Primary MyelofibrosisPhase 1/2NCT06327100
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)
A Phase 1/2 study testing Ruxolitinib and Tasquinimod in people with Primary Myelofibrosis and Post-polycythemia Vera Myelofibrosis, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
To learn if tasquinimod either alone or in combination with ruxolitinib can help to control PMF, post-PV MF, or post-ET MF. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-Essential Thrombocytosis Myelofibrosis
- What is being tested?
- Ruxolitinib and Tasquinimod
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Aug 2024to 1 May 2031
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants eligible for inclusion in this study have to meet the following criteria:
- Myelofibrosis MF Monotherapy:
- Participants who are not candidates for, intolerant of, or relapsed/refractory to approved JAKi (ruxolitinib and / or fedratinib / pacritinib) or when further benefit from therapy is not anticipated per investigator. Participants not eligible for JAKi therapy irrespectively of previous treatments. Prior JAKi therapy is not required.
- Myelofibrosis Ruxolitinib Combination MF participants treated with Ruxolitinib for at least 3 months on a stable, uninterrupted dose for at least 2 months prior to study enrollment AND have suboptimal response (palpable spleen of ≥5 cm, or total symptoms score of ≥10) or progressive anemia/thrombocytopenia/neutropenia.
- AND all the below criteria in both cohorts:
- Must be diagnosed with treatment requiring PMF or post ET/PV MF diagnosed according to the 2016 World Health Organization with intermediate -1, intermediate -2 or high-risk disease according to the DIPSS prognostic scoring system, or if with low risk disease then with symptomatic splenomegaly that is ≥ 5 cm below left costal margin by physical exam.
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- Peripheral or bone marrow blasts must be < 10%
- Participants must provide written informed consent.
- Age 18 years or older. Because no dosing or adverse event data are currently available on the use of tasquinimod as monotherapy or in combination with ruxolitinib in patients <18 years of age, children are excluded from this study.
- Willing and able to comply with scheduled visits, treatment plan and laboratory tests.
- Participant is able to swallow and retain oral medication.
- ECOG performance status 0-2.
- Required baseline laboratory status:
- Absolute neutrophil count (ANC) ≥ 1.0 x 109/L (1000/mm3)
- Serum direct bilirubin ≤ 1.0 x ULN (upper limit of normal)
- AST (SGOT) or ALT (SGPT) [if both measured, then this applies to both measurements] ≤ 2.5 x ULN, except for participants with MF involvement of the liver who must have levels ≤ 5 x ULN
- Glomerular Filtration Rate (GFR) of ≥ 30 ml/min based on Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation using serum or plasma creatinine or cystatin-C.
- Treatment-related toxicities from prior therapies must have resolved to Grade ≤ 1.
- At least 2 weeks from prior investigational MF-directed treatment (till the start of study drug). This excludes concurrent ruxolitinib which is allowed in combination cohort. Hydroxyurea is allowed as standard cytoreductive therapy up until one day prior to initiation of therapy with tasquinimod. No other standard of care therapy for MF is allowed (as specified in the exclusion criteria)
- For women of childbearing potential, a documented negative serum or urine pregnancy test within 14 days prior to the administration of study drug.
- The effects of tasquinimod on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. (Refer to Pregnancy Assessment Policy MD Anderson Institutional Policy # CLN1114). This includes all female participants, between the onset of menses (as early as 8 years of age) and 55 years unless the patient presents with an applicable exclusionary factor which may be one of the following:
- Postmenopausal (no menses in greater than or equal to 12 consecutive months).
- History of hysterectomy or bilateral salpingo-oophorectomy.
- Ovarian failure (Follicle Stimulating Hormone and Estradiol in menopausal range, who have received Whole Pelvic Radiation Therapy).
- History of bilateral tubal ligation or another surgical sterilization procedure.
- Approved methods of birth control are as follows: Hormonal contraception (i.e. birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject/Partner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the trial and the drug washout period is an acceptable practice; however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.
- Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 6 months after completion of tasquinimod administration.
- Ability to understand and the willingness to sign a written informed consent document.
Who cannot join
- Participants eligible for this study must not meet any of the following criteria:
- Any concurrent severe and/or uncontrolled medical conditions that could increase the participant's risk for toxicity while in the study or that could confound discrimination between disease- and study treatment-related toxicities.
- Impaired cardiac function or clinically significant cardiac diseases, including any of the following:
- History or presence of ventricular tachyarrhythmia.
- Presence of unstable atrial fibrillation (ventricular response > 100 bpm); Participants with stable atrial fibrillation are eligible, provided they do not meet any of the other cardiac exclusion criteria.
- Clinically significant resting bradycardia (< 50 bpm).
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- Angina pectoris or acute myocardial infarction ≤ 3 months prior to starting study drug.
- Other clinically significant heart disease (e.g., symptomatic congestive heart failure; uncontrolled arrhythmia or hypertension; history of labile hypertension or poor compliance with an antihypertensive regimen).
- Participants who are currently receiving chronic (> 14 days) treatment with corticosteroids at a dose ≥ 10 mg of prednisone (or its glucocorticoid equivalent) per day, or any other chronic immunosuppressive treatment that cannot be discontinued prior to starting study drug.
- Treatment with chemotherapy (except hydroxyurea within 1 day prior to study treatment), immunomodulatory drug therapy (e.g. thalidomide, interferon-alpha), platelet-reducing therapy (e.g. anagrelide), immunosuppressive therapy, and erythropoetin use within 28 days prior to study treatment
- Treatment with experimental therapy within the past 2 weeks or 5 half-lives, whichever is shorter
- Treatment with tasquinimod at any time
- Participants with impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of tasquinimod as per physician opinion
- Participants with known or active (acute and chronic) Hepatitis A, B, or C; and Hepatitis B and C carriers, HIV. Participants are excluded regardless of detectability of viral load (lack of safety data).
- Participants with clinically significant bacterial, fungal, parasitic or viral infection which require therapy
- History of pancreatitis
- History of malabsorption or other condition that would interfere with absorption of study drugs (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, small bowel resection)
- Systemic treatment within 14 days prior to the initiation of study treatment with any of the following moderate or strong inhibitor, or moderate or strong inducer of cytochrome P-3A4 (CYP3A4): imidazoles (e.g. ketoconazole), protease inhibitors (e.g. ritonavir), macrolides (e.g. erythromycin), rifampicin, rifabutin, phenytoin, carbamazepine, St. John's wort.
- Need for ongoing therapy with any of the following drug substances of narrow therapeutic range that are metabolized mainly by CYP3A4: alfentanil, fentanyl, quinidine, astemizole, terfenadine, sirolimus, tacrolimus, cyclosporine, cisapride, and ergotamine
- Need for ongoing therapy with any of the following drug substances of narrow therapeutic range metabolized mainly by CYP1A2: duloxetine, palonosetron, theophylline, tizanidine, and ondansetron.
- Need for ongoing therapy with any of the following drug substances of narrow therapeutic range metabolized mainly by CYP2D6: Dosulepin, Flecainide, Sotalol, Pimozide, Procainamide, Clonidine, Desipramine, Clomipramine, Amitriptyline, Imipramine, Nortriptyline, Trimipramine, Amoxapine, Dronedarone, Phenytoin
- Ongoing treatment with warfarin, unless the INR is <=3.0.
- Known hypersensitivity to tasquinimod or any excipients in the study treatments
- Any other condition that would, in the Investigator's judgment, contraindicate subject's participation in the clinical study due to safety concerns or compliance with clinical study procedures
- Prior inclusion in this study
- Pregnant women are excluded from this study because tasquinimod is an agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with tasquinimod, breastfeeding should be discontinued if the mother is treated with tasquinimod. These potential risks may also apply to other agents used in this study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
Lucia Masarova, MD, 832-750-4211, lmasarova@mdanderson.org
Lucia Masarova, MD
About this study
To learn if tasquinimod either alone or in combination with ruxolitinib can help to control PMF, post-PV MF, or post-ET MF.
Read the detailed description
Primary Objectives To determine anti-tumor activity of tasquinimod in patients with PMF, post-PV MF, and post-ET MF in a monotherapy and in combination with stable dose of ruxolitinib based on the measurement of the objective response rate (ORR) which is defined as the proportion of patients with CR (complete remission), PR (partial remission), or CI (clinical improvement) after six cycles of treatment, according to the International Working Group (IWG) consensus criteria.
Secondary Objectives To determine safety of tasquinimod in patients with PMF, post-PV MF, and post-ET MF in a monotherapy and in combination with stable dose of ruxolitinib
To determine time to response and response duration.
To assess changes in symptom burden as assessed by Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MFSAF v4.0).
To assess changes in bone marrow fibrosis grade.
To assess the pharmacokinetics (PK) of tasquinimod in blood to determine whether the systemic exposure of tasquinimod, when administered alone or in combination with ruxolitinib in patients with MF.
To assess correlation of response / resistance to tasquinimod with baseline genetic markers (cytogenetic alterations and mutations determined by NextGen sequencing of an 81-gene myeloid panel).
- Conditions
- Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-Essential Thrombocytosis Myelofibrosis
- Being tested
- Ruxolitinib, Tasquinimod
- Medical subject headings
- Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RuxolitinibDrug
Also called Jakafi, INCB018424, INC424
Given by PO
TasquinimodDrug
Given by PO
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Experimental | Participants have already been receiving ruxolitinib for at least 3 months and are not responding well to ruxolitinib or have low blood cell counts, participants will be assigned to Group 1. In Group 1, participants will receive tasquinimod alone daily each 28 day cycle. Receives: Drug: Tasquinimod |
| Group 2 | Experimental | Participants who are already on a stable dose of ruxolitinib for at least 8 weeks who have achieved a sub-optimal response will be assigned to Group 2. In Group 2, participants will receive tasquinimod daily on days 1-28 and ruxolitinib dose will be continued. Receives: Drug: Ruxolitinib; Drug: Tasquinimod |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and adverse events (AEs)
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Measured: Through study completion; an average of 1 year.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2024
- Study start1 Aug 2024
- Primary completion1 May 2029 (estimated)
- Study completion1 May 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary MyelofibrosisStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 33 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Active Biotech AB
- Responsible party
- Sponsor
- Investigators
- Lucia Masarova, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06327100
- Sponsor's protocol ID
- 2023-0934
- Other IDs
- NCI-2024-02551
- Status last verified
- May 2026
- First submitted
- 15 Mar 2024
- Last updated
- 26 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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)
- ClinicalTrials.gov ID
- NCT06327100
- Acronym / sponsor ID
- 2023-0934
- Conditions
- Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-Essential Thrombocytosis Myelofibrosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 33 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Collaborators
- Active Biotech AB
- Study start
- 1 Aug 2024 (actual)
- Primary completion
- 1 May 2029 (estimated)
- Study completion
- 1 May 2031 (estimated)
- First posted
- 25 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To learn if tasquinimod either alone or in combination with ruxolitinib can help to control PMF, post-PV MF, or post-ET MF.
Detailed description
Primary Objectives To determine anti-tumor activity of tasquinimod in patients with PMF, post-PV MF, and post-ET MF in a monotherapy and in combination with stable dose of ruxolitinib based on the measurement of the objective response rate (ORR) which is defined as the proportion of patients with CR (complete remission), PR (partial remission), or CI (clinical improvement) after six cycles of treatment, according to the International Working Group (IWG) consensus criteria.
Secondary Objectives To determine safety of tasquinimod in patients with PMF, post-PV MF, and post-ET MF in a monotherapy and in combination with stable dose of ruxolitinib
To determine time to response and response duration.
To assess changes in symptom burden as assessed by Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score (MFSAF v4.0).
To assess changes in bone marrow fibrosis grade.
To assess the pharmacokinetics (PK) of tasquinimod in blood to determine whether the systemic exposure of tasquinimod, when administered alone or in combination with ruxolitinib in patients with MF.
To assess correlation of response / resistance to tasquinimod with baseline genetic markers (cytogenetic alterations and mutations determined by NextGen sequencing of an 81-gene myeloid panel).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group 1 | Experimental | Drug: TasquinimodParticipants have already been receiving ruxolitinib for at least 3 months and are not responding well to ruxolitinib or have low blood cell counts, participants will be assigned to Group 1. In Group 1, participants will receive tasquinimod alone daily each 28 day cycle. |
| Group 2 | Experimental | Drug: Ruxolitinib, Drug: TasquinimodParticipants who are already on a stable dose of ruxolitinib for at least 8 weeks who have achieved a sub-optimal response will be assigned to Group 2. In Group 2, participants will receive tasquinimod daily on days 1-28 and ruxolitinib dose will be continued. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (28 listed)
- Participants who are not candidates for, intolerant of, or relapsed/refractory to approved JAKi (ruxolitinib and / or fedratinib / pacritinib) or when further benefit from therapy is not anticipated per investigator. Participants not eligible for JAKi therapy irrespectively of previous treatments. Prior JAKi therapy is not required.
- Myelofibrosis Ruxolitinib Combination MF participants treated with Ruxolitinib for at least 3 months on a stable, uninterrupted dose for at least 2 months prior to study enrollment AND have suboptimal response (palpable spleen of ≥5 cm, or total symptoms score of ≥10) or progressive anemia/thrombocytopenia/neutropenia.
- Must be diagnosed with treatment requiring PMF or post ET/PV MF diagnosed according to the 2016 World Health Organization with intermediate -1, intermediate -2 or high-risk disease according to the DIPSS prognostic scoring system, or if with low risk disease then with symptomatic splenomegaly that is ≥ 5 cm below left costal margin by physical exam.
- Peripheral or bone marrow blasts must be < 10%
- Participants must provide written informed consent.
Key exclusion criteria (26 listed)
- Any concurrent severe and/or uncontrolled medical conditions that could increase the participant's risk for toxicity while in the study or that could confound discrimination between disease- and study treatment-related toxicities.
- Impaired cardiac function or clinically significant cardiac diseases, including any of the following:
- Participants who are currently receiving chronic (> 14 days) treatment with corticosteroids at a dose ≥ 10 mg of prednisone (or its glucocorticoid equivalent) per day, or any other chronic immunosuppressive treatment that cannot be discontinued prior to starting study drug.
- Treatment with chemotherapy (except hydroxyurea within 1 day prior to study treatment), immunomodulatory drug therapy (e.g. thalidomide, interferon-alpha), platelet-reducing therapy (e.g. anagrelide), immunosuppressive therapy, and erythropoetin use within 28 days prior to study treatment
- Treatment with experimental therapy within the past 2 weeks or 5 half-lives, whichever is shorter
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RuxolitinibAlso: Jakafi, INCB018424, INC424Given by PO | Drug | Group 2 |
| TasquinimodGiven by PO | Drug | Group 1, Group 2 |
Primary outcome
Safety and adverse events (AEs)
Time frame: Through study completion; an average of 1 year.
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 May 2026.
- MD Anderson Cancer CenterLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Lucia Masarova, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Ruxolitinib and Tasquinimod in people with Primary Myelofibrosis and Post-polycythemia Vera Myelofibrosis, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 28 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 26 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lucia Masarova, MD ((832) 750-4211, lmasarova@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and adverse events (AEs) (time frame: Through study completion; an average of 1 year.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 May 2026.