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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05583552




Registration number
NCT05583552
Ethics application status
Date submitted
13/10/2022
Date registered
17/10/2022
Date last updated
19/09/2024

Titles & IDs
Public title
Study to Evaluate Imetelstat in Patients With High-Risk MDS or AML Failing HMA-based Therapy
Scientific title
A Phase II Study Evaluating the Efficacy and Safety of Imetelstat in Patients With HR Myelodysplastic Syndromes or AML Failing HMA-based Therapy
Secondary ID [1] 0 0
IMpress_001
Universal Trial Number (UTN)
Trial acronym
IMpress
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Myelodysplastic Syndromes 0 0
Acute Myeloid Leukemia 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Imetelstat

Experimental: Single-arm imetelstat -


Treatment: Drugs: Imetelstat
Intravenous injection
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Overall Hematological Response Rate of Participants after Treatment with Imetelstat
Timepoint [1] 0 0
After 4 Months of Treatment

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
5/06/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2026
Actual
Sample size
Target
46
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
Royal Brisbane and Women's Hospitals - Brisbane
Recruitment hospital [3] 0 0
Linear Clinical Research - Nedlands
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Brisbane
Recruitment postcode(s) [3] 0 0
- Nedlands
Recruitment outside Australia
Country [1] 0 0
France
State/province [1] 0 0
Nantes
Country [2] 0 0
France
State/province [2] 0 0
Nice
Country [3] 0 0
France
State/province [3] 0 0
Paris
Country [4] 0 0
France
State/province [4] 0 0
Toulouse
Country [5] 0 0
Germany
State/province [5] 0 0
Düsseldorf
Country [6] 0 0
Germany
State/province [6] 0 0
Jena
Country [7] 0 0
Germany
State/province [7] 0 0
Leipzig
Country [8] 0 0
Germany
State/province [8] 0 0
München

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
GCP-Service International West GmbH
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Geron Corporation
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Universitätsklinikum Leipzig
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
Saint-Louis Hospital, Paris, France
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Other
Name [4] 0 0
QIMR Berghofer Medical Research Institute
Address [4] 0 0
Country [4] 0 0
Other collaborator category [5] 0 0
Other
Name [5] 0 0
Australasian Leukaemia and Lymphoma Group
Address [5] 0 0
Country [5] 0 0
Other collaborator category [6] 0 0
Other
Name [6] 0 0
Groupe Francophone des Myelodysplasies
Address [6] 0 0
Country [6] 0 0
Other collaborator category [7] 0 0
Other
Name [7] 0 0
German Myelodysplastic Syndrome Study Group
Address [7] 0 0
Country [7] 0 0

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT05583552
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Uwe Platzbecker, MD
Address 0 0
Universitätsklinikum Leipzig
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Andreas Beust, Dr.
Address 0 0
Country 0 0
Phone 0 0
+49 (0) 421 89 67 66 11
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05583552