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)

NCT06327100 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: 2023-0934NCI-2024-02551
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 1

Brief Summary

To learn if tasquinimod either alone or in combination with ruxolitinib can help to control PMF, post-PV MF, or post-ET MF.

Conditions

Post-Essential Thrombocytosis Myelofibrosis; Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis

Outcome Measures

Primary Outcomes (1)

• 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

  Through study completion; an average of 1 year.

Study Arms (2)

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.

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.

Drug: Ruxolitinib; Drug: Tasquinimod

Interventions

Ruxolitinib DRUG

Given by PO

Also known as: Jakafi, INCB018424, INC424

Group 2

Tasquinimod DRUG

Given by PO

Group 1; Group 2

Eligibility Criteria

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

You may qualify if:

• 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.
• 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

You may not qualify if:

• For women of childbearing potential, a documented negative serum or urine pregnancy test within 14 days prior to the administration of study drug.
• 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.
• 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.
• Clinically significant resting bradycardia (\< 50 bpm).
• 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).

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• Active Biotech AB: collaborator

Study Sites (1)

MD Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Related Links

• MD Anderson Cancer Center

MeSH Terms

Conditions

Primary Myelofibrosis

Interventions

ruxolitinib; tasquinimod

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Lucia Masarova, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Lucia Masarova, MD

CONTACT

(832) 750-4211; lmasarova@mdanderson.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 15, 2024
• First Posted: March 25, 2024
• Study Start: August 1, 2024
• Primary Completion (Estimated): May 1, 2029
• Study Completion (Estimated): May 1, 2031
• Last Updated: February 27, 2026

Record last verified: 2026-02

Locations

US (1)