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Essential ThrombocythemiaPhase 2/3NCT02962388

The Ruxolitinib Versus Best Available Therapy Trial in Patients With High Risk ET in Second Line

A Phase 2/3 study testing Anagrelide, Ruxolitinib (JAKAVI®) and IFNα/ PegIFNα in people with Essential Thrombocythemia, run by French Innovative Leukemia Organisation at 1 site in France.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

Prospective national multicenter randomized open label phase IIb RUXBETA trial. Read more about this study

Who is it for?
Ages 18 to 74 years
Conditions the study is for
Essential Thrombocythemia
What is being tested?
Anagrelide, Ruxolitinib (JAKAVI®) and IFNα/ PegIFNα
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
13 participants
Where
1 siteFrance
Study dates
3 Jan 2017to 28 Jun 2021
Phase
Phase 2/3
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 to 74 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Target Population
  • Men and women, age more than or equal18 years and less than 75 years.
  • Confirmed diagnosis of Essential Thrombocythemia for at least 6 months, according to the 2008 WHO criteria, with a high-risk status.
  • Patients must have a treatment history for ET that meet the definition of resistance or intolerance to hydroxyurea therapy according to the ELN criteria as follow:
  • Platelets more than 600.0109/L after 3 months (12 weeks) of treatment at a dose over 2g/day.
  • Platelets more than 400.0 109/L and WBC less than 2.5109/L, whatever the dose of HU.
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  • Platelets more than 400.0 109/L and Hb less than 10g/dl whatever the dose of HU.
  • Leg ulcers or other unacceptable muco-cutaneous toxicity.
  • HU-related fever.
  • ECOG Performance Status (ECOG PS) less than or equal 2 at screening and at baseline.
  • Adequate Organ Function:
  • Direct bilirubin less than 2.0 times the institutional Upper Limit of Normal (ULN).
  • Hepatic enzymes (AST, ALT) less than or equal 2.5 times the institutional ULN.
  • Adequate renal function at screening as demonstrated by MDRD-eGFR more than 30 mL/min/1.73m2.
  • Women of childbearing potential (WOCBP) must be using an adequate method of contraception to avoid pregnancy during and after the study.
  • A male subject of fathering potential must use an adequate method of contraception to avoid conception during and after the study to minimize the risk of pregnancy.
  • For females and males, these restrictions apply for 24 hours after the last dose of study drug.
  • Women of childbearing potential must have a negative serum (beta-human chorionic gonadotropin hCG pregnancy test at Screening.
  • Signed Written Informed Consent.
  • Health insurance coverage.

Who cannot join

  • Patients with thrombocytosis related to another MPN than ET
  • Patients previously treated with a JAK2 inhibitor, Anagrelide or Interferon-alpha and prior history of therapy other than Hydroxyurea
  • Contraindication to Ruxolitinib, Anagrelide or Interferon-alpha (if no eligible for anagrelide), hypersensitivity to an excipient
  • Medical history and concurrent diseases:
  • Clinically significant cardiac disease (NYHA Class III or IV).
  • Chronic hepatocellular disease.
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  • Subjects with impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of Ruxolitinib
  • Subjects with clinically significant bacterial, fungal, parasitic or viral infection which requires therapy:
  • Subjects with acute bacterial infections requiring antibiotic use should delay screening/enrolment until the course of antibiotic therapy has been completed.
  • Subjects with active hepatitis A, B or C or with HIV positivity at screening.
  • Subjects with diagnosed primary immunodeficiency syndromes such as X-Linked a gammaglobulinemia and common variable immune deficiency.
  • Subject with medical history of tuberculosis
  • History of progressive multifocal leucoencephalopathy (PML).
  • Other malignant disease during the last 5 years prior to the inclusion except treated cervical intraepithelial neoplasia, basal cell carcinoma of the skin, or squamous cell carcinoma of the skin, with no evidence for recurrence in the past 3 years.
  • History of significant bleeding disorder not related to the ET.
  • Diagnosed congenital bleeding disorders,
  • Diagnosed acquired bleeding disorder within one year (e.g. acquired anti-factor VIII antibodies),
  • Ongoing or recent (3 months) significant gastrointestinal bleeding.
  • Subjects with an uncontrolled undercurrent illness or any concurrent condition that, in the investigator's opinion, would jeopardize the safety of the subject or compliance with the protocol.
  • Subjects being treated concurrently with a potent systemic inhibitor of CYP3A4 at the time of screening.
  • Subjects being treated concurrently with any prohibited medications.
  • Women who are pregnant or breastfeeding are not eligible for this study.
  • Inability to freely provide consent through judiciary or administrative condition.
  • Ongoing participation to another clinical investigational study.

Trial locations and contacts

1 site in 1 country.

France1 site

  • FILO

    Tours, 37044

Other trials in France

About this study

Official title: A Randomized, Multicenter Phase IIb Study to Evaluate the Efficacy and Safety of Ruxolitinib Versus Best Available Therapy in Patients With High Risk Essential Thrombocythemia, Who Are Resistant or Intolerant to Hydroxyurea: A FIM Study

Prospective national multicenter randomized open label phase IIb RUXBETA trial.

Read the detailed description

A randomized, open label, multicenter phase IIb study to evaluate the efficacy and safety of Ruxolitinib versus best available therapy in patients with high risk essential thrombocythemia, who are resistant or intolerant to hydroxyurea.

Being tested
Anagrelide, Ruxolitinib (JAKAVI®), IFNα/ PegIFNα
Keywords
efficacy, safety
Medical subject headings
Thrombocythemia, Essential

What participants receive

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

  • AnagrelideDrug

    Also called ARM A

    Anagrelide in the study, according to the investigator decision fom day 1 to 48 months

  • Ruxolitinib (JAKAVI®)Drug

    Also called ARM B

    Ruxolitinib (JAKAVI®) - Novartis. Tablets 5 mg. Starting dose 10 mg BID, orally. To be increased or decreased (5 or 10 mg steps) per standardized dosing Maximum dose 25 mg BID. fom day 1 to 48 months

  • IFNα/ PegIFNαDrug

    Also called ARM A

    IFNα/ PegIFNα in the study, according to the investigator decision fom day 1 to 48 months

Study groups in NCT02962388
Study groupRoleWhat they receive
Reference therapy armActive comparatorBest Available Therapy (BAT) in second line, after hydroxyurea. BAT restricted to anagrelide or IFNα/ PegIFNα in the study, according to the investigator decision
Receives: Drug: Anagrelide; Drug: IFNα/ PegIFNα
Investigational therapy armExperimentalRuxolitinib JAKAVI® Starting dose 10 mg BID, orally. To be increased or decreased (5 or 10 mg steps) per standardized dosing paradigm. Maximum dose 25 mg BID.
Receives: Drug: Ruxolitinib (JAKAVI®)

What is being measured

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

  1. Failure-free patients

    Failure is defined by the occurrence of either intolerance and/or resistance to the second line therapy according to the protocol criteria

    Measured: month 12

Secondary outcomes (5)

  1. Complete hematologic response

    Number of Participants With normal Laboratory Values

    Measured: 48 months

  2. AE/SAE

    Rates, types and grades of AE/SAE related to the therapy, according to the NCI-CTCAE v4.0 classification

    Measured: 48 months

  3. Median dose

    Median dose of the treatment received

    Measured: 48 months

  4. Thrombotic and hemorrhagic events

    Cumulative incidence of thrombotic and hemorrhagic events incidence of progression into PV, secondary MF and MDS/acute leukemia

    Measured: 48 months

  5. Quality of life questionnaire

    Quality of life

    Measured: 48 months

Timeline

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

  1. First posted11 Nov 2016 (estimated)
  2. Study start3 Jan 2017
  3. Primary completion28 Jun 2021
  4. Study completion28 Jun 2021

Why it stopped: not enough recruitment

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 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
13 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02962388
Sponsor's protocol ID
RUXBETA trial
Status last verified
Jun 2021
First submitted
5 Oct 2016
Last updated
29 Jun 2021
Results posted
No
Data monitoring committee
Yes
Individual data sharing
Yes: e crf

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Anagrelide, Ruxolitinib (JAKAVI®) and IFNα/ PegIFNα in people with Essential Thrombocythemia, run by French Innovative Leukemia Organisation at 1 site in France.

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 20 inclusion and 24 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: stopped early, as of the sponsor's last update on 29 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in France. 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 combined 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.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 was stopped early (reason given: not enough recruitment). Results are not available in the source record.

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?

Failure-free patients (time frame: month 12). 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 NCT02962388.

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 29 Jun 2021; AzTrials last refreshed its copy on 8 Nov 2026.

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