A Phase II Study Evaluating the Efficacy and Safety of Imetelstat in Patients With HR Myelodysplastic Syndromes or AML Failing HMA-based Therapy

GCP-Service International West GmbH11 sites in 3 countries46 target enrollmentJune 5, 2023

ConditionsMyelodysplastic Syndromes Acute Myeloid Leukemia

InterventionsImetelstat sodium

DrugsImetelstat sodium

Overview

Phase: Phase 2
Intervention: Imetelstat sodium
Conditions: Myelodysplastic Syndromes
Sponsor: GCP-Service International West GmbH
Enrollment: 46
Locations: 11
Primary Endpoint: Overall Hematological Response Rate of Participants after Treatment with Imetelstat
Status: Active, not recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The purpose of this study is to evaluate the efficacy, in terms of hematologic improvement, and safety of imetelstat in participants with high-risk (HR) myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) that is relapsed/refractory to hypomethylating agents (HMAs) treatment. Responding patients are eligible to continue treatment until loss of response/disease progression.

Registry

clinicaltrials.gov

Start Date

June 5, 2023

End Date

June 2026

Last Updated

last year

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

GCP-Service International West GmbH

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Signed written informed consent
•Male and female ≥ 18 years at the first screening
•Must be able to adhere to the study visit schedule and other protocol requirements
•Initial diagnosis of AML or MDS according to WHO 2016 classification
•At least one cytopenia
•Failure to achieve complete or partial response or hematological improvement observed after at least six azacitidine monotherapy or four decitabine monotherapy based 4-week treatment cycles administered during the past two years OR Failure to achieve complete or partial response or hematological improvement observed after at least two 4-week treatment cycles with azacitidine plus venetoclax or with decitabine plus venetoclax during the past two years OR Relapse after initial complete or partial response or hematological improvement observed after at least six (azacitidine) or four (decitabine) based 4-week treatment cycles administered during the past two years OR Relapse after initial complete or partial response or hematological improvement observed after at least two 4-week treatment cycles with azacitidine plus venetoclax or with decitabine plus venetoclax during the past two years OR Intolerance to treatment with HMA-based therapy during the past two years
•Not eligible for allogeneic stem cell transplantation
•≥ 5% bone marrow blasts at screening
•Off all other treatments for AML/MDS for at least 14 days; granulocyte colony-stimulating factor (G-CSF) and erythropoietin are allowed before and during the study as clinically indicated
•Eastern Cooperative Oncology Group (ECOG) performance status of 0-2

Exclusion Criteria

•Chemotherapy within the 14 days prior to the first dose of imetelstat being administered (other than hydroxyurea)
•Participant has known allergies, hypersensitivity, or intolerance to imetelstat or its excipients (refer to the Investigators Brochure (IB))
•Participant has received an experimental or investigational drug or used an invasive investigational medical device within 30 days prior to day 1 of Cycle 1
•Prior treatment with imetelstat
•Prior history of intensive chemotherapy or hematopoietic stem cell transplant
•Major surgery within 4 weeks prior to day 1 of Cycle 1 (excluding the placement of vascular access and other minor surgical procedures)
•Diagnosed or treated for malignancy other than MDS or AML, except:
•Malignancy treated with curative intent and with no known active disease present for 3 years before day 1 of Cycle 1 Adequately treated non-melanoma skin cancer or lentigo maligna without evidence of disease Adequately treated cervical carcinoma in situ without evidence of disease
•Clinically significant cardiovascular disease such as uncontrolled or symptomatic arrhythmias, congestive heart failure, or myocardial infarction within 6 months of day 1 of Cycle 1, or any Class 3 (moderate) or Class 4 (severe) cardiac disease as defined by the New York Heart Association Functional Classification
•Known history of human immunodeficiency virus (HIV) or any uncontrolled active systemic infection requiring IV antibiotics

Arms & Interventions

Single-arm imetelstat

Intervention: Imetelstat sodium

Outcomes

Primary Outcomes

Overall Hematological Response Rate of Participants after Treatment with Imetelstat

Time Frame: After 4 Months of Treatment

The combined response assessment criteria for MDS and AML based on IWG 2018 criteria (MDS) and the criteria of the European LeukemiaNet (AML) will be used to define responders. The response rate is calculated as number of responders divided by the number of all participants of the analysis set.