Trial Title:
Study to Evaluate Imetelstat in Patients With High-Risk MDS or AML Failing HMA-based Therapy
NCT ID:
NCT05583552
Condition:
Myelodysplastic Syndromes
Acute Myeloid Leukemia
Conditions: Official terms:
Preleukemia
Myelodysplastic Syndromes
Syndrome
Imetelstat
Motesanib diphosphate
Study type:
Interventional
Study phase:
Phase 2
Overall status:
Recruiting
Study design:
Allocation:
N/A
Intervention model:
Single Group Assignment
Primary purpose:
Treatment
Masking:
None (Open Label)
Intervention:
Intervention type:
Drug
Intervention name:
Imetelstat sodium
Description:
Intravenous injection
Arm group label:
Single-arm imetelstat
Other name:
GRN163L
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.
Criteria for 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
- Biochemical laboratory test values must be within the defined limits.
- Availability of blood counts and transfusion events for previous 16 weeks
- Women of childbearing potential and practicing a highly effective method of birth
control consistent with local regulations regarding the use of birth control methods
for subjects participating in clinical studies. For females, these restrictions
apply for 3 months after the end of dosing.
- A woman of childbearing potential must have a negative serum or urine pregnancy test
at screening and agree to be tested on day 1 of every cycle and at End of Treatment
(EOT)
- A man who is sexually active with a woman of childbearing potential and has not had
a vasectomy must agree to use a barrier method of birth control. For males, these
restrictions apply for 3 months after the end of dosing
- Patients who are relapsed or refractory to, or not eligible for, therapy with
approved and available FLT3 or IDH1/IDH2 inhibitors or other approved targeted
therapies.
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
- Active systemic hepatitis infection requiring treatment (carriers of hepatitis virus
are permitted to enter the study), or known acute or chronic liver disease including
cirrhosis
- Any life-threatening illness, medical condition, or organ system dysfunction which,
in the investigator's opinion, could compromise the participant 's safety, interfere
with the imetelstat metabolism, or put the study outcomes at undue risk; Participant
has any condition for which, in the opinion of the investigator, participation would
not be in the best interest of the participant (eg, compromise the well-being) or
that could prevent, limit, or confound the protocol-specified assessments
- Females who are pregnant or are currently breastfeeding or planning to become
pregnant while enrolled in this study or within 3 months after the end of dosing
- Participant is a man who plans to father a child while enrolled in this study or
within 3 months after the end of dosing
- Other:
Participant is in custody by order of an authority or a court of law Participation in
another interventional clinical study within the last 3 months prior to signing the
Informed consent form (ICF) or simultaneous participation in other interventional
clinical studies Previous assignment to treatment during this study Close affiliation
with the investigator (e.g., a close relative) or persons working at the study site
Participant is an employee of the sponsor or involved Contract Research Organization
(CRO) Criteria which in the opinion of the investigator preclude participation for
scientific reasons, for reasons of compliance, or for reasons of the Participant's safety
Gender:
All
Minimum age:
18 Years
Maximum age:
N/A
Healthy volunteers:
No
Locations:
Facility:
Name:
Royal Adelaide Hospital
Address:
City:
Adelaide
Country:
Australia
Status:
Recruiting
Contact:
Last name:
Deepak Singhal, Dr.
Facility:
Name:
Royal Brisbane and Women's Hospitals
Address:
City:
Brisbane
Country:
Australia
Status:
Recruiting
Contact:
Last name:
Steven Lane, Prof. Dr.
Facility:
Name:
Linear Clinical Research
Address:
City:
Nedlands
Country:
Australia
Status:
Recruiting
Contact:
Last name:
Carolyn Grove, Dr.
Facility:
Name:
CHU Nantes - Hôtel Dieu
Address:
City:
Nantes
Country:
France
Status:
Recruiting
Contact:
Last name:
Alice Garnier, Dr.
Facility:
Name:
Hôpital Archet 1
Address:
City:
Nice
Country:
France
Status:
Recruiting
Contact:
Last name:
Thomas Cluzeau, Prof.
Facility:
Name:
Hôpital Saint-Louis
Address:
City:
Paris
Country:
France
Status:
Recruiting
Contact:
Last name:
Lionel Adès, Prof.
Facility:
Name:
CHU de Toulouse
Address:
City:
Toulouse
Country:
France
Status:
Recruiting
Contact:
Last name:
Odile Rauzy, Prof.
Facility:
Name:
Marien Hospital Düsseldorf
Address:
City:
Düsseldorf
Country:
Germany
Status:
Recruiting
Contact:
Last name:
Aristoteles Giagounidis, Prof. Dr.
Facility:
Name:
Universität Jena, Medizinische Fakultät
Address:
City:
Jena
Country:
Germany
Status:
Recruiting
Contact:
Last name:
Sebastian Scholl, Prof. Dr.
Facility:
Name:
Universität Leipzig, Medizinische Fakultät
Address:
City:
Leipzig
Country:
Germany
Status:
Recruiting
Contact:
Last name:
Uwe Platzbecker, Prof. Dr.
Facility:
Name:
Klinikum rechts der Isar
Address:
City:
München
Country:
Germany
Status:
Recruiting
Contact:
Last name:
Katharina Götze, Prof. Dr.
Start date:
June 5, 2023
Completion date:
June 2026
Lead sponsor:
Agency:
GCP-Service International West GmbH
Agency class:
Industry
Collaborator:
Agency:
Geron Corporation
Agency class:
Industry
Collaborator:
Agency:
Universitätsklinikum Leipzig
Agency class:
Other
Collaborator:
Agency:
Saint-Louis Hospital, Paris, France
Agency class:
Other
Collaborator:
Agency:
QIMR Berghofer Medical Research Institute
Agency class:
Other
Collaborator:
Agency:
Australasian Leukaemia and Lymphoma Group
Agency class:
Other
Collaborator:
Agency:
Groupe Francophone des Myelodysplasies
Agency class:
Other
Collaborator:
Agency:
German Myelodysplastic Syndrome Study Group
Agency class:
Other
Source:
GCP-Service International West GmbH
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT05583552
