Hematopoietic NeoplasmPhase 2NCT01420770
Phase 2 Study of SAR302503 in Patients With Myelofibrosis
A Phase 2 study testing SAR302503 in people with Hematopoietic Neoplasm, run by Bristol-Myers Squibb at 4 sites in United States.
Trial summary
Primary Objective: To evaluate the efficacy of daily oral doses of 300 mg, 400 mg, and 500 mg SAR302503 for the reduction of spleen volume as determined by magnetic resonance imaging (MRI). Secondary Objectives: To evaluate the safety of SAR302503. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematopoietic Neoplasm
- 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
- 30 participants
- Where
- 4 sitesUnited States
- Study dates
- Aug 2011to Apr 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 18 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 (Post-ET MF) according to the 2008 World Health Organization (WHO) criteria
- Myelofibrosis classified as high-risk or intermediate-risk level 2, as defined by International Working Group - Myelofibrosis Research and Treatment (IWG-MRT)
- Enlarged spleen, palpable at least 5 cm below costal margin
- At least 18 years of age.
- Eastern Collaborative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2 at study entry.
- Adequate organ function
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- Absence of active malignancy other than MF, except adequately treated basal cell carcinoma and squamous cell carcinoma of the skin, cervical carcinoma in situ or other malignancies that have been stable and off therapy for 5 years.
- Written informed consent to participate.
- Willing to comply with scheduled visits, treatment plans, laboratory assessments, and other study-related procedures.
Who cannot join
- Splenectomy.
- Any 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; darbepoetin use within 28 days prior to initiation of study drug.
- Major surgery therapy within 28 days or radiation within 6 months prior to initiation of study drug.
- Concomitant treatment with or use of pharmaceutical or herbal agents known to be at least moderate inhibitors or inducers Cytochrome P450 3A4 (CYP3A4), unless approved by the sponsor.
- 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 Janus kinase 2 (JAK 2) Inhibitor,
- Contraindications for undergoing Magnetic resonance imaging (MRI) (eg. metal implants).
- Pregnant or lactating female.
- Women of childbearing potential, unless using effective contraception while on study drug.
- Men who partner with a woman of childbearing potential, unless they agree to use effective contraception while on study drug.
- Known human immunodeficiency virus or acquired immunodeficiency syndrome-related illness.
- Clinically active hepatitis B or C.
- Any severe acute or chronic medical, neurological, or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or study drug administration, may interfere with the informed consent process and/or with compliance with the requirements of the study, or may interfere with interpretation of study results and, in the Investigator's opinion, would make the patient inappropriate for entry into this study.
- Unable to swallow capsules
- Presence of any gastric or other disorder that would inhibit absorption of oral medication.
- 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
4 sites in 1 country.
United States4 sites
Investigational Site Number 840003
Ann Arbor, Michigan, 48109-0759
Investigational Site Number 840007
Canton, Ohio, 44718
Investigational Site Number 840006
Rochester, Minnesota, 55905
Investigational Site Number 840001
San Francisco, California, 94143
About this study
Official title: A Phase 2 Randomized, Open-Label, Dose-Ranging Study of the Efficacy and Safety of Orally Administered SAR302503 in 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 for the reduction of spleen volume as determined by magnetic resonance imaging (MRI).
Read more
Secondary Objectives:
- To evaluate the safety of SAR302503.
- To evaluate the pharmacokinetics (PK) of SAR302503 after single and repeat doses.
- To evaluate the pharmacodynamics of SAR302503 as measured by changes in JAK2V617F allele burden in those patients with JAK2V617F mutation, changes in substrate phosphorylation in the JAK-STAT signal transduction pathway, and the expression of cytokines.
- To measure improvement in baseline Myeloproliferative Neoplasm (MPN) associated symptoms, as well as overall impact in quality of life (QOL), through serial administration of the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF).
- To measure generic health-related quality of life (HRQL) and utility values using the EQ-5D questionnaire.
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.
The study duration will be approximately 16 months which includes a 3-month enrollment period followed by a 12-month treatment period following the last patient enrolled followed by a 30-day follow-up period. The cut-off date for the analysis of the primary endpoint of response will be in maximum at the end of 3 months after the date of first dose of study drug of the last treated patient. The final analysis will be performed after the last enrolled patient completes the Cycle12 assessment.
- Conditions
- Hematopoietic Neoplasm
- Being tested
- SAR302503
- Medical subject headings
- Hematologic Neoplasms
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 |
|---|---|---|
| SAR02503 300 mg qd | Experimental | daily X 28 days Receives: Drug: SAR302503 |
| SAR302503 400 mg qd | Experimental | daily X 28 days Receives: Drug: SAR302503 |
| SAR302503 500 mg qd | Experimental | daily X 28 days Receives: Drug: SAR302503 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The percent change in spleen volume based on MRI at the end of Cycle 3 relative to baseline
Measured: 1 year
Secondary outcomes (7)
The proportion of patients who achieve ≥35% reduction in spleen volume from baseline, to Cycle 6 end of cycle (EOC)
Measured: 1 year
The percent change in spleen volume based on MRI at Cycle 6 EOC compared to baseline
Measured: 1 year
Duration of maintenance of ≥35% reduction in spleen volume relative to baseline, as measured at Cycle 3 EOC, at Cycle 6 EOC, after a year, after 18 months and after two years and at end of treatment (EOT).
Measured: 1 year
Characterization of the safety profile of SAR302503, including the frequency, duration, and severity of adverse events (AEs) 2 years
Measured: 1 year
Area under the plasma concentration versus time curve (AUC) of SAR302503
Measured: 1 year
Peak plasma concentration (CMax) of SAR302503
Measured: 1 year
In patients with the JAK2V617F mutation, change in peripheral blood granulocyte JAK2V617F allele burden from baseline to Cycle 3 EOC, to Cycle 6 EOC and at the end of every third cycle thereafter until Cycle 12 EOC and EOT
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Aug 2011 (estimated)
- Study startAug 2011
- Primary completionApr 2014
- Study completionApr 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematopoietic NeoplasmStudies 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
- 30 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
- NCT01420770
- Sponsor's protocol ID
- ARD11936
- Other IDs
- U1111-1119-2965
- Status last verified
- Feb 2016
- First submitted
- 11 Aug 2011
- 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 Randomized, Open-Label, Dose-Ranging Study of the Efficacy and Safety of Orally Administered SAR302503 in Patients With Intermediate-2 or High Risk Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis With Splenomegaly
- ClinicalTrials.gov ID
- NCT01420770
- Acronym / sponsor ID
- ARD11936
- Conditions
- Hematopoietic Neoplasm
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Bristol-Myers SquibbIndustry (pharma, biotech, devices)
- Study start
- Aug 2011
- Primary completion
- Apr 2014 (actual)
- Study completion
- Apr 2014 (actual)
- First posted
- 22 Aug 2011 (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 for the reduction of spleen volume as determined by magnetic resonance imaging (MRI).
Secondary Objectives:
- To evaluate the safety of SAR302503.
- To evaluate the pharmacokinetics (PK) of SAR302503 after single and repeat doses.
- To evaluate the pharmacodynamics of SAR302503 as measured by changes in JAK2V617F allele burden in those patients with JAK2V617F mutation, changes in substrate phosphorylation in the JAK-STAT signal transduction pathway, and the expression of cytokines.
- To measure improvement in baseline Myeloproliferative Neoplasm (MPN) associated symptoms, as well as overall impact in quality of life (QOL), through serial administration of the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF).
- To measure generic health-related quality of life (HRQL) and utility values using the EQ-5D questionnaire.
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.
The study duration will be approximately 16 months which includes a 3-month enrollment period followed by a 12-month treatment period following the last patient enrolled followed by a 30-day follow-up period. The cut-off date for the analysis of the primary endpoint of response will be in maximum at the end of 3 months after the date of first dose of study drug of the last treated patient. The final analysis will be performed after the last enrolled patient completes the Cycle12 assessment.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| SAR02503 300 mg qd | Experimental | Drug: SAR302503daily X 28 days |
| SAR302503 400 mg qd | Experimental | Drug: SAR302503daily X 28 days |
| SAR302503 500 mg qd | Experimental | Drug: SAR302503daily X 28 days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Diagnosis of primary or post-polycythemia vera or post-essential thrombocythemia myelofibrosis (Post-ET MF) according to the 2008 World Health Organization (WHO) criteria
- Myelofibrosis classified as high-risk or intermediate-risk level 2, as defined by International Working Group - Myelofibrosis Research and Treatment (IWG-MRT)
- Enlarged spleen, palpable at least 5 cm below costal margin
- At least 18 years of age.
- Eastern Collaborative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2 at study entry.
Key exclusion criteria (18 listed)
- Splenectomy.
- Any 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; darbepoetin use within 28 days prior to initiation of study drug.
- Major surgery therapy within 28 days or radiation within 6 months prior to initiation of study drug.
- Concomitant treatment with or use of pharmaceutical or herbal agents known to be at least moderate inhibitors or inducers Cytochrome P450 3A4 (CYP3A4), unless approved by the sponsor.
- 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 | SAR02503 300 mg qd, SAR302503 400 mg qd, SAR302503 500 mg qd |
Primary outcome
The percent change in spleen volume based on MRI at the end of Cycle 3 relative to baseline
Time frame: 1 year
7 secondary outcomes
The proportion of patients who achieve ≥35% reduction in spleen volume from baseline, to Cycle 6 end of cycle (EOC)
Time frame: 1 year
The percent change in spleen volume based on MRI at Cycle 6 EOC compared to baseline
Time frame: 1 year
Duration of maintenance of ≥35% reduction in spleen volume relative to baseline, as measured at Cycle 3 EOC, at Cycle 6 EOC, after a year, after 18 months and after two years and at end of treatment (EOT).
Time frame: 1 year
Characterization of the safety profile of SAR302503, including the frequency, duration, and severity of adverse events (AEs) 2 years
Time frame: 1 year
Area under the plasma concentration versus time curve (AUC) of SAR302503
Time frame: 1 year
Peak plasma concentration (CMax) of SAR302503
Time frame: 1 year
In patients with the JAK2V617F mutation, change in peripheral blood granulocyte JAK2V617F allele burden from baseline to Cycle 3 EOC, to Cycle 6 EOC and at the end of every third cycle thereafter until Cycle 12 EOC and EOT
Time frame: 1 year
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
4 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 Hematopoietic Neoplasm, run by Bristol-Myers Squibb at 4 sites in United States.
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 9 inclusion and 18 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 4 sites in United States. 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?
The percent change in spleen volume based on MRI at the end of Cycle 3 relative to baseline (time frame: 1 year). 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.