ClinConnect ClinConnect Logo
Search / Trial NCT04576156

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

Launched by GERON CORPORATION · Oct 2, 2020

Trial Information

Current as of August 22, 2025

Recruiting

Keywords

Myeloproliferative Neoplasms Polycythemia Thrombocythemia Primary Myelofibrosis Janus Kinase Inhibitor

ClinConnect Summary

This clinical trial is studying a new treatment called imetelstat for patients with myelofibrosis (MF), a type of blood cancer, who have not responded well to standard treatments known as JAK inhibitors. The goal is to see if imetelstat can help these patients live longer compared to the best available therapy currently offered. This study is specifically looking for adults aged 65 and older who have intermediate-2 or high-risk MF and have experienced a relapse or worsening of their condition after JAK inhibitor treatment.

To be eligible for the trial, participants should have a confirmed diagnosis of myelofibrosis and show specific signs of the disease, such as a large spleen and active symptoms that impact their daily life. They should not have received certain treatments or have other serious health conditions that could interfere with the study. Participants in the trial can expect to receive either imetelstat or the best available therapy, and they will be closely monitored for their overall health and response to treatment throughout the study. This trial is actively recruiting participants who meet the criteria and are looking for new treatment options.

Gender

ALL

Eligibility 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

About Geron Corporation

Geron Corporation is a clinical-stage biopharmaceutical company focused on developing innovative therapies for cancer treatment, particularly through its proprietary telomerase inhibitor platform. With a commitment to advancing science and improving patient outcomes, Geron is dedicated to addressing unmet medical needs in oncology. The company actively conducts clinical trials to evaluate the safety and efficacy of its lead product candidates, leveraging cutting-edge research to bring transformative therapies to patients with hematologic malignancies and solid tumors. Through strategic partnerships and a robust pipeline, Geron aims to establish itself as a leader in cancer therapeutics.

Locations

Tampa, Florida, United States

Durham, North Carolina, United States

Sioux Falls, South Dakota, United States

Linz, , Austria

Houston, Texas, United States

Hobart, Tasmania, Australia

Herston, Queensland, Australia

Haifa, , Israel

New York, New York, United States

Canton, Ohio, United States

Salt Lake City, Utah, United States

San Diego, California, United States

Tacoma, Washington, United States

Zurich, , Switzerland

Tacoma, Washington, United States

Tampa, Florida, United States

London, , United Kingdom

Curitiba, , Brazil

St Leonards, New South Wales, Australia

Milano, , Italy

Louisville, Kentucky, United States

Tübingen, , Germany

Wels, , Austria

Seoul, , Korea, Republic Of

São Paulo, , Brazil

Taichung City, , Taiwan

Goshen, Indiana, United States

Seoul, , Korea, Republic Of

New Orleans, Louisiana, United States

Kuching, Sarawak, Malaysia

Taipei, , Taiwan

Bangalore, Karnataka, India

Durham, North Carolina, United States

Alicante, , Spain

Roma, , Italy

Richmond, Victoria, Australia

Leipzig, Sachsen, Germany

Córdoba, , Argentina

Incheon, , Korea, Republic Of

Kota Bharu, Kelantan, Malaysia

Long Beach, California, United States

Taipei, , Taiwan

Houston, Texas, United States

Maidstone, Kent, United Kingdom

Seoul, , Korea, Republic Of

Curitiba, Paraná, Brazil

Roseville, Michigan, United States

Gent, Oost Vlaanderen, Belgium

Seoul, , Korea, Republic Of

Seoul, , Korea, Republic Of

Sioux Falls, South Dakota, United States

Floridablanca, , Colombia

Seoul, , Korea, Republic Of

York, Pennsylvania, United States

Omaha, Nebraska, United States

Ogden, Utah, United States

Pleven, , Bulgaria

Valencia, , Spain

New Delhi, Delhi, India

Porto, , Portugal

Georgetown, Pulau Pinang, Malaysia

Firenze, , Italy

Madrid, , Spain

Watertown, South Dakota, United States

Columbus, Ohio, United States

Fort Wayne, Indiana, United States

Fredericksburg, Virginia, United States

Edegem, Antwerpen, Belgium

Tübingen, Baden Württemberg, Germany

Ampang, Selangor, Malaysia

Busan, , Korea, Republic Of

Leipzig, Saxony, Germany

Antwerpen, , Belgium

Torino, , Italy

Pune, , India

Izmir, , Turkey

Hartford, Connecticut, United States

Ankara, , Turkey

Cáceres, , Spain

Katowice, , Poland

Rockville, Maryland, United States

Genova, Liguria, Italy

Istanbul, , Turkey

Antwerpen, , Belgium

Haifa, , Israel

Varese, , Italy

Seoul, , Korea, Republic Of

Valencia, , Spain

Istanbul, , Turkey

Torino, , Italy

Long Beach, California, United States

Saint Joseph, Missouri, United States

Omsk, , Russian Federation

Springfield, Missouri, United States

Bologna, , Italy

Salem, Oregon, United States

Meldola, , Italy

Busan, Busan Gwang'yeogsi [Pusan Kwangyokshi], Korea, Republic Of

Milano, Lombard, Italy

Genova, , Italy

Milano, , Italy

Bhubaneswar, Orissa, India

Rosario, Santa Fe, Argentina

Goiânia, , Brazil

Alessandria, , Italy

Zaragoza, , Spain

Wels, Oberösterreich, Austria

'Aiea, Hawaii, United States

Johor Bahru, Johor, Malaysia

Fayetteville, Arkansas, United States

La Jolla, California, United States

Los Angeles, California, United States

Plantation, Florida, United States

Houston, Texas, United States

Ciudad De Buenos Aires, Buenos Aires, Argentina

Rosario, Santa Fe, Argentina

Wein, Burgenland, Austria

Linz, Oberösterreich, Austria

Haine Saint Paul, Hainaut, Belgium

Antwerpen, , Belgium

Antwerpen, , Belgium

Jette, , Belgium

Fortaleza, Ceará, Brazil

Goiânia, Goiás, Brazil

Porto Alegre, Rio Grande Do Sul, Brazil

Porto Alegre, Rio Grande Do Sul, Brazil

Florianópolis, , Brazil

São Paulo, , Brazil

São Paulo, , Brazil

Pleven, , Bulgaria

Plovdiv, , Bulgaria

Sofia, , Bulgaria

Pereira, Risaralda, Colombia

Antioquia, , Colombia

Cali, , Colombia

Floridablanca Santander, , Colombia

Aarhus, , Denmark

Odense, , Denmark

Nimes Cedex 09, Gard, France

Tours, Indre Et Loire, France

Nantes, Loire Atlantique, France

Avignon Cedex, Provence Alpes Côte D'azur, France

Pierre Benite, Rhône Alpes, France

Le Mans, Sarthe, France

Bobigny, Seine Saint Denis, France

Brest Cedex, , France

Paris, , France

Paris, île De France, France

Georgia, , Georgia

Tbilisi, , Georgia

Tbilisi, , Georgia

Tbilisi, , Georgia

Tbilisi, , Georgia

Tübingen, Baden Württemberg, Germany

Dresden, Hamburg, Germany

Halle (Saale), Sachsen Anhalt, Germany

Kiel, Schleswig Holstein, Germany

Kempten, , Germany

Budapest, , Hungary

Budapest, , Hungary

Nyíregyháza, , Hungary

Székesfehérvár, , Hungary

New Delhi, Delhi, India

Surat, Gujarat, India

Bengaluru, Karnataka, India

Gurgaon, New Delhi, India

Kolkata, West Bengal, India

Hyderabad, , India

Pune, , India

Ashdod, Hadarom, Israel

Ashkelon, Hadarom, Israel

Rehovot, Hamerkaz, Israel

Zerifin, Hamerkaz, Israel

Nahariya, Hazafon, Israel

Tel Aviv, Tel Aviv, Israel

Jerusalem, Yerushalayim, Israel

Cona, Ferrara, Italy

Rozzano, Milano, Italy

Orbassano, Torino, Italy

Catania, , Italy

Genova, , Italy

Milano, , Italy

Monza, , Italy

Napoli, , Italy

Ravenna, , Italy

Reggio Calabria, , Italy

Reggio Calabria, , Italy

Rimini, , Italy

Roma, , Italy

Vicenza, , Italy

Daegu, Daegu Gwang'yeogsi [Taegu Kwan], Korea, Republic Of

Seoul, Seoul Teugbyeolsi [Seoul T'ukp, Korea, Republic Of

Busan, , Korea, Republic Of

Seongdong, , Korea, Republic Of

Kelantan, , Malaysia

Slupsk, Pomorskie, Poland

Piła, Wielkopolskie, Poland

Skorzewo, Wielkopolskie, Poland

Katowice, śląskie, Poland

Braga, , Portugal

Coimbra, , Portugal

Lisboa, , Portugal

Lisboa, , Portugal

Lisboa, , Portugal

Lisboa, , Portugal

Moscow, Moskovskaya Oblast', Russian Federation

Novosibirsk, Novosibirskaya Oblast', Russian Federation

Saratov, Saratovskaya Oblast', Russian Federation

Tula, Tul'skaya Oblast', Russian Federation

Omsk, , Russian Federation

Saint Petersburg, , Russian Federation

Saint Petersburg, , Russian Federation

Saint Petersburg, , Russian Federation

Singapore, Central Singapore, Singapore

Singapore, Central Singapore, Singapore

Badalona, Barcelona, Spain

Sabadell, Barcelona, Spain

Las Palmas De Gran Canaria, Canarias, Spain

Alzira, Valencia, Spain

Girona, , Spain

Granada, , Spain

Madrid, , Spain

Madrid, , Spain

Málaga, , Spain

Salamanca, , Spain

Chiayi City, Chiayi, Taiwan

Ankara, , Turkey

London, , United Kingdom

Oxford, , United Kingdom

Seoul, , Korea, Republic Of

South Bend, Indiana, United States

Saint Petersburg, , Russian Federation

Canton, Ohio, United States

Tbilisi, , Georgia

Halle (Saale), Sachsen Anhalt, Germany

Pulau Pinang, Georgetown, Malaysia

Kempten, Rheinland Pfalz, Germany

Taichung City, , Taiwan

Greenville, South Carolina, United States

Whittier, California, United States

Herston, Queensland, Australia

Badalona, Barcelona, Spain

Linz, Oberösterreich, Austria

Liege, Liège, Belgium

Haute Vienne, Limoges, France

Nice, Nice Cedex 3, France

Loire, Saint Priest En Jarez, France

Mannheim, Baden Württemberg, Germany

Beersheba, , Israel

Busan, Busan Gwang'yeogsi [Pusan Kwangyokshi], Korea, Republic Of

Seoul, Seoul Teugbyeolsi [Seoul T'ukpyolshi], Korea, Republic Of

Málaga, Marbella, Spain

Adapazarı, , Turkey

Istanbul, , Turkey

Yenisehir/Mersin, , Turkey

Goyang Si, Gyeonggido, Korea, Republic Of

Incheon, , Korea, Republic Of

Seoul, Seoul Teugbyeolsi [Seoul T'ukpyolshi], Korea, Republic Of

Seoul, Seoul Teugbyeolsi [Seoul T'ukpyolshi], Korea, Republic Of

Seoul, Seoul Teugbyeolsi [Seoul T'ukpyolshi], Korea, Republic Of

New Haven, Connecticut, United States

Moscow, Moskva, Russian Federation

East Syracuse, New York, United States

São Paulo, , Brazil

Paris, Le Kremlin Bicêtre, France

Biała Podlaska, , Poland

Baton Rouge, Louisiana, United States

Novosibirsk, Novosibirskaya Oblast', Russian Federation

Saint Petersburg, , Russian Federation

Saratov, , Russian Federation

Los Alamitos, California, United States

Mersin, Içel, Turkey

Novosibirsk, Novosibirskaya Oblast', Russian Federation

Maumee, Ohio, United States

Tacoma, Washington, United States

Tbilisi, , Georgia

Székesfehérvár, , Hungary

Ravenna, , Italy

Rimini, , Italy

Daegu, Daegu Gwang'yeogsi [Taegu Kwan], Korea, Republic Of

Coimbra, , Portugal

Salamanca, , Spain

Clermont, Florida, United States

Reno, Nevada, United States

Saint Louis, Missouri, United States

Linz, , Austria

Liege, , Belgium

Fortaleza, , Brazil

Paris, , France

Nice, , France

Avignon Cedex, , France

Pierre Benite, , France

Paris, , France

Mannheim, , Germany

Busan, , Korea, Republic Of

Daegu, , Korea, Republic Of

Mersin, , Turkey

Covington, Louisiana, United States

Patients applied

0 patients applied

Trial Officials

Faye Feller

Study Director

Geron Corporation

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

Similar Trials