Active not recruitingPHASE2, PHASE3INTERVENTIONAL

Study to Evaluate Imetelstat (GRN163L) in Participants With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

This research study is investigating a new medication called imetelstat for people with a specific type of blood disorder called Myelodysplastic Syndromes (MDS). Specifically, it's for those with lower-risk MDS who need regular blood transfusions and whose previous treatment (erythropoiesis-stimulating agents, or ESAs) is no longer effective. The study has two main parts: Phase 2, which looks at how well imetelstat works and its safety, and Phase 3, which compares imetelstat to a dummy drug (placebo) to see if it can help patients become less reliant on blood transfusions. There's also a long-term follow-up to understand the drug's extended effects.

At a glance

Status

Active not recruiting

Phase

PHASE2, PHASE3

Sponsor

Geron Corporation

Enrolment target

289

Start

12 Jan 2016

Estimated completion

13 Oct 2026

Myelodysplastic Syndromes

Results

Results from this study

Posted February 2025

Results have been published for this study.

Primary outcome

Phase 2: Percentage of Participants Without Any Red Blood Cell (RBC) Transfusion During Any Consecutive 8-Weeks Period (All Participants)

Percentage of participants without any RBC transfusion during any consecutive 8 weeks (56 days) starting from Study Day 1 until subsequent anti-cancer therapy if any were reported. Study Day 1 is defined as the day of the first dose for participants enrolled in Phase 2. The 95% confidence interval (CI) was calculated using Clopper-Pearson method. Percentages were rounded off to the nearest single decimal place.

Full results on the registry

What is this study about?

This study is looking into a new medication called imetelstat for people living with Myelodysplastic Syndromes (MDS). MDS is a condition where the bone marrow, which is the spongy tissue inside some bones, doesn’t make enough healthy blood cells. Many people with MDS, especially those with lower-risk types, need regular blood transfusions to top up their red blood cells. Often, they first try treatments called erythropoiesis-stimulating agents (ESAs) to help their body make more red blood cells on its own. This study is specifically for those who still need transfusions even after trying ESAs, or for whom ESAs are no longer working.

The main goal of this study is to find out if imetelstat can help people with MDS reduce or even stop their need for blood transfusions. To do this, the study is split into two phases. The first part, Phase 2, focused on understanding how effective imetelstat is and what its side effects might be. The second, larger part, Phase 3, is comparing imetelstat directly with a placebo (a dummy drug that looks like the real thing but has no active ingredients). This comparison helps researchers determine if any improvements seen are truly due to imetelstat rather than other factors. There's also a special smaller study looking at how imetelstat might affect the heart's electrical activity.

For those who seem to benefit from imetelstat during the main study, there's an optional extension phase. This allows patients to continue receiving the treatment for a longer period. This extension is crucial for gathering information on the long-term safety of imetelstat, how long its benefits last, and to monitor for any changes in the condition, including if it progresses into a more aggressive form of leukaemia.

Key takeaways

The study is for adults with a specific type of Myelodysplastic Syndrome (MDS).
It's for people who need regular blood transfusions and whose previous treatment didn't work well.
The main goal is to see if a new drug, imetelstat, can reduce the need for blood transfusions.
Some participants will receive imetelstat, while others might receive a placebo.
Participation involves regular clinic visits for tests and monitoring over several years.
You can stop participating in the study at any time.

Who may be eligible?

To be considered for this study, you would generally need to be an adult, aged 18 or older, with a confirmed diagnosis of Myelodysplastic Syndrome (MDS). Your MDS should be categorised as 'low' or 'intermediate-1' risk according to standard medical guidelines. A key requirement is that you also need to be dependent on regular red blood cell transfusions, meaning you've had at least four transfusions over an eight-week period in the lead-up to the study. Your general health and ability to carry out daily activities, as assessed by a doctor, should be good enough to participate.

There are also some reasons why you might not be able to join. For example, if you've had a known allergy to imetelstat or its ingredients, or if you've previously taken imetelstat. You also couldn't have participated in another research study involving an experimental drug or device within the last month. Certain recent treatments, like high-dose steroids, specific growth factors, or certain chemotherapy and immune-modifying drugs within the last four to eight weeks, would also make you ineligible. For the Phase 3 part of the study, if you've previously been treated with certain other MDS medications like hypomethylating agents or lenalidomide, you would not be able to join.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a diagnosis of 'low' or 'intermediate-1' risk MDS?
Do you regularly need red blood cell transfusions (at least 4 units in the last 8 weeks)?
Has your previous treatment (like ESA) not worked well or stopped working?
Have you not received specific other MDS treatments recently?
Are you generally well enough to participate in a study?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would first go through a screening process to ensure you meet all the requirements. Once enrolled, you would receive either imetelstat or a placebo (in Phase 3 or the heart study) as an infusion. The study involves regular visits to the clinic for assessments, which will include blood tests, bone marrow checks, and other evaluations of your health and how the treatment is affecting you. For those in the Phase 3 part and the heart study, you wouldn't know whether you're receiving the active drug or the placebo; this is called 'double-blind'. If you are doing well and benefitting from the treatment, you could have the option to continue receiving imetelstat in an 'Extension Phase' for up to five years from your first dose. Throughout your participation, your study doctor and team would monitor your progress and any side effects. You are free to withdraw from the study at any time.

Potential risks and benefits

Participating in a clinical trial may offer potential benefits, such as gaining access to a new treatment before it is widely available, which might help reduce your need for blood transfusions. However, there's no guarantee that the treatment will work for you. There are also potential risks, including side effects from imetelstat, which would be explained in detail by the study team. Some participants might receive a placebo and would not get the active drug. Your contribution helps advance medical knowledge, potentially benefiting others in the future. It's important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (126)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

UAB Comprehensive Cancer Center
Verified postcode
Birmingham, United States
Acrc/Arizona Clinical Research, Inc.
Verified postcode
Tucson, United States
CBCC Global Research, Inc.
Verified postcode
Bakersfield, United States
UCLA Ronald Regan Medical Center
Verified postcode
Los Angeles, United States
Yale-New Haven Hospital (YNHH) - Smilow Cancer Hospital
Verified postcode
New Haven, United States
BRCR Medical Center
Verified postcode
Plantation, United States
University of South Florida (USF) - H. Lee Moffitt Cancer Center
Verified postcode
Tampa, United States
Franciscan Health
Verified postcode
Indianapolis, United States
St. Agnes Healthcare, Inc
Verified postcode
Baltimore, United States
Center for Cancer and Blood Disorders
Verified postcode
Bethesda, United States
Washington University School of Medicine
Verified postcode
St Louis, United States
University of New Mexico Cancer Center
Verified postcode
Albuquerque, United States

Common questions

What is Myelodysplastic Syndrome (MDS)?

MDS is a condition where your bone marrow doesn't produce enough healthy blood cells, leading to issues like anaemia, easy bleeding, or infections.

What does 'transfusion-dependent' mean?

It means you regularly need blood transfusions to keep your red blood cell count at a healthy level because your body isn't making enough on its own.

What is imetelstat?

Imetelstat is a new investigational drug being tested to see if it can help people with MDS reduce their need for blood transfusions.

What is a 'placebo'?

A placebo is an inactive substance or dummy treatment that looks just like the real drug but contains no active ingredients. It's used in some studies to compare against the real drug.

Can I stop participating in the study at any time?

Yes, absolutely. Your participation is voluntary, and you can withdraw from the study at any point without it affecting your standard medical care.