Clinical trial NCT03196297: A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors
Official title: A Multi-Centre Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Haemostasis
Haemophilia A
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2017-06-22
Enrollment: 36
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This trial is conducted in Asia, Europe and the United States of America (USA). The aim of the trial is to assess the efficacy of concizumab administered s.c. (subcutaneously, under the skin) once daily in preventing bleeding episodes in patients with severe haemophilia A without inhibitors.


Eligibility criteria: 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 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

Primary outcomes:
measure: The Number of Bleeding Episodes During at Least 24 Weeks From Treatment Onset
timeFrame: During at least 24 weeks from treatment onset
Secondary outcomes:
measure: The Number of Bleeding Episodes During at Least 76 Weeks From Treatment Onset
timeFrame: During at least 76 weeks from treatment onset
measure: The Number of Spontaneous Bleeding Episodes During at Least 24 Weeks From Treatment Onset
timeFrame: During at least 24 weeks from treatment onset
measure: The Number of Spontaneous Bleeding Episodes During at Least 76 Weeks From Treatment Onset
timeFrame: During at least 76 weeks from treatment onset
measure: Number of Treatment-emergent Adverse Events (TEAEs) During at Least 24 Weeks From Treatment Onset
timeFrame: During at least 24 weeks from treatment onset (week 0)
measure: Number of Treatment-emergent Adverse Events (TEAEs) During at Least 76 Weeks From Treatment Onset
timeFrame: During at least 76 weeks from treatment onset (week 0)
measure: Occurrence of Anti-concizumab Antibodies During at Least 24 Weeks From Treatment Onset
timeFrame: During at least 24 weeks from treatment onset (week 0)
measure: Occurrence of Anti-concizumab Antibodies During at Least 76 Weeks From Treatment Onset
timeFrame: During at least 76 weeks from treatment onset (week 0)
measure: Change in Fibrinogen During 24 Weeks From Treatment Onset
timeFrame: During 24 weeks from treatment onset (week 0)
measure: Change in Fibrinogen During at Least 76 Weeks From Treatment Onset
timeFrame: During at least 76 weeks from treatment onset (week 0)
measure: Change in D-dimer During 24 Weeks From Treatment Onset
timeFrame: During 24 weeks from treatment onset (week 0)
measure: Change in D-dimer During at Least 76 Weeks From Treatment Onset
timeFrame: During at least 76 weeks from treatment onset (week 0)
measure: Change in Prothrombin Fragment 1 + 2 (F1 + F2) During 24 Weeks From Treatment Onset
timeFrame: During 24 weeks from treatment onset (week 0)
measure: Change in Prothrombin Fragment 1 + 2 (F1 + F2) During at Least 76 Weeks From Treatment Onset
timeFrame: During at least 76 weeks from treatment onset (week 0)
measure: Change in Prothrombin Time (PT) During 24 Weeks From Treatment Onset
timeFrame: During 24 weeks from treatment onset (week 0)
measure: Change in Prothrombin Time (PT) During at Least 76 Weeks From Treatment Onset
timeFrame: During at least 76 weeks from treatment onset (week 0)
measure: Change in Activated Partial Thromboplastin Time (APTT) During 24 Weeks From Treatment Onset
timeFrame: During 24 weeks from treatment onset (week 0)
measure: Change in Activated Partial Thromboplastin Time (APTT) During at Least 76 Weeks From Treatment Onset
timeFrame: During at least 76 weeks from treatment onset (week 0)
measure: Change in Anti-thrombin (AT) During 24 Weeks From Treatment Onset
timeFrame: During 24 weeks from treatment onset (week 0)
measure: Change in Anti-thrombin (AT) After at Least 76 Weeks From Treatment
timeFrame: During at least 76 weeks from treatment onset (week 0)
measure: Concentration of Concizumab Prior to the Last Dose Administration at 24 Weeks
timeFrame: Prior to the last dose administration at 24 weeks
measure: Concentration of Concizumab Prior to the Last Dose Administration After at Least 76 Weeks
timeFrame: Prior to the last dose administration after at least 76 weeks
measure: Free Tissue Factor Pathway Inhibitor (TFPI) Concentration Value Prior to the Last Dose Administration at 24 Weeks
timeFrame: Prior to the last dose administration at 24 weeks
measure: Free Tissue Factor Pathway Inhibitor (TFPI) Concentration Value Prior to the Last Dose Administration After at Least 76 Weeks
timeFrame: Prior to the last dose administration after at least 76 weeks
measure: Peak Thrombin Generation Prior to the Last Dose Administration at 24 Weeks
timeFrame: Prior to the last dose administration at 24 weeks
measure: Peak Thrombin Generation Prior to the Last Dose Administration After at Least 76 Weeks
timeFrame: Prior to the last dose administration after at least 76 weeks
measure: Endogenous Thrombin Potential Prior to the Last Dose Administration at 24 Weeks
timeFrame: Prior to the last dose administration at 24 weeks
measure: Endogenous Thrombin Potential Prior to the Last Dose Administration After at Least 76 Weeks
timeFrame: Prior to the last dose administration after at least 76 weeks
measure: Thrombin Generation Velocity Index Prior to the Last Dose Administration at 24 Weeks
timeFrame: Prior to the last dose administration at 24 weeks
measure: Thrombin Generation Velocity Index Prior to the Last Dose Administration After at Least 76 Weeks
timeFrame: Prior to the last dose administration after at least 76 weeks
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Indianapolis
country: United States
facility: Novo Nordisk Investigational Site
state: Indiana
city: Oklahoma City
country: United States
facility: Novo Nordisk Investigational Site
state: Oklahoma
city: Nashville
country: United States
facility: Novo Nordisk Investigational Site
state: Tennessee
city: Salt Lake City
country: United States
facility: Novo Nordisk Investigational Site
state: Utah
city: Brest
country: France
facility: Novo Nordisk Investigational Site
city: Caen
country: France
facility: Novo Nordisk Investigational Site
city: Nantes Cedex 1
country: France
facility: Novo Nordisk Investigational Site
city: Bonn
country: Germany
facility: Novo Nordisk Investigational Site
city: Homburg
country: Germany
facility: Novo Nordisk Investigational Site
city: Milano
country: Italy
facility: Novo Nordisk Investigational Site
city: Rome
country: Italy
facility: Novo Nordisk Investigational Site
city: Aichi
country: Japan
facility: Novo Nordisk Investigational Site
city: Nara
country: Japan
facility: Novo Nordisk Investigational Site
city: Tokyo
country: Japan
facility: Novo Nordisk Investigational Site
city: Tokyo
country: Japan
facility: Novo Nordisk Investigational Site
city: Madrid
country: Spain
facility: Novo Nordisk Investigational Site
city: Málaga
country: Spain
facility: Novo Nordisk Investigational Site
city: Valencia
country: Spain
facility: Novo Nordisk Investigational Site
city: Malmö
country: Sweden
facility: Novo Nordisk Investigational Site
city: Solna
country: Sweden
facility: Novo Nordisk Investigational Site
city: Bangkok
country: Thailand
facility: Novo Nordisk Investigational Site
city: Ankara
country: Turkey
facility: Novo Nordisk Investigational Site
city: Bornova-IZMIR
country: Turkey
facility: Novo Nordisk Investigational Site
city: Edirne
country: Turkey
facility: Novo Nordisk Investigational Site
city: İstanbul
country: Turkey
facility: Novo Nordisk Investigational Site
city: Lviv
country: Ukraine
facility: Novo Nordisk Investigational Site
city: Belfast
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: Cambridge
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: London
country: United Kingdom
facility: Novo Nordisk Investigational Site
city: London
country: United Kingdom
facility: Novo Nordisk Investigational Site
Study officials:
References:
citation: Shapiro AD, Angchaisuksiri P, Astermark J, Benson G, Castaman G, Eichler H, Jimenez-Yuste V, Kavakli K, Matsushita T, Poulsen LH, Wheeler AP, Young G, Zupancic-Salek S, Oldenburg J, Chowdary P. Long-term efficacy and safety of subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors. Blood Adv. 2022 Jun 14;6(11):3422-3432. doi: 10.1182/bloodadvances.2021006403.
citation: Shapiro AD, Angchaisuksiri P, Astermark J, Benson G, Castaman G, Chowdary P, Eichler H, Jimenez-Yuste V, Kavakli K, Matsushita T, Poulsen LH, Wheeler AP, Young G, Zupancic-Salek S, Oldenburg J. Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial results. Blood. 2019 Nov 28;134(22):1973-1982. doi: 10.1182/blood.2019001542.