A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia Vera
This research study is investigating a new medication called sapablursen, in people diagnosed with a blood disorder called polycythemia vera. Specifically, it's looking at patients who need regular blood removal procedures (phlebotomies) to manage their condition. The main goal of this study is to find out if sapablursen can help reduce how often people need these blood removal treatments. It also aims to see if the new medicine can improve their quality of life. Participants will receive the study drug through injections under the skin over a period of many months. This is a Phase 2 study, meaning it's an early stage of testing new medicines to understand their effectiveness and safety in a larger group of people after initial safety checks.
At a glance
What is this study about?
This study is exploring a new treatment called sapablursen for a blood condition known as polycythemia vera (PV). PV is a long-term illness where your body makes too many red blood cells, making your blood thicker than normal. This can lead to serious health problems like blood clots. One common way to manage PV is through a procedure called phlebotomy, which is like giving blood to reduce the number of red blood cells and thin out your blood. For many people, these phlebotomies are needed regularly, sometimes every few weeks or months.
The main purpose of this research is to see if sapablursen can reduce the need for these frequent phlebotomies. We also want to understand if taking this new medicine can make a difference to how people feel day-to-day and improve their overall quality of life. This is important because managing PV can be quite demanding, and new treatments that offer relief or improved daily living are always being sought.
This is a 'Phase 2' study, which means it’s still in the early stages of testing, but it has already passed initial safety checks. It involves comparing different doses of the new medicine to see which works best and is safest. Participants will receive sapablursen, and everyone in the trial will get the active study drug; there won't be a placebo (a dummy treatment).
Key takeaways
- This study is testing a new medicine called sapablursen for polycythemia vera (PV).
- It aims to reduce the need for regular blood removal (phlebotomies) and improve quality of life.
- Participation involves injections and regular clinic visits over many months.
- Everyone in the study will receive the active drug, no placebo.
- Open to adults aged 18-90 who need regular phlebotomies for their PV.
Who may be eligible?
To join this study, you must have a confirmed diagnosis of polycythemia vera (PV) and currently need regular blood removal treatments (phlebotomies) to manage your condition. You can be taking other PV medications, such as hydroxyurea, interferon, or ruxolitinib, as long as you've been on a stable dose for at least three months before the study starts. If you've stopped other PV treatments, you'd need to have done so at least three months ago, and any side effects from those treatments should have settled.
There are certain reasons why you might not be able to join. For example, if your PV has progressed to a more advanced stage called post-PV myelofibrosis, or if you have severe spleen pain. Also, if you have certain other serious health issues like active infections (including HIV, Hepatitis B or C, or current COVID-19 requiring treatment), bleeding problems, certain cancers within the last five years (unless it's a very common, easily treated type like some skin cancers), or if you've recently had major surgery, you might not be eligible.
The study is open to both men and women, aged between 18 and 90 years old.
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.
- Do you have a confirmed diagnosis of polycythemia vera?
- Do you currently need regular blood removal (phlebotomy)?
- Are you between 18 and 90 years old?
- Do you have stable doses of any current PV medications (if applicable)?
- Do you have any serious infections or bleeding problems right now?
- Have you had certain types of cancer within the last 5 years?
What does participation involve?
If you decide to take part, the study will involve several stages over a significant period. First, there's a 'screening period' of up to 7 weeks to check if you're suitable. After that, the main 'treatment period' lasts for about 37 weeks (around 8-9 months), during which you'll receive the study drug. This is given as injections under your skin, with a total of 9 doses over this time. Following this, there's an 'extension period' for another 36 weeks (about 8-9 months), where you'll receive another 9 doses of the study drug.
Throughout the study, you'll be assigned either a higher or a lower dose of sapablursen, chosen randomly, but everyone will receive the active drug – there's no placebo. There will be regular visits to the clinic for assessments, blood tests, and to receive your injections. After your final dose in the extension period, there will be a 'post-treatment period' of 13 weeks (about 3 months) for follow-up and final checks. In total, your involvement could last for well over a year and a half, with regular check-ups to monitor your health and the treatment's effects.
Potential risks and benefits
Locations (13)
- O'Neal Comprehensive Cancer Center University of Alabama at BirminghamVerified postcodeBirmingham, United States
- Mayo Clinic HospitalVerified postcodePhoenix, United States
- John Theurer Cancer CenterVerified postcodeHackensack, United States
- Cleveland Clinic FoundationVerified postcodeCleveland, United States
- University of Texas MD Anderson Cancer CenterVerified postcodeHouston, United States
- Calvary Mater Newcastle HospitalVerified postcodeWaratah, Australia
- Border Medical Oncology Research UnitVerified postcodeAlbury, Australia
- McGill University Health CentreVerified postcodeMontreal, Canada
- Samodzielny Publiczny Szpital Kliniczny Nr 1 w Lublinie, Klinika Hematoonkologii i Transplantacji SzpikuVerified postcodeLublin, Poland
- Wojewódzki Szpital Specjalistyczny Sp. z o.o.Verified postcodeSłupsk, Poland
- MICS Centrum Medyczne ToruńVerified postcodeTorun, Poland
- Oxford University Hospitals NHS Foundation TrustVerified postcodeOxford, United Kingdom
Common questions
What is polycythemia vera?
It's a rare blood condition where your body makes too many red blood cells, making your blood thicker and increasing the risk of clots.
What is a phlebotomy?
It's a procedure similar to giving blood, used to reduce the number of red blood cells in your body and thin your blood.
Will I get a placebo (dummy drug) in this study?
No, everyone in this study will receive the active study drug, sapablursen, either at a higher or lower dose.
How will the medicine be given?
The medicine will be given as injections under your skin.
How long will I be in the study?
Including screening and follow-up, your participation could last for over a year and a half.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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