A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients with Severe Haemophilia A without Inhibitors

Phase 1

• Conditions: Haemophilia A; MedDRA version: 20.0Level: LLTClassification code 10018938Term: Haemophilia A (Factor VIII)System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2016-000614-29-IT
• Lead Sponsor: OVO NORDISK. S.P.A.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Completion: 2020-06-03
• Study Start: 2021-06-17
• Last Update Posted: 21 July 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 36

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 the suitability for the trial
- Male patients aged 18 years or older at the time of signing informed consent, diagnosed with severe haemophilia A (FVIII activity below 1%), based on medical records or results at screening
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 30
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 3

Exclusion Criteria

- Known or suspected hypersensitivity to trial product(s) or related products
- Known inherited or acquired bleeding disorder other than haemophilia A
- Presence of inhibitors (neutralising antibodies) to Factor VIII (equal to or above 0.6 Bethesda Units) at screening measured by the Nijmegen method

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the efficacy of concizumab administered s.c. once daily in preventing bleeding episodes in patients with severe haemophilia A without inhibitors;Secondary Objective: To assess the longer-term efficacy of concizumab in patients with severe haemophilia A without inhibitors<br><br>To assess the safety of concizumab in patients with severe haemophilia A without inhibitors <br><br>To assess the immunogenicity of concizumab in patients with severe haemophilia A without inhibitors.;Primary end point(s): The number of bleeding episodes during at least 24 weeks from treatment onset.;Timepoint(s) of evaluation of this end point: During at least 24 weeks from treatment onset.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): 1. The number of spontaneous bleeding episodes <br><br>2. The number of treatment emergent adverse events (TEAEs);Timepoint(s) of evaluation of this end point: 1.+ 2. During at least 24 weeks from treatment onset<br><br>All endpoints referring to the time frame of at least 24 weeks will be evaluated in the main part of the trial, defined to end when the last patient has completed a minimum of 24 weeks of treatment with trial product (or have withdrawn). In addition, number of bleeding episodes during 76 weeks of treatment with prophylactic concizumab will be analysed.