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Experimental drug combo aims to tame bone marrow cancer

NCT ID NCT05371964

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 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

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

30 people

The number who actually took part.

Started

May 2022

Expected to finish

Aug 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 (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

  • City of Hope

    Duarte, California, 91010, United States

  • City of Hope

    Irvine, California, 92618, United States

  • Fred Hutchinson Cancer Center

    Seattle, Washington, 98109, United States

  • H. Lee Moffitt Cancer Center and Research Institute, Inc.

    Tampa, Florida, 33612, United States

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029, United States

  • University of Miami

    Coral Gables, Florida, 33146, United States

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