Study on the Effectiveness of Concizumab for Children Under 12 with Hemophilia A or B, With or 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 children under 12 years old who have Hemophilia A or Hemophilia B, with or without inhibitors. Hemophilia is a condition where the blood does not clot properly, leading to excessive bleeding. Inhibitors are substances that can interfere with the treatment of hemophilia. The medication being tested, Concizumab, is given as an injection under the skin using a special pen-injector device.

The purpose of the study is to see how well Concizumab works in preventing bleeding episodes compared to the previous treatment the children were receiving. The study will involve children who have not used Concizumab before. Participants will receive regular injections of Concizumab over a period of time, and the number of bleeding episodes they experience will be monitored and compared to their previous treatment.

Throughout the study, researchers will also keep track of any side effects or reactions to the medication, such as injection site reactions or the development of antibodies against Concizumab. The study aims to provide valuable information on the safety and effectiveness of Concizumab in managing bleeding episodes in young children with hemophilia.

1 joining the study

Upon joining the study, informed consent is required. This means understanding the study and agreeing to participate.

Eligibility is based on a diagnosis of severe haemophilia A or B, with or without inhibitors, and being under 12 years of age.

2 treatment initiation

The treatment involves the use of concizumab, a medication administered as a solution for injection under the skin (subcutaneous).

The specific dosage forms available are 40 mg/mL, 100 mg/mL, and 10 mg/mL.

3 treatment duration

The treatment is ongoing and will continue until the primary analysis cut-off, which is determined by the study’s timeline.

The study aims to compare the effectiveness of concizumab in reducing bleeding episodes compared to previous treatments.

4 monitoring and assessments

Throughout the study, the number of bleeding episodes, both spontaneous and traumatic, will be recorded.

Additional assessments include monitoring for any adverse events, such as reactions at the injection site or the development of antibodies to concizumab.

5 end of study

The study is estimated to conclude by November 2029.

Final assessments will be conducted to evaluate the overall effectiveness and safety of the treatment.

Who Can Join the Study?

Must have given permission to participate in the study. This means agreeing to take part after understanding what the study involves.
Must have a diagnosis of congenital severe haemophilia A (a condition where the blood doesn’t clot properly due to very low levels of a protein called factor VIII) or moderate/severe congenital haemophilia B (similar condition but due to low levels of factor IX), or have haemophilia with inhibitors (substances that prevent clotting treatments from working).
For one part of the study: Must be a boy under 12 years old at the time of agreeing to participate.
For boys with inhibitors: Must have medical records showing at least 26 weeks of treatment for bleeding episodes in the past year. This treatment involves using products that help blood clot.
For boys without inhibitors: Must have medical records showing at least 52 weeks of treatment for bleeding episodes in the past year, with at least 3 treated bleeding episodes documented. Alternatively, must have at least 26 weeks of preventive treatment in the past year.
For another part of the study: Must be a male patient who has previously been treated with a medication called concizumab through a special program.

Who Cannot Join the Study?

Children who are not male cannot participate in the study.
Children who do not have Haemophilia A or B cannot participate. Haemophilia is a condition where the blood does not clot properly, leading to excessive bleeding.
Children who are 12 years or older cannot participate in the study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Uniwersytecki Szpital Kliniczny Im Jana Mikulicza Radeckiego We Wroclawiu	Wroclaw	Poland
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Oslo Universitetssykehus HF	Oslo	Norway
Uniwersytecki Szpital Dzieciecy W Lublinie	Lublin	Poland

Other Sites

Site Name	City	Country
Spitalul Clinic De Urgenta Pentru Copii Cluj-Napoca	Cluj Napoca	Romania
Uniteversity Muliprofile Hospital For Active Treatment Tsaritsa Yoanna-Isul EAD	Sofia	Bulgaria
Hopital Beaujon	Clichy	France
Fundeni Clinical Institute	Bucharest	Romania
Afjldoi Oixxaxqwuyp dx Pjjrfi	Padua	Italy
Uirvolfdqj Mzpudnfytalc Heifunml Fyk Aoempq Tbfmjnpfa Spfby Gojouh Ebx	Plovdiv	Bulgaria
Adtgdeq Orynitmqkkaheouxcxqpmrzdq Pckrrnohzyp Gq Rxfztgyoqoda Mwclp Dz Cyvdkwo	Catania	Italy
Scfmhlao Cloihm Jhjgxcia Dr Uunwfva Bzxmi	Oradea	Romania
Hhlannzi Vimd dtpkxtay	Barcelona	Spain
Vlbpeccm uofvjryunwqw lcjdncmd Sueobhpf kjtdxhms Vjy	Vilnius	Lithuania
Ulsnutjuwd Cajhgypv Hpioutjb Vkchwp Dj Lv Ammnydti	Murcia	Spain
Civexp Hjiadyodioe Mlxsgjvys Sbwqva	Chambery	France
Ucfwjzeniruwyp Ckvtqsf Kqfavezck	Gdansk	Poland
Atbtdmh Osqymacnsqx Uiawqrwnjszxd Pqszt	Parma	Italy
Mndk Skpbw Mcgvim Esf	Varna	Bulgaria
Qfdjp Shllkz Cryngxwmp Hbqoirzd &rihktr Shfwgaghkxe Ufovxgvkrx Hsjaoyrc &fiwgkl Vgngsbp Gktxqdoktapeyrwbua	Gothenburg	Sweden
Fkwqqzsbr Prth Lt Ienemqclhtegc Bgepudssd Dhe Hdekprvl Ucfjylhvcgxbo Ld Pyn	Madrid	Spain
Nukzudgicz Ponyiu I Amzt Sjrhn	Athens	Greece
Uttkdeohyadubf Cbwgqnf Ktwxrlboj Wvsswtbqvghvr Upyqjbtzamgn Mxmajqooyj	Warsaw	Poland
Izgduhpued Glbqgsj Hhxqwmjb Oe Tgjjybaynkfc	Thessaloniki	Greece

Trial status

Country	Status	Recruitment Start
Bulgaria	Not recruiting	10.08.2022
France	Not recruiting	10.08.2022
Greece	Not recruiting	10.08.2022
Italy	Recruiting	10.08.2022
Lithuania	Recruiting	10.08.2022
Norway	Not recruiting	10.08.2022
Poland	Recruiting	10.08.2022
Romania	Not recruiting	10.08.2022
Spain	Recruiting	10.08.2022
Sweden	Recruiting	10.08.2022

Trial locations

Investigated Drugs:

Concizumab

Concizumab is a medication being studied for its ability to prevent bleeding episodes in children under 12 years old who have hemophilia A or B. This medication is being tested to see if it can reduce the number of bleeding episodes compared to the treatment the children were receiving before. The study includes children with and without inhibitors, which are substances that can make it harder for some treatments to work.

Investigated Diseases:

Hemophilia A – Hemophilia 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 hemophilia A may experience frequent nosebleeds, easy bruising, and joint bleeding, which can cause pain and swelling. Over time, repeated joint bleeding can lead to joint damage. The severity of bleeding episodes can vary, with some individuals experiencing more frequent and severe episodes than others.

Hemophilia B – Hemophilia B is a genetic bleeding disorder caused by a deficiency of clotting factor IX. Similar to hemophilia A, this condition results in prolonged bleeding after injuries or surgeries and can also cause spontaneous bleeding episodes. People with hemophilia B may experience symptoms such as frequent nosebleeds, easy bruising, and bleeding into joints and muscles. Joint bleeding can lead to pain, swelling, and over time, joint damage. The severity of symptoms can vary, with some individuals experiencing more frequent and severe bleeding episodes.

Trial ID:

2023-506925-13-00

Protocol code:

NN7415-4616

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Effectiveness of Concizumab for Children Under 12 with Hemophilia A or B, With or Without Inhibitors

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