AzTrials
AzTrials

Myelofibrosis (MF)Phase 3TRANSFORM-1NCT04472598

Study of Oral Navitoclax Tablet In Combination With Oral Ruxolitinib Tablet When Compared With Oral Ruxolitinib Tablet To Assess Change In Spleen Volume In Adult Participants With Myelofibrosis

A Phase 3 study testing Ruxolitinib and Navitoclax in people with Myelofibrosis (MF), run by AbbVie at 194 sites in 25 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

Myelofibrosis is a type of bone marrow cancer that usually develops slowly and disrupts body's normal production of blood cells. It causes bone marrow scarring, leading to severe anemia that can cause weakness and fatigue. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelofibrosis (MF)
What is being tested?
Ruxolitinib and Navitoclax
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
252 participants
Where
194 sitesin 25 countries
Study dates
29 Sep 2020to 29 Jan 2025
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

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

  • Documented diagnosis of Primary MyeloFibrosis (MF) as defined by World Health Organization (WHO) classification or Secondary MF (post polycythemia vera [PPV] - MF or Post Essential Thrombocythemia [PET] - MF) .
  • Must be able to complete the MF Symptom Assessment Form (MFSAF) v4.0 on at least 4 out of 7 days immediately preceding the date of randomization.
  • -- Must have at least 2 symptoms with a score >=3 or a total score of >=12, as measured by the MFSAF v4.0.
  • Classified as intermediate-2, or high-Risk MF as defined by the Dynamic International Prognostic Scoring System Plus (DIPSS+).
  • Has splenomegaly defined as spleen palpation measurement >= 5 centimeters (cm) below costal margin or spleen volume greater than or equal to 450 cubic cm as assessed centrally by magnetic resonance imaging (MRI) or computed tomography (CT) scan.
  • Ineligible for stem cell transplantation at time of study entry due to age, comorbidities, or unfit for unrelated or unmatched donor transplant and other criteria per National Comprehensive Cancer Network guidelines.
  • Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.

Who cannot join

  • Prior treatment with a Janus Kinase-2 (JAK-2) inhibitor.
  • Prior treatment with a B-cell lymphoma 2 homology 3 (BH3)-mimetic compound or bromodomain and extra-terminal motif (BET) inhibitor or stem cell transplant.
  • Receiving medication that interferes with coagulation or platelet function within 3 days prior to the first dose of study drug or during the study treatment period except for low dose aspirin (up to 100 milligram daily) and low molecular weight heparin (LMWH).

Trial locations and contacts

194 sites in 25 countries.

194 sites in 25 countries

United States35 sites

  • University of Michigan /ID# 221658

    Ann Arbor, Michigan, 48109-5000

  • Duplicate_Emory University /ID# 221562

    Atlanta, Georgia, 30322-1013

  • Augusta University Georgia Cancer Center /ID# 221551

    Augusta, Georgia, 30912-0003

  • Lynn Cancer Institute, Boca /ID# 230687

    Boca Raton, Florida, 33486

  • Massachusetts General Hospital /ID# 221559

    Boston, Massachusetts, 02114

  • Beth Israel Deaconess Medical Center /ID# 224261

    Boston, Massachusetts, 02215-5400

  • Dana-Farber Cancer Institute /ID# 218010

    Boston, Massachusetts, 02215

  • Gabrail Cancer Center Research /ID# 230488

    Canton, Ohio, 44718

  • Duplicate_Rush University Medical Center /ID# 221581

    Chicago, Illinois, 60612

  • Oncology Hematology Care, Inc. /ID# 222556

    Cincinnati, Ohio, 45236-2725

  • Columbus Regional Research Institute /ID# 227272

    Columbus, Georgia, 31904-8915

  • The Ohio State University /ID# 221584

    Columbus, Ohio, 43210

  • Minnesota Oncology Hematology /ID# 227357

    Edina, Minnesota, 55435

  • Virginia Cancer Specialists - Fairfax /ID# 242682

    Fairfax, Virginia, 22031

  • University of Kansas Cancer Center /ID# 218144

    Fairway, Kansas, 66205-2528

  • Florida Cancer Specialist - South /ID# 221726

    Fort Myers, Florida, 33901-8108

  • Providence - St. Jude Medical Center /ID# 241646

    Fullerton, California, 92835

  • Hackensack Univ Med Ctr /ID# 221654

    Hackensack, New Jersey, 07601

  • University of Texas MD Anderson Cancer Center /ID# 217994

    Houston, Texas, 77030

  • Indiana Blood & Marrow Transpl /ID# 221586

    Indianapolis, Indiana, 46237

  • MidAmerica Division, Inc. /ID# 221743

    Kansas City, Missouri, 64132

  • Thompson Cancer Survival Ctr /ID# 231689

    Knoxville, Tennessee, 37916

  • Moores Cancer Center at UC San Diego /ID# 218012

    La Jolla, California, 92093

  • Northwell Health - Monter Cancer Center /ID# 222996

    Lake Success, New York, 11042

  • Rocky Mountain Cancer Centers - Littleton /ID# 222562

    Littleton, Colorado, 80120

  • Weill Cornell Medical College /ID# 220933

    New York, New York, 10065

  • Mid Illinois Hematology & Oncology Associates, Ltd /ID# 224204

    Normal, Illinois, 61761

  • UPMC Hillman Cancer Ctr /ID# 218134

    Pittsburgh, Pennsylvania, 15232

  • Utah Cancer Specialists Salt Lake Clinic /ID# 221961

    Salt Lake City, Utah, 84106

  • University of Utah /ID# 221009

    Salt Lake City, Utah, 84112-5500

  • VA Puget Sound Health Care System /ID# 231691

    Seattle, Washington, 98108-1597

  • Highlands Oncology Group, PA /ID# 221824

    Springdale, Arkansas, 72762

  • Florida Cancer Specialists - North /ID# 221727

    St. Petersburg, Florida, 33705-1449

  • Texas Oncology - Northeast Texas /ID# 241813

    Tyler, Texas, 75702

  • Florida Cancer Specialists - East /ID# 221728

    West Palm Beach, Florida, 33401

Other trials in United States

Japan28 sites

  • Juntendo University Hospital /ID# 221405

    Bunkyo-ku, Tokyo, 113-8431

  • Nippon Medical School Hospital /ID# 221674

    Bunkyo-ku, Tokyo, 113-8602

  • Duplicate_Chiba University Hospital /ID# 239345

    Chiba, Chiba, 260-8677

  • University of Yamanashi Hospital /ID# 221701

    Chuo-shi, Yamanashi, 409-3821

  • Kyushu University Hospital /ID# 221783

    Fukuoka, Fukuoka, 812-8582

  • Fukushima Medical University Hospital /ID# 222752

    Fukushima, Fukushima, 960-1295

  • Kansai Medical University Hospital /ID# 221482

    Hirakata-shi, Osaka, 573-1191

  • Hitachi General Hospital /ID# 240048

    Hitachi-shi, Ibaraki, 317-0077

  • Juntendo University Shizuoka Hospital /ID# 221782

    Izunokuni-shi, Shizuoka, 410-2295

  • Kanazawa University Hospital /ID# 238424

    Kanazawa, Ishikawa-ken, 920-8641

  • Medical Corporation Seijinkai Ikeda Hospital /ID# 242172

    Kanoya-shi, Kagoshima-ken, 893-0024

  • National Cancer Center Hospital East /ID# 226093

    Kashiwa-shi, Chiba, 277-8577

  • Kobe University Hospital /ID# 246236

    Kobe, Hyōgo, 650-0017

  • Dokkyo Medical University Saitama Medical Center /ID# 222333

    Koshigaya, Saitama, 343-0845

  • Duplicate_Kawasaki Medical School Hospital /ID# 221481

    Kurashiki-shi, Okayama-ken, 701-0192

  • Kurashiki Central Hospital /ID# 221692

    Kurashiki-shi, Okayama-ken, 710-8602

  • Kyoto University Hospital /ID# 238423

    Kyoto, Kyoto, 606-8507

  • Gunmaken Saiseikai Maebashi Hospital /ID# 242806

    Maebashi, Gunma, 371-0821

  • Gunma University Hospital /ID# 221480

    Maebashi, Gunma, 371-8511

  • University of Miyazaki Hospital /ID# 221483

    Miyazaki, Miyazaki, 889-1692

  • Japanese Red Cross Aichi Medical Center Nagoya Daiichi Hospital /ID# 239100

    Nagoya, Aichi-ken, 453-8511

  • Ogaki Municipal Hospital /ID# 240173

    Ogaki-shi, Gifu, 503-8502

  • Kindai University Hospital /ID# 221479

    Osakasayama-shi, Osaka, 589-8511

  • Duplicate_Hokkaido University Hospital /ID# 242667

    Sapporo, Hokkaido, 060-8648

  • The University of Osaka Hospital /ID# 221478

    Suita-shi, Osaka, 565-0871

  • Ehime University Hospital /ID# 221443

    Toon-shi, Ehime, 791-0295

  • Fujita Health University Hospital /ID# 221537

    Toyoake, Aichi-ken, 470-1192

  • Mie University Hospital /ID# 221664

    Tsu, Mie-ken, 514-8507

Other trials in Japan

France11 sites

  • Chu Angers /Id# 219115

    Angers, 49933

  • Hopital Avicenne - APHP /ID# 221286

    Bobigny, Île-de-France Region, 93000

  • Centre Hospitalier Métropole Savoie - Site Hôpital de Chambéry /ID# 224506

    Chambéry, Savoie, 73007

  • CHU de Nantes, Hotel Dieu -HME /ID# 219113

    Nantes, Pays de la Loire Region, 44000

  • CHU NIMES - Hopital Caremeau /ID# 219114

    Nîmes, Gard, 30029

  • Hôpital Saint-Louis /ID# 221288

    Paris, 75010

  • AP-HP - Hopital Necker /ID# 231318

    Paris, 75015

  • Centre Hospitalier Universitaire de Bordeaux /ID# 222518

    Pessac, Gironde, 33604

  • HCL - Hopital Lyon Sud /ID# 222913

    Pierre-Bénite, Rhone, 69495

  • CH Roubaix - Hopital Victor Provo /ID# 219116

    Roubaix, Hauts-de-France, 59100

  • ICANS - Institut de Cancérologie Strasbourg Europe /ID# 229978

    Strasbourg, 67033

Other trials in France

Spain11 sites

  • Hospital Universitario Germans Trias i Pujol /ID# 229936

    Badalona, Barcelona, 08916

  • Hospital Parc de Salut del Mar /ID# 220913

    Barcelona, 08003

  • Hospital Universitario Vall d'Hebron /ID# 229690

    Barcelona, 08035

  • Hospital Universitario Dr. Negrin /ID# 220897

    Las Palmas de Gran Canaria, Las Palmas, 35019

  • CLINICA UNIVERSIDAD DE NAVARRA-Madrid /ID# 230721

    Madrid, 28027

  • Hospital Universitario Ramon y Cajal /ID# 220877

    Madrid, 28034

  • Hospital Universitario 12 de Octubre /ID# 229691

    Madrid, 28041

  • Hospital Universitario Virgen de la Victoria /ID# 220878

    Málaga, 29010

  • Clinica Universidad de Navarra - Pamplona /ID# 230720

    Pamplona, Navarre, 31008

  • Duplicate_Hospital Clínico Universitario de Santiago-CHUS /ID# 222264

    Santiago de Compostela, A Coruna, 15706

  • Hospital Clinico Universitario de Valencia /ID# 220875

    Valencia, 46010

Other trials in Spain

South Korea9 sites

  • Duplicate_Inje University Busan Paik Hospital /ID# 231667

    Busan, Busan Gwang Yeogsi, 47392

  • Pusan National University Hospital /ID# 222087

    Busan, Busan Gwang Yeogsi, 49241

  • Duplicate_Kyungpook National University Hospital /ID# 231666

    Daegu, Daegu Gwang Yeogsi, 41944

  • Gachon University Gil Medical Center /ID# 222089

    Incheon, Gyeonggido, 21565

  • Duplicate_Seoul National University Bundang Hospital /ID# 219053

    Seongnam, Gyeonggido, 13620

  • Seoul National University Hospital /ID# 219055

    Seoul, Seoul Teugbyeolsi, 03080

  • Asan Medical Center /ID# 219054

    Seoul, Seoul Teugbyeolsi, 05505

  • Samsung Medical Center /ID# 221068

    Seoul, Seoul Teugbyeolsi, 06351

  • The Catholic University of Korea, Seoul St. Marys Hospital /ID# 219056

    Seoul, Seoul Teugbyeolsi, 06591

Other trials in South Korea

Australia8 sites

  • The Kinghorn Cancer Centre /ID# 221503

    Darlinghurst, New South Wales, 2010

  • Townsville University Hospital /ID# 229794

    Douglas, Queensland, 4814

  • Border Medical Oncology Research Unit Albury Wodonga Regiona /ID# 231311

    East Albury, New South Wales, 2640

  • Gosford Hospital /ID# 221499

    Gosford, New South Wales, 2250

  • Liverpool Hospital /ID# 221803

    Liverpool, New South Wales, 2170

  • Peter MacCallum Cancer Ctr /ID# 229795

    Melbourne, Victoria, 3000

  • The Alfred Hospital /ID# 221501

    Melbourne, Victoria, 3004

  • Royal Perth Hospital /ID# 223203

    Perth, Western Australia, 6000

Other trials in Australia

Belgium8 sites

  • ZAS Cadix /ID# 221465

    Antwerp, Antwerpen, 2030

  • AZ Sint-Jan Brugge /ID# 218805

    Bruges, 8000

  • UZ Gent /ID# 221125

    Ghent, Oost-Vlaanderen, 9000

  • Universitair Ziekenhuis Leuven /ID# 218806

    Leuven, Vlaams-Brabant, 3000

  • CHU de Liège /ID# 218874

    Liège, Liege, 4000

  • AZ-Delta /ID# 221466

    Roeselare, West-Vlaanderen, 8800

  • Vitaz /Id# 229861

    Sint-Niklaas, Oost-Vlaanderen, 9100

  • Université Catholique de Louvain-Namur - Centre Hospitalier Universitaire Dinant /ID# 221127

    Yvoir, Namur, 5530

Other trials in Belgium

Germany7 sites

  • Universitaetsklinikum Aachen /ID# 221519

    Aachen, North Rhine-Westphalia, 52074

  • BAG Freiberg-Richter, Jacobasch, Illmer, Wolf /ID# 221347

    Dresden, Saxony, 01307

  • Universitaetsklinikum Essen /ID# 221522

    Essen, 45147

  • OncoResearch Lerchenfeld GmbH /ID# 230867

    Hamburg, 22081

  • Universitatsklinikum Mannheim /ID# 221523

    Mannheim, Baden-Wurttemberg, 68167

  • Haemato-Onkologie /ID# 221061

    Munich, Bavaria, 81241

  • Klinikum rechts der Isar /ID# 221520

    Munich, 81675

Other trials in Germany

Italy7 sites

  • ASST Papa Giovanni XXIII /ID# 221907

    Bergamo, 24127

  • IRCCS AOU di Bologna - Policlinico Sant'Orsola-Malpighi /ID# 220867

    Bologna, Emilia-Romagna, 40138

  • ASST degli Spedali Civili di Brescia /ID# 241273

    Brescia, 25123

  • AOU Policlinico G. Rodolico - San Marco /ID# 219085

    Catania, 95123

  • Azienda Ospedaliero Universitaria Careggi /ID# 219086

    Florence, Firenze, 50134

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS-Università Cattolica /ID# 219083

    Rome, Roma, 00168

  • Duplicate_ASST Sette Laghi /ID# 219084

    Varese, 21100

Other trials in Italy

Russia7 sites

  • Moscow State budget healthcare /ID# 221025

    Moscow, Moscow, 125284

  • Republican hospital named after V.A. Baranov /ID# 221412

    Petrozavodsk, Murmansk Oblast, 185019

  • Clinic UZI 4D /ID# 221024

    Pyatigorsk, Stavropol Kray, 357502

  • Russian Research Institute of Hematology and Transfusiology of the FMBA /ID# 221029

    Saint Petersburg, 191024

  • Leningrad Regional Clinical Hospital /ID# 221028

    Saint Petersburg, 194291

  • Almazov National Medical Research Centre /ID# 221033

    Saint Petersburg, 197341

  • Tula Regional Clinical Hospital /ID# 221027

    Tula, 300053

Other trials in Russia

Israel6 sites

  • Rambam Health Care Campus /ID# 219120

    Haifa, H_efa, 3109601

  • Hadassah Medical Center-Hebrew University /ID# 219110

    Jerusalem, Jerusalem, 91120

  • Meir Medical Center /ID# 221374

    Kfar Saba, Central District, 4428164

  • The Chaim Sheba Medical Center /ID# 219137

    Ramat Gan, Tel Aviv, 5265601

  • Tel Aviv Sourasky Medical Center /ID# 219134

    Tel Aviv, Tel Aviv, 6423906

  • Yitzhak Shamir Medical Center /ID# 222957

    Tsrifin, Central District, 70300

Other trials in Israel

Taiwan6 sites

  • Kaohsiung Chang Gung Memorial Hospital /ID# 218984

    Kaohsiung City, Kaohsiung, 833

  • China Medical University Hospital /ID# 218978

    Taichung, 40447

  • Chi Mei Hospital - Liouying /ID# 221145

    Tainan, Tainan, 73657

  • Taipei Veterans General Hosp /ID# 221146

    Taipei, 11217

  • National Taiwan University Hospital /ID# 218976

    Taipei City, Taipei, 100

  • Linkou Chang Gung Memorial Hospital /ID# 218983

    Taoyuan City, 333

Other trials in Taiwan

Austria5 sites

  • Duplicate_Medizinische Universitaet Graz /ID# 220910

    Graz, Styria, 8010

  • Ordensklinikum Linz GmbH Elisabethinen /ID# 220813

    Linz, Upper Austria, 4010

  • Medizinische Universitaet Wien /ID# 220906

    Vienna, State of Vienna, 1090

  • Hanusch Krankenhaus /ID# 220909

    Vienna, 1140

  • Klinikum Wels-Grieskirchen GmbH /ID# 220901

    Wels, Upper Austria, 4600

Other trials in Austria

Bulgaria5 sites

  • UMHAT Dr Georgi Stranski EAD /ID# 231161

    Pleven, 5800

  • UMHAT Sveti Georgi /ID# 231053

    Plovdiv, 4002

  • Acibadem City Clinic Tokuda University Hospital EAD /ID# 231036

    Sofia, 1407

  • UMHAT Sveti Ivan Rilski /ID# 231028

    Sofia, 1431

  • UMHAT Alexandrovska EAD /ID# 231056

    Sofiya, Sofia, 1431

Other trials in Bulgaria

Canada5 sites

  • Royal Victoria Hospital /ID# 222636

    Barrie, Ontario, L4M 6M2

  • Juravinski Cancer Centre /ID# 221752

    Hamilton, Ontario, L8V 1C3

  • Lakeridge Health - Oshawa /ID# 222080

    Oshawa, Ontario, L1G 2B9

  • CHUQ- Hôpital de l'Enfant-Jesus /ID# 221754

    Québec, Quebec, G1J 1Z4

  • Niagara Health System /ID# 230994

    St. Catharines, Ontario, L2S 0A9

Other trials in Canada

Turkey (Türkiye)5 sites

  • Hacettepe University Medical Faculty /ID# 230759

    Ankara, 06230

  • Trakya University Medical Facu /ID# 230754

    Edirne, Istanbul, 22030

  • Bagcilar Medipol Mega Universite Hastanesi /ID# 230757

    Istanbul, 34214

  • Ege University Medical Faculty /ID# 230753

    Izmir, 35040

  • Inonu University Medical Faculty /ID# 230758

    Malatya, 44280

Other trials in Turkey (Türkiye)

United Kingdom5 sites

  • University Hospitals Birmingham NHS Foundation Trust /ID# 221333

    Birmingham, B15 2TH

  • United Lincolnshire Hospitals NHS Trust /ID# 231471

    Lincoln, Lincolnshire, LN2 4AX

  • Guys and St Thomas NHS Foundation Trust /ID# 219185

    London, Greater London, SE1 9RT

  • The Christie Hospital /ID# 219191

    Manchester, M20 4BX

  • Oxford University Hospitals NHS Foundation Trust /ID# 219192

    Oxford, Oxfordshire, OX3 9DU

Other trials in United Kingdom

Croatia4 sites

  • Duplicate_Klinicki bolnicki centar Split /ID# 230796

    Split, Split-Dalmatia County, 21000

  • Clinical Hospital Dubrava /ID# 230795

    Zagreb, City of Zagreb, 10000

  • Klinicka bolnica Merkur /ID# 231155

    Zagreb, City of Zagreb, 10000

  • Klinicki bolnicki centar Zagreb /ID# 230793

    Zagreb, City of Zagreb, 10000

Other trials in Croatia

Netherlands4 sites

  • Albert Schweitzer Ziekenhuis /ID# 224015

    Dordrecht, South Holland, 3318 AT

  • Universitair Medisch Centrum Groningen /ID# 218947

    Groningen, 9713 GZ

  • Radboud Universitair Medisch Centrum /ID# 218948

    Nijmegen, Gelderland, 6525 GA

  • Universitair Medisch Centrum Utrecht /ID# 218949

    Utrecht, 3584 CX

Other trials in Netherlands

Serbia4 sites

  • University Clinical Center Serbia /ID# 230854

    Belgrade, Beograd, 11000

  • Clin Hosp Ctr Bezanijska Kosa /ID# 230946

    Belgrade, Beograd, 11080

  • University Clinical Center Kragujevac /ID# 230855

    Kragujevac, Sumadijski Okrug, 34000

  • Duplicate_Clinical Center Vojvodina /ID# 230853

    Novi Sad, Vojvodina, 21000

Other trials in Serbia

Ukraine4 sites

  • Communal non-profit enterprise Regional Center of Oncology /ID# 230832

    Kharkiv, Kharkiv Oblast, 61070

  • Medical Center OK Clinic LLC, International Institute of Clinical Research /ID# 230834

    Kyiv, 02091

  • Feofaniya Clinical Hospital of State Management of Affairs /ID# 232370

    Kyiv, 03143

  • SI Institute of Blood Pathology and Transfusion Medicine of NAMS of Ukraine /ID# 230833

    Lviv, 79044

Other trials in Ukraine

Greece3 sites

  • General Hospital of Athens Laiko /ID# 230785

    Athens, Attica, 11527

  • Duplicate_University General Hospital Attikon /ID# 230784

    Athens, Attica, 12462

  • General Hospital of Athens Evaggelismos and Ophthalmiatrio of Athens Polyclinic /ID# 230786

    Athens, 10676

Other trials in Greece

South Africa3 sites

  • Wits Clinical Research /ID# 232072

    Johannesburg, Gauteng, 1864

  • Duplicate_Wits Clinical Research Site /ID# 232071

    Johannesburg, Gauteng, 2193

  • Alberts Cellular Therapy /ID# 232073

    Pretoria, Gauteng, 0044

Other trials in South Africa

Sweden3 sites

  • Duplicate_Sahlgrenska University Hospital /ID# 218776

    Gothenburg, Västra Götaland County, 413 45

  • Duplicate_Skane University Hospital Lund /ID# 220835

    Lund, Skåne County, 221 41

  • Orebro Universitetssjukhuset /ID# 220829

    Örebro, Örebro County, 701 85

Other trials in Sweden

New Zealand1 site

  • Aotearoa Clinical Trials /ID# 230770

    Papatoetoe, Auckland, 2025

Other trials in New Zealand

About this study

Official title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study Of Navitoclax In Combination With Ruxolitinib Versus Ruxolitinib In Subjects With Myelofibrosis (TRANSFORM-1)

Myelofibrosis is a type of bone marrow cancer that usually develops slowly and disrupts body's normal production of blood cells. It causes bone marrow scarring, leading to severe anemia that can cause weakness and fatigue. It can also cause a low number of blood-clotting cells called platelets, which increases risk of bleeding. Myelofibrosis often causes an enlarged spleen. The purpose of this study is to see if a combination of navitoclax and ruxolitinib is more effective and safe in assessment of change in spleen volume when compared to ruxolitinib in participants with myelofibrosis.

Navitoclax is an investigational drug for the treatment of myelofibrosis. Participants in this study are divided into two groups, called treatment arms. Each group receives a different treatment. Adult participants with a diagnosis of myelofibrosis will be enrolled. Around 230 participants will be enrolled in approximately 190 sites worldwide.

Read more

Participants will receive oral navitoclax tablet with oral ruxolitinib tablet or oral ruxolitinib tablet with oral placebo (no active drug) tablet and treatment may continue untill the participant cannot tolerate the study drug, or benefit is not achieved, or other reasons which qualify for discontinuation of the study drug.

There may be a higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the course of the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, magnetic resonance imaging (MRI) or computed tomography (CT) scan, bone marrow tests, checking for side effects, and completing questionnaires.

Being tested
Placebo for Navitoclax, Ruxolitinib, Navitoclax
Keywords
Myelofibrosis, Navitoclax, Ruxolitinib, Cancer, ABT-263, TRANSFORM-1
Medical subject headings
Primary Myelofibrosis, Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Placebo for NavitoclaxDrug

    Film-coated tablet; Oral

  • RuxolitinibDrug

    Also called Jakafi

    Tablet; Oral

  • NavitoclaxDrug

    Also called ABT-263

    Film-coated tablet; Oral

Study groups in NCT04472598
Study groupRoleWhat they receive
Placebo for Navitoclax + RuxolitinibActive comparatorPlacebo for navitoclax tablets were administered orally once daily (QD) per Baseline platelet count (>150 × 10^9/L starting dose of 200 mg; ≤150 × 10^9/L starting dose of 100 mg, which could be increased to 200 mg QD after 7 days provided platelet count is ≥75 × 10^9/L). Placebo for navitoclax didn't exceed 200 mg QD for first 24 weeks of treatment. After Week 25, Day 1 visit, placebo for navitoclax dose may be increased to 300 mg QD at Investigator's discretion for those with suboptimal spleen response defined as failure to achieve spleen volume reduction of at least 10% per imaging. Ruxolitinib tablets were administered orally twice daily (BID) per Baseline platelet count (>200 × 10^9/L starting dose of 20 mg; 100-200 × 10^9/L starting dose of 15 mg). Participants will continue their treatment until end of clinical benefit, unacceptable toxicity, or they meet other protocol criteria for discontinuation (whichever occurs first).
Receives: Drug: Placebo for Navitoclax; Drug: Ruxolitinib
Navitoclax + RuxolitinibExperimentalNavitoclax tablets were administered orally once daily (QD) per Baseline platelet count (>150 × 10^9/L starting dose of 200 mg; ≤150 × 10^9/L starting dose of 100 mg, which could be increased to 200 mg QD after 7 days provided platelet count is ≥75 × 10^9/L). Navitoclax didn't exceed 200 mg QD for first 24 weeks of treatment. After Week 25, Day 1 visit, navitoclax dose may be increased to 300 mg QD at Investigator's discretion for those with suboptimal spleen response defined as failure to achieve spleen volume reduction of at least 10% per imaging. Ruxolitinib tablets were administered orally twice daily (BID) per Baseline platelet count (>200 × 10^9/L starting dose of 20 mg; 100-200 × 10^9/L starting dose of 15 mg). Participants will continue their treatment until end of clinical benefit, unacceptable toxicity, or they meet other protocol criteria for discontinuation (whichever occurs first).
Receives: Drug: Ruxolitinib; Drug: Navitoclax

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage of Participants With ≥ 35% Reduction From Baseline in Spleen Volume at Week 24 (SVR35W24)

    Reduction in spleen volume is measured by magnetic resonance imaging (MRI) or computed tomography (CT), per International Working Group (IWG) criteria.

    Measured: Baseline, Week 24

Secondary outcomes (9)

  1. Change From Baseline in Total Symptom Score (TSS) at Week 24 as Measured by Myelofibrosis Symptom Assessment Form (MFSAF) v4.0

    TSS is assessed by the Myelofibrosis Symptom Assessment Form (MFSAF) version 4.0. Participants complete a symptom diary and rate the following seven MF symptoms: fatigue, night sweats, abdominal discomfort, pruritus, pain under the ribs on the left side, early satiety, and bone pain daily using a scale from 0 (absent) to 10 (worst imaginable), and the scores are averaged over 7 days, with a minimum of 4 days required to calculate the average score. Participants for whom a valid average score cannot be calculated either at baseline or post-baseline are considered non-responders. The TSS reflects the sum of the scores of these symptoms, for a maximum possible score of 70 (i.e., most severe symptom experience). Negative changes from Baseline indicate improvement.

    Measured: Baseline, Week 24

  2. Percentage of Participants With ≥ 35% Reduction From Baseline in Spleen Volume (SVR35) at Any Time

    Reduction in spleen volume is measured by magnetic resonance imaging (MRI) or computed tomography (CT), per International Working Group (IWG) criteria.

    Measured: Up to Week 97

  3. Duration of 35% Spleen Volume Reduction (SVR35)

    Duration of SVR35 is defined as the time between the date of first response of spleen volume reduction of 35% achievement to the date of the first assessment where the spleen volume is less than 35% reduction from Baseline and is at least 25% increase from the nadir (the lowest spleen volume), confirmed relapse, or leukemic transformation per International Working Group (IWG) criteria, whichever is earlier.

    Measured: Baseline (Week 0) Up to Month 48

  4. Change From Baseline In Fatigue at Week 24 as Measured by the PROMIS Fatigue Short Form (SF) 7a

    The PROMIS Fatigue SF 7a is a 7-item patient-reported outcome measure that assesses the impact and experience of participants with fatigue over the past 7 days. Each item is scored on a 5-point Likert scale (1 = Never, 2 = Rarely, 3 = Sometimes, 4 = Often, and 5 = Always). Raw scores range from 7 to 35 and are subsequently transformed into a standardized T-score using the PROMIS wave 1 calibration (where a score of 50 represents the U.S. general population mean with a standard deviation of 10). Higher T-scores indicate greater fatigue severity. A decrease in T-score from Baseline represents a clinical improvement in fatigue symptoms.

    Measured: Baseline, Week 24

  5. Change From Baseline at Week 24 in Physical Functioning as Measured by the Physical Functioning Domain of the European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30

    EORTC QLQ-C30 is a 30-item participant self-report questionnaire composed of both multi-item and single scales, including a physical functioning scale. Participants rate items and a score ranging from 0 to 100 is calculated. A higher score on the physical functioning scale indicates a better level of functioning, and positive changes from Baseline indicate improvement.

    Measured: Baseline, Week 24

  6. Percentage of Participants Achieving Anemia Response

    For a participant who is transfusion independent (TI) at Baseline with hemoglobin value < 10 g/dL, anemia response is achieved if the post-Baseline hemoglobin level increases by ≥2 g/dL without receiving packed red blood cells (PRBC) transfusion (for any reason) within 2 weeks and without any erythropoietin or mimetics within the last 4 weeks prior to the increase in hemoglobin level by ≥2g/dL was observed. Hemoglobin values more than 30 days after the last dose of study treatment or after the start of post-study treatment or disease progression, whichever is earlier, will not be considered in the analysis of anemia response.

    For a participant who is transfusion dependent (TD) at Baseline, anemia response is defined as a period of at least 12 consecutive weeks without PRBC transfusion at any time after the first dose of study drug and on or prior to 30 days post last dose of study drug, the start of post-study treatment, disease progression or death, whichever occurs earlier.

    Measured: Up to Week 97

  7. Overall Survival (OS)

    OS is defined as the time from the date of randomization to the date of death from any cause.

    Measured: Up to 50 months

  8. Leukemia-Free Survival

    Leukemia-free survival is defined as the number of days from the date of randomization to the onset date of documented leukemia, disease progression due to leukemia, or death due to leukemia, whichever occurs first.

    Measured: Up to 50 months

  9. Percentage of Participants Who Achieved Reduction in Grade of Bone Marrow Fibrosis From Baseline at Any Time

    Change in grade of bone marrow fibrosis was measured per the European consensus grading system through bone marrow biopsy. The percentage of participants who achieved reduction of at least 1 grade in bone marrow fibrosis compared to Baseline is reported.

    Measured: Up to Week 97

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted15 Jul 2020
  2. Study start29 Sep 2020
  3. Primary completion13 Apr 2023
  4. Study completion29 Jan 2025

Resources

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 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
252 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04472598
Sponsor's protocol ID
M16-191
Other IDs
2020-000097-15
Acronym
TRANSFORM-1
Status last verified
Mar 2026
First submitted
14 Jul 2020
Last updated
23 Mar 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: AbbVie is committed to responsible clinical trial data sharing. This includes access to anonymized, individual and trial-level data (analysis data sets), as well as other information.
Expanded access
Available

Publications

  • Pemmaraju N, Mead AJ, Somervaille TCP, Palandri F, Koschmieder S, Delage R, Kiladjian JJ, Benton C, Tantravahi SK, Lavi N, Yeh SP, Gomez Casares MT, Ammatuna E, McDonald A, Yoon SS, Kirito K, Devos T, Perkins A, Radinoff A, Sevindik OG, Bogdanovic A, Moskal R, Harb JG, Murray B, Jiang Q, Chopra AS, Papadopoulos EJ, Potluri J, Passamonti F. TRANSFORM-1 phase 3 study: efficacy and safety of navitoclax plus ruxolitinib in patients with untreated myelofibrosis. Blood. 2026 Oct 15;148(16):2029-2042. doi: 10.1182/blood.2025032360.

    PubMed 42378247

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Ruxolitinib and Navitoclax in people with Myelofibrosis (MF), run by AbbVie at 194 sites in 25 countries.

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 7 inclusion and 3 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: finished, as of the sponsor's last update on 23 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 194 sites in United States, Japan, France, Spain, South Korea and 20 more countries. 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 phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 23 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage of Participants With ≥ 35% Reduction From Baseline in Spleen Volume at Week 24 (SVR35W24) (time frame: Baseline, Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04472598, which also lists 1 linked publication.

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 23 Mar 2026.

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