Plain English Summary
Background and study aims
Haemophilia A is an inherited condition where there's not enough of a substance called clotting factor VIII in the blood as there should be, so the patient bleeds for longer than usual. Nuwiq is an engineered version of clotting factor VIII. The aim of this study is to find out whether treatment with Nuwiq prevents bleeding in patients with haemophilia A.
Who can participate?
Haemophilia A patients being treated with Nuwiq
What does the study involve?
The routine treatment of haemophilia A patients is documented, including all treatments with Nuwiq, any bleeding episodes, surgical procedures, and quality of life.
What are the possible benefits and risks of participating?
Not provided at time of registration
Where is the study run from?
Vivantes Klinikum (Germany)
When is the study starting and how long is it expected to run for?
March 2015 to March 2020
Who is funding the study?
Investigator initiated and funded
Who is the main contact?
Jennifer Feddern
Trial website
Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
GENA-100
Study information
Scientific title
Prevention of bleeding in haemophilia A by prophylactic treatment with Nuwiq® : a prospective, multi-national, non-interventional study to evaluate routine practice prophylactic treatment schedules – NIS-Previq
Acronym
NIS-Previq
Study hypothesis
To assess the influence of weekly FVII dose on annualized bleeding rate.
Ethics approval
Ethics Committee of the Medical Association Berlin, 18/11/2015, ref: Eth-11/15
Study design
Open prospective multi-centre multi-national non-interventional study
Primary study design
Observational
Secondary study design
Cohort study
Trial setting
Other
Trial type
Treatment
Patient information sheet
Not available in web format, please use the contact details below to request a patient information sheet
Condition
Haemophilia A
Intervention
NIS-Previq is a non-interventional study for which the routine treatment of haemophilia A patients on prophylaxis is documented. All treatments with Nuwiq, any bleeding episode occuring or surgical procedures are carefully documented. Additionally, a joint score (HJHS), quality of life questionnaire (SF-36) and/or a pharmacokinetic evaluation with individually calculated dosing schedules can be documented on an optional basis.
Intervention type
Drug
Phase
Not Applicable
Drug names
Simoctocog alfa
Primary outcome measures
Annual bleeding rate (ABR) during prophylaxis with Nuwiq and ABR by weekly Nuwiq doses (IU/kg)
Secondary outcome measures
1. Frequency distribution of the infusion intervals used – e.g. once, twice, thrice weekly or more frequently.
1.1. FVIII Dose/kg body weight per injection
1.2. Weekly FVIII Dose/kg body weight
2. Changes in SF-36 results over time and after PK-based adjustments of prophylactic schedule.
3. Changes in HJHS Score results over time and after PK-based adjustments of prophylactic schedule.
4 . Efficacy assessments for treatment of bleeding episodes according to a 4-point verbal rating scale
5. ADR rates per infusion and per patient
Overall trial start date
01/03/2015
Overall trial end date
31/03/2020
Reason abandoned
Eligibility
Participant inclusion criteria
1. Haemophilia A
2. Treated with Nuwiq
3. Prophylactic treatment schedule for at least 6 month is to be expected
4. Good compliance regarding documentation of FVIII treatments and bleeding episodes (in case home treatment is performed)
Participant type
Patient
Age group
All
Gender
Both
Target number of participants
200
Participant exclusion criteria
Patients with known contraindications as specified in the SPC
Recruitment start date
01/03/2015
Recruitment end date
31/03/2020
Locations
Countries of recruitment
Germany
Trial participating centre
Vivantes Klinikum
Rudower Straße 48
Berlin
12351
Germany
Funders
Funder type
Not defined
Funder name
Investigator initiated and funded
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration.
Intention to publish date
Participant level data
Not provided at time of registration
Results - basic reporting
Publication summary