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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

Must not have

Known history of human immunodeficiency virus or any uncontrolled active systemic infection requiring IV antibiotics

Prior treatment with imetelstat

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to end of study (approximately 3 years)

Awards & highlights

No Placebo-Only Group

Pivotal Trial

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.

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.

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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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.

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My condition is considered high-risk by myelofibrosis standards.

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My condition did not improve with JAK-inhibitor treatment and I cannot have a stem cell transplant.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have HIV or a current severe infection needing IV antibiotics.

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I have been treated with imetelstat before.

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I have not had major surgery in the last 4 weeks.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to end of study (approximately 3 years)

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Overall survival (OS)

Secondary study objectives

Assessment of AUC

Assessment of Cmax

Assessment of Tmax

+9 more

Side effects data

From 2018 Phase 2 trial • 80 Patients • NCT01731951

100%

Diarrhoea

100%

Neutrophil count decreased

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%

Constipation

22%

Blood amylase increased

22%

Upper respiratory tract infection

22%

Hypokalaemia

22%

Muscular weakness

22%

Neck pain

22%

Insomnia

22%

Hyperhidrosis

22%

Cough

22%

Cardiac failure

22%

Oedema peripheral

22%

Weight decreased

22%

Anorexia

22%

Decreased appetite

22%

Hypernatraemia

22%

Hypocalcaemia

22%

Hyponatraemia

22%

Dyspnoea

22%

Pain in extremity

22%

Hypertension

22%

Headache

22%

Alopecia

22%

Arthralgia

22%

Weight increased

11%

Sepsis

11%

Lymphocyte count decreased

11%

Atrial fibrillation

11%

Abdominal distension

11%

Night sweats

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%

Hyperkalaemia

11%

Hyperuricaemia

11%

Epistaxis

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%

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 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 B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF)

Arm A: Imetelstat 9.4 mg/kg (MF)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: ImetelstatExperimental Treatment1 Intervention

Participants will receive imetelstat sodium 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

0 / 11Eligibility criteria met