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

NCT ID: NCT03333187

Last Updated: 2025-03-30

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 87 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-12-21
• Study Completion Date: 2025-10-07

Brief 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.

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.

Conditions

Bone Marrow Fibrosis

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Arm A

Treatment with Allogeneic Stem cell Transplantation after 3 months of Ruxolitinib induction therapy

Group Type: EXPERIMENTAL

Allogeneic stem cell transplantation

Intervention Type: PROCEDURE

Arm B

Treatment with Ruxolitinib continuous therapy

Group Type: ACTIVE_COMPARATOR

Ruxolitinib continuous therapy

Intervention Type: DRUG

Interventions

Allogeneic stem cell transplantation

Intervention Type: PROCEDURE

Ruxolitinib continuous therapy

Intervention Type: DRUG

Other Intervention Names

Jakavi

Eligibility Criteria

Inclusion Criteria

1. 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

2. Patients age: 18 - 70 years at time of inclusion (female and male)

3. Patients understand and voluntarily sign an informed consent form

4. Platelet count ≥ 50 x 109/L

5. No prior Ruxolitinib treatment

6. ECOG ≤ 2

Exclusion Criteria

1. 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

2. Positive serology for HIV

3. Pregnant or lactating women (positive serum pregnancy test)

4. Age < 18 and ≥ 71 years.

5. Uncontrolled invasive fungal infection at time of screening (baseline)

6. Serious psychiatric or psychological disorders

7. Participation in another study with ongoing use of unlicensed investigational product from 28 days before study enrollment

8. Transformation to AML

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis

INDUSTRY

Sponsor Role: collaborator

Clinical Trial Center North (CTC North GmbH & Co. KG)

OTHER

Sponsor Role: collaborator

Universitätsklinikum Hamburg-Eppendorf

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Nicolaus Kröger, Prof. Dr.

Role: PRINCIPAL_INVESTIGATOR

Universitätsklinikum Hamburg-Eppendorf

Locations

Universitätsklinkum Aachen

Aachen, , Germany

HELIOS Klinikum Berlin-Buch

Berlin, , Germany

Universitätsklinikum Bonn

Bonn, , Germany

Universitätsklinikum Düsseldorf

Düsseldorf, , Germany

Universitätsklinkum Halle

Halle, , Germany

University Medical Center Hamburg-Eppendorf

Hamburg, , Germany

Universitätsklinikum Jena

Jena, , Germany

Universitätsmedizin der Johannes Gutenberg-Universität Mainz

Mainz, , Germany

Johannes Wesling Klinikum Minden

Minden, , Germany

Universitätsklinikum Münster

Münster, , Germany

Klinikum Nürnberg

Nuremberg, , Germany

Robert-Bosch-Krankenhaus Stuttgart

Stuttgart, , Germany

Universitätsmedizin Tübingen

Tübingen, , Germany

Universitätsklinkum Ulm

Ulm, , Germany

Countries

Germany

Other Identifiers

MMM 02 study / RuxoAlloStudy

Identifier Type: -

Identifier Source: org_study_id