Not recruitingRare disease

Study on the Effects of Sapablursen for Patients with Phlebotomy Dependent Polycythemia Vera

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called Polycythemia Vera, which is a blood disorder where the body produces too many red blood cells. This can lead to various health issues, including the need for frequent blood removal, known as phlebotomy, to manage the condition. The trial will test a treatment called sapablursen, also known by its code name ISIS 702843. This treatment is given as an injection under the skin.

The purpose of the study is to see if sapablursen can reduce the number of times patients need phlebotomy over a specific period. Participants in the study will receive the treatment and be monitored for its effects on their condition. The study will last for a total of 37 weeks, with a focus on the last 20 weeks to assess the treatment's effectiveness. During this time, researchers will also look at how the treatment affects symptoms and overall health.

Throughout the study, various health checks will be conducted, including blood tests and heart monitoring, to ensure the safety and well-being of participants. The study aims to provide valuable information on whether sapablursen can help manage Polycythemia Vera more effectively, potentially reducing the need for frequent phlebotomy and improving the quality of life for those affected by this condition.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, eligibility is confirmed based on specific criteria related to polycythemia vera, a condition characterized by an increased number of red blood cells.
Participants must be dependent on phlebotomy, a procedure to remove blood, and meet certain diagnostic criteria.
Step 2
Treatment period
The treatment involves the administration of sapablursen, also known as ISIS 702843, through subcutaneous injection.
The treatment period lasts for 37 weeks, during which the frequency of phlebotomy is monitored and compared to the baseline.
Step 3
Medication administration
The medication is administered via injection under the skin. The specific dosage and frequency are determined by the study protocol.
Step 4
Monitoring and assessments
Throughout the study, various assessments are conducted to evaluate the effectiveness and safety of the treatment.
These assessments include monitoring symptoms, vital signs, and laboratory tests such as blood chemistry and thyroid function.
Step 5
End of treatment period
At the end of the 37-week treatment period, the primary goal is to assess the reduction in the frequency of phlebotomy.
Secondary goals include evaluating symptom changes and the proportion of patients achieving specific reductions in phlebotomy frequency.
Step 6
Treatment extension period
An optional extension period may follow, lasting up to 73 weeks, to further evaluate the long-term effects of the treatment.
During this period, the focus is on maintaining control of hematocrit levels, which measure the proportion of red blood cells in the blood, without the need for phlebotomy.

Who can join the trial?

6 criteria

You must have been diagnosed with **Polycythemia Vera**, a condition where your body makes too many red blood cells, according to the modified World Health Organization (WHO) 2016 criteria.
You need to be **phlebotomy dependent**, meaning you require regular blood removal to manage your condition.
You do not need to be on **cytoreductive therapy**, which is treatment to reduce the number of blood cells. If you have been on this therapy before, it must have been stopped at least 3 months before the study starts. If you are currently on this therapy, your dose must have been stable for at least 3 months before the study starts.
Both men and women can participate in the study.
The study includes people from certain age groups, but the specific ages are not listed here.
The study may include people who are considered part of a vulnerable population, which means they might need extra protection or care.

Who cannot join the trial?

7 criteria

Patients who have any other serious health conditions that could interfere with the study.
Patients who are currently participating in another clinical trial.
Patients who have had a recent major surgery or are planning to have surgery during the study period.
Patients who are pregnant or breastfeeding.
Patients who have a history of allergic reactions to similar medications.
Patients who have not been diagnosed with Polycythemia Vera, a condition where the body makes too many red blood cells.
Patients who are unable to follow the study procedures or attend the required visits.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

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Samodzielny Publiczny Szpital Kliniczny Nr 1 W Lublinie
Lublin, Poland
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Szpital Wojewodzki W Opolu Sp. z o.o.
Opole, Poland
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Wojewodzki Szpital Specjalistyczny Im. Janusza Korczaka W Slupsku Sp. z o.o.
Slupsk, Poland

Trial locations

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Investigated drugs

Sapablursen is a medication being studied for its ability to help patients with a condition called phlebotomy-dependent polycythemia vera. This condition causes the body to produce too many red blood cells, leading to the need for frequent blood removal, known as phlebotomy. The trial aims to see if sapablursen can reduce the need for these blood removal procedures over a period of time, making it easier for patients to manage their condition.

Sapablursen

What is already known about the treatment

Sapablursen – Sapablursen is administered through injections and is currently being studied in clinical trials for its effectiveness in treating Polycythemia Vera, a condition characterized by an overproduction of red blood cells. The medication is in the experimental phase, with ongoing research to determine its safety and efficacy. It is designed to reduce the need for phlebotomy, a procedure to remove excess blood, by targeting specific molecular pathways involved in blood cell production. Sapablursen belongs to a class of drugs known as antisense oligonucleotides, which work by binding to RNA molecules and altering gene expression.

Investigated diseases

Polycythemia Vera – Polycythemia Vera is a rare blood disorder characterized by the overproduction of red blood cells in the bone marrow. This excess of red blood cells thickens the blood, slowing its flow and potentially leading to complications such as blood clots. Symptoms may include headaches, dizziness, and a ruddy complexion. Over time, the increased blood volume and viscosity can strain the heart and other organs. The disease progresses slowly, and individuals may experience periods of stability followed by symptom flare-ups. Regular monitoring and management are essential to control the condition and prevent complications.