AzTrials
AzTrials

MyelofibrosisPhase 3NCT04576156

A Study Comparing Imetelstat Versus Best Available Therapy for the Treatment of Intermediate-2 or High-risk Myelofibrosis (MF) Who Have Not Responded to Janus Kinase (JAK)-Inhibitor Treatment

A Phase 3 study testing Imetelstat and Best Available Therapy (BAT) in people with Myelofibrosis, run by Geron Corporation at 144 sites in 26 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

The purpose of the study is to evaluate the overall survival of participants treated with imetelstat compared to best available therapy with intermediate-2 or high-risk Myelofibrosis (MF) who are relapsed/refractory (R/R) to Janus Kinase (JAK)-Inhibitor treatment. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelofibrosis
What is being tested?
Imetelstat and Best Available Therapy (BAT)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
327 participants
Where
144 sitesin 26 countries
Study dates
12 Apr 2021to 30 Jun 2028
Phase
Phase 3
Are there results?
Not yet: the study is under way

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

  • 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:
  • no decrease in spleen volume (< 10% by MRI or CT) from the start of treatment with JAK-inhibitor
  • no decrease in spleen size (< 30% by palpation or length by imaging) from the start of treatment with JAK-inhibitor
Show all 22Show fewer
  • no decrease in symptoms (< 20% by Myelofibrosis Symptom Assessment Form [MFSAF] or myeloproliferative neoplasm SAF) from the start of treatment with JAK-inhibitor
  • 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
  • Increase in spleen volume from time of best response by 25% measured by MRI or CT, or
  • 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

Who cannot join

  • 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
Show all 12Show fewer
  • 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

Trial locations and contacts

144 sites in 26 countries.

144 sites in 26 countries

Italy21 sites

  • PO Civile SS.Antonio e Biagio

    Alessandria, Alessandria, 15121

  • A.O.di Bologna Policl.S.Orsola

    Bologna, Bologna, 40138

  • Presidio Ospedaliero Garibaldi

    Catania, 95122

  • Arcispedale S.Anna - Ematologi

    Cona, Ferrara, 44124

  • AOU Careggi

    Florence, Firenze, 50134

  • Clinica Ematologica, Univ. Deg

    Genova, Genova, 6- 16132

  • Irccs Irst

    Meldola, 47014

  • Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

    Milan, Milano, 20122

  • ASST Grande Ospedale Metropoli

    Milan, 20162

  • Azienda Ospedaliera San Gerard

    Monza, 20900

  • AOU Federico II

    Naples, 80122

  • AOU San Luigi Gonzaga

    Orbassano, Torino, 10043

  • Ospedale Civile S.Maria delle Croci, AUSL Ravenna

    Ravenna, Ravenna, 48121

  • Arcispedale S Maria Nuova, AO di Reggio Emilia

    Reggio Calabria, Reggio Calabria, 42123

  • Azienda Ospedaliera Bianchi-Me

    Reggio Calabria, Reggio Calabria, 89124

  • AUSL di Rimini Ospedale Infermi di Rimini

    Rimini, Rimini, 47900

  • Policlinico Universitario Agostino Gemelli

    Roma, Roma, 00168

  • ASL Roma 2 - PO "S. Eugenio"

    Roma, 00144

  • AOU Città della Salute e della Scienza di Torino

    Torino, Torino, 10126

  • Ospedale di Circolo, PO Varese

    Varese, Varese, 21100

  • Ospedale S.Bortolo, AULSS n.6

    Vicenza, Vicenza, 36100

Other trials in Italy

United States12 sites

  • Gabrail Cancer Center

    Canton, Ohio, 44718

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • The University of Texas MD

    Houston, Texas, 77030-4000

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

    La Jolla, California, 92093-1503

  • UCLA David Geffen School of Medicine

    Los Angeles, California, 90096

  • Norton Cancer Institute

    Louisville, Kentucky, 40207

  • Smilow Cancer Center at YNHH

    New Haven, Connecticut, 06511

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029

  • BRCR Medical Center Inc

    Plantation, Florida, 33326

  • Northwest Medical Specialties PLLC

    Seattle, Washington, 98405

  • University of South Florida

    Tampa, Florida, 33612

  • Prairie Lakes Health Care System, Inc.

    Watertown, South Dakota, 57201

Other trials in United States

India10 sites

  • St. John's Medical College Hospital

    Bangalore, Karnataka, 560 034

  • Fortis Hospital 154/9

    Bengaluru, Karnataka, 560076

  • All India Institute of Medical Sciences

    Bhubaneswar, Odisha, 751019

  • Fortis Memorial Research Institute

    Gūrgaon, New Delhi, 122002

  • Nilratan Sircar Medical College

    Kolkata, West Bengal, 700014

  • 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

  • Sir Ganga Ram Hospital

    New Delhi, National Capital Territory of Delhi, 110060

  • Deenanath Mangeshkar Hospital & Research Center

    Pune, 411004

  • Sahyadri Specialty Hospital

    Pune

  • Nirmal Hospital - Hematology

    Surat, Gujarat, 395002

Other trials in India

Brazil9 sites

  • Hospital Erasto Gaertner

    Curitiba, Paraná, 81520-060

  • CEPON Centro de Pesquisas Oncologicas SC

    Florianópolis, 88034-000

  • Centro de oncologia Leonardo da Vinci

    Fortaleza, Ceará, 60135285

  • Hospital das Clínicas UFG

    Goiânia, Goiás, 74605

  • Hospital de Clinicas de Porto Alegre - UFRGS

    Porto Alegre, Rio Grande do Sul, 90035-903

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

    Porto Alegre, Rio Grande do Sul, 90470340

  • Hospital Israelita Albert Einstein

    São Paulo, São Paulo, 05651-901

  • Fundacao Doutor Amaral Carvalho / Hospital Amaral Carvalho

    São Paulo, São Paulo, 17210-120

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

    São Paulo

Other trials in Brazil

Spain9 sites

  • H.G.U. Alicante

    Alicante, Alicante, 03010

  • Institut Català d'Oncologia-Ho

    Badalona, Barcelona, 08916

  • H. San Pedro de Alcántara

    Cáceres, Cáceres, 10003

  • Hospital Universitario Virgen

    Granada, Granada, 18014

  • Hospital Universitario de Gran

    Las Palmas de Gran Canaria, Canary Islands, 35019

  • Hospital U. 12 Octubre

    Madrid, Madrid, 28041

  • H.U. La Paz

    Madrid, Madrid, 28046

  • H.U.V. de la Victoria

    Málaga, Málaga, 29010

  • H. Quirón Zaragoza

    Zaragoza, Zaragoza, 50012

Other trials in Spain

France8 sites

  • CHRU Brest - Hôpital Morvan

    Brest, 29609

  • Centre Hospitalier Du Mans - Cancérologie Médicale

    Le Mans, Sarthe, 72037

  • CHU De Nantes - Hematologie

    Nantes, Loire-Atlantique, 44000

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

    Nice, Nice Cedex 3, 06202

  • Hopital Bicetre

    Paris, Le Kremlin-Bicêtre, 94270

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

    Paris, 75010

  • Centre Hospitalier Lyon

    Pierre-Bénite, Auvergne-Rhône-Alpes, 69495

  • CHU Bretonneau

    Tours, Indre-et-Loire, 37044

Other trials in France

Israel8 sites

  • Assuta Ashdod University Hospi

    Ashdod, Southern District, 7747629

  • Barzilai Medical Center

    Ashkelon, Southern District, 78278

  • Soroka Medical Center - Hematology Institute

    Beersheba, 84101

  • Bnai Zion Medical Center

    Haifa, 31048

  • Carmel MC

    Haifa, 3436212

  • Hadassah Medical Organization

    Jerusalem, Jerusalem, 9112001

  • Kaplan Medical Center

    Rehovot, Central District, 7610001

  • Shamir Medical Center (Assaf Harofeh)

    Ẕerifin, Central District, 7030000

Other trials in Israel

Turkey (Türkiye)8 sites

  • Sakarya Research and Training Hospital - Medical Oncology

    Adapazarı, 54100

  • Ankara University Medical Facu

    Ankara, Ankara, 6590

  • Gazi University Medical Faculty

    Ankara, 06560

  • Istanbul üniversitesi cerrahpaşa

    Istanbul, Istanbul, 34098

  • Medipol Bagcilar Mega Hospital

    Istanbul, Istanbul, 34214

  • Marmara university pendik training and research hospital

    Istanbul, Istanbul, 34899

  • Ege Universitesi Tip Fakultesi

    Izmir, İzmir, 35100

  • Medical Park Mersin Hastanesi

    Mersin, Mersin

Other trials in Turkey (Türkiye)

Belgium5 sites

  • AZ Klina

    Antwerp, Antwerpen, 2930

  • UZ Antwerpen

    Edegem, Antwerpen, 2650

  • Universitair Ziekenhuis Gent

    Ghent, Oost-Vlaanderen, 9000

  • Centre Hospitalier de Jolimont

    Haine-Saint-Paul, Hainaut, 7100

  • Universitair Ziekenhuis Brussel - Myeloom Centrum Brussel (MCB)

    Jette

Other trials in Belgium

Georgia5 sites

  • J.S.C."K.Eristavi National Cen

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

  • Ltd "Medinvest - Institute of Hematology and Trans

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

  • LTD Israeli-Georgian Medical R

    Tbilisi, 0112

  • M.Zodelava Hematology Center L

    Tbilisi, 0112

  • Multi Profile Clinic Consilium

    Tbilisi, 0179

Other trials in Georgia

Germany5 sites

  • Universitätsklinikum Carl Gust

    Dresden, Free and Hanseatic City of Hamburg, 1307

  • Martin-Luther-Universität Halle-Wittenberg

    Halle, Saxony-Anhalt, 6120

  • Universitätsklinikum Leipzig AöR

    Leipzig, Saxony, 04103

  • Universitätsklinikum Mannheim - University of Heidelberg

    Mannheim, Baden-Wurttemberg, 68167

  • Universitätsklinikum Tübingen

    Tübingen, Baden-Wurttemberg, 72076

Other trials in Germany

Malaysia5 sites

  • Hospital Ampang

    Ampang, Selangor, 68000

  • Hospital Sultanah Aminah Johor Bahru

    Johor Bahru, Johor, 80000

  • Hospital Raja Perempuan Zainab II

    Kota Bharu, Kelantan, 15586

  • Sarawak General Hospital

    Kuching, Sarawak, 93586

  • Hospital Pulau Pinang

    Pulau Pinang, Georgetown, 10990

Other trials in Malaysia

South Korea5 sites

  • Inje University Busan Paik Hos

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

  • Inje University Haeundae Paik Hospital

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

  • The Catholic University of Korea

    Seongdong, 6591

  • Seoul National University Hospital - Department of Internal

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

  • Severance Hospital, Yonsei Uni

    Seoul, 3722

Other trials in South Korea

Australia4 sites

  • Royal Brisbane and Women's Hospital

    Herston, Queensland, 4011

  • Royal Hobart Hospital

    Hobart, Tasmania, 7000

  • Epworth Healthcare

    Richmond, Victoria, 3121

  • Royal North Shore Hospital

    St Leonards, New South Wales, 2065

Other trials in Australia

Austria4 sites

  • Krankenhaus der Elisabethinen

    Linz, Upper Austria, 4020

  • Kepler Universitätsklinikum Gm

    Linz, Upper Austria, 4021

  • Krankenhaus Hietzing mit Neurologischem Zentrum Rosenhügel

    Wein, Burgenland, 1130

  • Klinikum Wels-Grieskirchen GmbH

    Wels, Upper Austria, 4600

Other trials in Austria

Poland4 sites

  • Wojewodzki Szpital Specjalistyczny

    Biała Podlaska, 21-500

  • Pratia Onkologia Katowice

    Katowice, Silesian Voivodeship, 40-519

  • Ars Medical Sp. z o.o.

    Piła, Greater Poland Voivodeship, 64-920

  • Centrum Medyczne Pratia Poznan

    Skórzewo, Greater Poland Voivodeship, 60-185

Other trials in Poland

Russia4 sites

  • MONIKI - Oncology

    Moscow, Moscow Oblast, 129110

  • MUZ City Clinical Hospital # 2

    Novosibirsk, Novosibirsk Oblast, 630091

  • Budgetary Healthcare Institution of Omsk Region

    Omsk, 644013

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

    Saratov, 410012

Other trials in Russia

Argentina3 sites

  • Hospital Aleman

    Ciudad de Buenos Aires, Buenos Aires, C118AAT

  • Sanatorio Allende

    Córdoba, X5000JHQ

  • Sanatorio de la Mujer

    Rosario, Santa Fe Province, S2000ORE

Other trials in Argentina

United Kingdom3 sites

  • St Bartholomew's Hospital

    London, EC1A 7BE

  • Guys and St Thomas' Hospital

    London, SE1 9RT

  • Oxford University Hospitals

    Oxford, OX3 9DS

Other trials in United Kingdom

Bulgaria2 sites

  • UMBAL Sveti Georgi

    Plovdiv, Plovdiv, 4002

  • Specialized Hospital for Active Therapy of Hematological dis

    Sofia, Sofia, 1756

Other trials in Bulgaria

Colombia2 sites

  • Hospital Pablo Tobon Uribe

    Antioquia

  • Oncologos del Occidente S.A

    Pereira, Risaralda Department, 660000

Other trials in Colombia

Hungary2 sites

  • Dél-pesti Centrumkórház Ország

    Budapest, H-1097

  • Szabolcs-Szatmár-Bereg Megyei Önkormányzat Jósa András

    Nyíregyháza, 4400

Other trials in Hungary

Singapore2 sites

  • National University Cancer Institute (NCIS)

    Singapore, Central Singapore, 119074

  • Singapore General Hospital

    Singapore, Central Singapore, 169608

Other trials in Singapore

Taiwan2 sites

  • Chang Gung Medical Foundation

    Chiayi City, Chiayi, 613

  • China Medical University Hospital - Hematology/Onc

    Taichung, 404

Other trials in Taiwan

Denmark1 site

  • Odense University Hospital - Hematology

    Odense, DK-5000 C

Other trials in Denmark

Portugal1 site

  • Centro Clinico Fundacao Champalimaud

    Lisbon, Lisbon District, 1400-038

Other trials in Portugal

About this study

Official title: A Randomized Open-Label, Phase 3 Study to Evaluate Imetelstat (GRN163L) Versus Best Available Therapy (BAT) in Patients With Intermediate-2 or High-risk Myelofibrosis (MF) Relapsed / Refractory (R/R) to Janus Kinase (JAK) Inhibitor

The purpose of the study is to evaluate the overall survival of participants treated with imetelstat compared to best available therapy with intermediate-2 or high-risk Myelofibrosis (MF) who are relapsed/refractory (R/R) to Janus Kinase (JAK)-Inhibitor treatment.

Read the detailed description

This is a multicenter study with 2 arms, and will include 3 phases: a) screening phase of up to 28 days before randomization during which participants will complete a 14-day washout period from all prior therapies including JAK-inhibitor treatment, and the participant's eligibility will be reviewed; b) treatment phase, from randomization until study treatment (imetelstat or BAT) discontinuation; and c) post treatment follow-up phase, that begins when the participant discontinues treatment, and will continue until death, lost to follow-up, withdrawal of consent, or study end, whichever occurs first. Participants will be randomized (2:1) into 2 Arms (Arm A will receive imetelstat and Arm B will receive BAT).

Participants who meet progressive disease criteria and discontinue BAT, may crossover to receive imetelstat treatment after sponsor's approval.

Conditions
Myelofibrosis
Being tested
Imetelstat, Best Available Therapy (BAT)
Keywords
Myeloproliferative neoplasms, Polycythemia, Thrombocythemia, Primary myelofibrosis, Janus Kinase-Inhibitor
Medical subject headings
Primary Myelofibrosis, Myeloproliferative Disorders, Polycythemia, Thrombocytosis

What participants receive

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

  • ImetelstatDrug

    Also called GRN163L

    Imetelstat sodium will be given intravenously at 9.4 mg/kg every 21 days, until disease progression or unacceptable toxicity, treatment discontinuation or study end.

  • Best Available Therapy (BAT)Drug

    Non-JAK-inhibitor treatment will be given, which may include but is not limited to hydroxyurea, thalidomide or an analog of thalidomide, interferon, danazol, hypomethylating agents, chemotherapy or radiotherapy.

Study groups in NCT04576156
Study groupRoleWhat they receive
ImetelstatExperimentalParticipants will receive imetelstat sodium at 9.4 mg/kg intravenous (IV) every 21 days (±3 days), until disease progression or unacceptable toxicity, treatment discontinuation or study end.
Receives: Drug: Imetelstat
Best Available Therapy (BAT)Active comparatorParticipants will receive BAT (investigator-selected non-JAK-inhibitor treatment), until disease progression or unacceptable toxicity, treatment discontinuation or study end. Participants on BAT who meet protocol-defined criteria for progressive disease may crossover to receive imetelstat treatment after sponsor's approval.
Receives: Drug: Best Available Therapy (BAT)

What is being measured

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

  1. Overall survival (OS)

    Overall survival is defined as the time interval from randomization date to date of death from any cause.

    Measured: Baseline (Day 1) until End of Study (EOS) (approximately 3 years )]

Secondary outcomes (12)

  1. Symptom response rate

    The proportion of participants achieving a ≥50% reduction in Total Symptom Score (TSS) measured at Week 24 compared to baseline

    Measured: Baseline (Day 1), and at Week 24

  2. Progression-free survival

    Progression-free survival is defined as the time interval from randomization date to the first date of disease progression (worsening splenomegaly or leukemic transformation per 2013 International Working Group - Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria) or death from any cause, whichever occurs first.

    Measured: Baseline (Day 1) until End of Study (EOS) (approximately 3 years)

  3. Spleen response rate

    The proportion of participants who achieve a reduction in spleen volume of ≥ 35% from baseline at Week 24.

    Measured: Baseline (Day 1), and at Week 24

  4. Complete remission (CR), partial remission (PR), clinical improvement (CI), spleen response, symptoms response, and anemia response per modified 2013 IWG-MRT criteria

    The proportion of participants achieving CR or PR, CI, spleen response, symptom response, and anemia response per modified 2013 IWG-MRT criteria.

    Measured: Baseline (Day 1) until End of Treatment (approximately 3 years)

  5. Reduction in the degree of bone marrow fibrosis

    Reduction in the degree of bone marrow fibrosis will be assessed.

    Measured: Baseline (Day 1) until End of Treatment (approximately 3 years)

  6. Number of Participants with Adverse Events

    Safety will be assessed based on the incidence and severity (according to the Common Terminology Criteria for Adverse Events) of treatment emergent adverse events from the time of randomization until 30 days after completion of treatment

    Measured: Screening (Day -28 to -1) until End of Study (approximately 3 years)

  7. Assessment of Cmax

    Maximum Observed Plasma Concentration (Cmax).

    Measured: Day 1 of all cycles (each cycle is 21 days)

  8. Assessment of Tmax

    Time to reach the maximum observed plasma concentration

    Measured: Day 1 of all cycles (each cycle is 21 days)

  9. Assessment of t1/2

    Elimination half-life.

    Measured: Day 1 of all cycles (each cycle is 21 days)

  10. Assessment of AUC

    Area under the drug concentration-plasma time curve (AUC) from time zero to last measurable concentration

    Measured: Day 1 of all cycles (each cycle is 21 days)

  11. European Organization for Research and Treatment of Cancer Quality-of-Life-Questionnaire-Core-30 (EORTC QLQ-C30) scores

    Patient-reported outcomes including health-related quality of life, pain, and overall change in participant's health will be assessed using the EORTC QLQ-C30. The EORTC QLQ-C30 includes 30 items resulting in 5 functional scales (physical functioning, role functioning, emotional functioning, cognitive functioning, and social functioning), 1 Global Health Status scale, 3 symptom scales (fatigue, nausea and vomiting, and pain), and 6 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties). Scores are transformed to a 0 to 100 scale. Higher scores indicated worse outcome.

    Measured: Baseline to End of Study (approximately 3 years)

  12. EuroQol-EQ-5D (EQ-5D-5L) questionnaire scores

    EQ-5D-5L is a 5 item questionnaire that assesses 5 domains including mobility, self-care, usual activities, pain/discomfort and anxiety/depression plus a visual analog scale rating "health today" with anchors ranging from 0 (worst imaginable health state) to 100 (best imaginable health state).

    Measured: Baseline to End of Study (approximately 3 years)

Timeline

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

  1. First posted6 Oct 2020
  2. Study start12 Apr 2021
  3. Primary completion30 Jun 2028 (estimated)
  4. Study completion30 Jun 2028 (estimated)

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
None (open label)
Enrolment
327 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04576156
Sponsor's protocol ID
MYF3001
Other IDs
2020-003288-24, 2023-509120-17-00, 2023
Status last verified
Jun 2026
First submitted
18 Sep 2020
Last updated
8 Jul 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Mascarenhas J, Harrison CN, Kiladjian JJ, Komrokji RS, Koschmieder S, Vannucchi AM, Berry T, Redding D, Sherman L, Dougherty S, Peng L, Sun L, Huang F, Wan Y, Feller FM, Rizo A, Verstovsek S. Imetelstat in intermediate-2 or high-risk myelofibrosis refractory to JAK inhibitor: IMpactMF phase III study design. Future Oncol. 2022 Jul;18(22):2393-2402. doi: 10.2217/fon-2022-0235. Epub 2022 May 5.

    PubMed 35510486

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Imetelstat and Best Available Therapy (BAT) in people with Myelofibrosis, run by Geron Corporation at 144 sites in 26 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 22 inclusion and 12 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: under way, not looking for participants, as of the sponsor's last update on 8 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 144 sites in Italy, United States, India, Brazil, Spain and 21 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?

This study is still under way. Results are not available in the source record yet.

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?

Overall survival (OS) (time frame: Baseline (Day 1) until End of Study (EOS) (approximately 3 years )]). 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 NCT04576156, 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 8 Jul 2026.

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