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New hope for myelofibrosis patients who fail current treatments

NCT ID NCT04576156

Disease control Sponsor: Geron Corporation Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 09, 2026 · Updated 2 times

Summary

This phase 3 trial tests a drug called imetelstat against the best available treatments for people with advanced myelofibrosis whose disease has stopped responding to JAK inhibitors. The study includes 327 participants and aims to see if imetelstat can help them live longer and feel better. The drug is given intravenously every three weeks.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
imetelstat
What this could lead to
If successful, imetelstat could offer a new treatment option that extends survival and reduces symptoms for people with advanced myelofibrosis who have stopped responding to current therapies.
What could go wrong
This is a late-stage trial, but the drug may still fail to improve survival or cause significant side effects. Results may not apply to all patients with myelofibrosis.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

327 people

The number who actually took part.

Started

Apr 2021

Expected to finish

Jun 2028

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.

Inclusion Criteria: * Diagnosis of primary myelofibrosis according to the revised World Health Organization criteria or post-essential thrombocythemia-MF or post-polycythemia vera-MF according to the IWG-MRT criteria * Dynamic International Prognostic Scoring System intermediate-2 or high-risk MF * Relapsed/refractory to JAK-inhibitor treatment as defined in either inclusion (i), (ii) or (iii) and not eligible for allogeneic stem cell transplantation (ASCT) at screening: * (i) Treatment with JAK-inhibitor for \>= 6 months duration, including at least 2 months at an optimal dose as assessed by the investigator for that participant and at least one of the following: 1. no decrease in spleen volume (\< 10% by MRI or CT) from the start of treatment with JAK-inhibitor 2. no decrease in spleen size (\< 30% by palpation or length by imaging) from the start of treatment with JAK-inhibitor 3. no decrease in symptoms (\< 20% by Myelofibrosis Symptom Assessment Form \[MFSAF\] or myeloproliferative neoplasm SAF) from the start of treatment with JAK-inhibitor 4. a score of at least 15 on TSS assessed using the MFSAF v4.0 during screening. * (ii) Treatment with JAK-inhibitor treatment for\>= 3 months duration with maximal doses (e.g., 20-25 mg twice daily ruxolitinib) for that participant and no decrease in spleen volume/size or symptoms as defined in inclusion criterion (i \[a, b, or c\]). * (iii) Following maximum tolerated doses of JAK inhibitor therapy for ≥3 months duration, having documented relapsed disease defined as either 1. Increase in spleen volume from time of best response by 25% measured by MRI or CT, or 2. Increase in spleen size by palpation, CT, or ultrasound * (b.i) For splenomegaly of 5-10 cm at the start of JAK inhibitor treatment, at least 100% increase in palpable spleen size from time of best response; * (b.ii) For splenomegaly of \> 10 cm at the start of JAK inhibitor treatment, at least 50% increase in palpable spleen size from time of best response; AND not a candidate for further JAK inhibitor at screening per investigator. * Measurable splenomegaly demonstrated by a palpable spleen measuring \>= 5 cm below the left costal margin or a spleen volume \>= 450 cm\^3 by MRI or CT * Active symptoms of MF on the MFSAF v4.0 demonstrated by a symptom score of at least 5 points (on a 0 to 10 scale) * Hematology laboratory test values within the protocol defined limits * Biochemical laboratory test values must be within protocol defined limits * Eastern Cooperative Oncology Group Performance Status score of 0, 1, or 2 * Participants should follow protocol defined contraceptives procedures * A woman of childbearing potential must have a negative serum or urine pregnancy test at screening Exclusion Criteria: * Peripheral blood blast count of \>= 10% or bone marrow blast count of \>=10% * Known allergies, hypersensitivity, or intolerance to imetelstat or its excipients * Prior treatment with imetelstat * Any chemotherapy or MF directed therapy, including investigational drug regardless of class or mechanism of action, immunomodulatory or immunosuppressive therapy, corticosteroids greater than 30 mg/day prednisone or equivalent, and JAK-inhibitor treatment less than equal to 14 days prior to randomization * Diagnosis or treatment for malignancy other than MF except: * Malignancy treated with curative intent and with no known active disease present for \>= 3 years before randomization * Adequately treated non-melanoma skin cancer or lentigo maligna without evidence of disease * Adequately treated cervical carcinoma in situ without evidence of disease * Known history of human immunodeficiency virus or any uncontrolled active systemic infection requiring IV antibiotics * Active systemic hepatitis infection requiring treatment (carriers of hepatitis virus are permitted to enter the study), or any known acute or chronic liver disease requiring treatment unless related to underlying hepatosplenomegaly due to MF * Major surgery within 28 days prior to randomization * Any life-threatening illness (e.g., coronavirus disease-2019), medical condition, or organ system dysfunction which, in the investigator's opinion, could compromise the participant's safety, interfere with the imetelstat metabolism, or put the study outcomes at undue risk

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Conditions

The condition(s) this trial relates to.

myelofibrosis Myeloproliferative Disorders polycythemia primary myelofibrosis thrombocytosis disease

As listed by the trial registrant

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

Myelofibrosis

Contacts and locations

Locations

  • A.O.di Bologna Policl.S.Orsola

    Bologna, Bologna, 40138, Italy

  • AOU Careggi

    Florence, Firenze, 50134, Italy

  • AOU Città della Salute e della Scienza di Torino

    Torino, Torino, 10126, Italy

  • AOU Federico II

    Naples, 80122, Italy

  • AOU San Luigi Gonzaga

    Orbassano, Torino, 10043, Italy

  • ASL Roma 2 - PO "S. Eugenio"

    Roma, 00144, Italy

  • ASST Grande Ospedale Metropoli

    Milan, 20162, Italy

  • AUSL di Rimini Ospedale Infermi di Rimini

    Rimini, Rimini, 47900, Italy

  • AZ Klina

    Antwerp, Antwerpen, 2930, Belgium

  • All India Institute of Medical Sciences

    Bhubaneswar, Odisha, 751019, India

  • All India Institute of Medical Sciences, Dept. of Hematology, New Delhi (All India Institute Of Medical Sciences)

    New Delhi, National Capital Territory of Delhi, 110029, India

  • Ankara University Medical Facu

    Ankara, Ankara, 6590, Turkey (Türkiye)

  • Arcispedale S Maria Nuova, AO di Reggio Emilia

    Reggio Calabria, Reggio Calabria, 42123, Italy

  • Arcispedale S.Anna - Ematologi

    Cona, Ferrara, 44124, Italy

  • Ars Medical Sp. z o.o.

    Piła, Greater Poland Voivodeship, 64-920, Poland

  • Assuta Ashdod University Hospi

    Ashdod, Southern District, 7747629, Israel

  • Azienda Ospedaliera Bianchi-Me

    Reggio Calabria, Reggio Calabria, 89124, Italy

  • Azienda Ospedaliera San Gerard

    Monza, 20900, Italy

  • BRCR Medical Center Inc

    Plantation, Florida, 33326, United States

  • Barzilai Medical Center

    Ashkelon, Southern District, 78278, Israel

  • Bnai Zion Medical Center

    Haifa, 31048, Israel

  • Budgetary Healthcare Institution of Omsk Region

    Omsk, 644013, Russia

  • CEPON Centro de Pesquisas Oncologicas SC

    Florianópolis, 88034-000, Brazil

  • CHRU Brest - Hôpital Morvan

    Brest, 29609, France

  • CHU - Hôpital Saint Louis - Centre D'Investigations Cliniq

    Paris, 75010, France

  • CHU Bretonneau

    Tours, Indre-et-Loire, 37044, France

  • CHU De Nantes - Hematologie

    Nantes, Loire-Atlantique, 44000, France

  • CHU de Nice - Hopital de l'Archet II - Pharmacie

    Nice, Nice Cedex 3, 06202, France

  • Carmel MC

    Haifa, 3436212, Israel

  • Centre Hospitalier Du Mans - Cancérologie Médicale

    Le Mans, Sarthe, 72037, France

  • Centre Hospitalier Lyon

    Pierre-Bénite, Auvergne-Rhône-Alpes, 69495, France

  • Centre Hospitalier de Jolimont

    Haine-Saint-Paul, Hainaut, 7100, Belgium

  • Centro Clinico Fundacao Champalimaud

    Lisbon, Lisbon District, 1400-038, Portugal

  • Centro Gaucho Integrado de Oncologia, Hematologia, Ensino e Pesquisa LTDA

    Porto Alegre, Rio Grande do Sul, 90470340, Brazil

  • Centro de oncologia Leonardo da Vinci

    Fortaleza, Ceará, 60135285, Brazil

  • Centrum Medyczne Pratia Poznan

    Skórzewo, Greater Poland Voivodeship, 60-185, Poland

  • Chang Gung Medical Foundation

    Chiayi City, Chiayi, 613, Taiwan

  • China Medical University Hospital - Hematology/Onc

    Taichung, 404, Taiwan

  • Clinica Ematologica, Univ. Deg

    Genova, Genova, 6- 16132, Italy

  • Deenanath Mangeshkar Hospital & Research Center

    Pune, 411004, India

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Dél-pesti Centrumkórház Ország

    Budapest, H-1097, Hungary

  • Ege Universitesi Tip Fakultesi

    Izmir, İzmir, 35100, Turkey (Türkiye)

  • Epworth Healthcare

    Richmond, Victoria, 3121, Australia

  • Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

    Milan, Milano, 20122, Italy

  • Fortis Hospital 154/9

    Bengaluru, Karnataka, 560076, India

  • Fortis Memorial Research Institute

    Gurgaon, New Delhi, 122002, India

  • Fundacao Doutor Amaral Carvalho / Hospital Amaral Carvalho

    São Paulo, São Paulo, 17210-120, Brazil

  • GOU VPO Saratov State Medical University n.a. V.I.

    Saratov, 410012, Russia

  • Gabrail Cancer Center

    Canton, Ohio, 44718, United States

  • Gazi University Medical Faculty

    Ankara, 06560, Turkey (Türkiye)

  • Guys and St Thomas' Hospital

    London, SE1 9RT, United Kingdom

  • H. Quirón Zaragoza

    Zaragoza, Zaragoza, 50012, Spain

  • H. San Pedro de Alcántara

    Cáceres, Cáceres, 10003, Spain

  • H.G.U. Alicante

    Alicante, Alicante, 03010, Spain

  • H.U. La Paz

    Madrid, Madrid, 28046, Spain

  • H.U.V. de la Victoria

    Málaga, Málaga, 29010, Spain

  • Hadassah Medical Organization

    Jerusalem, Jerusalem, 9112001, Israel

  • Hopital Bicetre

    Paris, Le Kremlin-Bicêtre, 94270, France

  • Hospital A.C.Camargo Cancer Center - Clinical Oncology

    São Paulo, Brazil

  • Hospital Aleman

    Ciudad de Buenos Aires, Buenos Aires, C118AAT, Argentina

  • Hospital Ampang

    Ampang, Selangor, 68000, Malaysia

  • Hospital Erasto Gaertner

    Curitiba, Paraná, 81520-060, Brazil

  • Hospital Israelita Albert Einstein

    São Paulo, São Paulo, 05651-901, Brazil

  • Hospital Pablo Tobon Uribe

    Antioquia, Colombia

  • Hospital Pulau Pinang

    Pulau Pinang, Georgetown, 10990, Malaysia

  • Hospital Raja Perempuan Zainab II

    Kota Bharu, Kelantan, 15586, Malaysia

  • Hospital Sultanah Aminah Johor Bahru

    Johor Bahru, Johor, 80000, Malaysia

  • Hospital U. 12 Octubre

    Madrid, Madrid, 28041, Spain

  • Hospital Universitario Virgen

    Granada, Granada, 18014, Spain

  • Hospital Universitario de Gran

    Las Palmas de Gran Canaria, Canary Islands, 35019, Spain

  • Hospital das Clínicas UFG

    Goiânia, Goiás, 74605, Brazil

  • Hospital de Clinicas de Porto Alegre - UFRGS

    Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029, United States

  • Inje University Busan Paik Hos

    Busan, Busan Gwang'Yeogsi [Pusan-Kwangyokshi], 47392, South Korea

  • Inje University Haeundae Paik Hospital

    Busan, Busan Gwang'Yeogsi [Pusan-Kwangyokshi], 48108, South Korea

  • Institut Català d'Oncologia-Ho

    Badalona, Barcelona, 08916, Spain

  • Irccs Irst

    Meldola, 47014, Italy

  • Istanbul üniversitesi cerrahpaşa

    Istanbul, Istanbul, 34098, Turkey (Türkiye)

  • J.S.C."K.Eristavi National Cen

    Tbilisi, K'alak'i T'bilisi, 0159, Georgia

  • Kaplan Medical Center

    Rehovot, Central District, 7610001, Israel

  • Kepler Universitätsklinikum Gm

    Linz, Upper Austria, 4021, Austria

  • Klinikum Wels-Grieskirchen GmbH

    Wels, Upper Austria, 4600, Austria

  • Krankenhaus Hietzing mit Neurologischem Zentrum Rosenhügel

    Wein, Burgenland, 1130, Austria

  • Krankenhaus der Elisabethinen

    Linz, Upper Austria, 4020, Austria

  • LTD Israeli-Georgian Medical R

    Tbilisi, 0112, Georgia

  • Ltd "Medinvest - Institute of Hematology and Trans

    Tbilisi, K'alak'i T'bilisi, 186, Georgia

  • M.Zodelava Hematology Center L

    Tbilisi, 0112, Georgia

  • MONIKI - Oncology

    Moscow, Moscow Oblast, 129110, Russia

  • MUZ City Clinical Hospital # 2

    Novosibirsk, Novosibirsk Oblast, 630091, Russia

  • Marmara university pendik training and research hospital

    Istanbul, Istanbul, 34899, Turkey (Türkiye)

  • Martin-Luther-Universität Halle-Wittenberg

    Halle, Saxony-Anhalt, 6120, Germany

  • Medical Park Mersin Hastanesi

    Mersin, Mersin, Turkey (Türkiye)

  • Medipol Bagcilar Mega Hospital

    Istanbul, Istanbul, 34214, Turkey (Türkiye)

  • Multi Profile Clinic Consilium

    Tbilisi, 0179, Georgia

  • National University Cancer Institute (NCIS)

    Singapore, Central Singapore, 119074, Singapore

  • Nilratan Sircar Medical College

    Kolkata, West Bengal, 700014, India

  • Nirmal Hospital - Hematology

    Surat, Gujarat, 395002, India

  • Northwest Medical Specialties PLLC

    Seattle, Washington, 98405, United States

  • Norton Cancer Institute

    Louisville, Kentucky, 40207, United States

  • Odense University Hospital - Hematology

    Odense, DK-5000 C, Denmark

  • Oncologos del Occidente S.A

    Pereira, Risaralda Department, 660000, Colombia

  • Ospedale Civile S.Maria delle Croci, AUSL Ravenna

    Ravenna, Ravenna, 48121, Italy

  • Ospedale S.Bortolo, AULSS n.6

    Vicenza, Vicenza, 36100, Italy

  • Ospedale di Circolo, PO Varese

    Varese, Varese, 21100, Italy

  • Oxford University Hospitals

    Oxford, OX3 9DS, United Kingdom

  • PO Civile SS.Antonio e Biagio

    Alessandria, Alessandria, 15121, Italy

  • Policlinico Universitario Agostino Gemelli

    Roma, Roma, 00168, Italy

  • Prairie Lakes Health Care System, Inc.

    Watertown, South Dakota, 57201, United States

  • Pratia Onkologia Katowice

    Katowice, Silesian Voivodeship, 40-519, Poland

  • Presidio Ospedaliero Garibaldi

    Catania, 95122, Italy

  • Royal Brisbane and Women's Hospital

    Herston, Queensland, 4011, Australia

  • Royal Hobart Hospital

    Hobart, Tasmania, 7000, Australia

  • Royal North Shore Hospital

    St Leonards, New South Wales, 2065, Australia

  • Sahyadri Specialty Hospital

    Pune, India

  • Sakarya Research and Training Hospital - Medical Oncology

    Adapazarı, 54100, Turkey (Türkiye)

  • Sanatorio Allende

    Córdoba, X5000JHQ, Argentina

  • Sanatorio de la Mujer

    Rosario, Santa Fe Province, S2000ORE, Argentina

  • Sarawak General Hospital

    Kuching, Sarawak, 93586, Malaysia

  • Seoul National University Hospital - Department of Internal

    Seoul, Seoul Teugbyeolsi [Seoul-T'Ukp, 03080, South Korea

  • Severance Hospital, Yonsei Uni

    Seoul, 3722, South Korea

  • Shamir Medical Center (Assaf Harofeh)

    Ẕerifin, Central District, 7030000, Israel

  • Singapore General Hospital

    Singapore, Central Singapore, 169608, Singapore

  • Sir Ganga Ram Hospital

    New Delhi, National Capital Territory of Delhi, 110060, India

  • Smilow Cancer Center at YNHH

    New Haven, Connecticut, 06511, United States

  • Soroka Medical Center - Hematology Institute

    Beersheba, 84101, Israel

  • Specialized Hospital for Active Therapy of Hematological dis

    Sofia, Sofia, 1756, Bulgaria

  • St Bartholomew's Hospital

    London, EC1A 7BE, United Kingdom

  • St. John's Medical College Hospital

    Bangalore, Karnataka, 560 034, India

  • Szabolcs-Szatmár-Bereg Megyei Önkormányzat Jósa András

    Nyíregyháza, 4400, Hungary

  • The Catholic University of Korea

    Seongdong, 6591, South Korea

  • The University of Texas MD

    Houston, Texas, 77030-4000, United States

  • UCLA David Geffen School of Medicine

    Los Angeles, California, 90096, United States

  • UMBAL Sveti Georgi

    Plovdiv, Plovdiv, 4002, Bulgaria

  • UZ Antwerpen

    Edegem, Antwerpen, 2650, Belgium

  • Universitair Ziekenhuis Brussel - Myeloom Centrum Brussel (MCB)

    Jette, Belgium

  • Universitair Ziekenhuis Gent

    Ghent, Oost-Vlaanderen, 9000, Belgium

  • University of California-San Diego/Moores UCSD Cancer Center

    La Jolla, California, 92093-1503, United States

  • University of South Florida

    Tampa, Florida, 33612, United States

  • Universitätsklinikum Carl Gust

    Dresden, Free and Hanseatic City of Hamburg, 1307, Germany

  • Universitätsklinikum Leipzig AöR

    Leipzig, Saxony, 04103, Germany

  • Universitätsklinikum Mannheim - University of Heidelberg

    Mannheim, Baden-Wurttemberg, 68167, Germany

  • Universitätsklinikum Tübingen

    Tübingen, Baden-Wurttemberg, 72076, Germany

  • Wojewodzki Szpital Specjalistyczny

    Biała Podlaska, 21-500, Poland

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