MyelofibrosisPhase 2NCT01692366
Phase 2 Study in Japanese Patients With Intermediate-2 or High Risk Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis With Splenomegaly
A Phase 2 study testing SAR302503 in people with Myelofibrosis, run by Bristol-Myers Squibb at 7 sites in Japan.
Trial summary
Primary Objective: To evaluate the efficacy of daily oral doses of 300 mg, 400 mg, and 500 mg SAR302503 and combined for the response rate defined with the ≥35% reduction of spleen volume as determined by magnetic resonance imaging (MRI or computed tomography scan [CT] in patients with contraindications for MRI). Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Myelofibrosis
- What is being tested?
- SAR302503
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 7 sitesJapan
- Study dates
- Nov 2012to Mar 2014
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of primary or post-polycythemia vera or post-essential thrombocythemia myelofibrosis
- Myelofibrosis classified as high-risk or intermediate-risk level 2
- Enlarged spleen, palpable at least 5 cm below costal margin
- Active symptoms of myelofibrosis
- At least 20 years of age
- Eastern Collaborative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2 at study entry
- Absence of active malignancy other than myelofibrosis
- Written informed consent to participate.
Who cannot join
- Splenectomy.
- Any recent chemotherapy (eg, hydroxyurea), immunomodulatory drug therapy (eg, thalidomide, interferon-alpha), immunosuppressive therapy, corticosteroids >10 mg/day prednisone or equivalent, or growth factor treatment (eg, erythropoietin), hormones (eg, androgens, danazol) within 14 days prior to initiation of study drug.
- Major surgery therapy within 28 days or radiation including spleen radiation within 6 months prior to initiation of study drug.
- Concomitant treatment with or use of pharmaceutical or herbal agents known to be moderate or severe inhibitors or inducers CYP3A4.
- Active acute infection requiring antibiotics.
- Uncontrolled congestive heart failure (New York Heart Association Classification 3 or 4), angina, myocardial infarction, cerebrovascular accident, coronary/peripheral artery bypass graft surgery, transient ischemic attack, or pulmonary embolism within 3 months prior to initiation of study drug.
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- Participation in any study of an investigational agent (drug, biologic, device) within 30 days, unless during nontreatment phase.
- Prior treatment with a JAK 2 Inhibitor.
- Treatment with aspirin in doses >150 mg/day
- Known human immunodeficiency virus or acquired immunodeficiency syndrome-related illness.
- Pregnant or lactating female. Once the lactating female stop and participate in the study, she cannot re-start feeding the baby.
- Women of childbearing potential, unless using effective contraception while on study drug. Otherwise patients must be post-menopausal (at least 1 years from last menstruation without other medical reason), or surgically sterile.
- Known active (acute or chronic) Hepatitis A, B, or C; and hepatitis B and C carriers.
- Prior history of chronic liver disease (eg, chronic alcoholic liver disease, autoimmune hepatitis, sclerosing cholangitis, primary biliary cirrhosis, hemachromatosis, non-alcoholic steatohepatitis [NASH])
- The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
Japan7 sites
Investigational Site Number 392010
Akita
Investigational Site Number 392002
Bunkyō City
Investigational Site Number 392006
Bunkyō City
Investigational Site Number 392004
Sendai
Investigational Site Number 392008
Shinjuku-Ku
Investigational Site Number 392009
Shinjuku-Ku
Investigational Site Number 392003
Suita-Shi
About this study
Official title: A Phase 2 Open-Label, Dose-Ranging Study of the Efficacy and Safety of Orally Administered SAR302503 in Japanese Patients With Intermediate-2 or High Risk Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis With Splenomegaly
Primary Objective:
- To evaluate the efficacy of daily oral doses of 300 mg, 400 mg, and 500 mg SAR302503 and combined for the response rate defined with the ≥35% reduction of spleen volume as determined by magnetic resonance imaging (MRI or computed tomography scan [CT] in patients with contraindications for MRI).
Read more
Secondary Objectives:
- To evaluate the safety of SAR302503 for both pooled (300, 400, and 500mg) and individual doses population.
- To evaluate the pharmacokinetics (PK) of SAR302503 after single and repeat-dose.
- To evaluate the effect on Myelofibrosis (MF)-associated symptoms (Key MF symptoms) as measured by the modified Myelofibrosis Symptom Assessment Form (MFSAF).
- To evaluate the durability of splenic response.
- To evaluate the effect of SAR302503 on bone marrow with regard to changes on reticulin fibrosis.
Read the detailed description
The duration of the study for an individual patient will include a period to assess eligibility (screening period 28 days), followed by a treatment period of at least 1 cycle (28 days) of study treatment, and an end-of-treatment visit at least 30 days following the last administration of study drug. However, treatment may continue if patients are deriving benefit and do not have unacceptable toxicity or meet study withdrawal criteria.
- Conditions
- Myelofibrosis
- Being tested
- SAR302503
- Medical subject headings
- Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SAR302503Drug
Pharmaceutical form:Capsule
Route of administration: oral
| Study group | Role | What they receive |
|---|---|---|
| SAR302503 300mg | Experimental | SAR302503 will be self-administered, orally, once daily, as a single agent, in consecutive, 28-day cycles at the dose level of 300mg. SAR302503 will be taken on an empty stomach at approximately the same time each day Receives: Drug: SAR302503 |
| SAR302503 400 mg | Experimental | SAR302503 will be self-administered, orally, once daily, as a single agent, in consecutive, 28-day cycles at the dose level of 400 mg. SAR302503 will be taken on an empty stomach at approximately the same time each day Receives: Drug: SAR302503 |
| SAR302503 500 mg | Experimental | SAR302503 will be self-administered, orally, once daily, as a single agent, in consecutive, 28-day cycles at the dose level of 500 mg. SAR302503 will be taken on an empty stomach at approximately the same time each day Receives: Drug: SAR302503 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response Rate (RR), defined as the proportion of subjects who have a ≥35% reduction as measured by MRI (or CT scan in subjects with contraindications for MRI). - Time Frame:
Measured: 24 weeks
Secondary outcomes (7)
Number of patients with Serious Adverse events using NCI CTCAE v4.03, clinical parameters and vital signs
Measured: From baseline to the 30 days after last drug administration
Measurements of SAR302503 pharmacokinetic endpoints including Cmax, Tmax, and AUC0-24
Measured: SAR302503, pre-dose and post-dose plasma collections will be obtained on Cycle 1 Day 1, Cycle 1 Day 2, Cycle 1 Day 15, Cycle 2 Day 1, Cycle 2 Day 2, and Cycle 3 Day 1
Symptom Response Rate (SRR): Proportion of subjects with a ≥50% reduction in the total symptom score using the modified MFSAF
Measured: 24 weeks
Duration of maintenance of ≥35% reduction in spleen volume
Measured: From baseline to the 30 days after last drug administration
Percent change from baseline in spleen volume measured by MRI
Measured: 24 weeks
Percent change from baseline in spleen size measured by palpation
Measured: 24 weeks
Proportion of patients with any grade reduction in reticulin fibrosis
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Sep 2012 (estimated)
- Study startNov 2012
- Primary completionMar 2014
- Study completionMar 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MyelofibrosisStudies of the same condition, open and under way.
- More trials run by Bristol-Myers SquibbThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bristol-Myers Squibb
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01692366
- Sponsor's protocol ID
- ARD12888
- Other IDs
- U1111-1130-3710
- Status last verified
- Sep 2014
- First submitted
- 10 Sep 2012
- Last updated
- 5 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Open-Label, Dose-Ranging Study of the Efficacy and Safety of Orally Administered SAR302503 in Japanese Patients With Intermediate-2 or High Risk Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis With Splenomegaly
- ClinicalTrials.gov ID
- NCT01692366
- Acronym / sponsor ID
- ARD12888
- Conditions
- Myelofibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Bristol-Myers SquibbIndustry (pharma, biotech, devices)
- Study start
- Nov 2012
- Primary completion
- Mar 2014 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 25 Sep 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Primary Objective:
- To evaluate the efficacy of daily oral doses of 300 mg, 400 mg, and 500 mg SAR302503 and combined for the response rate defined with the ≥35% reduction of spleen volume as determined by magnetic resonance imaging (MRI or computed tomography scan [CT] in patients with contraindications for MRI).
Secondary Objectives:
- To evaluate the safety of SAR302503 for both pooled (300, 400, and 500mg) and individual doses population.
- To evaluate the pharmacokinetics (PK) of SAR302503 after single and repeat-dose.
- To evaluate the effect on Myelofibrosis (MF)-associated symptoms (Key MF symptoms) as measured by the modified Myelofibrosis Symptom Assessment Form (MFSAF).
- To evaluate the durability of splenic response.
- To evaluate the effect of SAR302503 on bone marrow with regard to changes on reticulin fibrosis.
Detailed description
The duration of the study for an individual patient will include a period to assess eligibility (screening period 28 days), followed by a treatment period of at least 1 cycle (28 days) of study treatment, and an end-of-treatment visit at least 30 days following the last administration of study drug. However, treatment may continue if patients are deriving benefit and do not have unacceptable toxicity or meet study withdrawal criteria.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| SAR302503 300mg | Experimental | Drug: SAR302503SAR302503 will be self-administered, orally, once daily, as a single agent, in consecutive, 28-day cycles at the dose level of 300mg. SAR302503 will be taken on an empty stomach at approximately the same time each day |
| SAR302503 400 mg | Experimental | Drug: SAR302503SAR302503 will be self-administered, orally, once daily, as a single agent, in consecutive, 28-day cycles at the dose level of 400 mg. SAR302503 will be taken on an empty stomach at approximately the same time each day |
| SAR302503 500 mg | Experimental | Drug: SAR302503SAR302503 will be self-administered, orally, once daily, as a single agent, in consecutive, 28-day cycles at the dose level of 500 mg. SAR302503 will be taken on an empty stomach at approximately the same time each day |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Diagnosis of primary or post-polycythemia vera or post-essential thrombocythemia myelofibrosis
- Myelofibrosis classified as high-risk or intermediate-risk level 2
- Enlarged spleen, palpable at least 5 cm below costal margin
- Active symptoms of myelofibrosis
- At least 20 years of age
Key exclusion criteria (15 listed)
- Splenectomy.
- Any recent chemotherapy (eg, hydroxyurea), immunomodulatory drug therapy (eg, thalidomide, interferon-alpha), immunosuppressive therapy, corticosteroids >10 mg/day prednisone or equivalent, or growth factor treatment (eg, erythropoietin), hormones (eg, androgens, danazol) within 14 days prior to initiation of study drug.
- Major surgery therapy within 28 days or radiation including spleen radiation within 6 months prior to initiation of study drug.
- Concomitant treatment with or use of pharmaceutical or herbal agents known to be moderate or severe inhibitors or inducers CYP3A4.
- Active acute infection requiring antibiotics.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SAR302503Pharmaceutical form:Capsule Route of administration: oral | Drug | SAR302503 300mg, SAR302503 400 mg, SAR302503 500 mg |
Primary outcome
Response Rate (RR), defined as the proportion of subjects who have a ≥35% reduction as measured by MRI (or CT scan in subjects with contraindications for MRI). - Time Frame:
Time frame: 24 weeks
7 secondary outcomes
Number of patients with Serious Adverse events using NCI CTCAE v4.03, clinical parameters and vital signs
Time frame: From baseline to the 30 days after last drug administration
Measurements of SAR302503 pharmacokinetic endpoints including Cmax, Tmax, and AUC0-24
Time frame: SAR302503, pre-dose and post-dose plasma collections will be obtained on Cycle 1 Day 1, Cycle 1 Day 2, Cycle 1 Day 15, Cycle 2 Day 1, Cycle 2 Day 2, and Cycle 3 Day 1
Symptom Response Rate (SRR): Proportion of subjects with a ≥50% reduction in the total symptom score using the modified MFSAF
Time frame: 24 weeks
Duration of maintenance of ≥35% reduction in spleen volume
Time frame: From baseline to the 30 days after last drug administration
Percent change from baseline in spleen volume measured by MRI
Time frame: 24 weeks
Percent change from baseline in spleen size measured by palpation
Time frame: 24 weeks
Proportion of patients with any grade reduction in reticulin fibrosis
Time frame: 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing SAR302503 in people with Myelofibrosis, run by Bristol-Myers Squibb at 7 sites in Japan.
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 8 inclusion and 15 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 5 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Japan. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Response Rate (RR), defined as the proportion of subjects who have a ≥35% reduction as measured by MRI (or CT scan in subjects with contraindications for MRI). - Time Frame: (time frame: 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 5 Mar 2025.