AzTrials
AzTrials

MyelofibrosisNCT02469974

Ruxolitinib in Combination With Autotransplant

A study testing RUXOLITINIB / INC 424, Filgrastim and Busulfan in people with Myelofibrosis and MF, run by Marina Kremyanskaya.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
Search all trials

Trial summary

To determine the safety of the approach of giving RUXOLITINIB before and after an autologous stem cell transplant, as measured by graft failure or death. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Myelofibrosis, MF
What is being tested?
RUXOLITINIB / INC 424, Filgrastim and Busulfan
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
No sites listed yet
Study dates
May 2015to Aug 2016
Phase
Interventional
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 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Histologically documented diagnosis of MF (idiopathic or post PV/ET)
  • Age 18-75 years
  • Intermediate-2/ high-risk disease as per Dynamic IPSS (DIPSS) criteria or Intermediate-1 risk disease with one of the following features within one year from screening:
  • Red cell transfusion dependency
  • unfavorable Karyotype
  • platelet count <100 x 109/L
Show all 21Show fewer
  • symptomatic splenomegaly
  • PB blasts > 1%
  • Blasts in PB <20% prior to study enrollment
  • No available suitable matched related (6/6 or 5/6) or unrelated donor (8/8 or 7/8 allele matched) or unwilling or unable to pursue allogeneic stem cell transplant
  • WBC <50,000/ml at screening
  • Able to give informed written consent
  • ECOG Performance status of 0-2
  • Life expectancy >6 months
  • Off all myelofibrosis-related investigational or standard agents (except for ruxolitinib) for at least 4 weeks prior to study enrollment and recovered from all toxicities. If patient is already on ruxolitinib for a minimum of 16 weeks prior to study enrollment, patient can proceed to mobilization and collection
  • Adequate organ function defined as the following (*unless clearly disease related):
  • Adequate renal function - creatinine <2 x ULN
  • Adequate hepatic function - AST/ALT <3 x ULN, Total Bilirubin <3 x ULN, exception is elevated indirect bilirubin attributed to Gilbert's syndrome or hemolysis
  • Adequate hematopoietic function - Platelet ≥50 x 109/L (without transfusion) and ANC ≥1.0 x 109/L
  • LVEF >40% (MUGA or echocardiogram)
  • Adequate pulmonary function with DLCO >40%

Who cannot join

  • Hypersensitivity to JAK inhibitor
  • Clinical evidence of cirrhosis
  • Leukemic transformation (>20% blasts in PB or BM any time prior to HCT)
  • Platelet count <50 x 109/L
  • Active uncontrolled infection
  • History of another malignancy within 5-years of date of HCT except history of basal cell or squamous cell carcinoma of skin or PV or ET
  • Known HIV positive
  • Woman of childbearing potential unwilling or unable to use adequate contraception Pregnant or nursing females Known active infection with hepatitis A, B or C virus

About this study

Official title: Ruxolitinib in Combination With High Dose Therapy and Autologous Stem Cell Transplantation for Myelofibrosis

To determine the safety of the approach of giving RUXOLITINIB before and after an autologous stem cell transplant, as measured by graft failure or death.

Read the detailed description

This is a pilot, single arm, single center study with no stratification to assess the safety (measured by graft failure or death) and feasibility (measured by adequacy of stem cell collection) of combining ruxolitinib with autologous Hematopoeitic Stem Cell Transplantation (HSCT) in patients with advanced myelofibrosis (MF). Patients will receive a short course of ruxolitinib prior to and during mobilization of HSCT with Filgrastim. Conditioning for the autologous HSCT will consist of Bulsulfan. Post-transplant patients will receive ruxolitinib maintenance.

Conditions
Myelofibrosis, MF
Being tested
RUXOLITINIB / INC 424, Filgrastim, Busulfan
Keywords
Myelofibrosis, RUXOLITINIB, Autologous stem cell transplant
Medical subject headings
Primary Myelofibrosis

What participants receive

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

  • RUXOLITINIB / INC 424Drug

    Also called RUX, Jakafi®

    Administered orally 5-20 mg twice daily x 16 weeks of therapy prior to attempted peripheral blood stem cells (PBSC) collection, during the collection and rest period and 3 months of therapy after high dose chemotherapy (HDC).

  • FilgrastimDrug

    Also called Neupogen®

    Peripheral blood stem cells (PBSC) will be mobilized with filgrastim 10 mcg/kg/day IV

  • BusulfanDrug

    Also called Busulfex®

    Conditioning for autologous Hematopoeitic Stem Cell Transplantation (HSCT) will consist of IV busulfan 2.0 mg/KBW once daily x 4 for days -5 to -2

Study groups in NCT02469974
Study groupRoleWhat they receive
Ruxolitinib / INC 424ExperimentalRuxolitinib, Jakafi ®, will be given orally at standard dose daily for 16 weeks pre ASCT and up to 3 months post-ASCT for 10 patients (allowing for 2 additional screen failures). Patients will restart ruxolitinib at 100 days after the ASCT as long as their platelet count is at least 50 x103. For patients whose platelet count is below 50 x103 at day 100, ruxolitinib should be restarted once platelet count reaches 50 x103. The dose of ruxolitinib can be titrated up as per clinical guidelines. PBSC mobilization will include G-CSF 10 mcg/kg/day. HDC for ASCT will consist of IV busulfan 2.0 mg/KBW once daily x 4 for days -5 to -2.
Receives: Drug: RUXOLITINIB / INC 424; Drug: Filgrastim; Drug: Busulfan

What is being measured

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

  1. Safety of combining ruxolitinib with autologous HSCT measured by graft failure or death

    Safety of this approach as measured by graft failure or death

    Measured: 2 years

Secondary outcomes (11)

  1. CD34 cells

    Total CD34+ cell dose will be calculated based on results of flow cytometric analysis and patient's weight.

    Measured: 4 years

  2. The regimen related mortality (RRM)

    Measured: day 100

  3. The regimen related mortality (RRM)

    Measured: day 365

  4. Rate of engraftment/graft failure

    Measured: 4 years

  5. Time of engraftment for neutrophils and platelets

    Measured: 4 years

  6. The incidence of serious infectious complications

    Measured: up to 1 year post transplant

  7. Changes in marrow fibrosis score

    The myelofibrosis score will be assessed as per the European Consensus Grading published by Thiele Grading Description at 365 days as compared to 180 days

    Measured: at 180 and 365 days post-transplant

  8. Change in FISH allele

    Changes in FISH abnormalities when present will be measured by cytogenetics.

    Measured: at 365 days post-transplant

  9. Change in JAK allele

    Changes in Jak 2 V617F allele burden when present will be measured by quantitative RT-PCR

    Measured: at 365 days post-transplant

  10. Rate of response

    Overall efficacy will be rated on a scale as complete remission, partial remission, clinical improvement, or stable disease

    Measured: at 6 months post-transplant

  11. Rate of response

    Overall efficacy will be rated on a scale as complete remission, partial remission, clinical improvement, or stable disease

    Measured: at 1 year post-transplant

Timeline

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

  1. First posted12 Jun 2015 (estimated)
  2. Study startMay 2015
  3. Primary completionAug 2016
  4. Study completionAug 2016

Why it stopped: no enrollments

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 N/A
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02469974
Sponsor's protocol ID
GCO 14-2106
Status last verified
Aug 2016
First submitted
28 May 2015
Last updated
12 Aug 2016
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A study testing RUXOLITINIB / INC 424, Filgrastim and Busulfan in people with Myelofibrosis and MF, run by Marina Kremyanskaya.

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 21 inclusion and 8 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: withdrawn, as of the sponsor's last update on 12 Aug 2016. Status can change between updates, so confirm with the study contact before making plans.

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.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

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?

Safety of combining ruxolitinib with autologous HSCT measured by graft failure or death (time frame: 2 years). 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 NCT02469974.

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 12 Aug 2016.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in