Tasquinimod in Patients with Myelofibrosis Refractory to or Intolerant for JAK2 Inhibition

ConditionMyelofibrosis
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorStichting Hemato-Oncologie voor Volwassenen Nederland

About this trial

The goal of this clinical trial is to learn if therapy can be improved in patients with myelofibrosis (MF) who have primary resistance or who have progressed after treatment with a Janus kinase (JAK) inhibitor or are intolerant for this category of drugs.

The main questions it aims to answer are:

* To evaluate the feasibility and safety of once daily dose of tasquinimod for 24 weeks (6 cycles) * To determine the optimal dose

Patients will be treated once daily with tasquinimod for a maximum period of 24 weeks (6 cycles).

During the study most (diagnostic) procedures are part of the standard of care. Different from standard of care:

* Participation may lead to extra visits to the outpatient clinic * Additional blood will be drawn when blood is already taken per standard of care * Bone marrow sampling at entry and at the end of the trial * MRI scans (or CT-scans) have to be performed * Quality-of-life questionnaires have to be filled out

Eligibility criteria

Qualifiers

Diagnosis of PMF or Post-PV MF or Post-ET MF based on a bone marrow (BM) biopsy not older than 6 months, according to the 2016 World Health Organization.

Refactory or intolerant to treatment with an approved JAK inhibitor or ineligible for JAK inhibitor treatment.

MF classified as Intermediate-1 with disease-related symptoms (e.g. symptomatic splenomegaly), Intermediate-2 or high-risk by Dynamic International Prognostic Scoring System Plus

Spleen ≥5 cm below costal margin as measured by palpation.

Disqualifiers

Patients eligible for hematopoietic stem cell transplantation (suitable candidate and a suitable donor is available).

Splenectomy.

Splenic irradiation within the last 6 months.

Prior allogeneic stem cell transplantation.

Trial design

Treatments tested in this trial

  • Tasquinimod

Treatment groups

20 Participants
are divided into 1 treatment group