Primary MyelofibrosisPhase 2NCT01790295
Ruxolitinib Prior to Transplant in Patients With Myelofibrosis
A Phase 2 study testing Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in people with Primary Myelofibrosis and Post Polycythemia Vera Myelofibrosis, run by John Mascarenhas at 8 sites in 3 countries.
Trial summary
The purpose of this study is to find out if giving the study drug Ruxolitinib (INC424) prior to a combination of other chemotherapeutic drugs (Fludarabine and Busulfan) before infusing another person's hematopoietic stem cells (bone marrow transplantation) will be successful in people who have advanced primary… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, Post Essential Thrombocythemia Myelofibrosis
- What is being tested?
- Ruxolitinib Pre- Hematopoietic cell transplantation (HCT)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 8 sitesin 3 countries
- Study dates
- Nov 2013to 26 Oct 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Documented diagnosis of primary myelofibrosis according to WHO criteria or post PV myelofibrosis or post ET myelofibrosis as per IWG-MRT criteria
- Age 18-70 years
- Intermediate-2/ high-risk disease as per Dynamic IPSS (DIPSS) criteria OR Intermediate-1 risk disease with one of the following additional unfavorable features known to impact the survival adversely
- Red cell transfusion dependency
- Unfavorable Karyotype
- Platelet count <100 x 109/l
Show all 19Show fewer
- Blasts in the PB and BM ≤10% prior to study enrollment
- Availability of a suitable matched related (6/6 or 5/6) or unrelated donor (10/10 or 9/10 antigen or allele matched).
- Able to give informed written consent
- ECOG Performance status of 0-2.
- Life expectancy >3 months
- Off all MF-directed therapy including investigational agents for at least 2 weeks prior to study enrollment and recovered from all toxicities*
- Adequate organ function
- Adequate renal function - creatinine <1.5 x IULN
- Adequate hepatic function - AST/ALT <2.5 x IULN, Total Bilirubin <1.5 x IULN
- Adequate hematopoietic function - Platelet ≥50 x 109/l and ANC ≥1.0 x 109/l
- LVEF >40% (MUGA or echocardiogram) Normal per Institutional standard
- Adequate pulmonary function with DLCO >50%
- A patient who has been on stable dose of Ruxolitinib and has received ruxolitinib ≤6 months prior to the study entry will be considered potentially eligible for the study with the caveat that there is no evidence of loss of response (>5cm increase in spleen size from the nadir).
Who cannot join
- Any previous JAK2 inhibitor treatment prior to study enrollment, with the exception of Ruxolitinib
- Hypersensitivity to JAK inhibitor
- Clinical or laboratory evidence of cirrhosis
- Prior allogeneic transplant for any hematopoietic disorder
- >20% blast in the PB or BM prior to HCT or had leukemic transformation (>20% blasts in PB or BM any time prior to HCT)
- Syngeneic donor
Show all 15Show fewer
- Cord Blood transplant
- Active uncontrolled infection
- H/o another malignancy within 5-years of date of HCT except h/o basal cell or squamous cell carcinoma of skin or PV or ET
- Known HIV positive
- Pregnancy at the time of BMT
- Any other concurrent illness which in investigator's opinion puts the patient at excessive risk of treatment related toxicities
- Unable to give informed consent
- Active infection with hepatitis A,B or C virus
- Subjects who require therapy with a strong CYP3A4 inhibitor prior to enrollment to this study
Trial locations and contacts
8 sites in 3 countries.
8 sites in 3 countries
United States6 sites
Emory Hospital
Atlanta, Georgia, 30322
Northwestern University, Robert h. Lurie Comprehensive Cancer Center
Chicago, Illinois, 60611
Ohio State University
Columbus, Ohio, 43210
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
University of Kansas Cancer Center
Westwood, Kansas, 66205
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina, 27103
Canada1 site
Princess Margaret Cancer Centre, University of Toronto
Toronto, M5G 2M9
About this study
Official title: Exploring the Potential of Dual Kinase JAK 1/2 Inhibitor Ruxolitinib (INC424) With Reduced Intensity Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis
The purpose of this study is to find out if giving the study drug Ruxolitinib (INC424) prior to a combination of other chemotherapeutic drugs (Fludarabine and Busulfan) before infusing another person's hematopoietic stem cells (bone marrow transplantation) will be successful in people who have advanced primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF) or post-essential thrombocythemia myelofibrosis (PET-MF), collectively known as myelofibrosis (MF). MF is a disorder in which bone marrow tissue develops in abnormal sites because the bone marrow itself undergoes fibrosis or scarring. This study plans to evaluate whether adding the drug Ruxolitinib will further aid in reducing pre-transplant spleen size, improve physical performance levels and reduce adverse events (side effects) related to the transplant. Ruxolitinib is a drug that is approved by the FDA for the treatment of patients with advanced forms of myelofibrosis. Using Ruxolitinib prior to stem cell transplantation is experimental.
Read the detailed description
A two- stage Simon Phase II study will be conducted in each of two groups of patients: related and unrelated donor transplants. In each donor transplant group, the first stage of this design will include 11 patients evaluated for death or graft failure by 100 days post-transplant. In each stratum, we will enroll additional patients (up to 20%) of stratum total to take into account exclusions due to donor failure (such as donor deemed unsuitable for stem cell donation due to medical or other reasons) only. Those patients who have toxicities related to Ruxolitinib and not been able to reach HCT due to these toxicities will be included in the estimation of overall failure rates. Only those patients who are excluded based on donor related issues without any regimen related complications will be excluded from the estimation of failure rates. However, all data on these patients will be reported.
- Conditions
- Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, Post Essential Thrombocythemia Myelofibrosis
- Being tested
- Ruxolitinib Pre- Hematopoietic cell transplantation (HCT)
- Keywords
- Myelofibrosis, Stem cell transplant, Ruxolitinib
- 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 Pre- Hematopoietic cell transplantation (HCT)Drug
Also called Ruxolitinib \Pre- Hematopoietic cell transplantation (HCT), INC424
Ruxolitinib (INC424) tablets will be started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib will be determined according to baseline platelet count and will be modified according to platelet count at follow-up. The drug will be given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and will be stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug will be supplied as 5 mg tablets.
| Study group | Role | What they receive |
|---|---|---|
| Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) | Experimental | Ruxolitinib (INC424) tablets will be started 62 days (day -67) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib will be determined according to baseline platelet count and will be modified according to platelet count at follow-up. The drug will be given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and will be stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug will be supplied as 5 mg tablets. Receives: Drug: Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent of Participants With 100-day Survival Without Graft Failure
The feasibility of combining Ruxolitinib (INC424) with a Reduced intensity conditioning (RIC) regimen likely to produce success post transplantation, success being defined as patient being alive, and without graft failure at day 100-post allogeneic stem cell transplantation (in patients who receive (a) related donor transplant and in those who receive (b) an unrelated donor transplant.
Measured: Day 100-post allogeneic stem cell transplantation
Secondary outcomes (19)
Time to Neutrophil Recovery
Neutrophil recovery will be defined as first of the three consecutive days with neutrophil count ≥0.5 x 109/l.
Measured: up to 4 years
Platelet Recovery
Platelet recovery will be defined as first of the 7 days with platelet count ≥20 x 109/l, without platelet transfusion support and both maintained for 30 days without transfusion support or myeloid cytokine support.
Measured: up to 4 years
Percent of Participants With Non-relapse Mortality (NRM)
NRM will be defined as death in first 30 days due to any cause, and subsequently death due to any cause without the recurrence or progression of myelofibrosis. Cumulative incidence of NRM will be calculated taking relapse/progression as competing event.
Measured: 100 days
Percent of Participants With Non-relapse Mortality (NRM)
NRM will be defined as death in first 30 days due to any cause, and subsequently death due to any cause without the recurrence or progression of myelofibrosis. Cumulative incidence of NRM will be calculated taking relapse/progression as competing event.
Measured: 1-year post transplant
Percent of Participants With Graft Versus Host Disease (GvHD)
Acute and chronic GvHD. GvHD is a potentially serious complication of allogeneic stem cell transplantation.
Stage Skin Liver (bilirubin) Gut (stool output/day)
0 No GVHD rash < 2 mg/dl < 500 ml/day or persistent nausea.
- Maculopapular rash< 25% body surface area (BSA) 2-3 mg/dl 500-999 ml/day
- Maculopapular rash 25 - 50% BSA 3.1-6 mg/dl 1000-1500 ml/day
- Maculopapular rash > 50% BSA 6.1-15 mg/dl Adult: >1500 ml/day
- Generalized erythroderma plus bullous formation >15 mg/dl Severe abdominal pain with or without ileus Grade I Stage 1-2 None None II Stage 3 or Stage 1 or Stage 1 III - Stage 2-3 or Stage 2-4 IV Stage 4 or Stage 4 -
Measured: 1-year post transplant
Chimerism Studies
Will check percentage of donor versus recipient blood cells to determine efficacy of donor engraftment
Measured: 30 days post transplant
Chimerism Studies
Will check percentage of donor versus recipient blood cells to determine efficacy of donor engraftment
Measured: 60 days post transplant
Chimerism Studies
Will check percentage of donor versus recipient blood cells to determine efficacy of donor engraftment
Measured: 100 days post transplant
Number of Participants With Remission Status According to IWG-MRT Criteria
Remission status according to International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) criteria
Remission defined as:
Bone marrow:* Age-adjusted normocellularity; <5% blasts; ≤grade 1 MF and Peripheral blood: Hemoglobin ≥100 g/L and <UNL; neutrophil count ≥ 1 × 109/L and <UNL; Platelet count ≥100 × 109/L and <UNL; <2% immature myeloid cells and Clinical: Resolution of disease symptoms; spleen and liver not palpable; no evidence of extramedullary hematopoiesis (EMH)
Measured: Day 100 post transplant
Number of Participants With Remission Status at 6 Months Post Transplant
Remission defined as:
Bone marrow:* Age-adjusted normocellularity; <5% blasts; ≤grade 1 MF† and Peripheral blood: Hemoglobin ≥100 g/L and <UNL; neutrophil count ≥ 1 × 109/L and <UNL; Platelet count ≥100 × 109/L and <UNL; <2% immature myeloid cells‡ and Clinical: Resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH
Measured: 6 months post transplant
Number of Participants With Remission Status at 12 Months Post Transplant
Remission defined as:
Bone marrow:* Age-adjusted normocellularity; <5% blasts; ≤grade 1 MF† and Peripheral blood: Hemoglobin ≥100 g/L and <UNL; neutrophil count ≥ 1 × 109/L and <UNL; Platelet count ≥100 × 109/L and <UNL; <2% immature myeloid cells‡ and Clinical: Resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH
Measured: 12 months post transplant
Number of Participants With Relapse/Progression (Defined as Per IWG-MRT Criteria)
Relapse/progression defined as:
Peripheral blood: Hemoglobin ≥100 g/L and <UNL; neutrophil count ≥1 × 109/L and <UNL; platelet count ≥100 × 109/L and <UNL; <2% immature myeloid cells‡ and Clinical: Resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH or Bone marrow:* Age-adjusted normocellularity; <5% blasts; ≤grade 1 MF†, and peripheral blood: Hemoglobin ≥85 but <100 g/L and <UNL; neutrophil count ≥1 × 109/L and <UNL; platelet count ≥50, but <100 × 109/L and <UNL; <2% immature myeloid cells‡ and Clinical: Resolution of disease symptoms; spleen and liver not palpable; no evidence of EMH
Measured: 1-year post transplant
Number of Participants With Progression-free Survival
Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions.
Measured: 1-year post transplant
Number of Overall Survival
Measured: 1-year post transplant
Mean Change in the Brief Fatigue Inventory Score
Mean change in the Brief Fatigue Inventory score (BFI) from baseline to 48 months to assess impact of allogeneic stem cell transplant on myelofibrosis associated symptoms and overall quality of life. The BFI is a 9 item scored from 0 (no fatigue) -10 (as bad as you can imagine), items are averaged with total score from 0-10, with higher score indicating more fatigue.
Measured: baseline and 48 months
Expression Profiling and Measurements of Cytokines Prior to Start of Ruxolitinib, Prior to Start of Chemotherapy for Conditioning
Measured: 30 days post transplant
Expression Profiling and Measurements of Cytokines Prior to Start of Ruxolitinib, Prior to Start of Chemotherapy for Conditioning
Measured: 100 days post transplant
Association of Cytokines Levels With Acute and Chronic GvHD
Measured: 30 days post transplant
Association of Cytokines Levels With Acute and Chronic GvHD
Measured: 100 days post transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2013 (estimated)
- Study startNov 2013
- Primary completion26 Oct 2017
- Study completion26 Oct 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary MyelofibrosisStudies of the same condition, open and under way.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- John Mascarenhas
- Sponsor type
- University, hospital or other
- Collaborators
- Myeloproliferative Disorders-Research Consortium, National Cancer Institute (NCI), Incyte Corporation, Novartis
- Responsible party
- Sponsor-investigator, John Mascarenhas, Icahn School of Medicine at Mount Sinai
- Investigators
- John Mascarenhas, MD, Principal investigator, Icahn School of Medicine at Mount SinaiVikas Gupta, MD, FRCP, FRCPath, Study chair, University of TorontoAdam Mead, MD, Study chair, University of Oxford, John Radcliffe Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01790295
- Sponsor's protocol ID
- GCO 12-1809
- Other IDs
- MPD-RC 114
- Status last verified
- Feb 2019
- First submitted
- 8 Feb 2013
- Last updated
- 20 Mar 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Gupta V, Kosiorek HE, Mead A, Klisovic RB, Galvin JP, Berenzon D, Yacoub A, Viswabandya A, Mesa RA, Goldberg J, Price L, Salama ME, Weinberg RS, Rampal R, Farnoud N, Dueck AC, Mascarenhas JO, Hoffman R. Ruxolitinib Therapy Followed by Reduced-Intensity Conditioning for Hematopoietic Cell Transplantation for Myelofibrosis: Myeloproliferative Disorders Research Consortium 114 Study. Biol Blood Marrow Transplant. 2019 Feb;25(2):256-264. doi: 10.1016/j.bbmt.2018.09.001. Epub 2018 Sep 8.
PubMed 30205231
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Exploring the Potential of Dual Kinase JAK 1/2 Inhibitor Ruxolitinib (INC424) With Reduced Intensity Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis
- ClinicalTrials.gov ID
- NCT01790295
- Acronym / sponsor ID
- GCO 12-1809
- Conditions
- Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, Post Essential Thrombocythemia Myelofibrosis
- Keywords
- Myelofibrosis, Stem cell transplant, Ruxolitinib
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 21 participants (actual)
- Lead sponsor
- John MascarenhasUniversity, hospital or other
- Collaborators
- Myeloproliferative Disorders-Research Consortium, National Cancer Institute (NCI), Incyte Corporation, Novartis
- Study start
- Nov 2013
- Primary completion
- 26 Oct 2017 (actual)
- Study completion
- 26 Oct 2017 (actual)
- First posted
- 13 Feb 2013 (estimated)
- Results
- Posted 20 Mar 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to find out if giving the study drug Ruxolitinib (INC424) prior to a combination of other chemotherapeutic drugs (Fludarabine and Busulfan) before infusing another person's hematopoietic stem cells (bone marrow transplantation) will be successful in people who have advanced primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF) or post-essential thrombocythemia myelofibrosis (PET-MF), collectively known as myelofibrosis (MF). MF is a disorder in which bone marrow tissue develops in abnormal sites because the bone marrow itself undergoes fibrosis or scarring. This study plans to evaluate whether adding the drug Ruxolitinib will further aid in reducing pre-transplant spleen size, improve physical performance levels and reduce adverse events (side effects) related to the transplant. Ruxolitinib is a drug that is approved by the FDA for the treatment of patients with advanced forms of myelofibrosis. Using Ruxolitinib prior to stem cell transplantation is experimental.
Detailed description
A two- stage Simon Phase II study will be conducted in each of two groups of patients: related and unrelated donor transplants. In each donor transplant group, the first stage of this design will include 11 patients evaluated for death or graft failure by 100 days post-transplant. In each stratum, we will enroll additional patients (up to 20%) of stratum total to take into account exclusions due to donor failure (such as donor deemed unsuitable for stem cell donation due to medical or other reasons) only. Those patients who have toxicities related to Ruxolitinib and not been able to reach HCT due to these toxicities will be included in the estimation of overall failure rates. Only those patients who are excluded based on donor related issues without any regimen related complications will be excluded from the estimation of failure rates. However, all data on these patients will be reported.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) | Experimental | Drug: Ruxolitinib Pre- Hematopoietic cell transplantation (HCT)Ruxolitinib (INC424) tablets will be started 62 days (day -67) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib will be determined according to baseline platelet count and will be modified according to platelet count at follow-up. The drug will be given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and will be stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug will be supplied as 5 mg tablets. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (19 listed)
- Documented diagnosis of primary myelofibrosis according to WHO criteria or post PV myelofibrosis or post ET myelofibrosis as per IWG-MRT criteria
- Age 18-70 years
- Intermediate-2/ high-risk disease as per Dynamic IPSS (DIPSS) criteria OR Intermediate-1 risk disease with one of the following additional unfavorable features known to impact the survival adversely
- Blasts in the PB and BM ≤10% prior to study enrollment
- Availability of a suitable matched related (6/6 or 5/6) or unrelated donor (10/10 or 9/10 antigen or allele matched).
Key exclusion criteria (15 listed)
- Any previous JAK2 inhibitor treatment prior to study enrollment, with the exception of Ruxolitinib
- Hypersensitivity to JAK inhibitor
- Clinical or laboratory evidence of cirrhosis
- Prior allogeneic transplant for any hematopoietic disorder
- >20% blast in the PB or BM prior to HCT or had leukemic transformation (>20% blasts in PB or BM any time prior to HCT)
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 |
|---|---|---|
| Ruxolitinib Pre- Hematopoietic cell transplantation (HCT)Also: Ruxolitinib \Pre- Hematopoietic cell transplantation (HCT), INC424Ruxolitinib (INC424) tablets will be started 60 days (day -65) prior to start of conditioning chemotherapy. The starting dose of Ruxolitinib will be determined according to baseline platelet count and will be modified according to platelet count at follow-up. The drug will be given in the maximum tolerated dose as defined in the protocol for 56 days, followed by 4 days of taper, and will be stopped completely at the planned start of conditioning therapy (starting on day -5) i.e. 5 days prior to stem cell infusion. The drug will be supplied as 5 mg tablets. | Drug | Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) |
Primary outcome
Percent of Participants With 100-day Survival Without Graft Failure
Time frame: Day 100-post allogeneic stem cell transplantation
The feasibility of combining Ruxolitinib (INC424) with a Reduced intensity conditioning (RIC) regimen likely to produce success post transplantation, success being defined as patient being alive, and without graft failure at day 100-post allogeneic stem cell transplantation (in patients who receive (a) related donor transplant and in those who receive (b) an unrelated donor transplant.
19 secondary outcomes
Time to Neutrophil Recovery
Time frame: up to 4 years
Platelet Recovery
Time frame: up to 4 years
Percent of Participants With Non-relapse Mortality (NRM)
Time frame: 100 days
Percent of Participants With Non-relapse Mortality (NRM)
Time frame: 1-year post transplant
Percent of Participants With Graft Versus Host Disease (GvHD)
Time frame: 1-year post transplant
Chimerism Studies
Time frame: 30 days post transplant
Chimerism Studies
Time frame: 60 days post transplant
Chimerism Studies
Time frame: 100 days post transplant
Number of Participants With Remission Status According to IWG-MRT Criteria
Time frame: Day 100 post transplant
Number of Participants With Remission Status at 6 Months Post Transplant
Time frame: 6 months post transplant
Number of Participants With Remission Status at 12 Months Post Transplant
Time frame: 12 months post transplant
Number of Participants With Relapse/Progression (Defined as Per IWG-MRT Criteria)
Time frame: 1-year post transplant
Number of Participants With Progression-free Survival
Time frame: 1-year post transplant
Number of Overall Survival
Time frame: 1-year post transplant
Mean Change in the Brief Fatigue Inventory Score
Time frame: baseline and 48 months
Expression Profiling and Measurements of Cytokines Prior to Start of Ruxolitinib, Prior to Start of Chemotherapy for Conditioning
Time frame: 30 days post transplant
Expression Profiling and Measurements of Cytokines Prior to Start of Ruxolitinib, Prior to Start of Chemotherapy for Conditioning
Time frame: 100 days post transplant
Association of Cytokines Levels With Acute and Chronic GvHD
Time frame: 30 days post transplant
Association of Cytokines Levels With Acute and Chronic GvHD
Time frame: 100 days post transplant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Mar 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 30205231Gupta V, Kosiorek HE, Mead A, Klisovic RB, Galvin JP, Berenzon D, Yacoub A, Viswabandya A, Mesa RA, Goldberg J, Price L, Salama ME, Weinberg RS, Rampal R, Farnoud N, Dueck AC, Mascarenhas JO, Hoffman R. Ruxolitinib Therapy Followed by Reduced-Intensity Conditioning for Hematopoietic Cell Transplantation for Myelofibrosis: Myeloproliferative Disorders Research Consortium 114 Study. Biol Blood Marrow Transplant. 2019 Feb;25(2):256-264. doi: 10.1016/j.bbmt.2018.09.001. Epub 2018 Sep 8.
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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.
John Mascarenhas, MD principal investigator
Icahn School of Medicine at Mount Sinai
Vikas Gupta, MD, FRCP, FRCPath study chair
University of Toronto
Adam Mead, MD study chair
University of Oxford, John Radcliffe Hospital
8 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ruxolitinib Pre- Hematopoietic cell transplantation (HCT) in people with Primary Myelofibrosis and Post Polycythemia Vera Myelofibrosis, run by John Mascarenhas at 8 sites in 3 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 19 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: stopped early, as of the sponsor's last update on 20 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States, Canada, United Kingdom. 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?
Yes. Results are posted in the registry (first posted 20 Mar 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percent of Participants With 100-day Survival Without Graft Failure (time frame: Day 100-post allogeneic stem cell transplantation). 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 NCT01790295, which also lists 1 linked publication.
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 20 Mar 2019.