Study on How Concizumab Works for Patients with Hemophilia A or B Without Inhibitors

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What is this study about?

This clinical trial is focused on studying the effects of a medication called Concizumab in people with a bleeding disorder known as hemophilia. Hemophilia is a condition where the blood does not clot properly, leading to excessive bleeding. The study specifically looks at two types of hemophilia: hemophilia A and hemophilia B, both without inhibitors. Inhibitors are substances that can interfere with the treatment of hemophilia, so this study focuses on patients who do not have these inhibitors.

The purpose of the study is to see how well Concizumab works in reducing the number of bleeding episodes in patients with hemophilia A and B. The medication is given as a solution for injection using a device called the PDS290 pen-injector. Participants in the study will receive either the medication or a placebo, which is a substance with no active drug. The study will compare the effects of taking Concizumab regularly (prophylaxis) to not taking it regularly (on-demand treatment) to see which approach is more effective in preventing bleeding episodes.

Throughout the study, participants will receive injections of Concizumab and will be monitored for any changes in their condition, including the number of bleeding episodes they experience. The study will last for several months, allowing researchers to gather enough information to determine the effectiveness and safety of the medication. This research aims to provide better treatment options for people living with hemophilia, helping them manage their condition more effectively.

1 joining the study

Upon joining the study, informed consent is required. This involves understanding the trial’s purpose and agreeing to participate.

Eligibility criteria include being male, aged 12 years or older, with a body weight over 25 kg, and having severe haemophilia A or moderate/severe haemophilia B without inhibitors.

2 initial assessment

An initial assessment is conducted to confirm eligibility and gather baseline health information.

This may include medical history review and physical examination.

3 randomization

Participants are randomly assigned to one of two groups: one receiving concizumab prophylaxis and the other receiving on-demand treatment.

Randomization ensures that each participant has an equal chance of being assigned to either group.

4 treatment phase

For those in the concizumab group, the medication is administered as a subcutaneous injection.

The dosage is either 100 mg/mL or 40 mg/mL, depending on the specific requirements of the study.

The treatment duration is at least 32 weeks, with regular monitoring and adjustments as needed.

5 monitoring and follow-up

Participants are monitored for bleeding episodes, injection site reactions, and any hypersensitivity reactions.

Regular blood tests are conducted to measure concizumab levels and assess the body’s response to the treatment.

6 end of trial

The trial is expected to conclude by June 2026.

Final assessments are conducted to evaluate the overall effectiveness and safety of concizumab.

Who Can Join the Study?

You must give your permission to participate in the study before any study-related activities begin. These activities include any procedures done to see if you are suitable for the study.
You must be a male who is at least 12 years old at the time you give your permission to participate.
Your body weight must be more than 25 kilograms at the time of screening.
You must have been born with severe haemophilia A (which means your Factor VIII level is less than 1%) or moderate/severe haemophilia B (which means your Factor IX level is 2% or less). Haemophilia is a condition where your blood doesn’t clot properly.
You must have a record of using a product that contains a coagulation factor in the last 24 weeks. This requirement does not apply to certain patients who were enrolled before a treatment pause.

Who Cannot Join the Study?

Patients with haemophilia A or haemophilia B who have inhibitors. Inhibitors are substances in the blood that stop the treatment from working properly.
Female patients cannot participate in this study.
Patients who are considered part of a vulnerable population. This means people who might need extra protection or care, like those who cannot make decisions for themselves.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Centro Hospitalar Universitario Sao Joao E.P.E.	Porto	Portugal
Centre Hospitalier Regional Et Universitaire De Brest	Brest	France

Other Sites

Site Name	City	Country
Central Hospital Of Northern Pest Military Hospital	Budapest	Hungary
Instytut Hematologii I Transfuzjologii	Warsaw	Poland
Virgen del Rocío University Hospital	Sevilla	Spain
Vilniaus universiteto ligonine Santaros klinikos VšĮ	Vilnius	Lithuania
Saarland University Hospital	Homburg	Germany
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie	Cracow	Poland
Samodzielny Publiczny Szpital Kliniczny Nr 1 W Lublinie	Lublin	Poland
Uniwersytecki Szpital Kliniczny W Poznaniu	Poznan	Poland
Rigshospitalet	Copenhagen	Denmark
North Estonia Medical Centre Foundation	Tallin	Estonia
Rmgnaz Soxzt Sjgeue Undifxzneccvotqrplf	Malmo	Sweden
Assqpsi Oobwjxhujcg Ugbhnuelxfakh Ccfmundryvqg Drphi Svcugd E Duwxj Sbiaqxz Dj Taicqg	Turin	Italy
Frkmlfcsm Piwr Lq Iyswgjmkrcqrg Buqfqypep Dfm Htoxgfmc Utudjqrpaacwl Ln Pgu	Madrid	Spain

Trial status

Country	Status	Recruitment Start
Denmark	Not recruiting	13.11.2019
Estonia	Not recruiting	13.11.2019
France	Not recruiting	13.11.2019
Germany	Not recruiting	13.11.2019
Hungary	Not recruiting	13.11.2019
Italy	Not recruiting	13.11.2019
Lithuania	Not recruiting	13.11.2019
Poland	Not recruiting	13.11.2019
Portugal	Not recruiting	13.11.2019
Spain	Not recruiting	13.11.2019
Sweden	Not recruiting	13.11.2019

Trial locations

Investigated drugs:

Concizumab

Concizumab is a medication being studied for its ability to prevent bleeding episodes in patients with hemophilia A or B who do not have inhibitors. It is used as a prophylactic treatment, meaning it is given regularly to help prevent bleeding rather than treating bleeding episodes after they occur. The trial aims to see how effective concizumab is compared to not using any prophylactic treatment.

Haemophilia A – Haemophilia A is a genetic disorder where the blood does not clot properly due to a deficiency of clotting factor VIII. This condition leads to prolonged bleeding after injuries, surgeries, or even spontaneously without any apparent cause. Individuals with haemophilia A may experience frequent nosebleeds, easy bruising, and bleeding into joints and muscles, which can cause pain and swelling. Over time, repeated joint bleeds can lead to joint damage. The severity of bleeding episodes can vary, with some individuals experiencing mild symptoms and others having more severe bleeding issues.

Haemophilia B – Haemophilia B is a genetic disorder characterized by a deficiency of clotting factor IX, leading to improper blood clotting. Similar to haemophilia A, this condition results in prolonged bleeding after injuries or surgeries and can also cause spontaneous bleeding episodes. Common symptoms include frequent nosebleeds, easy bruising, and bleeding into joints and muscles, which can result in pain and swelling. Repeated joint bleeds may lead to joint damage over time. The severity of symptoms can vary among individuals, with some experiencing mild symptoms and others having more severe bleeding problems.

Trial ID:

2023-506831-13-00

Protocol code:

NN7415-4307

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on How Concizumab Works for Patients with Hemophilia A or B Without Inhibitors

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?