Actively Recruiting
Open Label Phase 1/2 Study of Tasquinimod in Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocytosis Myelofibrosis
Led by M.D. Anderson Cancer Center · Updated on 2026-05-26
33
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
Sponsors
M
M.D. Anderson Cancer Center
Lead Sponsor
A
Active Biotech AB
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the effects of tasquinimod alone or combined with ruxolitinib in patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (post-PV MF), or post-essential thrombocytosis myelofibrosis (post-ET MF). This open-label Phase 1/2 study aims to determine the anti-tumor activity of tasquinimod and its safety, along with assessing symptom changes, bone marrow fibrosis grade, pharmacokinetics, and genetic markers related to treatment response. Participants are assigned to one of two groups based on prior ruxolitinib treatment and response. Group 1 includes patients not responding well or with low blood counts after at least 3 months of ruxolitinib, receiving daily tasquinimod alone in 28-day cycles. Group 2 includes those on a stable ruxolitinib dose for at least 8 weeks with suboptimal response, receiving daily tasquinimod alongside continued ruxolitinib dosing. Both drugs are taken orally. During the study, participants will be monitored for safety and adverse events over about one year. Researchers will evaluate treatment response using objective remission criteria after six treatment cycles, symptom scores, and laboratory tests. Genetic and pharmacokinetic assessments will help understand treatment effects. Participants must attend scheduled visits, comply with treatment plans and tests, and provide informed consent throughout the study period.
CONDITIONS
Brief Title
Open Label Phase 1/2 Study of Tasquinimod in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), or Post-Essential Thrombocytosis Myelofibrosis (Post-ET MF)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosed with treatment-requiring primary myelofibrosis or post-ET/PV myelofibrosis per 2016 WHO criteria with intermediate-1, intermediate-2, or high-risk disease by DIPSS, or low-risk disease with symptomatic splenomegaly 65 cm below left costal margin
- Age 18 years or older
- Able to swallow and retain oral medication
- ECOG performance status of 0 to 2
- Peripheral or bone marrow blasts less than 10%
- For monotherapy group: Not candidates for, intolerant of, or relapsed/refractory to approved JAK inhibitors or no prior JAKi required
- For combination group: On stable ruxolitinib dose for at least 3 months with suboptimal response or progressive blood count issues
- Absolute neutrophil count 6 1.0 x 10^9/L
- Serum direct bilirubin 4 x upper limit of normal
- AST/ALT 4 2.5 x upper limit of normal (or 5 x ULN if liver involved)
- Glomerular filtration rate 30 ml/min or higher
- Prior treatment toxicities resolved to Grade 1
- At least 2 weeks from prior investigational myelofibrosis therapy
- Negative pregnancy test for women of childbearing potential
- Use of effective contraception for women of childbearing potential and men during and after study
- Willing and able to comply with study visits, treatment, and testing
- Able to provide informed consent
You will not qualify if you...
- Severe or uncontrolled medical conditions increasing risk or confounding toxicity assessment
- Significant cardiac conditions including ventricular tachyarrhythmia, unstable atrial fibrillation, resting bradycardia below 50 bpm, recent angina or heart attack within 3 months, symptomatic congestive heart failure, uncontrolled arrhythmia or hypertension
- Chronic corticosteroid or immunosuppressive treatment above specified doses
- Recent chemotherapy, immunomodulatory, platelet-reducing, immunosuppressive therapy, or erythropoetin use within specified timeframes
- Treatment with tasquinimod previously
- Gastrointestinal disorders affecting tasquinimod absorption
- Active or carrier status of Hepatitis A, B, or C; HIV infection
- Ongoing clinically significant infections
- History of pancreatitis or malabsorption conditions interfering with drug absorption
- Recent use of drugs strongly affecting cytochrome P-3A4, CYP1A2, or CYP2D6 enzymes
- Use of warfarin unless INR 3.0
- Known hypersensitivity to tasquinimod or its components
- Pregnant or breastfeeding women
- Any other condition deemed unsafe or noncompliant by investigator
- Prior participation in this study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Repeated 28-day cycles until disease progression or discontinuation
Participants receive tasquinimod daily in 28-day cycles either alone or in combination with a stable dose of ruxolitinib.
1 visit per cycle (in-person)
Trial Site Locations
Total: 1 location
1
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
L
Lucia Masarova, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
2
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