Myelodysplastic SyndromesNCT05937568
Expanded Access for Treatment With Imetelstat
A study testing Imetelstat in people with Myelodysplastic Syndromes, run by Geron Corporation.
Trial summary
The objective of this expanded access protocol (EAP) is to provide access to treatment with imetelstat, the Investigational Product (IP), for eligible adult participants diagnosed with very low, low, intermediate risk (by Revised International Prognostic Scoring System, IPSS-R) myelodysplastic syndromes (MDS) who are… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- Imetelstat
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
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
- Adult, Older adult
Who may be able to join
- Documented diagnosis of MDS according to World Health Organization (WHO) criteria confirmed by bone marrow aspirate and/or biopsy preferably within 24 but no more than 52 weeks prior to EAP start.
- Very Low, Low, or Intermediate risk MDS (≤3.5) per IPSS-R.
- Red blood cell (RBC) transfusion dependent, defined as requiring at least 4 RBC units over an 8-week period during the 16 weeks prior to EAP entry; pre-transfusion hemoglobin (Hb) should be less than or equal to 9.0 grams per deciliter (g/dL) to count towards the 4 units total.
- Has MDS that has failed to respond or has lost response or are ineligible for ESAs.
- To ensure participants have adequate iron stores they must have transferrin saturation greater than 20% and serum ferritin greater than 400 nanograms per milliliter (ng/mL).
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1 or 2.
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- Hematology and biochemical laboratory test values within the protocol defined limits.
- Have no approved alternative treatment available.
- Inability to participate in a current imetelstat clinical study for the disease or conditions.
- Have a treating physician who has appropriately evaluated the benefit/risk profile for potential imetelstat treatment.
Who cannot join
- Have previously assessed as having IPSS-R high or very high risk MDS.
- Participant with del(5q) karyotype.
- Participant with MDS/myeloproliferative neoplasm Overlap Syndromes.
- Participant has known allergies, hypersensitivity, or intolerance to imetelstat or its excipients.
- Participant has received an experimental or investigational product or used an invasive investigational medical device within 30 days prior to EAP entry or is currently enrolled in an investigational study.
- Prior treatment with imetelstat; or eligible for enrollment in an ongoing imetelstat clinical trial.
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- Prior treatment with lenalidomide or a hypomethylating agent (example, azacitidine, decitabine, or decitabine/cedazuridine).
- Has received an ESA or any anti-MDS therapy, chemotherapy, immunomodulatory, or immunosuppressive therapy within 4 weeks prior to EAP entry.
- Prior history of hematopoietic stem cell transplant.
- Note: Other protocol specified inclusion/exclusion criteria may apply.
About this study
Official title: Expanded Access Treatment With Imetelstat For Adult Participants With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Are Transfusion-Dependent And Have Failed to Respond or Have Lost Response or Are Ineligible For Erythropoiesis-Stimulating Agents (ESAs)
The objective of this expanded access protocol (EAP) is to provide access to treatment with imetelstat, the Investigational Product (IP), for eligible adult participants diagnosed with very low, low, intermediate risk (by Revised International Prognostic Scoring System, IPSS-R) myelodysplastic syndromes (MDS) who are red blood cell (RBC) transfusion-dependent, have failed to respond or have lost response or are ineligible for ESAs, had not received prior treatment with either a hypomethylating agent or lenalidomide and were non-del(5q), until such time that imetelstat becomes commercially available.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Imetelstat
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ImetelstatDrug
Participants will receive imetelstat, intravenously (IV) by infusion every 4 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Expanded access
Sponsor and investigators
- Lead sponsor
- Geron Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05937568
- Sponsor's protocol ID
- MDSEAP001
- Status last verified
- Jun 2024
- First submitted
- 30 Jun 2023
- Last updated
- 24 Jun 2024
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Treatment With Imetelstat For Adult Participants With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Are Transfusion-Dependent And Have Failed to Respond or Have Lost Response or Are Ineligible For Erythropoiesis-Stimulating Agents (ESAs)
- ClinicalTrials.gov ID
- NCT05937568
- Acronym / sponsor ID
- MDSEAP001
- Conditions
- Myelodysplastic Syndromes
- Study type
- Expanded access
- Recruitment status
- Approved For Marketing
- Lead sponsor
- Geron CorporationIndustry (pharma, biotech, devices)
- First posted
- 10 Jul 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this expanded access protocol (EAP) is to provide access to treatment with imetelstat, the Investigational Product (IP), for eligible adult participants diagnosed with very low, low, intermediate risk (by Revised International Prognostic Scoring System, IPSS-R) myelodysplastic syndromes (MDS) who are red blood cell (RBC) transfusion-dependent, have failed to respond or have lost response or are ineligible for ESAs, had not received prior treatment with either a hypomethylating agent or lenalidomide and were non-del(5q), until such time that imetelstat becomes commercially available.
Study design
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Documented diagnosis of MDS according to World Health Organization (WHO) criteria confirmed by bone marrow aspirate and/or biopsy preferably within 24 but no more than 52 weeks prior to EAP start.
- Very Low, Low, or Intermediate risk MDS (≤3.5) per IPSS-R.
- Red blood cell (RBC) transfusion dependent, defined as requiring at least 4 RBC units over an 8-week period during the 16 weeks prior to EAP entry; pre-transfusion hemoglobin (Hb) should be less than or equal to 9.0 grams per deciliter (g/dL) to count towards the 4 units total.
- Has MDS that has failed to respond or has lost response or are ineligible for ESAs.
- To ensure participants have adequate iron stores they must have transferrin saturation greater than 20% and serum ferritin greater than 400 nanograms per milliliter (ng/mL).
Key exclusion criteria (10 listed)
- Have previously assessed as having IPSS-R high or very high risk MDS.
- Participant with del(5q) karyotype.
- Participant with MDS/myeloproliferative neoplasm Overlap Syndromes.
- Participant has known allergies, hypersensitivity, or intolerance to imetelstat or its excipients.
- Participant has received an experimental or investigational product or used an invasive investigational medical device within 30 days prior to EAP entry or is currently enrolled in an investigational study.
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 |
|---|---|---|
| ImetelstatParticipants will receive imetelstat, intravenously (IV) by infusion every 4 weeks. | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2024.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Imetelstat in people with Myelodysplastic Syndromes, run by Geron Corporation.
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 10 inclusion and 10 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: status not confirmed, as of the sponsor's last update on 24 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
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.
Are results available for this study?
Results are not available in the source record.
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.
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 24 Jun 2024; AzTrials last refreshed its copy on 31 Oct 2026.