Clinical trial NCT01392443: Asian Phase II Study of INC424 in Patients With Primary Myelofibrosis (MF), Post-PV MF or Post-ET MF
Official title: A Multi-national Open-label Phase II Study of the JAK Inhibitor INC424 in Patients With Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis or Post-essential Thrombocythemia Myelofibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Primary Myelofibrosis (MF)
Post-Polycythemia Vera (PV) MF
Post-Essential Thrombocythemia (ET) MF
Keywords: Primary Myelofibrosis (MF)
Post-Polycythemia Vera (PV) MF
Post-Essential Thrombocythemia (ET) MF
post-PV MF
post-ET MF
INC424
Ruxolitinib
Myelofibrosis
MF
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2011-07-12
Enrollment: 120
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The objective of this study was to determine the efficacy of INC424 as assessed by reduction in spleen volume in patients with primary myelofibrosis (MF), post-polycythemia vera (PV) MF, or post-essential thrombocythemia (ET) MF. The safety and tolerability of INC424 and the effects of INC424 on patient reported outcomes and the duration of response as assessed by reduction in spleen volume was also assessed.


Eligibility criteria: Inclusion Criteria:

1. 18 years or older
2. Diagnosis of primary myelofibrosis (MF), post-polycythemia vera (PV) MF, or post-essential thrombocythemia (ET) MF
3. Enlarged spleen, measuring 5 cm or greater from the costal margin
4. Must have two or more of the following risk factors:

   1. Over 65 years old
   2. Have the following symptoms often associated with MF: loss of weight, fever, night sweats
   3. Have a low red blood cell count (anemia - hemoglobin \< 10 g/dL)
   4. Have a high white blood cell count (history of white blood cell count \> 25,000/uL)
   5. Have high circulating blasts (\> or = 1%) as measured by blood tests
5. Should have circulating blasts \<10% (as measured by blood tests)
6. Should be capable of self-care
7. Should have adequate bone marrow reserve
8. Should not have the option of stem cell transplantation
9. Should discontinue any prior or ongoing treatment for myelofibrosis prior to entering the study
10. Had no prior treatment with another JAK inhibitor

Exclusion Criteria:

1. Does not have adequate liver or kidney function (as measured by blood tests)
2. Has an active infection (bacterial, viral, etc.)
3. Has active hepatitis A, B, or C or positive for HIV
4. Has another cancer that needs active intervention
5. Had a history of bleeding disorder
6. Had a history of very low platelet counts (as measured by blood tests) not related to treatment of MF
7. Had radiation of the spleen within 1 year of joining the study
8. Does not have adequate heart function
9. Sufficient time has elapsed between stopping previous treatment for MF and joining the study
10. Females who are pregnant or breast-feeding
11. Not able to sign informed consent
12. Has any other active medical conditions that the doctor deems may compromise your safety or ability to join in the study

Primary outcomes:
measure: Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Week 24
timeFrame: 24 weeks
Secondary outcomes:
measure: Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Each Scheduled Time Point - Best Response
timeFrame: Weeks 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, at any time point
measure: Kaplan Meier Estimates of Duration of Response of at Least ≥ 35% Reduction From Baseline in Spleen Volume Per Kaplan Meier Estimates
timeFrame: Weeks 12, 24, 36, 48, 60, 72, 84, 96, 120, 144, 168, 192, 216, 240
measure: Change in EORTC QLQ-C30 Scores From Baseline in at Week 24
timeFrame: Baseline, Week 24
measure: Change in Total Symptom Score From Baseline at Week 24 as Measured by Seven-day Modified MFSAF v2.0
timeFrame: Baseline, Week 24
Other outcomes:
Locations:
city: Beijing
country: China
facility: Novartis Investigative Site
state: Beijing
city: Guangzhou
country: China
facility: Novartis Investigative Site
state: Guangdong
city: Wuhan
country: China
facility: Novartis Investigative Site
state: Hubei
city: Nanjing
country: China
facility: Novartis Investigative Site
state: Jiangsu
city: Suzhou
country: China
facility: Novartis Investigative Site
state: Jiangsu
city: Chengdu
country: China
facility: Novartis Investigative Site
state: Sichuan
city: Tianjin
country: China
facility: Novartis Investigative Site
state: Tianjin
city: Hangzhou
country: China
facility: Novartis Investigative Site
state: Zhejiang
city: Jinan
country: China
facility: Novartis Investigative Site
city: Shanghai
country: China
facility: Novartis Investigative Site
city: Nagoya
country: Japan
facility: Novartis Investigative Site
state: Aichi
city: Fukuoka city
country: Japan
facility: Novartis Investigative Site
state: Fukuoka
city: Maebashi city
country: Japan
facility: Novartis Investigative Site
state: Gunma
city: Kanazawa-city
country: Japan
facility: Novartis Investigative Site
state: Ishikawa
city: Tsu-city
country: Japan
facility: Novartis Investigative Site
state: Mie
city: Suita city
country: Japan
facility: Novartis Investigative Site
state: Osaka
city: Bunkyo ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Shinjuku-ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Shinjuku-ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Seoul
country: Korea, Republic of
facility: Novartis Investigative Site
state: Korea
city: Seoul
country: Korea, Republic of
facility: Novartis Investigative Site
state: Seocho Gu
city: Seoul
country: Korea, Republic of
facility: Novartis Investigative Site
city: Seoul
country: Korea, Republic of
facility: Novartis Investigative Site
city: Kaohsiung
country: Taiwan
facility: Novartis Investigative Site
city: Taipei
country: Taiwan
facility: Novartis Investigative Site
city: Taoyuan
country: Taiwan
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Jin J, Du X, Zhou DB, Li JM, Li JY, Hou M, Liu T, Wu DP, Hu Y, Xiao ZJ. [Efficacy and safety of JAK inhibitor ruxolitinib in Chinese patients with myelofibrosis: results of a 1-year follow-up of A2202]. Zhonghua Xue Ye Xue Za Zhi. 2016 Oct 14;37(10):858-863. doi: 10.3760/cma.j.issn.0253-2727.2016.10.007. Chinese.