Condition category
Haematological Disorders
Date applied
23/02/2015
Date assigned
23/03/2015
Last edited
30/03/2016
Prospective/Retrospective
Retrospectively registered
Overall trial status
Ongoing
Recruitment status
Recruiting

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

Contact information

Type

Scientific

Primary contact

Mrs Jennifer Feddern

ORCID ID

Contact details

Elisabeth-Selbert-Strasse 11
Langenfeld
40764
Germany

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

Sponsor information

Organisation

Octapharma GmbH

Sponsor details

Elisabeth-Selbert-Strasse 11
Langenfeld
40764
Germany

Sponsor type

Industry

Website

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

Publication citations

Additional files

Editorial Notes

30/03/2016: Ethics approval information added. 15/03/2016: Plain English summary added.