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Hematopoietic NeoplasmPhase 2NCT01420783

Study With SAR302503 in Patients With Polycythemia Vera or Essential Thrombocythemia

A Phase 2 study testing SAR302503 in people with Hematopoietic Neoplasm, run by Bristol-Myers Squibb at 35 sites in 9 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

Primary Objective: Dose Ranging Phase: To evaluate the efficacy of daily oral doses of 100, 200, and 400 mg SAR302503 in patients with PV and ET who are resistant or intolerant to hydroxyurea (per European LeukemiaNet criteria) for : Inducing absence of phlebotomy and a hematocrit below 45% for a minimum of 3 months… 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
81 participants
Where
35 sitesin 9 countries
Study dates
Oct 2011to May 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

  • Has had a diagnosis of hydroxyurea resistant or intolerant polycythemia vera (PV) or essential thrombocythemia (ET) documented at Screening.
  • Polycythemia vera or essential thrombocythemia defined according to the revised WHO criteria.
  • Polycythemia vera resistance or intolerance to hydroxyurea is defined as polycythemia vera patients on hydroxyurea with a hematocrit >45%, or phlebotomy twice in the last 6 months and at least once in the last 3 months.
  • Essential thrombocythemia resistance or intolerance to hydroxurea is defined as essential thrombocythemia patients on HU with platelet count >600 x 10x9/L.
  • Dose Expansion Phase (polycythemia vera) and 600 mg/day group (essential thrombocythemia):
  • Has had a diagnosis of polycythemia vera or essential thrombocythemia according to the revised WHO 2008 criteria.
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  • PV patients must be resistant or intolerant to hydroxyurea.
  • ET patients must be resistant or intolerant to hydroxyurea.
  • Provide written informed consent to participate.

Who cannot join

  • Less than 18 years of age.
  • Participation in any study of an investigational agent (drug, biologic, device) within 30 days prior to initiation of study drug, unless during non-treatment phase. (Prior treatment with another JAK2 inhibitor is allowed.)
  • Unwilling to comply with scheduled visits, treatment plans, laboratory assessments, and other study-related procedures.
  • Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 3 or 4 at study entry.
  • Splenectomy.
  • Active malignancy other than polycythemia vera or essential thrombocythemia, 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.
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  • Major surgery within 28 days or radiation within 3 months prior to initiation of study drug.
  • Active acute infection requiring antibiotics.
  • Known human immunodeficiency virus or acquired immunodeficiency syndrome-related illness.
  • Uncontrolled congestive heart failure (New York Heart Association Classification 3 or 4), angina, myocardial infarction, cerebrovascular accident, coronary/peripheral artery bypass surgery, transient ischemic attack, or pulmonary embolism within 3 months prior to initiation of study drug.
  • 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.
  • Inadequate organ function.
  • 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]).
  • Concomitant treatment with or use of drugs or herbal agents known to be at least moderate inhibitors or inducers cytochrome P450 3A4 (CYP3A4).
  • Presence of any gastric or other disorder that would inhibit absorption of oral medication.
  • Known hypersensitivity to any excipients in the study drug formulation.
  • 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.
  • 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

35 sites in 9 countries.

35 sites in 9 countries

United States8 sites
  • Investigational Site Number 840010

    Ann Arbor, Michigan, 48109-0759

  • Investigational Site Number 840001

    Houston, Texas, 77030

  • Investigational Site Number 840004

    La Jolla, California, 92093

  • Investigational Site Number 840005

    Los Angeles, California, 90033

  • Investigational Site Number 840011

    Palo Alto, California, 94301

  • Investigational Site Number 840007

    Rochester, Minnesota, 55905

  • Investigational Site Number 840008

    Scottsdale, Arizona, 85259-5499

  • Investigational Site Number 840003

    St Louis, Missouri, 63110

Other trials in United States

Australia4 sites
  • Investigational Site Number 036001

    Clayton, 3168

  • Investigational Site Number 036002

    Kingswood, 2747

  • Investigational Site Number 036004

    Kogarah, 2217

  • Investigational Site Number 036003

    Randwick, 2031

Other trials in Australia

South Korea4 sites
  • Investigational Site Number 410001

    Seongnam, 463-707

  • Investigational Site Number 410003

    Seoul, 110-744

  • Investigational Site Number 410004

    Seoul, 120-752

  • Investigational Site Number 410002

    Seoul, 135-710

Other trials in South Korea

Spain4 sites
  • Investigational Site Number 724004

    Badalona, 08916

  • Investigational Site Number 724001

    Barcelona, 08036

  • Investigational Site Number 724003

    Madrid, 28046

  • Investigational Site Number 724002

    Valencia

Other trials in Spain

United Kingdom4 sites
  • Investigational Site Number 826001

    Belfast, BT9 7AB

  • Investigational Site Number 826006

    Birmingham, B9 5SS

  • Investigational Site Number 826003

    London, SE1 7EH

  • Investigational Site Number 826004

    London, W12 0HS

Other trials in United Kingdom

Canada3 sites
  • Investigational Site Number 124002

    Montreal, H3T 1E2

  • Investigational Site Number 124003

    Toronto, M5G 2M9

  • Investigational Site Number 124001

    Vancouver, V6Z 1Y6

Other trials in Canada

France3 sites
  • Investigational Site Number 250004

    Brest, 29609

  • Investigational Site Number 250003

    Marseille, 13273

  • Investigational Site Number 250001

    Paris, 75475

Other trials in France

Italy3 sites
  • Investigational Site Number 380003

    Bologna, 40138

  • Investigational Site Number 380001

    Florence, 50134

  • Investigational Site Number 380004

    Orbassano, 10043

Other trials in Italy

Germany2 sites
  • Investigational Site Number 276004

    Frankfurt am Main, 60590

  • Investigational Site Number 276003

    Mannheim, 68167

Other trials in Germany

About this study

Official title: A Randomized Phase II, Open-Label Study of the Efficacy and Safety of Orally Administered SAR302503 in Patients With Polycythemia Vera (PV) or Essential Thrombocythemia (ET) Who Are Resistant or Intolerant to Hydroxyurea

Primary Objective:

  • Dose Ranging Phase: To evaluate the efficacy of daily oral doses of 100, 200, and 400 mg SAR302503 in patients with PV and ET who are resistant or intolerant to hydroxyurea (per European LeukemiaNet criteria) for :
  • Inducing absence of phlebotomy and a hematocrit below 45% for a minimum of 3 months in patients with polycythemia vera, and
  • Reduction of platelet count to ≤400 x 10x9/L for a minimum of 3 months in patients with essential thrombocythemia.
  • PV Dose Expansion Phase and ET Dose Ranging Phase (only 600 mg dose group): To evaluate the efficacy of daily oral SAR302503 in patients with PV and ET who are resistant or intolerant to hydroxyurea (per European LeukemiaNet criteria) for:
  • Inducing absence of phlebotomy eligibility beginning at Day 1 of Cycle 4 visit and continuing through Day 1 of Cycle 6 visit in patients with PV, and
  • Reduction of platelet count to ≤400 x 10x9/L beginning at Day 1 of Cycle 4 visit and continuing through Day 1 of Cycle 6 visit in patients with ET.
Read more

Secondary Objectives:

  • To evaluate the safety of SAR302503.
  • To evaluate the efficacy of SAR302503 in patients with PV who are resistant or intolerant to hydroxyurea for inducing absence of phlebotomy eligibility.
  • To evaluate the efficacy of SAR302503 in patients with ET who are resistant or intolerant to hydroxyurea for reduction of platelet counts.
  • To evaluate the efficacy of SAR302503 in inducing complete and partial responses beginning at Day 1 of Cycle 6 visit through Cycle 8.
  • To evaluate splenic response as measured by spleen volume using MRI or CT.
  • To evaluate the pharmacokinetics of SAR302503 after single and repeat doses.
  • To evaluate the pharmacodynamics of SAR302503 as measured by changes in JAK2V617F allele burden in patients with JAK2V617F mutation, and STAT3 phosphorylation inhibition.
  • To measure improvement in baseline myeloproliferative neoplasm (MPN)-associated symptoms, as well as overall impact on quality of life.
  • To measure generic health-related quality of life and utility value using the EuroQol Group (EQ-5DTM) questionnaire.
Read the detailed description

The duration of the study for an individual patient is at least 40 weeks and will include a period to assess eligibility (screening period) of up to 4 weeks (28 days), a treatment period of up to 8, 28-day cycles (32 weeks), and a follow-up visit 30 days following the last administration of study drug. Treatment may continue if the patient is deriving benefit and does not experience disease progression, unacceptable toxicity, or meet other study withdrawal criteria.

Per Protocol Amendment No. 5, accrual of patients with essential thrombocythemia is closed.

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 groups in NCT01420783
Study groupRoleWhat they receive
SAR302503 100 mgExperimentalonce daily X 28 days
Receives: Drug: SAR302503
SAR302503 200 mgExperimentalonce daily X 28 days
Receives: Drug: SAR302503
SAR302503 400 mgExperimentalonce daily X 28 days
Receives: Drug: SAR302503
SAR302503 600 mgExperimentalonce daily X 28 days
Receives: Drug: SAR302503

What is being measured

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

  1. Dose Ranging Phase: Proportion of PV patients with absence of phlebotomy and hematocrit below 45% and proportion of ET patients with a platelet count ≤ 400 x 10x9/L for a minimum of 3 months during the first 8 cycles of therapy.

    Measured: 2 years

  2. PV Dose Expansion Phase: Proportion of PV patients with absence of phlebotomy eligibility beginning at Day 1 of Cycle 4 visit and continuing through Day 1 of Cycle 6 visit.

    Measured: 2 years

  3. ET Dose Ranging Phase (only 600 mg dose group): Proportion of ET patients with a platelet count ≤400 x 10x9/L beginning at Day 1 of Cycle 4 visit and continuing through Day 1 of Cycle 6 visit.

    Measured: 2 years

Secondary outcomes (11)
  1. Proportion of PV patients with absence of phlebotomy eligibility beginning at Day 1 of Cycle 4 visit through Cycle 8 (for PV dose expansion phase only).

    Measured: 2 years

  2. Proportion of ET patients with platelet count ≤400 x 10x9/L beginning at Day 1 of Cycle 4 visit and continuing through Cycle 8.

    Measured: 2 years

  3. Characterization of clinicohematologic response (CR, PR, and no Response) defined by European LeukemiaNet beginning at Day 1 of Cycle 6 visit through Cycle 8.

    Measured: 2 years

  4. Percent change in spleen volume (per Magnetic Resonance Imaging (MRI)) at the end of Cycles 4 and 8 or end of treatment (EOT) relative to baseline.

    Measured: 2 years

  5. Proportion of patients with a ≥ 35% reduction in spleen volume (per Magnetic Resonance Imaging (MRI)) at the end of Cycles 4 and 8 or end of treatment (EOT) relative to baseline.

    Measured: 2 years

  6. Number of participants who have changes in histological, cytogenetic, and molecular responses in bone marrow.

    Measured: 2 years

  7. Response (defined as either a 2-point improvement in or resolution of a symptom present at baseline) at the end of Cycles 1, 4, and 8 or end of treatment (EOT), as measured by the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF).

    Measured: 2 years

  8. Cumulative Distribution Function of responses between treatment groups at the end of Cycles 1, 4, and 8 or EOT on the MPN-SAF.

    Measured: 2 years

  9. For each MPN-associated symptom present at baseline on the MPN-SAF, proportion of patients with resolution of that symptom at the end of Cycles 1, 4, and 8.

    Measured: 2 years

  10. To measure generic health-related quality of life and utility values using the EQ-5D questionnaire after completion of 8 cycles of therapy.

    Measured: 2 years

  11. Characterization of the safety profile of SAR302503, including the frequency, duration, and severity of adverse events graded using the National Cancer Institute (NCI) - CTCAE version 4.03, clinical laboratory parameters, ECG, and vital signs.

    Measured: 2 years

Timeline

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

  1. First posted22 Aug 2011 (estimated)
  2. Study startOct 2011
  3. Primary completionNov 2012
  4. Study completionMay 2014

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
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
81 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01420783
Sponsor's protocol ID
ARD12042
Other IDs
2011-001847-58, U1111-1121-4203
Status last verified
Feb 2016
First submitted
11 Aug 2011
Last updated
5 Mar 2025
Results posted
No
Data monitoring committee
No

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 35 sites in 9 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 9 inclusion and 20 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 35 sites in United States, Australia, South Korea, Spain, United Kingdom and 4 more countries. 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?

Dose Ranging Phase: Proportion of PV patients with absence of phlebotomy and hematocrit below 45% and proportion of ET patients with a platelet count ≤ 400 x 10x9/L for a minimum of 3 months during the first 8 cycles of therapy. (time frame: 2 years). The study lists 3 primary outcomes in all. 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.

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