Experimental drug combo aims to tame bone marrow cancer
NCT ID NCT05371964
First seen Jun 27, 2026 · Last updated Aug 27, 2026 · Updated 2 times
Summary
This early-stage trial tests two drugs—imetelstat and ruxolitinib—together in people with myelofibrosis, a type of bone marrow cancer. The study has two parts: first, finding a safe dose of the combination, and second, checking if it improves symptoms like fatigue and spleen discomfort. About 36 adults with intermediate or high-risk myelofibrosis are being recruited.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- imetelstat and ruxolitinib
- What this could lead to
- If it works, this combination could offer better symptom control and possibly slow disease progression for people with myelofibrosis.
- What could go wrong
- This is an early Phase 1 trial with only 36 participants, so safety and effectiveness are not yet proven. Side effects from the drug combination are unknown.
Why investors are watching
Geron is testing its drug imetelstat combined with ruxolitinib in 30 patients with myelofibrosis, a type of bone marrow cancer. For a small company like Geron, this early-stage trial matters because a positive safety and activity signal could expand the use of imetelstat beyond its current approved setting. The result is consequential because Geron's value depends heavily on how many patients can eventually use this drug.
If it works: If the combination proves safe and shows signs of shrinking or controlling the disease, Geron could move to a larger trial and potentially win approval for a broader patient group. That would give the company a second revenue source and reduce its reliance on a single use of imetelstat.
If it fails: Early-stage trials often fail to show enough benefit or reveal safety problems, and this one has only 30 participants, so the results may be unclear or disappointing. A negative or delayed readout could set back Geron's plans and hurt investor confidence, since the company has few other products in late-stage development.
AI-written from the trial record. Speculative, and not investment advice.
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
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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30 people
The number who actually took part.
- Started
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May 2022
- Expected to finish
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Aug 2028
An estimate. End dates often move.
- Lead sponsor
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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
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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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.
Inclusion Criteria: * Diagnosis of primary myelofibrosis (PMF) according to the revised World Health Organization (WHO) criteria or post-essential thrombocythemia-MF or post-polycythemia vera according to the International Working Group for Myelofibrosis Research and Treatment (IWG-MRT) criteria. * Dynamic International Prognostic Scoring System (DIPSS) intermediate-1, intermediate-2 or high-risk MF. * Candidate for ruxolitinib treatment: * Part 1 participants: On ruxolitinib treatment for at least 12 weeks with at least 4 consecutive weeks immediately prior to enrollment at a stable dose. * Part 2 participants: Candidate for ruxolitinib treatment as assessed by the investigator and has not previously been treated with a JAK inhibitor (Cohort A) OR currently receiving ruxolitinib per standard of care for at least 12 weeks with at least 4 consecutive weeks at a stable dose prior to enrollment (Cohort B). Note that the study will no longer recruit participants into Cohort A. * Active symptoms of MF on the MFSAF v4.0 demonstrated by: * Part 1 participants only: At least 2 symptoms with a score ≥ 1 * Part 2 participants only: At least 2 symptoms with a score of ≥ 3, or a total score of at least 10. * Ineligible for or unwilling to undergo hematopoietic stem cell transplant at time of study entry. * Hematology laboratory test values within protocol defined limits. * Biochemical laboratory test values 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%. * Prior treatment with JAK inhibitor (except for participants being dosed optimized on ruxolitinib treatment prior to screening and enrollment in part 1 or Part 2 Cohort B). * Known allergies, hypersensitivity, or intolerance to imetelstat or ruxolitinib or excipients. * Prior treatment with imetelstat. * Major surgery within 28 days prior to enrollment. * Any investigational drug regardless of class or mechanism of action, hydroxyurea, chemotherapy, (except for ruxolitinib for participants being dose optimized prior to enrollment), immunomodulatory or immunosuppressive therapy, corticosteroids \>30 mg/day prednisone or equivalent ≤14 days prior to enrollment. * Prior history of hematopoietic stem cell transplant. * 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 enrollment. * Adequately treated non-melanoma skin cancer or lentigo maligna without evidence of disease. * Adequately treated cervical carcinoma in situ without evidence of disease. * Clinically significant cardiovascular disease. * Known history of human immunodeficiency virus (HIV) or any uncontrolled active systemic infection requiring IV antibiotics. * Active systemic hepatitis infection requiring treatment or any known acute or chronic liver disease unless related to MF. Carriers of hepatitis virus are permitted to enter the study.
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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
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City of Hope
Duarte, California, 91010, United States
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City of Hope
Irvine, California, 92618, United States
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Fred Hutchinson Cancer Center
Seattle, Washington, 98109, United States
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H. Lee Moffitt Cancer Center and Research Institute, Inc.
Tampa, Florida, 33612, United States
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Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
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University of Miami
Coral Gables, Florida, 33146, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Half-Matched stem cells tested as cure for myelofibrosis
- Can blood tests predict transplant complications?
- Can a new daily pill shrink the spleen and ease myelofibrosis symptoms?
- Can a new pill tame myelofibrosis?
- MRI as a window into bone marrow disease: a new biomarker test?
- Can a menin inhibitor tame myelofibrosis when standard drugs fall short?