior to screening or 4 weeks prior to baseline
4. Patients currently participating in COMFORT-I and COMFORT -II trials
5.Patients receiving any medications listed in the "prohibited medications" listing
6. Impairment of GI function or GI disease that may alter the absorption of INC424
7. Patients with cardiac disease which my jeopardize the safety of the patient
8. Patients with currently uncontrolled or unstable angina, rapid or paroxysmal fibrillation or recent myocardial infarction or acute coronary syndrome
9. Patients with clinically significant infections (for further details see protocol)
10. Patients with known active hepatitis A, B, C or who are HIV -positive
11. Patients with coagulation parameters
12.Pregnant or nursing women


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

Health Condition(s) or Problem(s) studied

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

primary myelofibrosis (PMF), post polycythemia vera myelofibrosis (PPV MF) or Post essential thrombocythemia myelofibrosis (PET-MF)

Intervention(s)

Product Name: ruxolitinib
Product Code: INC424
Pharmaceutical Form: Tablet
Current Sponsor code: INC424
Other descriptive name: ruxolitinib
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-mg

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: Monthly for the first 3 months, then every 3 months and at study discontinuation

Secondary Objective: To document the best overall response rate to INC424 in patients with PMF, PPV MF , or PET MF as evaluated by the investigator

To collect quality of life (QoL) endpoints

To document medical resource utilization

Main Objective: to collect additional safety of INC424 in patients with PMF, PPV MF, or PET MF, who have either received prior treatment with commercially available agents or never received treatment

Primary end point(s): Safety:
Clinical and laboratory parameters will be collected to evaluate study drug safety and toxicity.
Safety and tolerability will be collected by monitoring the frequency, duration and severity of all grade adverse events (AEs) by CTCAE v.3.0, performing physical exams (PE), and evaluating changes in vital signs (VS), ECOG performance status (PS), ECGs and serum chemistry and hematology results.
Grade 3 and 4 AEs, Serious Adverse Events (SAEs).
Frequency of dose interruptions and discontinuations due to AEs.

Secondary Outcome(s)

Secondary end point(s): Quality of life:
Change in ECOG PS from baseline
Change in FACT-Lym
Change in Functional Assessment of Chronic Illness Therapy

Timepoint(s) of evaluation of this end point: Monthly for the first 3 months, then every 3 months and at study discontinuation

Secondary ID(s)

CINC424A2401

2010-024473-39-AT

Source(s) of Monetary Support

Novartis Pharma Services AG