Comparing Imetelstat to Best Available Therapy for Myelofibrosis
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
Run by Geron Corporation · NCT04576156 · Data updated 2026-05-14
This study is no longer in our active list. It may have stopped recruiting or finished. The details below are as of 2026-05-14 and are kept for reference. Check ClinicalTrials.gov for its current status, or see similar studies below.
This study is testing if a new drug called imetelstat can help people with myelofibrosis who haven't improved with standard treatments live longer compared to the best available therapy.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 320 (estimated) |
| Sponsor | Geron Corporation Industry-sponsored |
| Drugs / interventions | ruxolitinib, chemotherapy, prednisone |
| Locations | 215 sites (La Jolla, California and 214 other locations) |
| Trial ID | NCT04576156 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are individuals diagnosed with primary myelofibrosis or post-essential thrombocythemia/polycythemia vera myelofibrosis who are relapsed/refractory to JAK-inhibitor treatment.
✕Probably not a fit
Patients who are not relapsed/refractory to JAK-inhibitor treatment or those eligible for allogeneic stem cell transplantation may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This multicenter Phase 3 trial evaluates the overall survival of patients with intermediate-2 or high-risk myelofibrosis who have not responded to Janus Kinase (JAK)-inhibitor treatment. Participants will undergo a screening phase followed by randomization into two arms: one receiving imetelstat and the other receiving the best available therapy. The study includes a treatment phase and a post-treatment follow-up phase to monitor outcomes until death or study end. Patients who show progressive disease while on best available therapy may have the option to crossover to imetelstat treatment.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option for patients with myelofibrosis who have not responded to existing therapies.
How similar studies have gone: Other studies have shown promise with similar approaches, but this specific comparison of imetelstat to best available therapy is novel.
Who can join: the full rules
Show full inclusion / exclusion criteria
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
Where it's running
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University of California-San Diego/Moores UCSD Cancer Center
La Jolla, California, United States Recruiting -
UCLA David Geffen School of Medicine
Los Angeles, California, United States Recruiting -
Smilow Cancer Center at YNHH
New Haven, Connecticut, United States Recruiting -
BRCR Medical Center Inc
Plantation, Florida, United States Recruiting -
University of South Florida
Tampa, Florida, United States Recruiting -
Norton Cancer Institute
Louisville, Kentucky, United States Recruiting
Show 44 more locations
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Icahn School of Medicine at Mount Sinai
New York, New York, United States Recruiting -
Duke University Medical Center
Durham, North Carolina, United States Recruiting -
Gabrail Cancer Center
Canton, Ohio, United States Recruiting -
Prairie Lakes Health Care System, Inc.
Watertown, South Dakota, United States Recruiting -
The University of Texas MD
Houston, Texas, United States Recruiting -
Hospital Aleman
Ciudad de Buenos Aires, Buenos Aires, Argentina Recruiting -
Sanatorio de la Mujer
Rosario, Santa Fe, Argentina Recruiting -
Sanatorio Allende
Córdoba, Argentina Recruiting -
Royal North Shore Hospital
St Leonards, New South Wales, Australia Recruiting -
Royal Brisbane and Women's Hospital
Herston, Queensland, Australia Recruiting -
Royal Hobart Hospital
Hobart, Tasmania, Australia Recruiting -
Epworth Healthcare
Richmond, Victoria, Australia Recruiting -
Krankenhaus Hietzing mit Neurologischem Zentrum Rosenhügel
Wein, Burgenland, Austria Recruiting -
Krankenhaus der Elisabethinen
Linz, Oberösterreich, Austria Recruiting -
Kepler Universitätsklinikum Gm
Linz, Oberösterreich, Austria Recruiting -
Klinikum Wels-Grieskirchen GmbH
Wels, Oberösterreich, Austria Recruiting -
Centre Hospitalier de Jolimont
Haine-Saint-Paul, Hainaut, Belgium Recruiting -
CHU De Liège
Liege, Liège, Belgium Recruiting -
Universitair Ziekenhuis Gent
Gent, Oost-Vlaanderen, Belgium Recruiting -
AZ Klina
Antwerpen, Belgium Recruiting -
Universitair Ziekenhuis Brussel - Myeloom Centrum Brussel (MCB)
Jette, Belgium Recruiting -
Centro de oncologia Leonardo da Vinci
Fortaleza, Ceará, Brazil Recruiting -
Hospital das Clínicas UFG
Goiânia, Goiás, Brazil Recruiting -
Hospital Erasto Gaertner
Curitiba, Paraná, Brazil Recruiting -
Hospital de Clinicas de Porto Alegre - UFRGS
Porto Alegre, Rio Grande Do Sul, Brazil Recruiting -
Centro Gaucho Integrado de Oncologia, Hematologia, Ensino e Pesquisa LTDA
Porto Alegre, Rio Grande Do Sul, Brazil Recruiting -
CEPON Centro de Pesquisas Oncologicas SC
Florianópolis, Brazil Recruiting -
Instituto de Educação, Pesquisa e Gestão em Saúde
São Paulo, Brazil Recruiting -
Hospital Israelita Albert Einstein
São Paulo, Brazil Recruiting -
Fundacao Doutor Amaral Carvalho / Hospital Amaral Carvalho
São Paulo, Brazil Recruiting -
Hospital A.C.Camargo Cancer Center - Clinical Oncology
São Paulo, Brazil Recruiting -
UMBAL Sveti Georgi
Plovdiv, Bulgaria Recruiting -
Oncologos del Occidente S.A
Pereira, Risaralda, Colombia Recruiting -
Hospital Pablo Tobon Uribe
Antioquia, Colombia Recruiting -
Centro Medico Imbanaco de Cali S.A.
Cali, Colombia Recruiting -
Foscal
Floridablanca, Colombia Recruiting -
Odense University Hospital - Hematology
Odense, Denmark Recruiting -
Institut de cancérologie du Gard - Hematologie clinique
Nimes Cedex 09, Gard, France Recruiting -
CHU Bretonneau
Tours, Indre-et-Loire, France Recruiting -
Hopital Bicetre
Paris, Le Kremlin-Bicêtre, France Recruiting -
CHU de Limoges Dupuytren
Haute-Vienne, Limoges, France Recruiting -
CHU De Nantes - Hématologie C
Nantes, Loire-Atlantique, France Recruiting -
CHU de Nice - Hopital de l'Archet II - Pharmacie
Nice, Nice Cedex 3, France Recruiting -
Hospital CENTRE HOSPITALIER AVIGNON
Avignon cedex, Provence-Alpes-Côte-d'Azur, France Recruiting
+165 more sites. See ClinicalTrials.gov for the full list.
Study contacts
- Study coordinator: Michelle Mudge-Riley, DO
- Email: myf3001-info@Geron.com
- Phone: +1 (650) 473-7793
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
MPN PROGRESSion Registry — tracking symptoms, treatments, and disease progression in people with MPNs.
Track long-term symptoms, treatments, and disease progression in adults with myeloproliferative neoplasms to see how these conditions evolve over time.
-
Nelfinavir to improve anemia in people with myelofibrosis by restoring iron delivery to the bone marrow
This trial tests whether the antiviral drug nelfinavir can help people with myelofibrosis and anemia by raising hemoglobin and lowering blood markers of fibrosis.
-
National registry for people with myelofibrosis and anemia in Germany.
This project will collect health, treatment, and quality-of-life information from people in Germany with primary or secondary myelofibrosis and anemia to see how their care and symptoms change over time.
-
Evaluating DISC-0974 for treating anemia in myelofibrosis patients
This study tests if a new treatment called DISC-0974 can help people with myelofibrosis who have anemia feel better and improve their condition.
-
Elritercept vs. placebo for adults with myelofibrosis and anemia on ruxolitinib
This study tests whether elritercept can improve anemia in adults with myelofibrosis who are taking ruxolitinib, compared with placebo.
-
TQ05105 tablets for intermediate/high-risk myelofibrosis
This phase 2 study tests TQ05105 tablets to see if they help people with intermediate or high-risk myelofibrosis who have not benefited from prior JAK inhibitors.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.