Ruxolitinib Vs Allogeneic SCT for Patients with Myelofibrosis According to Donor Availability

Universitätsklinikum Hamburg-Eppendorf

Status and phase: Active, not recruiting; Phase 2

Conditions: Bone Marrow Fibrosis

Treatments: Procedure: Allogeneic stem cell transplantation; Drug: Ruxolitinib continuous therapy

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT03333187

MMM 02 study / RuxoAlloStudy

Details and patient eligibility

About

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.

Full 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.

Enrollment: 87 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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87 participants in 2 patient groups

Arm A

Experimental group

Description:

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

Treatment:

Procedure: Allogeneic stem cell transplantation

Arm B

Active Comparator group

Description:

Treatment with Ruxolitinib continuous therapy

Treatment:

Drug: Ruxolitinib continuous therapy