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Telomerase Inhibitor

Imetelstat for Myelodysplastic Syndrome

Phase 2 & 3

Waitlist Available

Research Sponsored by Geron Corporation

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2

International Prognostic Scoring System (IPSS) low Risk or intermediate-1 risk MDS

Must not have

Have received corticosteroids greater than (>) 30 milligram per day (mg/day) prednisone or equivalent, or growth factor treatment within 4 weeks prior to study entry

Chronic or persistent atrial arrhythmia including atrial fibrillation and atrial flutter

Timeline

Screening 3 weeks

Treatment Varies

Follow Up during extension (up to approximately 3 years)

Summary

This trial is testing a new drug, imetelstat, to see if it works better than a placebo at treating anemia in people with myelodysplastic syndrome who have not responded to other treatments.

Who is the study for?

Adults over 18 with low or intermediate-1 risk Myelodysplastic Syndrome (MDS) who need regular blood transfusions and haven't responded to standard treatments can join. They must have an ECOG performance status of 0, 1, or 2, indicating they are fully active or have some limitations but don't require full-time care.

What is being tested?

The trial is testing Imetelstat's effectiveness in improving the condition of MDS patients who rely on blood transfusions after not responding to other treatments. Part of the group will receive Imetelstat while another part will get a placebo for comparison.

What are the potential side effects?

While specific side effects aren't listed here, participants should be aware that any new treatment like Imetelstat could cause unexpected reactions ranging from mild symptoms like nausea to more serious conditions affecting organ function.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can take care of myself and am up and about more than half of my waking hours.

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My MDS is classified as low or intermediate-1 risk.

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I needed at least 4 blood transfusions in the last 2 months due to low hemoglobin.

Exclusion Criteria

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

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I haven't taken high-dose steroids or growth factors in the last 4 weeks.

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I have a long-term irregular heartbeat.

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My family has a history of long QT syndrome.

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I have had chest surgery that may affect my heart's electrical signals.

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I have not used any experimental drugs or devices in the last 30 days.

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I have or might have long QT syndrome.

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I have been treated with imetelstat before.

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I have a history of a serious type of heart block.

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I am not on medications that affect heart rhythm.

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I have a history of significant heart rhythm problems.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ during extension (up to approximately 3 years)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~during extension (up to approximately 3 years)

This trial's timeline: 3 weeks for screening, Varies for treatment, and during extension (up to approximately 3 years) for reporting.

Treatment Details

Study Objectives

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

Secondary study objectives

Extension Phase: Progression Free Survival (PFS) Survival

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

Part 2 (Main Study): Assessment of EuroQol 5 Dimension Questionnaire (EQ-5D-5L)

+4 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)

Trial Design

5Treatment groups

Experimental Treatment

Placebo Group

Group I: Part 2 (Ventricular Repolarization Substudy): ImetelstatExperimental Treatment1 Intervention

Imetelstat will be administered at a starting dose of 7.5 mg/kg given intravenously every 4 weeks, until disease progression, unacceptable toxicity, or withdrawal of consent, or lack of response. Subjects receiving imetelstat who continue into the extension phase will continue to receive imetelstat treatment per this same schedule.

Group II: Part 2 (Main Study): ImetelstatExperimental Treatment1 Intervention

Group III: Part 1: ImetelstatExperimental Treatment1 Intervention

Imetelstat will be administered at a starting dose of 7.5 milligram per kilogram (mg/kg) given intravenously every 4 weeks, until disease progression, unacceptable toxicity, or withdrawal of consent, or lack of response.

Group IV: Part 2 (Main Study): PlaceboPlacebo Group1 Intervention

Matching Placebo to Imetelstat will be administered.

Group V: Part 2 (Ventricular Repolarization Substudy): PlaceboPlacebo Group1 Intervention

Matching Placebo to Imetelstat will be administered.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Imetelstat

2012

Completed Phase 2

~100