Expanded Access for Treatment With Imetelstat

June 20, 2024 updated by: Geron Corporation

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.

Study Overview

Status

Approved for marketing

Conditions

Intervention / Treatment

Study Type

Expanded Access

Expanded Access Type

  • Intermediate-size Population

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Description

Key Inclusion Criteria:

  • 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.
  • 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.

Key Exclusion Criteria:

  • 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.
  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Geron Corporation

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

June 30, 2023

First Submitted That Met QC Criteria

June 30, 2023

First Posted (Actual)

July 10, 2023

Study Record Updates

Last Update Posted (Actual)

June 24, 2024

Last Update Submitted That Met QC Criteria

June 20, 2024

Last Verified

June 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MDSEAP001

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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