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Bone Marrow FibrosisPhase 2NCT03333187

Ruxolitinib vs Allogeneic SCT for Patients With Myelofibrosis According to Donor Availability

A Phase 2 study testing Allogeneic stem cell transplantation and Ruxolitinib continuous therapy in people with Bone Marrow Fibrosis, run by Universitätsklinikum Hamburg-Eppendorf at 14 sites in Germany.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

The present study will be a multicenter, prospective phase II-study comparing efficacy of allogeneic SCT for patients with myelofibrosis who have a suitable stem cell donor after a 3 months Ruxolitinib induction therapy with patients who lack a suitable stem cell donor and will continue to receive Ruxolitinib. Read more about this study

Who is it for?
Ages 18 to 70 years
Conditions the study is for
Bone Marrow Fibrosis
What is being tested?
Allogeneic stem cell transplantation and Ruxolitinib continuous therapy
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
87 participants
Where
14 sitesGermany
Study dates
21 Dec 2016to 7 Oct 2025
Phase
Phase 2
Are there results?
Not yet: the study is under way

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

  • Symptomatic primary myelofibrosis or myelofibrosis post polycythaemia vera or essential thrombocythemia stage intermediate 2- or high-risk according to IPSS or DIPSS [46] or intermediate 1-risk with high risk cytogenetics, other than normal karyotype, sole del 20q, del 13q, or sole+9, or transfusion-dependency
  • Patients age: 18 - 70 years at time of inclusion (female and male)
  • Patients understand and voluntarily sign an informed consent form
  • Platelet count ≥ 50 x 109/L
  • No prior Ruxolitinib treatment
  • ECOG ≤ 2

Who cannot join

  • Severe renal, hepatic, pulmonary or cardiac disease, such as:
  • Total bilirubin, SGPT or SGOT > 3 times upper the normal level
  • Left ventricular ejection fraction < 30 %
  • Creatinine clearance < 30 ml/min
  • DLCO < 35 % and/or receiving supplementary continuous oxygen
  • Positive serology for HIV
Show all 12Show fewer
  • Pregnant or lactating women (positive serum pregnancy test)
  • Age < 18 and ≥ 71 years.
  • Uncontrolled invasive fungal infection at time of screening (baseline)
  • Serious psychiatric or psychological disorders
  • Participation in another study with ongoing use of unlicensed investigational product from 28 days before study enrollment
  • Transformation to AML

Trial locations and contacts

14 sites in 1 country.

14 sites in 1 country

Germany14 sites

  • Universitätsklinkum Aachen

    Aachen, 52074

  • HELIOS Klinikum Berlin-Buch

    Berlin, 13125

  • Universitätsklinikum Bonn

    Bonn, 53105

  • Universitätsklinikum Düsseldorf

    Düsseldorf, 40225

  • Universitätsklinkum Halle

    Halle, 06120

  • University Medical Center Hamburg-Eppendorf

    Hamburg, 20246

  • Universitätsklinikum Jena

    Jena, 07747

  • Universitätsmedizin der Johannes Gutenberg-Universität Mainz

    Mainz, 55131

  • Johannes Wesling Klinikum Minden

    Minden, 32429

  • Universitätsklinikum Münster

    Münster, 48149

  • Klinikum Nürnberg

    Nuremberg, 90419

  • Robert-Bosch-Krankenhaus Stuttgart

    Stuttgart, 70376

  • Universitätsmedizin Tübingen

    Tübingen, 72076

  • Universitätsklinkum Ulm

    Ulm, 89081

Other trials in Germany

About this study

Official title: Ruxolitinib Versus Allogeneic Stem Cell Transplantation for Patients With Myelofibrosis According to Donor Availability: A Prospective Phase II Trial (MMM 02 Study)

The present study will be a multicenter, prospective phase II-study comparing efficacy of allogeneic SCT for patients with myelofibrosis who have a suitable stem cell donor after a 3 months Ruxolitinib induction therapy with patients who lack a suitable stem cell donor and will continue to receive Ruxolitinib.

Read the detailed description

This study is a multicenter, prospective phase II-study compares efficacy of allogeneic SCT for patients with myelofibrosis who have a suitable stem cell donor after a 3 months Ruxolitinib induction therapy with patients who lack a suitable stem cell donor and will continue to receive Ruxolitinib.

In this study will further assess and compare the safety and efficacy of study treatments/ induction therapy in both study arms on spleen reduction, improvement of constitutional symptoms, QOL, toxicity, fibrosis regression, development of GvHD as well as chimerism, engraftment, relapse incidence, disease related mortality, outcome and overall survival.

Being tested
Allogeneic stem cell transplantation, Ruxolitinib continuous therapy
Keywords
Myelofibrosis, Myeloproliferative Disease
Medical subject headings
Primary Myelofibrosis, Myeloproliferative Disorders

What participants receive

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

  • Allogeneic stem cell transplantationProcedure or surgery

  • Ruxolitinib continuous therapyDrug

    Also called Jakavi

Study groups in NCT03333187
Study groupRoleWhat they receive
Arm AExperimentalTreatment with Allogeneic Stem cell Transplantation after 3 months of Ruxolitinib induction therapy
Receives: Procedure: Allogeneic stem cell transplantation
Arm BActive comparatorTreatment with Ruxolitinib continuous therapy
Receives: Drug: Ruxolitinib continuous therapy

What is being measured

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

  1. Event free survival

    Compare to event free survival of patients at 3 years after allogeneic SCT and in Ruxolitinib continuous therapy in patients without a suitable donor

    Measured: 3 years

Secondary outcomes (20)

  1. Spleen reduction

    Ultrasound measurement Spleen size, reduction of Spleen size after 3 months Ruxolitinib induction therapy

    Measured: 3 months

  2. Improvement of constitutional symptoms

    Improvement of constitutional symptoms (Loose of weight and night sweat) after 3 months Ruxolitinib induction therapy, questionnaire, medical history

    Measured: 3 months

  3. Improvement of bone marrow fibrosis

    bone marrow histology, Improvement of bone marrow fibrosis after 3 months of Ruxolitinib induction therapy

    Measured: 3 months

  4. Acute graft-versus-host disease

    Incidence of acute graft-versus-host disease on Day +100 after allogeneic SCT according to the Glucksberg scale revised by Przepiorka

    Measured: Day +100 after allogeneic SCT

  5. Chronic graft-versus-host disease

    Incidence of chronic graft-versus-host disease according to the NIH consensus criteria of Filipovich et al. at 1, 2 and 3 years after allogeneic SCT

    Measured: 1, 2 and 3 years after allogeneic SCT

  6. Toxicity of Ruxolitinib

    Toxicity of Ruxolitinib scored according to NCI CTCAE, Version 4.0

    Measured: till 3 years

  7. Toxicity of conditioning therapy

    Toxicity of conditioning therapy scored according to NCI CTCAE, Version 4.0

    Measured: till 3 years

  8. Relapse

    Cumulative incidence of relapse at 3 years after allogeneic SCT

    Measured: 3 years

  9. Disease-related mortality

    Disease-related mortality at 3 years after allogeneic SCT and Ruxolitinib continuous therapies

    Measured: 3 years

  10. Non-relapsed mortality

    Non-relapsed mortality at 1 and 3 years after allogeneic SCT and Ruxolitinib continuous therapy

    Measured: 1 and 3 years

  11. Discontinuation rate

    Discontinuation rate at 3 years after Ruxolitinib continuous therapy (End of study)

    Measured: 3 years

  12. Evaluation of Sorror Risk Score

    Evaluation of Sorror Risk Score on outcome after allogeneic SCT

    Measured: at baseline

  13. Chimerism on relapse

    Chimerism Analyse, Impact of chimerism on relapse incidence after allogeneic SCT

    Measured: 30d, 100d, 180 d, 1 year, 2 years and 3 years

  14. Bone marrow fibrosis regression

    bone marrow histology, Evaluation of bone marrow fibrosis regression after allogeneic SCT at 30d, 100d, 1 year, and 3 years

    Measured: 30d, 100d, 1 year, and 3 years

  15. Bone marrow fibrosis regression

    bone marrow histology, Evaluation of bone marrow fibrosis regression after Ruxolitinib continuous therapy at 30d, 100d, 1 year and 3 years

    Measured: 30d, 100d, 1 year and 3 years

  16. Evaluation of QOL (FACT-BMT)

    Questionnaire, Evaluation of QOL (FACT-BMT) before Ruxolitinib induction therapy (= baseline), at transplantation, and after transplantation at 6m, 1 year, 2 years and 3 years

    Measured: baseline, at transplantation, +180d, +1 year, +2 years and +3 years

  17. Evaluation of QOL (MPN-SAF-TSS)

    Questionaire, Evaluation of QOL (MPN-SAF-TSS) before Ruxolitinib induction therapy (= baseline), at transplantation, and after transplantation at 6m, 1 year, 2 years and 3 years

    Measured: baseline, at transplantation, +180d, +1 year, +2 years and +3 years

  18. Evaluation of QOL (FACT-BMT)

    Questionnaire, Evaluation of QOL (FACT-BMT) before Ruxolitinib induction therapy (= baseline), at confinement to Ruxolitinib continuous therapy and after confinement at 6 months, 1 year, 2 years and 3 years

    Measured: baseline, confinement to Ruxolitinib continous therapy, +180d, +1 year, +2 years and +3 years

  19. Evaluation of QOL (MPN-SAF-TSS)

    Questionnaire, Evaluation of QOL (MPN-SAF-TSS) before Ruxolitinib induction therapy (= baseline), at confinement to Ruxolitinib continuous therapy and after confinement at 6 months, 1 year, 2 years and 3 years

    Measured: baseline, confinement to Ruxolitinib continous therapy, +180d, +1 year, +2 years and +3 years

  20. Overall Survival

    Overall survival at 3 years after allogeneic SCT compared to Ruxolitinib continuous therapy in patients without a suit-able donor

    Measured: 3 years

Timeline

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

  1. First posted6 Nov 2017
  2. Study start21 Dec 2016
  3. Primary completion7 Oct 2024
  4. Study completion7 Oct 2025 (estimated)

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
Non-randomised
Intervention model
Parallel groups
Model description
Treatment A (only with a suitable stem cell donor): Allogeneic SCT after 3 months of Ruxolitinib induction therapy Treatment B: Ruxolitinib continuous therapy
Masking
None (open label)
Enrolment
87 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03333187
Sponsor's protocol ID
MMM 02 study / RuxoAlloStudy
Status last verified
Mar 2025
First submitted
22 Jun 2017
Last updated
30 Mar 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Allogeneic stem cell transplantation and Ruxolitinib continuous therapy in people with Bone Marrow Fibrosis, run by Universitätsklinikum Hamburg-Eppendorf at 14 sites in Germany.

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 6 inclusion and 12 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: under way, not looking for participants, as of the sponsor's last update on 30 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 14 sites in Germany. 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?

This study is still under way. Results are not available in the source record yet.

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?

Event free survival (time frame: 3 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 NCT03333187.

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 30 Mar 2025.

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