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Imetelstat for Myelofibrosis

Phase 3
Recruiting
Research Sponsored by Geron Corporation
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
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: no decrease in spleen volume (< 10% by MRI or CT) from the start of treatment with JAK-inhibitor, no decrease in spleen size (< 30% by palpation or length by imaging) from the start of treatment with JAK-inhibitor, no decrease in symptoms (< 20% by Myelofibrosis Symptom Assessment Form [MFSAF] or myeloproliferative neoplasm SAF) from the start of treatment with JAK-inhibitor, a score of at least 15 on TSS assessed using the MFSAF v4.0 during screening.
Eastern Cooperative Oncology Group Performance Status score of 0, 1, or 2
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to end of study (approximately 3 years)
Awards & highlights

Study Summary

This trial is testing a new drug for people with myelofibrosis who have not responded to other treatments. The goal is to see if it improves survival.

Who is the study for?
This trial is for adults with intermediate-2 or high-risk Myelofibrosis who haven't improved after treatment with JAK-inhibitor drugs. They should not be eligible for a stem cell transplant, have symptoms of MF, and meet certain blood test criteria. People can't join if they've had recent major surgery, other cancers (with some exceptions), uncontrolled infections, liver disease unrelated to MF, or previous imetelstat treatment.Check my eligibility
What is being tested?
The study compares the effectiveness of Imetelstat versus Best Available Therapy (BAT) in improving overall survival rates in patients whose Myelofibrosis hasn't responded to JAK-inhibitors. Participants will either receive Imetelstat or their doctor's choice of another therapy deemed best available.See study design
What are the potential side effects?
Imetelstat may cause side effects such as low blood counts leading to anemia and increased risk of infection, liver problems, nausea and vomiting. The specific side effects depend on the individual patient's reaction and the type of Best Available Therapy chosen.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I've been on a JAK-inhibitor for 6+ months without improvement in spleen size or symptoms.
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I am able to care for myself and perform daily activities.
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I have been diagnosed with a specific type of bone marrow disorder.
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I've been on a high-dose JAK-inhibitor for 3+ months without improvement in spleen size or symptoms.
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My spleen has grown after JAK inhibitor treatment and I can't have more of this treatment.
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I have symptoms of myelofibrosis with a score of 5 or more.
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My spleen is enlarged, confirmed by a doctor's exam or imaging.
Select...
My condition is considered high-risk by myelofibrosis standards.
Select...
My condition did not improve with JAK-inhibitor treatment and I cannot have a stem cell transplant.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to end of study (approximately 3 years)
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to end of study (approximately 3 years) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Overall survival (OS)
Secondary outcome measures
Assessment of AUC
Assessment of Cmax
Assessment of Tmax
+9 more

Side effects data

From 2018 Phase 2 trial • 80 Patients • NCT01731951
100%
Neutrophil count decreased
100%
Diarrhoea
100%
Fatigue
100%
Platelet count decreased
100%
White blood cell count decreased
89%
Anaemia
78%
Hyperglycaemia
56%
Aspartate aminotransferase increased
56%
Pain
56%
Alanine aminotransferase increased
44%
Nausea
44%
Back pain
44%
Blood creatinine increased
33%
Contusion
33%
Lipase increased
33%
Blood bilirubin increased
22%
Cough
22%
Cardiac failure
22%
Oedema peripheral
22%
Upper respiratory tract infection
22%
Weight decreased
22%
Anorexia
22%
Decreased appetite
22%
Hypernatraemia
22%
Hypocalcaemia
22%
Hyponatraemia
22%
Insomnia
22%
Dyspnoea
22%
Constipation
22%
Blood amylase increased
22%
Pain in extremity
22%
Hyperhidrosis
22%
Hypertension
22%
Hypokalaemia
22%
Muscular weakness
22%
Neck pain
22%
Headache
22%
Alopecia
22%
Arthralgia
22%
Weight increased
11%
Dizziness
11%
Anxiety
11%
Early satiety
11%
Vomiting
11%
Lung infection
11%
Infusion related reaction
11%
Pyrexia
11%
Blood alkaline phosphatase increased
11%
Gamma-glutamyltransferase increased
11%
Lymphocyte count decreased
11%
Hyperkalaemia
11%
Hyperuricaemia
11%
Night sweats
11%
Sepsis
11%
Epistaxis
11%
Atrial fibrillation
11%
Abdominal distension
11%
Abdominal pain upper
11%
Non-cardiac chest pain
11%
Fall
11%
Musculoskeletal pain
11%
Pulmonary hypertension
11%
Sinusitis
11%
Hypotension
100%
80%
60%
40%
20%
0%
Study treatment Arm
Arm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)
Arm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia
Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF)
Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts])
Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts])
Arm A: Imetelstat 9.4 mg/kg (MF)

Trial Design

2Treatment groups
Experimental Treatment
Active Control
Group I: ImetelstatExperimental Treatment1 Intervention
Participants will receive imetelstat at 9.4 mg/kg intravenous (IV) every 21 days (±3 days), until disease progression or unacceptable toxicity, treatment discontinuation or study end.
Group II: Best Available Therapy (BAT)Active Control1 Intervention
Participants will receive BAT (investigator-selected non-JAK-inhibitor treatment), until disease progression or unacceptable toxicity, treatment discontinuation or study end. Participants on BAT who meet protocol-defined criteria for progressive disease may crossover to receive imetelstat treatment after sponsor's approval.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Imetelstat
2012
Completed Phase 2
~100

Find a Location

Who is running the clinical trial?

Geron CorporationLead Sponsor
18 Previous Clinical Trials
1,132 Total Patients Enrolled
Faye FellerStudy DirectorGeron Corporation

Media Library

Best Available Therapy (BAT) Clinical Trial Eligibility Overview. Trial Name: NCT04576156 — Phase 3
Myelofibrosis Research Study Groups: Imetelstat, Best Available Therapy (BAT)
Myelofibrosis Clinical Trial 2023: Best Available Therapy (BAT) Highlights & Side Effects. Trial Name: NCT04576156 — Phase 3
Best Available Therapy (BAT) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04576156 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are we able to enroll patients in this clinical trial at the current time?

"That is accurate. The information available on clinicaltrials.gov affirms that this study, which was initially advertised on April 12th 2021, is still looking for patients to enroll. There are 320 participants needed in total and they will be recruited from 13 different locations."

Answered by AI

Could you walk me through other studies that have used Imetelstat in the past?

"Since 2015, when imetelstat was first studied at Nottingham City Hospital - Clinical Haematology, there have been 17 completed clinical trials. Out of these, 2 are actively recruiting patients. A significant number of these studies are based in New york City."

Answered by AI

Are there any foreseeable risks associated with Imetelstat?

"Imetelstat's safety is estimated to be a 3. This Phase 3 rating means that not only does efficacy have some supporting data, but there have been multiple rounds of tests affirming Imetelstat's safety."

Answered by AI

How many test subjects are involved in this experiment?

"This study demands 320 individuals that fit the pre-established inclusion criteria. The sponsor, Geron Corporation, plans to run the trial from different locations including Weill Cornell Medical College and Gabrail Cancer Center."

Answered by AI

Is this study popular in Canada?

"There are 13 clinical trial sites for this study, which can be found in cities such as New york, Canton and Durham. If you choose to participate in the trial, it may be helpful to select a location nearest you to reduce travel costs and time commitment."

Answered by AI

What are the goals that researchers hope to achieve with this trial?

"The primary goal of this medical study, which will last for around three years, is to observe the participants' overall survival rates. Additionally, secondary objectives include measuring the maximum plasma concentration (Cmax), elimination half-life (t1/2), and reduction in bone marrow fibrosis."

Answered by AI

How novel is this line of research?

"As of now, there are 2 ongoing Imetelstat clinical trials in 31 countries and 149 cities. The first trial began in 2015 and was sponsored by Geron Corporation. That initial study had 278 participants and completed Phase 2 & 3 drug approval. In the 5 years since then, 17 more studies have concluded."

Answered by AI
~128 spots leftby Apr 2026