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

NCT03333187 | Active Not Recruiting Phase 2 DMC

• 1 other identifier: MMM 02 study / RuxoAlloStudy
• Study Type: interventional
• Target: 87
• Locations: 1 country
• Sites: 14

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.

Conditions

Bone Marrow Fibrosis

Keywords

Myelofibrosis, Myeloproliferative Disease

Outcome Measures

Primary Outcomes (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

  3 years

Secondary Outcomes (20)

• Spleen reduction

  3 months

• Improvement of constitutional symptoms

  3 months

• Improvement of bone marrow fibrosis

  3 months

• Acute graft-versus-host disease

  Day +100 after allogeneic SCT

• Chronic graft-versus-host disease

  1, 2 and 3 years after allogeneic SCT

Study Arms (2)

Arm A

EXPERIMENTAL

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

Procedure: Allogeneic stem cell transplantation

Arm B

ACTIVE COMPARATOR

Treatment with Ruxolitinib continuous therapy

Drug: Ruxolitinib continuous therapy

Interventions

Allogeneic stem cell transplantation PROCEDURE

Arm A

Ruxolitinib continuous therapy DRUG

Also known as: Jakavi

Arm B

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 understand and voluntarily sign an informed consent form
• Platelet count ≥ 50 x 109/L
• No prior Ruxolitinib treatment
• ECOG ≤ 2

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Universitätsklinikum Hamburg-Eppendorf: lead
• Novartis: collaborator
• Clinical Trial Center North (CTC North GmbH & Co. KG): collaborator

Study Sites (14)

Universitätsklinkum Aachen

Aachen, 52074, Germany

Location

HELIOS Klinikum Berlin-Buch

Berlin, 13125, Germany

Location

Universitätsklinikum Bonn

Bonn, 53105, Germany

Location

Universitätsklinikum Düsseldorf

Düsseldorf, 40225, Germany

Location

Universitätsklinkum Halle

Halle, 06120, Germany

Location

University Medical Center Hamburg-Eppendorf

Hamburg, 20246, Germany

Location

Universitätsklinikum Jena

Jena, 07747, Germany

Location

Universitätsmedizin der Johannes Gutenberg-Universität Mainz

Mainz, 55131, Germany

Location

Johannes Wesling Klinikum Minden

Minden, 32429, Germany

Location

Universitätsklinikum Münster

Münster, 48149, Germany

Location

Klinikum Nürnberg

Nuremberg, 90419, Germany

Location

Robert-Bosch-Krankenhaus Stuttgart

Stuttgart, 70376, Germany

Location

Universitätsmedizin Tübingen

Tübingen, 72076, Germany

Location

Universitätsklinkum Ulm

Ulm, 89081, Germany

Location

MeSH Terms

Conditions

Primary Myelofibrosis; Myeloproliferative Disorders

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Nicolaus Kröger, Prof. Dr.

  Universitätsklinikum Hamburg-Eppendorf

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Treatment A (only with a suitable stem cell donor): Allogeneic SCT after 3 months of Ruxolitinib induction therapy Treatment B: Ruxolitinib continuous therapy

Study Record Dates

• First Submitted: June 22, 2017
• First Posted: November 6, 2017
• Study Start: December 21, 2016
• Primary Completion: October 7, 2024
• Study Completion: October 7, 2025
• Last Updated: March 30, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

DE (14)