Myelodysplastic SyndromesPhase 2IMpressNCT05583552
Study to Evaluate Imetelstat in Patients With High-Risk MDS or AML Failing HMA-based Therapy
A Phase 2 study testing Imetelstat sodium in people with Myelodysplastic Syndromes and Acute Myeloid Leukemia, run by GCP-Service International West GmbH at 11 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes, Acute Myeloid Leukemia
- What is being tested?
- Imetelstat sodium
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 46 participants
- Where
- 11 sitesin 3 countries
- Study dates
- 5 Jun 2023to 30 Sep 2025
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 (World Health Organization) 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
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- 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 (FMS-like tyrosine kinase 3) or IDH1/IDH2 (Isocitrate dehydrogenase) inhibitors or other approved targeted therapies.
Who cannot join
- 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)
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- 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
Trial locations and contacts
11 sites in 3 countries.
11 sites in 3 countries
France4 sites
CHU Nantes - Hôtel Dieu
Nantes
Hôpital Archet 1
Nice
Hôpital Saint-Louis
Paris
CHU de Toulouse
Toulouse
Germany4 sites
Marien Hospital Düsseldorf
Düsseldorf
Universität Jena, Medizinische Fakultät
Jena
Universität Leipzig, Medizinische Fakultät
Leipzig
Klinikum rechts der Isar
München
Australia3 sites
Royal Adelaide Hospital
Adelaide
Royal Brisbane and Women's Hospitals
Brisbane
Linear Clinical Research
Nedlands
About this study
Official title: A Phase II Study Evaluating the Efficacy and Safety of Imetelstat in Patients With HR Myelodysplastic Syndromes or AML Failing HMA-based Therapy
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.
- Conditions
- Myelodysplastic Syndromes, Acute Myeloid Leukemia
- Being tested
- Imetelstat sodium
- Medical subject headings
- Myelodysplastic Syndromes, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Imetelstat sodiumDrug
Also called GRN163L
Intravenous injection
| Study group | Role | What they receive |
|---|---|---|
| Single-arm imetelstat | Experimental | Not described Receives: Drug: Imetelstat sodium |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Hematological Response Rate of Participants After Treatment With Imetelstat
The combined response assessment criteria for MDS and AML based on IWG (International Working Group) 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.
Measured: Up to Week 17 (Visit 9; approximately 17 weeks after first dose).
Secondary outcomes (1)
Toxicity Measured by NCI CTCAE v5.0 (National Cancer Institute - Common Terminology Criteria for Adverse Events Version 5.0)
Toxicity was assessed using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0. A treatment-emergent adverse event (TEAE) was defined as any adverse event arising or worsening after the first dose of study drug. The number of participants experiencing at least one TEAE was summarized in the Safety Evaluation Set.
Measured: From first dose of imetelstat through the last dose received, including follow-up to 28 ± 5 days after the last dose (duration varies by subject).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Oct 2022
- Study start5 Jun 2023
- Primary completion27 Mar 2025
- Study completion30 Sep 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- GCP-Service International West GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Geron Corporation, Universitätsklinikum Leipzig, Saint-Louis Hospital, Paris, France, QIMR Berghofer Medical Research Institute, Australasian Leukaemia and Lymphoma Group, Groupe Francophone des Myelodysplasies, German Myelodysplastic Syndrome Study Group
- Responsible party
- Sponsor
- Investigators
- Uwe Platzbecker, MD, Principal investigator, Universitätsklinikum Leipzig
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05583552
- Sponsor's protocol ID
- IMpress_001
- Acronym
- IMpress
- Status last verified
- Sep 2026
- First submitted
- 13 Oct 2022
- Last updated
- 2 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study Evaluating the Efficacy and Safety of Imetelstat in Patients With HR Myelodysplastic Syndromes or AML Failing HMA-based Therapy
- ClinicalTrials.gov ID
- NCT05583552
- Acronym / sponsor ID
- IMpress / IMpress_001
- Conditions
- Myelodysplastic Syndromes, Acute Myeloid Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 46 participants (actual)
- Lead sponsor
- GCP-Service International West GmbHIndustry (pharma, biotech, devices)
- Collaborators
- Geron Corporation, Universitätsklinikum Leipzig, Saint-Louis Hospital, Paris, France, QIMR Berghofer Medical Research Institute, Australasian Leukaemia and Lymphoma Group, Groupe Francophone des Myelodysplasies, German Myelodysplastic Syndrome Study Group
- Study start
- 5 Jun 2023 (actual)
- Primary completion
- 27 Mar 2025 (actual)
- Study completion
- 30 Sep 2025 (actual)
- First posted
- 17 Oct 2022 (actual)
- Results
- Posted 12 May 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single-arm imetelstat | Experimental | Drug: Imetelstat sodium |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- 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 (World Health Organization) 2016 classification
- At least one cytopenia
Key exclusion criteria (16 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Imetelstat sodiumAlso: GRN163LIntravenous injection | Drug | Single-arm imetelstat |
Primary outcome
Overall Hematological Response Rate of Participants After Treatment With Imetelstat
Time frame: Up to Week 17 (Visit 9; approximately 17 weeks after first dose).
The combined response assessment criteria for MDS and AML based on IWG (International Working Group) 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.
1 secondary outcome
Toxicity Measured by NCI CTCAE v5.0 (National Cancer Institute - Common Terminology Criteria for Adverse Events Version 5.0)
Time frame: From first dose of imetelstat through the last dose received, including follow-up to 28 ± 5 days after the last dose (duration varies by subject).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Uwe Platzbecker, MD principal investigator
Universitätsklinikum Leipzig
11 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Imetelstat sodium in people with Myelodysplastic Syndromes and Acute Myeloid Leukemia, run by GCP-Service International West GmbH at 11 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in France, Germany, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 May 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Hematological Response Rate of Participants After Treatment With Imetelstat (time frame: Up to Week 17 (Visit 9; approximately 17 weeks after first dose).). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2026; AzTrials last refreshed its copy on 1 Nov 2026.