Clinical trial NCT04334057: Post-Marketing Surveillance (Use-results Surveillance) With Esperoct®
Official title: Post-Marketing Surveillance (Use-results Surveillance) With Esperoct®. A Multi-centre, Prospective, Observational, Non-interventional Post-marketing Study to Investigate the Long-term Safety and Effectiveness of Esperoct® in Haemophilia A Patients Under Routine Clinical Practice Conditions in Japan
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Haemophilia A
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2020-04-03
Enrollment: 23
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Haemophilia A patients in routine clinical practice in Japan

Brief summary: The purpose of this study is to assess the safety and effectiveness of Esperoct® for long-term routine use in patients with Haemophilia A. Participants will get Esperoct® as prescribed by their doctor. The study will last for about 2 years for each participant.


Eligibility criteria: Inclusion Criteria:

* Signed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol).
* The decision to initiate treatment with commercially available Esperoct® has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study.
* Diagnosis of haemophilia A in males or females, no age limitation.
* New patients who have not previously been exposed to Esperoct®.

Exclusion Criteria:

* Previous participation in this study. Participation is defined as having given informed consent in this study.
* Known or suspected hypersensitivity to study product or related products.
* Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.

Primary outcomes:
measure: Number of adverse reactions (ARs) reported during the observation period
timeFrame: From baseline (week 0) to end of study (week 104)
Secondary outcomes:
measure: Number of serious adverse events (SAEs) reported during the observation period
timeFrame: From baseline (week 0) to end of study (week 104)
measure: Number of serious adverse reactions (SARs) reported during the observation period
timeFrame: From baseline (week 0) to end of study (week 104)
measure: Number of patients who have confirmed inhibitory antibodies against FVIII during the observation period
timeFrame: From baseline (week 0) to end of study (week 104)
measure: Number of bleeding episodes requiring treatment for patients using Esperoct® during the observation period assessed by annual bleeding rate (ABR)
timeFrame: From baseline (week 0) to end of study (week 104)
measure: Evaluation of the haemostatic response of Esperoct® measured as number of successes for treatment requiring bleeds
timeFrame: From baseline (week 0) to end of study (week 104)
measure: Evaluation of the haemostatic response of Esperoct® measured as number of successes in treatment of bleeds in perioperative management during surgical procedures
timeFrame: From baseline (week 0) to end of study (week 104)
Other outcomes:
Locations:
city: Fukuoka
country: Japan
facility: Kurume University Hospital, Pediatrics
city: Gifu
country: Japan
facility: Gifu University Hospital
city: Iruma-gun, Saitama
country: Japan
facility: Saitama Medical University Hospital, Pediatrics
city: Kamigyo-ku, Kyoto
country: Japan
facility: University Hospital Kyoto Prefectual University of Medicine
city: Kanagawa
country: Japan
facility: St. Marianna University School of Medicine Hospital_Pediatrics
city: Kanagawa
country: Japan
facility: St. Marianna University School of Medicine Hospital
city: Nara
country: Japan
facility: Nara Medical University Hospital_Pediatrics
city: Okinawa
country: Japan
facility: Nanbu Medical Center & Children's Medical Center
city: Saitama
country: Japan
facility: Shibuya Children's Clinic, Department of Pediatric
city: Shiga
country: Japan
facility: Lake Children Clinic
city: Shizuoka
country: Japan
facility: Shizuoka Children's Hospital, Hematology-Oncology
city: Shizuoka
country: Japan
facility: Shizuoka Children's Hospital
city: Tokyo
country: Japan
facility: Nippon Medical School Hospital
city: Tokyo
country: Japan
facility: Tokyo Medical Univ. Hospital_Laboratory Medicine
city: Tokyo
country: Japan
facility: Tokyo Medical Univ. Hospital
city: Tokyo
country: Japan
facility: Ogikubo Hospital_Tokyo
city: Yamagata
country: Japan
facility: Nihonkai Sogo Hospital
city: Yokohama-shi, Kanagawa
country: Japan
facility: St. Marianna Univ., Yokohama City Seibu HP, Pediatrics Dept,
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Reporting Anchor & Disclosure (1452)
role: STUDY_DIRECTOR
References: