134 Participants Needed

Concizumab for Hemophilia

(explorer7 Trial)

Recruiting at 174 trial locations
Age: Any Age
Sex: Male
Trial Phase: Phase 3
Sponsor: Novo Nordisk A/S
Must be taking: Bypassing agents
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 4 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests concizumab, a new medicine, in people with hemophilia A or B who have inhibitors. It aims to see if concizumab can prevent bleeding by helping the blood clot more effectively. Concizumab has shown good results in earlier studies for hemophilia A and B.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications. However, if you usually take medicine to prevent bleeds, you will start the study medicine right away. If you take medicine only when you have a bleed, you might continue your normal medicine for the first 6 months.

How is the drug Concizumab unique for treating hemophilia?

Concizumab is unique because it is a monoclonal antibody that targets the tissue factor pathway inhibitor (TFPI), which helps restore normal blood clotting in hemophilia patients. Unlike traditional treatments that replace missing clotting factors, Concizumab is administered subcutaneously (under the skin) and works by inhibiting TFPI to promote clot formation, offering a novel approach to managing bleeding episodes.12345

What data supports the effectiveness of the drug Concizumab for treating hemophilia?

Concizumab has been shown to help reduce bleeding episodes in people with hemophilia by blocking a protein that normally slows down blood clotting. In studies, it increased the body's ability to form clots, which is important for preventing excessive bleeding.14567

Who Is on the Research Team?

CR

Clinical Reporting Anchor and Disclosure (1452)

Principal Investigator

Novo Nordisk A/S

Are You a Good Fit for This Trial?

This trial is for males aged 12 or older with Hemophilia A or B and inhibitors, who have used bypassing agents in the last 24 weeks. It's not for those with thromboembolic disease, high risk of blood clots, ongoing immune treatments, hypersensitivity to similar drugs, or other coagulation disorders.

Inclusion Criteria

Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
I am a male aged 12 or older.
I have needed treatment with special blood clotting agents in the last 6 months.
See 1 more

Exclusion Criteria

I am undergoing or planning to start treatment to boost my immune system.
I have a blood clotting disorder that is not congenital haemophilia.
I am currently being treated for or showing signs of blood clots.
See 3 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive daily concizumab injections under the skin, which they administer themselves at home. The treatment phase includes randomization into different groups, with some starting concizumab immediately and others after 6 months.

167 weeks
Visits every 4 weeks for the first 6-12 months, then every 8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment, including recording information in an electronic diary and possibly wearing an activity tracker.

4 weeks

Extension

Participants in the extension part receive daily concizumab injections, continuing the treatment regimen.

Up to 7 years or until the drug is available for purchase

What Are the Treatments Tested in This Trial?

Interventions

  • Concizumab
Trial Overview The study tests concizumab's effectiveness in preventing bleeds in people with hemophilia on-demand or prophylaxis treatment. Participants self-inject daily using a pen-injector and are monitored over six years with clinic visits and an electronic diary.
How Is the Trial Designed?
4Treatment groups
Experimental Treatment
Group I: Arm 4: Concizumab prophylaxisExperimental Treatment1 Intervention
Patients previously on prophylaxis with by-passing agents and on-demand patients who are screened at a timepoint where the required number of patients in arms 1 and 2 have been randomised. These patients will, if eligible, be enrolled into the trial and will initiate concizumab prophylaxis at visit 2a (week 0).
Group II: Arm 3: Concizumab prophylaxisExperimental Treatment1 Intervention
The HAwI and HBwI patients enrolled into the concizumab phase 2 trial (NN7415-4310) at time of transfer will be offered enrolment into this trial. It is required that these patients are on concizumab prophylaxis up until enrolment into the trial. These patients will continue concizumab prophylaxis.
Group III: Arm 2: Concizumab prophylaxisExperimental Treatment1 Intervention
HAwI and HBwI patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis.
Group IV: Arm 1: No prophylaxisExperimental Treatment1 Intervention
Haemophilia A with inhibitors (HAwI) and haemophilia B with inhibitors (HBwI) patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis. In the extension part, patients in arm 1 will receive daily concizumab subcutaneous (s.c., under the skin) injections.

Concizumab is already approved in European Union, United States, Canada for the following indications:

🇪🇺
Approved in European Union as Alhemo for:
  • Hemophilia A with inhibitors
  • Hemophilia B with inhibitors
🇺🇸
Approved in United States as Alhemo for:
  • Hemophilia A with factor VIII inhibitors
  • Hemophilia B with factor IX inhibitors
🇨🇦
Approved in Canada as Alhemo for:
  • Hemophilia A with inhibitors
  • Hemophilia B with inhibitors

Find a Clinic Near You

Who Is Running the Clinical Trial?

Novo Nordisk A/S

Lead Sponsor

Trials
1,578
Recruited
3,813,000+
Lars Fruergaard Jørgensen profile image

Lars Fruergaard Jørgensen

Novo Nordisk A/S

Chief Executive Officer since 2017

MSc in Finance and Business Administration, Aarhus School of Business, Aarhus University, Denmark

Martin Holst Lange profile image

Martin Holst Lange

Novo Nordisk A/S

Chief Medical Officer since 2021

MD from University of Copenhagen

Published Research Related to This Trial

Concizumab is a monoclonal antibody that effectively prevents tissue factor pathway inhibitor (TFPI) from inhibiting activated Factor X, which is crucial for blood clotting, making it a promising treatment for hemophilia A and B.
In March 2023, concizumab received approval in Canada for use in adolescents and adults with hemophilia B who have FIX inhibitors, marking a significant advancement in prophylactic treatment options to reduce bleeding episodes.
Concizumab: First Approval.Keam, SJ.[2023]
Concizumab, a monoclonal antibody that inhibits tissue factor pathway inhibitor (TFPI), effectively restores thrombin generation in hemophilia patients, showing promise in reducing bleeding episodes as demonstrated in both plasma studies and a rabbit model.
In Phase 1 trials, Concizumab led to a dose-dependent decrease in TFPI levels and increased thrombin generation without significant thrombosis risk, indicating its potential as a safe and effective alternative to traditional factor replacement therapies.
Inhibition of Tissue Factor Pathway Inhibitor (TFPI) as a Treatment for Haemophilia: Rationale with Focus on Concizumab.Chowdary, P.[2023]
Concizumab effectively binds to and neutralizes the inhibitory function of tissue factor pathway inhibitor (TFPI), promoting coagulation, which could be beneficial for patients with hemophilia.
Pharmacokinetic studies in rabbits showed a non-linear, dose-dependent clearance profile for concizumab, indicating that its distribution and elimination are influenced by its interaction with TFPI on the endothelium of blood vessels.
Target-mediated clearance and bio-distribution of a monoclonal antibody against the Kunitz-type protease inhibitor 2 domain of Tissue Factor Pathway Inhibitor.Hansen, L., Petersen, LC., Lauritzen, B., et al.[2019]

Citations

Concizumab: First Approval. [2023]
A randomized trial of safety, pharmacokinetics and pharmacodynamics of concizumab in people with hemophilia A. [2023]
Inhibition of Tissue Factor Pathway Inhibitor (TFPI) as a Treatment for Haemophilia: Rationale with Focus on Concizumab. [2023]
Target-mediated clearance and bio-distribution of a monoclonal antibody against the Kunitz-type protease inhibitor 2 domain of Tissue Factor Pathway Inhibitor. [2019]
Concizumab as a Subcutaneous Prophylactic Treatment Option for Patients with Hemophilia A or B: A Review of the Evidence and Patient's Perspectives. [2022]
Phase 3 Trial of Concizumab in Hemophilia with Inhibitors. [2023]
Safety and pharmacokinetics of anti-TFPI antibody (concizumab) in healthy volunteers and patients with hemophilia: a randomized first human dose trial. [2023]
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