JNJ-63935937 (imetelstat)
Research type
Research Study
Full title
A Randomized, Single-Blind, Multicenter Phase 2 Study to Evaluate the Activity of 2 Dose Levels of Imetelstat in Subjects with Intermediate-2 or High-Risk Myelofibrosis (MF) Relapsed/Refractory to Janus Kinase (JAK) Inhibitor
IRAS ID
181406
Contact name
Dragana Milojkovic
Contact email
Sponsor organisation
Geron Corporation
Eudract number
2015-000946-41
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 5 months, 24 days
Research summary
This is a randomised, single-blind, multicentre, phase 2 study in participants at least 18 years old with intermediate-2 or high-risk myselofibrosis (MF). MF is a fatal condition, with approximately 70% of individuals with MF diagnosed with intermediate-2 or high-risk MF. A key symptom of MF is splenomegaly (enlarged spleen leading to abdominal fullness). Allogeneic stem cell transplantation is the only treatment that can induce long term remission of MF. However, many patients will not be transplanted because of the advanced age at which MF is diagnosed (mean age of 65 years). Such patients are currently treated with hydroxyurea, interferon and Janus kinase (JAK) inhibitors, which may result in a spleen volume reduction. Ruxolitinib is the only JAK inhibitor currently approved for treatment of patients with intermediate or high risk MF. As such, for patients previously treated with a JAK inhibitor, there is no approved therapy and prognosis is poor. There is therefore an unmet need for a novel treatment for this patient population. Imetelstat is such a novel treatment which has been shown to reduce splenomegaly in such patients, and also has induced complete or partial remission in subjects with MF.
In this trial, 200 participants will be enrolled into one of two treatments arms involving imetelstat administration via 2 hour intravenous infusion every 3 weeks until disease progression or unacceptable toxicity. The two treatments arms are dosing regimens of imetelstat 9.4mg/kg or Imetelstat 4.7mg/kg.
The trial will be divided into three sections: a screening period of up to 21 days before randomisation to a treatment arm, the treatment period involving infusion every 3 weeks from randomisation to imetelstat discontinuation, and a follow up phase involving follow up every 16 weeks from imetelstat discontinuation to death unless participants are lost to follow up, withdraw consent or study ends.
REC name
London - South East Research Ethics Committee
REC reference
15/LO/1105
Date of REC Opinion
31 Jul 2015
REC opinion
Further Information Favourable Opinion