Prevention of bleeding in haemophilia A by prophylactic treatment with Nuwiq®
| ISRCTN | ISRCTN57549638 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN57549638 |
| Secondary identifying numbers | GENA-100 |
- Submission date
- 23/02/2015
- Registration date
- 23/03/2015
- Last edited
- 11/06/2025
- Recruitment status
- No longer recruiting
- Overall study status
- Ongoing
- Condition category
- Haematological Disorders
Plain English summary of protocol
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. Octanate® and Wilate® are human plasma-derived factor VIII concentrate, and Nuwiq® is an engineered version of clotting factor VIII. The aim of this study is to find out whether treatment with Octanate®, Wilate® or Nuwiq prevents bleeding in patients with haemophilia A.
Who can participate?
Haemophilia A patients being treated with Octanate®, Wilate® or 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 2030
Who is funding the study?
Investigator initiated and funded
Who is the main contact?
Jennifer Feddern
Contact information
Scientific
Elisabeth-Selbert-Strasse 11
Langenfeld
40764
Germany
Study information
| Study design | Open prospective multi-centre multi-national non-interventional study |
|---|---|
| Primary study design | Observational |
| Secondary study design | Cohort study |
| Study setting(s) | Other |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details to request a patient information sheet |
| Scientific title | Prevention and treatment of bleeding in haemophilia A with Nuwiq®: Octanate®, Wilate®: A prospective, non-interventional study to evaluate prophylactic and on-demand treatment schedules and factor VIII requirement in routine clinical practice |
| Study acronym | PEAQ-NOW |
| Study objectives | To assess the influence of weekly FVII dose on annualized bleeding rate. |
| Ethics approval(s) | Ethics Committee of the Medical Association Berlin, 18/11/2015, ref: Eth-11/15 |
| Health condition(s) or problem(s) studied | Haemophilia A |
| Intervention | Current interventions as of 19/03/2024: NIS-Previq is a non-interventional study for which the routine treatment of haemophilia A patients on prophylaxis is documented. All treatments with Octanate®, Wilate®or Nuwiq®, any bleeding episode occuring or surgical procedures are carefully documented. Additionally, a joint score (HJHS) and/or a pharmacokinetic evaluation with individually calculated dosing schedules can be documented on an optional basis. Previous interventions: 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 |
| Pharmaceutical study type(s) | |
| Phase | Not Applicable |
| Drug / device / biological / vaccine name(s) | Simoctocog alfa, Octanate®, Wilate® |
| Primary outcome measure | Current primary outcome measure as of 19/03/2024: Annual bleeding rate (ABR) during prophylaxis with Octanate®, Wilate® or Nuwiq® and ABR by weekly Nuwiq doses (IU/kg) Previous primary outcome measure: Annual bleeding rate (ABR) during prophylaxis with Nuwiq and ABR by weekly Nuwiq doses (IU/kg) |
| Secondary outcome measures | Current secondary outcome measures as of 19/03/2024: 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 HJHS Score results over time and after PK-based adjustments of prophylactic schedule. 3. Efficacy assessments for treatment of bleeding episodes according to a 4-point verbal rating scale 4. ADR rates per infusion and per patient Previous 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 study start date | 01/03/2015 |
| Completion date | 31/03/2030 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | All |
| Sex | Both |
| Target number of participants | 200 |
| Key inclusion criteria | Current inclusion criteria as of 11/06/2025: 1. The patient suffers from haemophilia A or reduced factor VIII levels with bleeding tendency 2. Treated with Octanate®, Wilate®or Nuwiq® 3. One of the following criteria is met: 3.1. The Patient receives prophylaxis with FVIII and a duration of 6 month is foreseen 3.2. The Patient is treated on demand with FVIII and treatment within the next 6 month is likely 3.3. The Patient receives prophylaxis with non-factor preparation and is in actual need of factor VIII treatment 4. Good compliance regarding documentation of FVIII treatments and bleeding episodes (in case home treatment is performed) _____ Previous inclusion criteria as of 19/03/2024: 1. Haemophilia A 2. Treated with Octanate®, Wilate®or Nuwiq® 3. Prophylactic treatment schedule for at least 6 months is to be expected 4. Good compliance regarding documentation of FVIII treatments and bleeding episodes (in case home treatment is performed) _____ Previous inclusion criteria: 1. Haemophilia A 2. Treated with Nuwiq 3. Prophylactic treatment schedule for at least 6 months is to be expected 4. Good compliance regarding documentation of FVIII treatments and bleeding episodes (in case home treatment is performed) |
| Key exclusion criteria | Patients with known contraindications as specified in the SPC |
| Date of first enrolment | 01/03/2015 |
| Date of final enrolment | 31/08/2024 |
Locations
Countries of recruitment
- Germany
Study participating centre
Berlin
10249
Germany
Sponsor information
Industry
Elisabeth-Selbert-Strasse 11
Langenfeld
40764
Germany
"ROR" |
https://ror.org/002k5fe57 |
|---|
Funders
Funder type
Not defined
No information available
Results and Publications
| Intention to publish date | 28/02/2026 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Data sharing statement to be made available at a later date |
| Publication and dissemination plan | Publication plans for the current study are not defined and will be made available at a later date |
| IPD sharing plan | The data-sharing plans for the current study are unknown and will be made available at a later date |
Editorial Notes
11/06/2025: The following changes were made to the trial record:
1. The scientific title was changed from "Prevention of bleeding in haemophilia A by prophylactic treatment with Octanate®, Wilate®, Nuwiq®: a prospective, multi-national, non-interventional study to evaluate routine practice prophylactic treatment schedules – NIS-Previq" to "Prevention and treatment of bleeding in haemophilia A with Nuwiq®: Octanate®, Wilate®: A prospective, non-interventional study to evaluate prophylactic and on-demand treatment schedules and factor VIII requirement in routine clinical practice".
2. The study acronym was changed from NIS-Previq to PEAQ-NOW.
3. The overall end date was changed from 28/02/2025 to 31/03/2030.
4.
19/03/2024: The following changes were made to the study record:
1. The scientific title was changed from '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' to 'Prevention of bleeding in haemophilia A by prophylactic treatment with Octanate®, Wilate®, Nuwiq®: a prospective, multi-national, non-interventional study to evaluate routine practice prophylactic treatment schedules – NIS-Previq'.
2. The interventions, primary and secondary outcome measures and inclusion criteria were updated.
3. The recruitment end date was changed from 31/03/2020 to 31/08/2024.
4. The overall study end date was changed from 31/03/2020 to 28/02/2025.
5. The study participating centre was changed from Vivantes Klinikum to Vivantes Klinikum Friedrichshain.
6. Octanate® and Wilate® were added to the drug/device/biological/vaccine name(s).
7. Publication and dissemination plan and IPD sharing plan added.
12/03/2024: Contact details updated.
30/03/2016: Ethics approval information added.
15/03/2016: Plain English summary added.
