Imetelstat for Myelofibrosis

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
MyelofibrosisImetelstat - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This trial is studying the recommended dosage of a combination of imetelstat and ruxolitinib to treat myelofibrosis, as well as the safety and effectiveness of this dosage.

Eligible Conditions
  • Myelofibrosis

Treatment Effectiveness

Effectiveness Progress

1 of 3

Similar Trials

Study Objectives

3 Primary · 13 Secondary · Reporting Duration: From first dose of imetelstat treatment up to approximately 5 years

Day 28
Part 1: Incidence, Type, and Severity of Adverse Events, Including Dose-limiting Toxicity (DLT) During the DLT Observation Period and/or Study Treatment
Year 5
Part 2: Number of Participants With Treatment-emergent Adverse Event (AE)
Year 5
Part 1 and 2: Imetelstat and Ruxolitinib Exposure From Time Zero to Last Measurable Concentration (AUC0-t)
Part 1 and 2: Pharmacokinetic Profile of Imetelstat and Ruxolitinib (Maximum Observed Plasma Concentration [Cmax]
Part 1 and 2: Pharmacokinetic Profile of Imetelstat and Ruxolitinib Area under the Plasma Concentration [AUC0-24]
Part 1 and 2: Pharmacokinetic Profile of Imetelstat and Ruxolitinib Terminal Disposition Half-life [T1/2]
Part 1 and 2: Pharmacokinetic Profile of Imetelstat and Ruxolitinib Time to Reach Maximum Plasma Concentration [Tmax])
Percentage of Participants with Anti-imetelstat Antibodies
Year 5
Part 1 and 2: Time to Response
Year 5
Part 1 and 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
Bone Marrow
Year 5
Part 1 and 2: Progression Free Survival (PFS)
Year 5
Part 1 and 2: Duration of Response (DOR) Per IWG-MRT Criteria
Week 24
Part 1 and 2: Spleen Response at Week 24
Part 1: Symptom Response at Week 24
Part 2: Symptom Response Rate at Week 24

Trial Safety

Safety Progress

1 of 3

Similar Trials

Side Effects for

Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts])
100%Fatigue
100%Platelet count decreased
100%Neutrophil count decreased
100%Diarrhoea
100%White blood cell count decreased
72%Hyperglycaemia
67%Nausea
61%Anaemia
44%Hyperuricaemia
44%Pain
39%Activated partial thromboplastin time prolonged
33%Aspartate aminotransferase increased
33%Alanine aminotransferase increased
33%Abdominal pain
33%Weight decreased
28%Blood bilirubin increased
28%Oedema peripheral
28%Cough
28%Blood alkaline phosphatase increased
28%Dyspnoea
28%Hypertension
28%Vomiting
22%Upper respiratory tract infection
22%Blood creatinine increased
22%Gamma-glutamyltransferase increased
22%Pruritus
22%Epistaxis
22%Eye disorders
22%Anorexia
17%Contusion
17%Lipase increased
17%Infusion related reaction
17%Hypokalaemia
17%Hypocalcaemia
17%Muscular weakness
17%Back pain
17%Sinus tachycardia
17%Hyperkalaemia
17%Decreased appetite
17%Headache
11%Lung infection
11%Bone pain
11%Cardiac failure
11%Arthralgia
11%Chills
11%Pyrexia
11%Blood amylase increased
11%Lymphocyte count decreased
11%Hyponatraemia
11%Hyperhidrosis
11%Weight increased
11%Pain in extremity
11%Hypernatraemia
11%Anxiety
6%Insomnia
6%Fall
6%Alopecia
6%Abdominal distension
6%Constipation
6%Non-cardiac chest pain
6%Pulmonary hypertension
6%Night sweats
6%Hypotension
6%Early satiety
6%Abdominal pain upper
6%Neck pain
6%Hypoglycaemia
6%Musculoskeletal pain
6%Myalgia
This histogram enumerates side effects from a completed 2018 Phase 2 trial (NCT01731951) in the Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts]) ARM group. Side effects include: Fatigue with 100%, Platelet count decreased with 100%, Neutrophil count decreased with 100%, Diarrhoea with 100%, White blood cell count decreased with 100%.

Trial Design

1 Treatment Group

Imetelstat + Ruxolitinib
1 of 1

Experimental Treatment

41 Total Participants · 1 Treatment Group

Primary Treatment: Imetelstat · No Placebo Group · Phase 1

Imetelstat + RuxolitinibExperimental Group · 2 Interventions: Imetelstat, Ruxolitinib · Intervention Types: Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Imetelstat
2012
Completed Phase 2
~100
Ruxolitinib
2018
Completed Phase 3
~740

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: from first dose of imetelstat treatment up to approximately 5 years

Who is running the clinical trial?

Geron CorporationLead Sponsor
17 Previous Clinical Trials
1,400 Total Patients Enrolled
Tymara Berry, MDStudy DirectorGeron Corporation

Eligibility Criteria

Age 18+ · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have been treated with a JAK inhibitor for at least 6 months prior to the study.
You have active symptoms of MF on the MFSAF v4.0.
You are not eligible for or unwilling to undergo hematopoietic stem cell transplant at time of study entry.
You have a performance status of 0, 1, or 2.