New pill shows promise for rare bone marrow cancer

NCT ID NCT04176198

Disease control Sponsor: Sumitomo Pharma America, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 04, 2026 · Updated 2 times

Summary

This study tests an experimental pill called nuvisertib for people with myelofibrosis, a rare bone marrow disorder. It is for patients whose disease is moderate to high risk and who have not responded well to standard treatments. The goal is to see if the drug is safe and can shrink an enlarged spleen and improve symptoms.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 240 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2019

Expected to finish

Apr 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Patients must meet all of the following inclusion criteria to be eligible: Nuvisertib (TP-3654) Monotherapy Arm: * Confirmed pathological diagnosis of primary myelofibrosis (PMF) or post-PV-MF/post-ET- MF and intermediate or high-risk primary or secondary MF * Previously treated with JAK inhibitor(s) and is intolerant, resistant, refractory or has lost response to the JAK inhibitor(s) or is ineligible to be treated with JAK inhibitor * Fulfill the following clinical laboratory parameters: * Platelet count ≥ 25 x 10\^9 /L, without assistance of growth factors or platelet transfusions * ANC ≥ 1 x 10\^9/L without assistance of granulocyte growth factors * Peripheral blood blast count \< 5% * ECOG performance status ≤ 1 * Life expectancy ≥ 6 months * Adequate renal function * Adequate hepatic function * Adequate coagulation function * Splenomegaly (spleen volume of ≥ 450 cm3 by MRI or CT scan) within 2 weeks prior to Cycle 1 Day 1. * Dose escalation: At least 2 symptoms measurable (score ≥ 1) using the MF-SAF * Dose expansion: At least 2 symptoms measurable with each score of ≥ 3 or a total average score of ≥ 10 per MFSAF Nuvisertib (TP-3654) + Ruxolitinib Arm: * Confirmed pathological diagnosis of PMF or post-PV-MF/post ET- MF and intermediate or high-risk primary or secondary MF * On ruxolitinib treatment for ≥ 6 months, and on a stable dose of ruxolitinib (5 to 25 mg BID) for ≥ 8 weeks prior to the first dose of nuvisertib, but has either lost response or had a suboptimal or plateau in response * Fulfills the following clinical laboratory parameters: * Platelet count ≥ 50 × 10\^9/L (without assistance of growth factors or platelet transfusions) * ANC ≥ 1 × 109/L without assistance of granulocyte growth factors * Peripheral blood blast count \< 5% at screening * Adequate renal function * Adequate hepatic function * Adequate coagulation function * Splenomegaly (spleen volume of ≥ 450 cm3 by MRI/CT scan) within 2 weeks prior to Cycle 1 Day 1 * At least 2 symptoms measurable with each score ≥ 3 or a total average score of ≥ 10 per MFSAF v4.0 * ECOG performance status ≤ 1 * Life expectancy ≥ 6 months Nuvisertib (TP-3654) + Momelotinib Arm * Confirmed pathological diagnosis of PMF or post-PV-MF/post ET-MF and intermediate or high-risk primary or secondary MF * Previously treated with an approved JAK inhibitor (except momelotinib) for PMF or Post-PV/ET MF for ≥ 12 weeks, or ≥ 4 weeks if JAK inhibitor therapy was complicated by a transfusion requirement of ≥ 4 units of red blood cells in 8 weeks, or Grade 3/4 AEs of thrombocytopenia, anemia, or hematoma * Fulfills the following clinical laboratory parameters: * Anemic, defined as Hb \<10 g/dL or requiring RBC transfusion at baseline * Platelet count ≥ 50 × 109/L (without assistance of growth factors or platelet transfusions) * ANC ≥ 1 × 109/L without assistance of granulocyte growth factors * Peripheral blood blast count \< 5% at screening * Adequate renal function * Adequate hepatic function * Adequate coagulation function * Splenomegaly (spleen volume of ≥ 450 cm3 by MRI/CT scan) within 2 weeks prior to Cycle 1 Day 1 * At least 2 symptoms measurable with each score of ≥ 3 or a total average score of ≥ 10 per MFSAF v4.0 * ECOG performance status ≤ 1 * Life expectancy ≥ 6 months Patients meeting any one of these exclusion criteria will be prohibited from participating in this study: Nuvisertib (TP-3654) Monotherapy Arm: * Received previous systemic antineoplastic therapy or any experimental therapy within 2 weeks or 5 half-lives, whichever is longer, prior to Cycle 1 Day 1. Hydroxyurea or anagrelide are allowed up to 24 hours prior to Cycle 1 Day 1). * Major surgery within 4 weeks prior to Cycle 1 Day 1 and/or not recovered adequately from from surgery prior to first dose. * Splenic irradiation within 6 months prior to Screening or prior splenectomy. * Prior allogeneic stem cell transplant within the last 6 months. * Eligible for allogeneic bone marrow or stem cell transplantation. * Unresolved Grade ≥ 2 non-hematological toxicity related to prior treatment * History of symptomatic congestive heart failure, or myocardial infarction, or uncontrolled arrhythmia within 6 months prior to Cycle 1 Day 1; left ventricular ejection fraction (LVEF) \< 45% by echocardiogram within 4 weeks prior to Cycle 1 Day 1. * Corrected QT interval \> 480msec. * Prior or concurrent malignancy that could interfere with the investigational regime. * Known history of chronic liver disease, e.g. portal hypertension or any of its complications, cirrhosis, Child-Pugh C, auto-immune hepatitis, alpha-1 anti-trypsin deficiency, Wilson's disease, etc. * Active, uncontrolled bacterial, viral, or fungal infections, requiring systemic antimicrobial within 1 week prior to Cycle 1 Day 1. * Chronic active or acute viral hepatitis A, B, or C infection (testing for hepatitis B and C are required) * Exhibited allergic reactions or sensitivity to nuvisertib, or similar compound. * Medical condition or GI tract surgery that could impair absorption or result in short bowel syndrome with diarrhea. * Systemic steroid therapy (\>10 mg daily prednisone or equivalent) within 1 week prior to the first dose of study treatment (note: topical, inhaled, nasal, and ophthalmic steroids are not prohibited). * Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or thalassemia, or severe GI bleeding. * Pregnant or breastfeeding * Currently receiving any other investigational agent. Nuvisertib (TP-3654) + Ruxolitinib Arm: * Received previous systemic antineoplastic therapy (other than ruxolitinib) or any other experimental therapy within 2 weeks or 5 half-lives, whichever is longer, prior to Cycle 1 Day 1 (Note: Prior treatment with nuvisertib is not allowed. Hydroxyurea or anagrelide are allowed up to 24 hours prior to Cycle 1 Day 1). * Received systemic steroid therapy (\>10 mg daily prednisone or equivalent) within 1 week prior to Cycle 1 Day 1 (Note: Topical, inhaled, nasal, and ophthalmic steroids are not prohibited) * Known allergic reactions or sensitivity to nuvisertib, or similar compound. * Splenic irradiation within 6 months prior to Screening or prior splenectomy * Prior allogeneic stem cell transplant within the last 6 months (Note: Patients who have relapsed after 6 months post-transplant and do not have active GVHD are eligible). * Eligible for allogeneic bone marrow or stem cell transplantation (Note: Patients who are not willing to undergo transplantation or for whom a suitable donor is not available are considered as transplant ineligible.) * Major surgery within 4 weeks prior to Cycle 1 Day 1 and/or have not recovered adequately prior to first dose. * Active, uncontrolled bacterial, viral, or fungal infections, requiring parenteral antimicrobial within 1 week prior to Cycle 1 Day 1 * Chronic active or acute viral hepatitis A, B, or C infection (testing for hepatitis B and C are required) * Known history of chronic liver disease (eg, portal hypertension or any of its complications, cirrhosis, Child-Pugh C, auto-immune hepatitis, alpha-1 anti-trypsin deficiency, Wilson's disease, etc) (Note: Abnormal liver morphology at baseline imaging may require additional testing, as needed). * Unresolved Grade ≥ 2 non-hematological adverse events related to prior treatment (stable Grade 2 conditions may be permitted in consultation with the Sponsor) * History of myocardial infarction or symptomatic congestive heart failure or uncontrolled arrhythmia within 6 months prior to Cycle 1 Day 1; LVEF \<45% by echocardiogram within 4 weeks prior to Cycle 1 Day 1 * Corrected QTcF of \> 480 msec * Prior or concurrent malignancy that could interfere with the safety or efficacy assessment of the study intervention * History of a medical condition or GI tract surgery that could impair absorption or could result in short bowel syndrome with diarrhea * Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or thalassemia, or severe GI bleeding * Pregnant or breastfeeding Nuvisertib (TP-3654) + Momelotinib Arm: * Received previous systemic antineoplastic therapy or any experimental therapy within 2 weeks or 5 half-lives, whichever is longer, prior to Cycle 1 Day 1 (Notes: Prior treatment with momelotinib or nuvisertib is not allowed; in patients with ongoing JAK inhibitor therapy, ie, ruxolitinib, at screening, JAK inhibitor therapy must be tapered over a period of at least 1 week. Patients on a low dose of ruxolitinib (eg, 5 mg QD) may have a reduced taper period or no taper; hydroxyurea or anagrelide are allowed up to 24 hours prior to Cycle 1 Day 1). * Received systemic steroid therapy (\>10 mg daily prednisone or equivalent) within 1 week prior to Cycle 1 Day 1 (Note: Topical, inhaled, nasal, and ophthalmic steroids are not prohibited). * Known allergic reactions or sensitivity to nuvisertib, momelotinib, or any structurally similar drug, or to any component of the formulations of either study intervention * Splenic irradiation within 6 months prior to screening or prior splenectomy * Prior allogenic stem cell transplant within the last 6 months (Note: Patients who have relapsed after 6 months post-transplant and do not have active GVHD are eligible). * Eligible for allogeneic bone marrow or stem cell transplantation (Note: Patients who are not willing to undergo transplantation or for whom a suitable donor is not available are considered as transplant ineligible). * Major surgery within 4 weeks prior to Cycle 1 Day 1 and/or have not recovered adequately from surgery prior to first dose. * Active, uncontrolled bacterial, viral, or fungal infections, requiring parenteral antimicrobial within 1 week prior to Cycle 1 Day 1 * Chronic active or acute viral hepatitis A, B, or C infection (testing for hepatitis B and C are required) * Known history of chronic liver disease (eg, portal hypertension or any of its complications, cirrhosis, Child-Pugh C, auto-immune hepatitis, alpha-1 anti-trypsin deficiency, Wilson's disease, etc) (Note: Abnormal liver morphology at baseline imaging may require additional testing, as needed) * Unresolved Grade ≥ 2 non-hematological adverse events related to prior treatment (stable Grade 2 conditions may be permitted in consultation with the Sponsor) * Presence of Grade ≥ 2 peripheral neuropathy * History of myocardial infarction or symptomatic congestive heart failure or uncontrolled arrhythmia within 6 months prior to Cycle 1 Day 1; LVEF \< 45% by echocardiogram within 4 weeks prior to Cycle 1 Day 1 * Corrected QTcF of \> 480 msec * Prior or concurrent malignancy that could interfere with the safety or efficacy assessment of the study intervention * History of a medical condition or GI tract surgery that could impair absorption or could result in short bowel syndrome with diarrhea * Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or thalassemia, or severe GI bleeding * Pregnant or breastfeeding

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Conditions

The condition(s) this trial relates to.

myelofibrosis primary myelofibrosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Myelofibrosis

How to take part

Only the study team decides who joins. These are the ways to reach them.

The places running it

86 sites in 10 countries. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

Open the record ↗
A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

ASST - Spedali Civili di Brescia
RECRUITING

Brescia, Italy
Aichi Medical University Hospital
RECRUITING

Aichi, Japan
Aomori Prefectural Central Hospital
RECRUITING

Aomori, Japan
Azienda Ospedaliera Nazionale SS. Antonio e Biagio e Cesare Arrigo
RECRUITING

Alessandria, 15121, Italy
Azienda Ospedaliera Universitaria Citta' Della Salute E della Scienza di Torino
RECRUITING

Torino, 10126, Italy
Baptist Health - Miami Cancer Institute
RECRUITING

Miami, Florida, 33176, United States
Blood Cancer Center
RECRUITING

Denver, Colorado, 80218, United States
CHU Angers
RECRUITING

Angers, 9000, France
CHU de Liege
RECRUITING

Liège, 4000, Belgium
Centre Hospitalier Lyon Sud
RECRUITING

Lyon, 69495, France
Centre Hospitalier Universitaire D'Amiens
RECRUITING

Amiens, 80054, France
City of Hope
RECRUITING

Duarte, California, 91010, United States
Duke Cancer Institute
RECRUITING

Durham, North Carolina, 27710, United States
Eastern Health Box Hill Hospital
RECRUITING

Box Hill, Victoria, Australia
Emory University
RECRUITING

Atlanta, Georgia, 30322, United States
Epworth Healthcare
RECRUITING

Richmond, Victoria, Australia
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
RECRUITING

Milan, 20122, Italy
Hoag Family Cancer Institute
RECRUITING

Newport Beach, California, 92663, United States
Hokkaido University Hospital
RECRUITING

Hokkaido, Japan
Hospital Saint Louis
RECRUITING

Paris, 75010, France
Hospital Universitario de Salamanca
RECRUITING

Salamanca, Spain
Hospital Universitario y Politecnico La Fe
RECRUITING

Barcelona, Spain
Hospitalier Universitaire (CHU) de Nice - Hopital de l'Archet
RECRUITING

Nice, 06200, France
Huntsman Cancer Institute
RECRUITING

Salt Lake City, Utah, 84112, United States
IRCCS Azienda Ospedaliero -Universitaria Di Bologna - Dipartimento Malattie Oncologiche ed Ematologiche - UO Ematologia
RECRUITING

Bologna, 40138, Italy
IRCCS istituto Romagnolo per lo studio dei tumori "Dino Amadori"
RECRUITING

Meldola, 47014, Italy
Icahn School of Medicine at Mount Sinai
RECRUITING

New York, New York, 10029, United States
Icon Cancer Centre (Ashford Cancer Centre Research)
RECRUITING

Adelaide, Australia
Institut Catala d'Oncologia
RECRUITING

Barcelona, Spain
Institut Gustave Roussy
RECRUITING

Villejuif, 94805, France
Institut de cancerologie du Gard
RECRUITING

Nîmes, 30029, France
Institut de cancerologie du Gard
RECRUITING

Nîmes, France
Istituto Nazionale Tumori, IRCCS Centro di Riferimento Oncologico di Aviano
RECRUITING

Aviano, 33081, Italy
Jewish General Hospital
RECRUITING

Montreal, Quebec, H3T 1E2, Canada
John Theurer Cancer Center at Hackensack University Medical Center
RECRUITING

Hackensack, New Jersey, 07601, United States
Juntendo University Hospital
RECRUITING

Tokyo, Japan
Juravinski Cancer Center
NOT_YET_RECRUITING

Hamilton, Ontario, L8V 5C2, Canada
Kyushu University Hospital
RECRUITING

Fukuoka, Japan
Lincoln County Hospital
RECRUITING

Lincoln, United Kingdom
MD Anderson Cancer Center
RECRUITING

Houston, Texas, 77054, United States
Massachusetts General Hospital
RECRUITING

Boston, Massachusetts, 02114, United States
Medical University of South Carolina
RECRUITING

Charleston, South Carolina, 29425, United States
Memorial Sloan Kettering Cancer Center
RECRUITING

New York, New York, 10065, United States
Mie University Hospital
RECRUITING

Tsu, Japan
Monash University
RECRUITING

Clayton, Victoria, Australia
Montefiore Cancer Center
RECRUITING

The Bronx, New York, 10461, United States
National Cancer Center Hospital East
RECRUITING

Chiba, Japan
Ohio State University
RECRUITING

Columbus, Ohio, 43210, United States
Okayama University Hospital
RECRUITING

Okayama, Japan
Oxford University Hospitals NHS Foundation
RECRUITING

Oxford, United Kingdom
Peter McCallum Center
RECRUITING

Melbourne, Victoria, Australia
Princess Margaret Cancer Center
RECRUITING

Toronto, Ontario, M5G 2M9, Canada
Roswell Park Comprehensive Cancer Center
COMPLETED

Buffalo, New York, 14263, United States
Royal Adelaide Hospital
RECRUITING

Adelaide, South Australia, Australia
Saitama Medical Center
COMPLETED

Saitama, Japan
Shizuoka Cancer Center
COMPLETED

Shizuoka, Japan
Shonan Kamakura General Hospital
RECRUITING

Kamakura, 247-8533, Japan
St Vincent's Hospital Melbourne
RECRUITING

Fitzroy, Victoria, Australia
St. Paul's Hospital Hematology/Oncology Research
RECRUITING

Vancouver, British Columbia, V6T 1Z3, Canada
The University of Arizona Cancer Center
RECRUITING

Tucson, Arizona, 85724, United States
The University of Osaka Hospital
RECRUITING

Osaka, Japan
Tohoku University Hospital
RECRUITING

Sendai, Japan
Tokyo Medical University Hospital
RECRUITING

Tokyo, 160-0023, Japan
Tri-Star Centennial Medical Center
RECRUITING

Nashville, Tennessee, 37203, United States
United Lincolnshire Hospitals NHS Trust - Pilgrim Hospital
RECRUITING

Lincoln, United Kingdom
Universitair Ziekenhuis Gent
RECRUITING

Ghent, 9000, Belgium
University College London Hospital's NHS foundation Trust
RECRUITING

London, United Kingdom
University Hospital of Poitiers
RECRUITING

Poitiers, 86021, France
University Hospitals Leuven
RECRUITING

Leuven, Vlaams-Brabant, 3000, Belgium
University of Alabama
RECRUITING

Birmingham, Alabama, 35294, United States
University of British Columbia
RECRUITING

Vancouver, British Columbia, V6T 1Z3, Canada
University of Calgary
RECRUITING

Calgary, Alberta, T2N 1N4, Canada
University of Chicago
RECRUITING

Chicago, Illinois, 60637, United States
University of Florida Health Shands Cancer Hospital
COMPLETED

Gainesville, Florida, 32608, United States
University of Maryland
RECRUITING

Baltimore, Maryland, 21201, United States
University of Miami
RECRUITING

Miami, Florida, 33136, United States
University of Michigan
RECRUITING

Ann Arbor, Michigan, 48109, United States
University of Minnesota
RECRUITING

Minneapolis, Minnesota, 55455, United States
University of Miyazaki Hospital
RECRUITING

Miyazaki, Japan
University of Southern California
RECRUITING

Los Angeles, California, 90033, United States
University of Virginia Cancer Center
RECRUITING

Charlottesville, Virginia, 22903, United States
University of Washington - Fred Hutchinson Cancer Center
RECRUITING

Seattle, Washington, 98109, United States
Universitätsklinikum Schleswig-Holstein
RECRUITING

Lübeck, Germany
Universitätsmedizin Halle
RECRUITING

Halle, Germany
Vanderbilt University
RECRUITING

Nashville, Tennessee, 37232, United States
Washington University of Medicine
RECRUITING

St Louis, Missouri, 63110, United States
Weill Cornell Medical Center
RECRUITING

New York, New York, 10065, United States
Yale School of Medicine
RECRUITING

New Haven, Connecticut, 06510, United States
ZNA Cadix
RECRUITING

Antwerp, 2020, Belgium
ZNA Middelheim
RECRUITING

Antwerp, 2030, Belgium

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