New drug combo shrinks spleens in bone marrow cancer patients
NCT ID NCT02158858
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a new drug called pelabresib in people with certain blood cancers, including myelofibrosis and leukemia. In the first part, the drug was given alone to find the safest dose. In the second part, it was given with or without another drug (ruxolitinib) to see if it could shrink an enlarged spleen and reduce the need for blood transfusions. The trial involved 336 adults and has been completed.
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
-
336 people
The number who actually took part.
- Started
-
Jul 2014
- Finished
-
Jan 2025
- 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 Hide 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.
Phase I (Dose Escalation) - Inclusion and Exclusion Criteria: 1. Inclusion Criteria (Phase I): * Age: Adults ≥18 years. * Diagnosis: Histologically or cytologically confirmed diagnosis of one of the following hematologic malignancies: * Acute myelogenous leukemia (AML) * Acute lymphocytic leukemia (ALL) * Acute undifferentiated or biphenotypic leukemia * Chronic myeloid leukemia (CML) in blast crisis * Myelodysplastic syndrome (MDS) * Myelodysplastic/myeloproliferative neoplasms (MDS/MPN) * Myelofibrosis (MF) * Performance Status: ECOG ≤2. * Organ Function: * Serum total bilirubin ≤1.5 × ULN * AST/ALT ≤2.5 × ULN (up to 5 × ULN if due to leukemic infiltration) * Serum creatinine ≤2.0 × ULN or CrCl ≥30 mL/min * Hematology (MF only): * Platelet count ≥50 × 10⁹/L and ANC ≥1 × 10⁹/L (MF not on ruxolitinib) * Platelet count ≥75 × 10⁹/L and ANC ≥1 × 10⁹/L (MF on ruxolitinib) * Other: * DIPSS-plus risk category of intermediate-2 or high (MF only) * Serum glucose ≤160 mg/dL (or HbA1C ≤7%) * Fully recovered from major surgery and acute toxic effects of prior therapy * Negative pregnancy test for women of childbearing potential * Agreement to use appropriate contraception * Written informed consent 2. Exclusion Criteria (Phase I): * Untreated newly diagnosed acute leukemia (unless AML with myelodysplasia-related changes and 20-30% blasts) * Relapsed/refractory acute leukemia where further induction chemotherapy is beneficial * Acute leukemia relapse \<6 months after allogeneic SCT * CML in blast crisis treated with only one TKI * Very low/low risk MDS without prior treatment * CNS involvement by leukemia (unless resolved) * Active HIV, Hepatitis B or C infection * GI impairment affecting absorption (unresolved nausea, vomiting, diarrhea \>CTCAE grade 1) * Significant cardiac disease (recent MI/angina, high cTn, QTcF \>470 ms, LVEF \<50%, uncontrolled arrhythmia, etc.) * Severe/uncontrolled comorbidities * Recent systemic anti-cancer therapy (other than hydroxyurea/radiotherapy) \<2 weeks prior * Ongoing or recent JAK inhibitor use (\<2 weeks prior, MF only) * Recent therapeutic antibody (\<4 weeks) or investigational agent (\<2 weeks or \<5 half-lives) * Use of strong CYP450 inhibitors/inducers or drugs with Torsades de Pointes risk * Immunosuppressive treatment that cannot be discontinued * Pregnant/lactating women * Inadequate contraception * Inability/unwillingness to comply with protocol Phase II (Expansion) - Inclusion \& Exclusion Criteria: 1. Inclusion Criteria (Phase II): 1. MF Arms (Prior JAKi, Add-on JAKi, JAKi Naïve) * Age: Adults ≥18 years * Diagnosis: Confirmed primary MF or MF evolved from ET or PV * Risk: DIPSS intermediate-2 or higher * Platelets: * ≥75 × 10⁹/L (Arms 1 \& 2) * ≥100 × 10⁹/L (Arm 3, JAKi naïve) * ANC: ≥1 × 10⁹/L * Spleen Volume: ≥450 cm³ by MRI/CT (non-TD cohorts) OR * Transfusion Dependence: Average ≥2 RBC transfusions/month (total ≥6 in prior 12 weeks) for TD cohorts * Peripheral Blood Blasts: \<10% * Symptoms: At least 2 symptoms measurable (score ≥1 for Arms 1 \& 2; score ≥3 or total ≥10 for Arm 3) using MFSAF v4.0 * Treatment History: * Arm 1 (Prior JAKi): Previously treated with JAKi and intolerant, resistant, refractory, or lost response, or ineligible for JAKi * Arm 2 (Add-on JAKi): On ruxolitinib ≥6 months, stable dose ≥8 weeks, not adequately controlled * Arm 3 (JAKi Naïve): No prior JAKi, eligible for ruxolitinib * Performance Status: ECOG ≤2 * Organ Function: Serum direct bilirubin \<2 × ULN, AST/ALT ≤2.5 × ULN (up to 5 × ULN if due to liver involvement), CrCl ≥45 mL/min * Other: Fully recovered from major surgery/acute toxic effects, effective contraception, written informed consent 2. ET Arm (High-Risk ET) * Age: Adults ≥18 years * Diagnosis: Confirmed ET (WHO 2016 criteria) * High-Risk: At least one of: * Age \>60 years * Platelets \>1500 × 10⁹/L * Prior thrombosis, erythromelalgia, or migraine (disease-related) * Prior hemorrhage related to ET * Diabetes/hypertension requiring therapy \>6 months * Symptoms: ≥2 symptoms with average score ≥3 or total score ≥15 (MPN-SAF) * Platelets: \>600 × 10⁹/L * Resistant/Intolerant to HU: As defined by ELN * Performance Status: ECOG ≤2 * Life Expectancy: \>24 weeks * ANC: ≥1 × 10⁹/L * Organ Function: Serum direct bilirubin \<2 × ULN, AST/ALT ≤2.5 × ULN, CrCl ≥45 mL/min * Other: Fully recovered from major surgery/acute toxic effects, effective contraception, written informed consent 2. Exclusion Criteria (Phase II) * Prior splenectomy (MF non-TD cohorts) * Splenic irradiation within 3 months * Active or chronic HIV, Hepatitis B/C infection * Active clinically significant infection (until recovery ≥2 weeks) * Anemia deemed clinically significant (iron/B12/folate deficiency, hemolytic anemia) * Major bleeding event (≥2 g/dL Hgb drop or ≥2 units transfused in last 6 months) * Liver cirrhosis Child-Pugh B or C * GI impairment affecting absorption (unresolved nausea, vomiting, diarrhea \>CTCAE grade 1) * Rare hereditary problems of galactose intolerance, Lapp lactase deficiency, or glucose-galactose malabsorption (Arm 3) * Hypersensitivity to ruxolitinib formulation (Arm 3) * History of PML (Arm 3) * Significant cardiac disease (recent MI/angina, QTcF \>500 ms \[\>450 ms in France/Germany\], uncontrolled arrhythmia, etc.) * Ongoing uncontrolled hypertension * Severe/uncontrolled comorbidities * Systemic anticancer treatment (other than ruxolitinib for Arm 2, HU/ANA up to 24h prior) \<2 weeks or \<5 half-lives prior * Prior treatment with any BET inhibitor * Hematopoietic growth factor or androgenic steroids \<4 weeks prior * Systemic corticosteroids ≥10 mg prednisone equivalent within 4 weeks (exceptions for short courses) * Concurrent/second malignancy (except certain adequately treated cancers) * Pregnant/lactating women, or planning pregnancy within protocol-defined window * Inability/unwillingness to comply with protocol
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Acute lymphocytic leukemia are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
AOU Maggiore della Carità
Novara, 28100, Italy
-
AOU S.Martino, IRCCS, IST-Istituto Nazionale Ricerca Sul Can
Genoa, Liguria, 16132, Italy
-
AZ Sint-Jan Burgge-Oostende AV- Campus Sint-Jan
Bruges, West-Vlaanderen, 8000, Belgium
-
Azienda Ospedaliero-Universitaria Careggi
Florence, 50134, Italy
-
Beatson West of Scotland Cancer Centre
Glasgow, G12 0YN, United Kingdom
-
Belfast City Hospital
Belfast, BT9 7AB, United Kingdom
-
CHRU de Lille - Hopital Claude Huriez
Toulouse, Haute-Garonne, 31059, France
-
CHRU de Lille - Hopital Claude Huriez - Maladies du Sang
Lille, Hauts-de-France, 59037, France
-
CHU - Hopital Saint Louis - Centre D'Investigations Clinique
Paris, 75010, France
-
Erasmus Universitair Medisch Centrum Rotterdam
Rotterdam, South Holland, 3015 AA, Netherlands
-
Froedtert & Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
-
Guys and St Thomas' Hospital - Haematology
London, SE1 9RT, United Kingdom
-
ICAHN School of Medicine at Mount Sinai
New York, New York, 10029, United States
-
IRCCS Policlinico San Matteo, Università degli studi di Pavi
Pavia, Lombardy, 27100, Italy
-
Institue of Hematology "L. and A. Seràgnoli"
Bologna, Emilia-Romagna, 40138, Italy
-
Institut Gustave Roussy
Villejuif, Île-de-France Region, 94805, France
-
Institut de cancérologie du Gard - Hematologie clinique
Nîmes, Gard, 30029, France
-
Instytut Hematologii i Transfuzjologii w Warszawie
Warsaw, Masovian Voivodeship, 02-776, Poland
-
Jewish General Hospital
Montreal, Quebec, H3T 1E2, Canada
-
Juravinski Cancer Centre
Hamilton, Ontario, L8V 5C2, Canada
-
Maastricht University Medical Center
Maastricht, Limburg, 6229 HX, Netherlands
-
Massachusetts General Hospital Cancer Center
Boston, Massachusetts, 02114, United States
-
Mayo Clinic Arizona
Phoenix, Arizona, 85054, United States
-
Mayo Clinic Jacksonville
Jacksonville, Florida, 32224, United States
-
Memorial Sloan Kettering Cancer Center
New York, New York, 10021, United States
-
Northwestern University - Lurie Comprehensive Cancer Center
Chicago, Illinois, 60611, United States
-
Ospedale Maggiore Policlinico, Fondazione IRCCS Ca' Granda
Milan, Lombardy, 20122, Italy
-
Ospedale di Circolo, PO Varese, AO Ospedale di Circolo e Fon
Varese, Lombardy, 21100, Italy
-
Oxford University Hospitals
Headington, Oxford, OX3 7LE, United Kingdom
-
Princess Margaret Cancer Centre
Toronto, Ontario, M5G 2M9, Canada
-
Servizio Sanitario Regionale Emilia-Romagna - Azienda Unita Sanitaria Locale (AUSL) di Rimini - Ospedale Infermi di Rimini
Rimini, Emilia-Romagna, 47923, Italy
-
St. Paul's Hospital
Vancouver, British Columbia, V6Z 2A5, Canada
-
The Christie Hospital
Manchester, M20 4BX, United Kingdom
-
The University of Texas MD Anderson Cancer Center
Houston, Texas, 77030, United States
-
UCLA Medical Center
Los Angeles, California, 90095, United States
-
UZ Leuven - Campus Gasthuisberg
Leuven, Viaams Braban, 3000, Belgium
-
University College London Hospital's NHS foundation Trust
London, NW1 2PG, United Kingdom
-
University Hospital of Wales
Cardiff, CF14 4XW, United Kingdom
-
University of Alberta Hospital
Edmonton, Alberta, T6G 2G3, Canada
-
University of Cambridge
Cambridge, CB2 0QQ, United Kingdom
-
University of Michigan Medical Center
Ann Arbor, Michigan, 48109, United States
-
Universitätsklinikum Bonn
Bonn, North Rhine-Westphalia, 53127, Germany
-
Universitätsklinikum Leipzig AöR
Leipzig, Saxony, 04103, Germany
-
Uniwersyteckie Centrum Kliniczne
Gdansk, Pomeranian Voivodeship, 80-952, Poland
-
VUmcResearch B.V.
Amsterdam, North Holland, 1081 HV, Netherlands
-
Washington University School of Medicne Neuromuscular Division Department of Neurology Research
St Louis, Missouri, 63110, United States
-
Weill Medical College and New York Presbyterian Hospital
New York, New York, 10065, United States
-
ZNA Stuyvenberg Antwerpen
Antwerp, 2060, Belgium
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Scientists map skin Cancer's inner workings to decode immunotherapy response
- When cancer blocks the airway, doctors lack a playbook. experts aim to write one.
- Half-Matched stem cells tested as cure for myelofibrosis
- Pens and vouchers: can small rewards boost participation in blood cancer research?
- Families rate dignity and support in final days of cancer care
- Can a Nine-Week group program help blood cancer patients grow through trauma?