Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT02962765 |
Recruitment Status :
Completed
First Posted : November 11, 2016
Last Update Posted : August 27, 2020
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease |
---|
Hemophilia A |
Study Type : | Observational |
Estimated Enrollment : | 200 participants |
Observational Model: | Cohort |
Time Perspective: | Prospective |
Official Title: | Prospective, Multinational, Non-interventional Post-authorisation Study to Document the Long-term Immunogenicity, Safety, and Efficacy of Human-cl rhFVIII (Simoctocog Alfa) in Patients With Haemophilia A Treated in Routine Clinical Practice |
Study Start Date : | January 2015 |
Actual Primary Completion Date : | August 20, 2020 |
Actual Study Completion Date : | August 20, 2020 |

- Incidence of FVIII inhibitors [ Time Frame: 100 exposure days ]FVIII inhibitors will be determined based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
- Incidence of adverse drug reactions [ Time Frame: 100 exposure days ]Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded in by patients in treatment diaries, which will be reviewed at each Follow-up Visit.
- Annualized rate of breakthrough bleeds to assess efficacy in prophylactic treatment [ Time Frame: 100 exposure days ]The occurrence of bleeding events during the study will documented in the treatment diary.
- Assessment of the efficacy of on-demand treatment [ Time Frame: 100 exposure days ]The treatment of bleeding episodes (BE) will be assessed by either by the patient (or the patient's parent or legal guardian) or by the treating physician in case of on-site treatment using a 4 point efficacy scale: 'excellent', 'good', 'moderate', 'none'. Based on this assessment, efficacy ratings assessed as either 'excellent' or 'good' will be considered 'successfully treated'.
- Overall assessment of the effectiveness of surgical prophylaxis by the treating physicians [ Time Frame: 100 exposure days ]At the end of the postoperative period, an overall assessment of the efficacy of treatment in the pre-, peri-, and postoperative periods using the 'excellent,' 'good,' moderate,' and 'none' scale will be done jointly by the surgeon and the haematologist. Based on this assessment, efficacy ratings assessed as either 'excellent' or 'good' will be considered 'successfully treated'.
Biospecimen Retention: Samples Without DNA

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
The goal is to collect data on 200 previously treated male patients of any age with haemophilia (FVIII:C ≤ 2%). Patients from pre-authorisation studies can be followed up to at least 100 EDs. Newly enrolled patients have to be treated and followed for at least 100 EDs.
- Of the 200 enrolled patients, at least 100 patients should have severe haemophilia A (FVIII:C < 1%).
- Of the 200 enrolled patients, approx. 60 patients should be < 12 years of age. At least 10 patients should be aged between 14-18 years.
- Patients with severe haemophilia A after successful immune tolerance induction (ITI) can also be included; the proportion of these ITI patients should not exceed 25% of the entire cohort.
Inclusion Criteria:
- Haemophilia A (FVIII:C ≤ 2%) based on medical history; at least 100 patients should have severe haemophilia A (FVIII:C < 1%)
- Male patients of any age
- Previous treatment with a FVIII concentrate for more than 150 EDs
- Availability of detailed documentation (patient diary, log book, etc.) covering either the last 50 EDs or the last 2 years per patient to confirm treatment modality (i.e., prophylaxis, on-demand, recent surgery, or immune tolerance induction)
- Inhibitor negative (< 0.6 BU) at study entry as confirmed by a recovery test with previous FVIII product and inhibitor test in a central laboratory
- Immunocompetence (CD4+ count > 200/µL), HIV-negative, or having a viral load < 200 particles/µL or < 400,000 copies/mL
- Decision to prescribe Human-cl rhFVIII before enrolment into the study
- Written informed consent by the patient or the patient's parent or legal guardian
Exclusion Criteria:
- Patients treated with any investigational medicinal product (IMP) except FVIII IMP within 30 days prior to the Screening Visit or patients planning to undergo treatment with any IMP other than Human-cl rhFVIII are not eligible for enrolment into the study.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02962765

Principal Investigator: | Kate Khair, PhD | Great Ormond Street Hospital |
Responsible Party: | Octapharma |
ClinicalTrials.gov Identifier: | NCT02962765 |
Other Study ID Numbers: |
GENA-99 |
First Posted: | November 11, 2016 Key Record Dates |
Last Update Posted: | August 27, 2020 |
Last Verified: | August 2020 |
Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn |