51 Participants Needed

Imetelstat + Ruxolitinib for Myelofibrosis

Recruiting at 7 trial locations
TB
SD
SN
JH
Overseen ByJudy Ho
Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: Geron Corporation
Must be taking: Ruxolitinib
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is testing a combination of two drugs, imetelstat and ruxolitinib, in patients with myelofibrosis, a type of bone marrow cancer. Imetelstat works by stopping cancer cells from multiplying, while ruxolitinib slows their growth. The study aims to find the best dose and evaluate the safety and effectiveness of this combination. Ruxolitinib is a targeted drug approved for treating myelofibrosis, known for reducing spleen size and improving survival rates.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop taking your current medications, but you cannot have taken any investigational drugs, hydroxyurea, chemotherapy, immunomodulatory or immunosuppressive therapy, or high-dose corticosteroids within 14 days before enrolling.

Is the combination of Imetelstat and Ruxolitinib safe for treating myelofibrosis?

Ruxolitinib, used for myelofibrosis, has been studied for over a decade and is generally safe, though it can cause anemia (low red blood cell count) and thrombocytopenia (low platelet count). These side effects are usually manageable and rarely lead to stopping treatment.12345

How is the drug combination of Imetelstat and Ruxolitinib unique for treating myelofibrosis?

The combination of Imetelstat and Ruxolitinib is unique because Imetelstat, a telomerase inhibitor, has shown to significantly improve overall survival in patients with myelofibrosis who have not responded to other treatments, like Ruxolitinib alone, which is a common JAK inhibitor. This combination offers a novel approach for patients with limited options after JAK inhibitor failure.678910

What data supports the effectiveness of the drug combination Imetelstat and Ruxolitinib for treating myelofibrosis?

Research shows that Imetelstat improved overall survival in myelofibrosis patients who had stopped responding to other treatments, with a median survival of 30 months compared to 12 months for those on other therapies. Ruxolitinib has been shown to improve symptoms and quality of life in myelofibrosis patients, making the combination potentially beneficial.235611

Who Is on the Research Team?

SN

Shyamala Navada, MD

Principal Investigator

Geron Corporation

Are You a Good Fit for This Trial?

This trial is for adults with a condition called myelofibrosis, who are either already on ruxolitinib treatment or haven't been treated with JAK inhibitors. They should have symptoms like an enlarged spleen or other related issues and must not be candidates for stem cell transplant. Participants need to meet certain blood and biochemical test criteria, have a performance status score of 0-2, and agree to use contraception.

Inclusion Criteria

I can take care of myself and am up and about more than half of my waking hours.
Participants should follow protocol defined contraceptives procedures
A woman of childbearing potential must have a negative serum or urine pregnancy test at screening
See 7 more

Exclusion Criteria

I have HIV or a severe infection needing IV antibiotics.
Peripheral blood blast count of ≥10% or bone marrow blast count of ≥10%
I have had surgery to remove all or part of my spleen.
See 9 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part 1

Participants who have received ruxolitinib for at least 12 weeks prior to Screening will initiate imetelstat therapy. Dose levels of imetelstat may include 4.7, 6, 7.5, 9.4mg, until a RP2D is established.

24 weeks
Regular visits for dose adjustments and monitoring

Treatment Part 2

JAK inhibitor naïve participants will receive initial treatment with ruxolitinib for at least 12 weeks, including 4 weeks at a stable dose, followed by imetelstat treatment at the RP2D in combination with ruxolitinib.

24 weeks
Regular visits for monitoring and assessment

Follow-up

Participants are monitored for safety and effectiveness after treatment

30 days after the last dose

What Are the Treatments Tested in This Trial?

Interventions

  • Imetelstat
  • Ruxolitinib
Trial Overview The study tests the combination of two drugs: Imetelstat and Ruxolitinib in people with myelofibrosis. The first part determines the best dose of Imetelstat when used with Ruxolitinib, while the second part assesses safety and how well this combined dose works.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Group I: Part 2: Imetelstat sodium + Ruxolitinib or other JAK inhibitorExperimental Treatment5 Interventions
Group II: Part 1: Imetelstat sodium + RuxolitinibExperimental Treatment2 Interventions

Imetelstat is already approved in United States for the following indications:

🇺🇸
Approved in United States as Rytelo for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Geron Corporation

Lead Sponsor

Trials
20
Recruited
1,500+

Published Research Related to This Trial

In the MYF2001 trial, patients with relapsed or refractory myelofibrosis treated with imetelstat showed a median overall survival of 29.9 months, indicating its potential efficacy in this challenging condition.
When compared to best available therapy (BAT) in a closely matched cohort, imetelstat significantly reduced the risk of death (hazard ratio: 0.35), suggesting it may offer a substantial survival advantage over traditional treatments after JAK inhibitor failure.
Favorable overall survival with imetelstat in relapsed/refractory myelofibrosis patients compared with real-world data.Kuykendall, AT., Sun, L., Mascarenhas, J., et al.[2022]
In a study of 219 patients with myelofibrosis, ruxolitinib significantly improved health-related quality of life (HRQoL) and reduced disease-related symptoms over 48 weeks compared to best available therapy (BAT).
Ruxolitinib treatment led to notable enhancements in physical functioning, fatigue, and appetite, with significant improvements observed as early as week 8 and continuing throughout the study, supporting its efficacy as a treatment for symptomatic myelofibrosis.
Health-related quality of life and symptoms in patients with myelofibrosis treated with ruxolitinib versus best available therapy.Harrison, CN., Mesa, RA., Kiladjian, JJ., et al.[2021]
In a phase II study, ruxolitinib, starting at a low dose of 5 mg twice daily, showed significant efficacy in myelofibrosis patients with low platelet counts (50-100 × 10^9/L), with 62% of patients achieving stable doses of 10 mg twice daily by week 24, and median reductions in spleen volume and symptoms of 24.2% and 43.8%, respectively.
While some patients experienced thrombocytopenia requiring dose adjustments, the treatment was generally manageable, with mean hemoglobin levels remaining stable, indicating that ruxolitinib can be safely administered to this subset of patients.
Interim analysis of safety and efficacy of ruxolitinib in patients with myelofibrosis and low platelet counts.Talpaz, M., Paquette, R., Afrin, L., et al.[2021]

Citations

Favorable overall survival with imetelstat in relapsed/refractory myelofibrosis patients compared with real-world data. [2022]
Health-related quality of life and symptoms in patients with myelofibrosis treated with ruxolitinib versus best available therapy. [2021]
Interim analysis of safety and efficacy of ruxolitinib in patients with myelofibrosis and low platelet counts. [2021]
JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. [2022]
Management of Myelofibrosis during Treatment with Ruxolitinib: A Real-World Perspective in Case of Resistance and/or Intolerance. [2022]
Primary analysis of JUMP, a phase 3b, expanded-access study evaluating the safety and efficacy of ruxolitinib in patients with myelofibrosis, including those with low platelet counts. [2021]
Ten years of treatment with ruxolitinib for myelofibrosis: a review of safety. [2023]
Fedratinib: a pharmacotherapeutic option for JAK-inhibitor naïve and exposed patients with myelofibrosis. [2022]
Ruxolitinib is manageable in patients with myelofibrosis and severe thrombocytopenia: a report on 12 Danish patients. [2021]
A subgroup analysis of JUMP, a phase IIIb, expanded-access study evaluating the safety and efficacy of ruxolitinib in patients with myelofibrosis in a Brazilian cohort. [2021]
11.United Statespubmed.ncbi.nlm.nih.gov
Randomized, Single-Blind, Multicenter Phase II Study of Two Doses of Imetelstat in Relapsed or Refractory Myelofibrosis. [2021]
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