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MyelofibrosisPhase 1NCT05371964

A Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of Imetelstat in Combination With Ruxolitinib in Participants With Myelofibrosis

A Phase 1 study testing Imetelstat sodium and Ruxolitinib in people with Myelofibrosis, run by Geron Corporation at 6 sites in United States.

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 identify the recommended Part 2 dose (R2PD) of imetelstat sodium in combination with ruxolitinib in participants with myelofibrosis (MF) in Part 1, and to evaluate the safety and preliminary clinical activity of the R2PD of imetelstat sodium in combination with ruxolitinib in… 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 sodium and Ruxolitinib
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
6 sitesUnited States
Study dates
4 May 2022to Aug 2028
Phase
Phase 1
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 (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:
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  • 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.

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

United States6 sites

  • University of Miami

    Coral Gables, Florida, 33146

  • City of Hope

    Duarte, California, 91010

  • City of Hope

    Irvine, California, 92618

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029

  • Fred Hutchinson Cancer Center

    Seattle, Washington, 98109

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

    Tampa, Florida, 33612

Other trials in United States

About this study

Official title: An Open Label, Phase 1/1b Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of Imetelstat in Combination With Ruxolitinib in Patients With Myelofibrosis

The purpose of the study is to identify the recommended Part 2 dose (R2PD) of imetelstat sodium in combination with ruxolitinib in participants with myelofibrosis (MF) in Part 1, and to evaluate the safety and preliminary clinical activity of the R2PD of imetelstat sodium in combination with ruxolitinib in participants with MF in Part 2.

Conditions
Myelofibrosis
Being tested
Imetelstat sodium, Ruxolitinib
Medical subject headings
Primary Myelofibrosis

What participants receive

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

  • Imetelstat sodiumDrug

    Also called GRN163L

    Imetelstat sodium will be administered as intravenous (IV) every 28 days.

  • RuxolitinibDrug

    Ruxolitinib will be administered, orally (PO), twice daily (BID) in cohort B as the standard of care per local prescribing guidelines.

Study groups in NCT05371964
Study groupRoleWhat they receive
Part 1: Imetelstat sodium + RuxolitinibExperimentalParticipants who have received ruxolitinib orally (PO) as part of standard of care (SOC) for at least 12 weeks prior to Screening will be enrolled. After enrollment, participants will initiate imetelstat sodium therapy. Dose levels of imetelstat sodium may include 4.7, 6, 7.5, 9.4 mg/kg, until a RP2D is established.
Receives: Drug: Imetelstat sodium; Drug: Ruxolitinib
Part 2: Imetelstat sodium + RuxolitinibExperimentalCohort A: Janus Kinase (JAK) inhibitor naive participants will receive initial treatment with ruxolitinib on study for at least 12 weeks, including 4 consecutive weeks at a stable dose, prior to the addition of imetelstat sodium. Participants can begin imetelstat sodium treatment after sponsor review and approval and meet the following requirements: platelet value is ≥75 x 10^9/L for two consecutive measurements, at least 1 week apart, and the participant does not meet criteria for dose delay. Note that the study will no longer recruit participants into Cohort A. Cohort B: Participants will receive treatment with ruxolitinib for 12 weeks with at least 4 consecutive weeks at a stable dose prior to enrollment and will start combination treatment with imetelstat on study.
Receives: Drug: Imetelstat sodium; Drug: Ruxolitinib

What is being measured

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

  1. Part 1: Incidence, Type, and Severity of Adverse Events, Including Dose-limiting Toxicity (DLT) During the DLT Observation Period and/or Study Treatment

    Measured: 28 days after first dose

  2. Part 2: Number of Participants With Treatment-emergent Adverse Event (AE)

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

    Measured: First dose of study treatment until 30 days after the last dose of study treatment (up to approximately 5 years)

  3. Part 2: Symptom Response Rate at Week 24

    Symptom response rate is defined as percentage of participants with >=50% reduction in the Total Symptom Score (TSS) measured by the Myelofibrosis Symptom Assessment Form (MFSAF) v4.0 e-diary at 24 week compared to baseline.

    Measured: Week 24

Secondary outcomes (15)

  1. Part 1: Pharmacokinetic Profile of Ruxolitinib (Maximum Observed Plasma Concentration [Cmax]

    Measured: From first dose of Ruxolitinib treatment up to approximately 5 years

  2. Part 1: Pharmacokinetic Profile of Ruxolitinib Time to Reach Maximum Plasma Concentration [Tmax])

    Measured: From first dose of imetelstat treatment up to approximately 5 years

  3. Part 1 and Part 2: Pharmacokinetic Profile of Imetelstat Sodium Maximum Observed Plasma Concentration [Cmax]

    Measured: From first dose of imetelstat treatment up to approximately 5 years

  4. Part 1 and Part 2: Pharmacokinetic Profile of Imetelstat Time to Reach Maximum Plasma Concentration [Tmax])

    Measured: From first dose of imetelstat treatment up to approximately 5 years

  5. Part 1 and Part 2: Percentage of Participants with Anti-imetelstat Antibodies

    Measured: From first dose of imetelstat treatment up to approximately 5 years

  6. Part 1: Symptom Response at Week 24

    Symptom response rate is defined as percentage of participants with >50% reduction in the TSS measured by the MFSAF v4.0 e-diary at 24 week compared to baseline.

    Measured: Baseline, Week 24

  7. Part 1 and Part 2: Absolute Change From Baseline in TSS at Week 24

    Absolute change is defined as change in total symptom score from baseline to week 24 as measured by the MFSAF v4.0 e-diary.

    Measured: Baseline, Week 24

  8. Part 1 and Part 2: Average Absolute Change in TSS Over 24 weeks

    Average absolute change in TSS is defined as change in the average of absolute change in total symptom score from week 1(baseline) to week 24 as measured by the MFSAF v4.0 e-diary.

    Measured: Baseline, Week 24

  9. Part 1 and Part 2: Spleen Response at Week 24

    Spleen response is the proportion of participants who achieve a reduction in spleen volume of ≥35% from baseline confirmed by magnetic resonance imaging (MRI) or computed tomography (CT).

    Measured: Week 24

  10. Part 1 and Part 2: Progression Free Survival (PFS)

    The time interval from start of study treatment date to the first date of disease progression or death from any cause, whichever occurs first.

    Measured: From start of study treatment date to the disease progression or death (up to approximately 5 years)

  11. Part 1 and Part 2: Percentage of Participants With Complete Remission (CR), Partial Remission (PR), Clinical Improvement (CI) Per the Modified 2013 International Working Group - Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) Criteria.

    Measured: From first dose to end of the treatment (up to approximately 5 years)

  12. Part 1 and Part 2: Time to Response

    Time to response was defined as the duration from first dose of study treatment to the earliest date that a response is first documented. The response CI/CR/PR was assessed by IWG-MRT criteria.

    Measured: From first dose of study treatment to the earliest date that a response was first documented (Up to approximately 5 years)

  13. Part 1 and Part 2: Duration of Response (DOR) Per IWG-MRT Criteria

    DOR measured from time of initial response (CR/PR/CI) until documented PD or death whichever occurs first.

    Measured: From time of initial response to PD or death whichever occurs first (up to approximately 5 years)

  14. Part 1 and Part 2: Reduction of Bone Marrow Fibrosis

    Reduction of bone marrow fibrosis is defined as the percentage of participants with a post-baseline bone marrow fibrosis degree smaller than the baseline fibrosis degree prior to start of subsequent anticancer therapy.

    Measured: From first dose to end of the treatment (up to approximately 5 years)

  15. Part 1 and Part 2: Time to Progression to Acute Myeloid Leukemia (AML)

    Time to progression to AML, defined as the time interval from the start of study treatment date to the first date of documented progression to AML or death from any cause, whichever occurs first.

    Measured: From first dose to end of the treatment (up to approximately 5 years)

Timeline

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

  1. First posted12 May 2022
  2. Study start4 May 2022
  3. Primary completionFeb 2027 (estimated)
  4. Study completionAug 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 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
30 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05371964
Sponsor's protocol ID
MYF1001
Status last verified
Aug 2026
First submitted
5 May 2022
Last updated
26 Aug 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Imetelstat sodium and Ruxolitinib in people with Myelofibrosis, run by Geron Corporation at 6 sites in United States.

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 14 inclusion and 14 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 26 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Part 1: Incidence, Type, and Severity of Adverse Events, Including Dose-limiting Toxicity (DLT) During the DLT Observation Period and/or Study Treatment (time frame: 28 days after first dose). The study lists 3 primary outcomes in all. 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 NCT05371964.

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 26 Aug 2026.

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