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Primary Myelofibrosis (MF)Phase 2NCT01392443

Asian Phase II Study of INC424 in Patients With Primary Myelofibrosis (MF), Post-PV MF or Post-ET MF

A Phase 2 study testing Ruxolitinib in people with Primary Myelofibrosis (MF) and Post-Polycythemia Vera (PV) MF, run by Novartis Pharmaceuticals at 26 sites in 4 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 65 years
What is being tested?
Ruxolitinib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
120 participants
Where
26 sitesin 4 countries
Study dates
14 Oct 2010to 31 Oct 2017
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 65 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 18 years or older
  • Diagnosis of primary myelofibrosis (MF), post-polycythemia vera (PV) MF, or post-essential thrombocythemia (ET) MF
  • Enlarged spleen, measuring 5 cm or greater from the costal margin
  • Must have two or more of the following risk factors:
  • Over 65 years old
  • Have the following symptoms often associated with MF: loss of weight, fever, night sweats
Show all 15Show fewer
  • Have a low red blood cell count (anemia - hemoglobin < 10 g/dL)
  • Have a high white blood cell count (history of white blood cell count > 25,000/uL)
  • Have high circulating blasts (> or = 1%) as measured by blood tests
  • Should have circulating blasts <10% (as measured by blood tests)
  • Should be capable of self-care
  • Should have adequate bone marrow reserve
  • Should not have the option of stem cell transplantation
  • Should discontinue any prior or ongoing treatment for myelofibrosis prior to entering the study
  • Had no prior treatment with another JAK inhibitor

Who cannot join

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

Trial locations and contacts

26 sites in 4 countries.

26 sites in 4 countries

China10 sites

  • Novartis Investigative Site

    Beijing, Beijing Municipality, 100730

  • Novartis Investigative Site

    Chengdu, Sichuan, 610041

  • Novartis Investigative Site

    Guangzhou, Guangdong, 51000

  • Novartis Investigative Site

    Hangzhou, Zhejiang, 310003

  • Novartis Investigative Site

    Jinan, 250012

  • Novartis Investigative Site

    Nanjing, Jiangsu, 210029

  • Novartis Investigative Site

    Shanghai, 200025

  • Novartis Investigative Site

    Suzhou, Jiangsu, 215006

  • Novartis Investigative Site

    Tianjin, Tianjin Municipality, 300020

  • Novartis Investigative Site

    Wuhan, Hubei, 430030

Other trials in China

Japan9 sites

  • Novartis Investigative Site

    Bunkyo Ku, Tokyo, 113 8655

  • Novartis Investigative Site

    Fukuoka, Fukuoka, 812-8582

  • Novartis Investigative Site

    Kanazawa, Ishikawa-ken, 920-8641

  • Novartis Investigative Site

    Maebashi, Gunma, 371 8511

  • Novartis Investigative Site

    Nagoya, Aichi-ken, 466 8560

  • Novartis Investigative Site

    Shinjuku-ku, Tokyo, 160 8582

  • Novartis Investigative Site

    Shinjuku-ku, Tokyo, 160-0023

  • Novartis Investigative Site

    Suita, Osaka, 565 0871

  • Novartis Investigative Site

    Tsu, Mie-ken, 514-8507

Other trials in Japan

South Korea4 sites

  • Novartis Investigative Site

    Seoul, Korea, 06351

  • Novartis Investigative Site

    Seoul, Seocho Gu, 06591

  • Novartis Investigative Site

    Seoul, 03080

  • Novartis Investigative Site

    Seoul, 03722

Other trials in South Korea

Taiwan3 sites

  • Novartis Investigative Site

    Kaohsiung City, 833

  • Novartis Investigative Site

    Taipei, 10048

  • Novartis Investigative Site

    Taoyuan, 333

Other trials in Taiwan

About this study

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

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.

Being tested
Ruxolitinib
Keywords
Primary Myelofibrosis (MF), Post-Polycythemia Vera (PV) MF, Post-Essential Thrombocythemia (ET) MF, post-PV MF, post-ET MF, INC424, Ruxolitinib, Myelofibrosis, MF
Medical subject headings
Primary Myelofibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RuxolitinibDrug

    Also called INC424

    INC424 Tablet for oral use, provided in 5 mg bottles. The dosage strength was 5 mg/tablet INC424 phosphate (free base equivalent).

Study groups in NCT01392443
Study groupRoleWhat they receive
RuxolitinibExperimentalRuxolitinib was taken twice daily, unless instructed. Starting dose 15 mg BID for patients with baseline platelet count of 100,000/μL to 200,000/μL (inclusive) or 20 mg BID for those with baseline platelet count >200,000/μL (approximately 12 hours apart: morning and night), increased or decreased per standardized dosing paradigm.
Receives: Drug: Ruxolitinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Week 24

    The primary measure of spleen size was by MRI. MRIs were performed with a body coil because the objective was to measure organ volume only, not to assess for lesions. MRIs were performed by local radiologists who were instructed not to provide a quantitative measure of spleen volume, but could provide a qualitative assessment such as enlarged, smaller, larger, etc. The scans from an individual patient were to be read by a central reader upon transfer from the site radiologist.

    Measured: 24 weeks

Secondary outcomes (4)

  1. Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Each Scheduled Time Point - Best Response

    The best response rate was defined as the proportion of patients achieving a ≥ 35% reduction in spleen volume from baseline at any post-baseline assessment. The best response rate was estimated with an associated 95% confidence interval.

    Measured: Weeks 24, 36, 48, 72, 96, 120, 144, 168, 192, 216, 240, at any time point

  2. Kaplan Meier Estimates of Duration of Response of at Least ≥ 35% Reduction From Baseline in Spleen Volume Per Kaplan Meier Estimates

    For patients who had at least one ≥ 35% reduction in spleen volume from baseline at postbaseline, the duration of response was calculated. The start date of the duration was defined as the first spleen volume measurement that was ≥ 35% reduction from baseline, and the end date of the response duration was defined as the earliest of the following: death, A ≥ 25% increase in spleen volume by MRI (or CT in applicable patients) compared to baseline, Splenic irradiation, Leukemic transformation as defined by a bone marrow or a peripheral blood blast count of ≥ 20%, Splenectomy. Duration of response is calculated only for participants who achieved at least one measured >= 35% reduction in spleen volume at any time.

    Measured: Weeks 12, 24, 36, 48, 60, 72, 84, 96, 120, 144, 168, 192, 216, 240

  3. Change in EORTC QLQ-C30 Scores From Baseline in at Week 24

    Patient reported outcomes regarding the impact of MF on patients were assessed using the EORTC QLQ-C30. Data from the EORTC QLQ-C30 questionnaire was analyzed using the standardized scores. There were 2 categories to this scale: Functional/QOL scale and Symptom and Other items scale. For each sub-scale, the raw scores were standardized in order to obtain scores ranging from 0 to 100. For Functional/QOL subscales: a higher score represents a higher/better level of functioning. For Symptoms and Other items subscales: a higher score represents worse level of symptoms. The absolute change from baseline was calculated for each scale and summarized descriptively by scheduled visit.

    Measured: Baseline, Week 24

  4. Change in Total Symptom Score From Baseline at Week 24 as Measured by Seven-day Modified MFSAF v2.0

    The Seven-day modified MFSAF v2.0 is a 7-item PRO instrument based on the modified MFSAF v2.0 diary administered at specified visits. Symptoms of myelofibrosis (MF) were assessed using this instrument \& included filling up quickly/early satiety, abdominal discomfort, pain under the ribs, night sweats, itching, bone/muscle pain \& inactivity. The first 6 items assessed MF symptom severity at its worst as recalled \& the seventh captured MF-related inactivity in the 7 days prior to the clinic visit assessment. All 7 items asked subjects to record their answers on an 11-point numeric rating scale (NRS) (0=Absent, 10=Worst Imaginable). The first 6 items of the instrument focus on MF symptoms \& are summed to create a Total Symptom score, defined as the sum of the 6 individual symptom scores other than the inactivity score (each with 0-10 point scale) collected on the same week. The total symptom scale ranges from 0 -60 where higher score indicates a worse level of the condition.

    Measured: Baseline, Week 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Jul 2011 (estimated)
  2. Study start14 Oct 2010
  3. Primary completion31 Oct 2017
  4. Study completion31 Oct 2017

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
120 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01392443
Sponsor's protocol ID
CINC424A2202
Status last verified
Aug 2019
First submitted
11 Jul 2011
Last updated
3 Sep 2019
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Publications

  • 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.

    PubMed 27801315

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ruxolitinib in people with Primary Myelofibrosis (MF) and Post-Polycythemia Vera (PV) MF, run by Novartis Pharmaceuticals at 26 sites in 4 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 15 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 3 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 26 sites in China, Japan, South Korea, Taiwan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 3 Sep 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Participants With at Least 35% Reduction in Spleen Volume From Baseline at Week 24 (time frame: 24 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01392443, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2019.

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