N/A
N=78
Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ
Hemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT02962765 ↗Enrolled (actual)
78
Serious AEs
0.0%
Results posted
Sep 2021
Primary outcome: Primary: Number of Patients With FVIII Inhibitors — 0; 0; 0 Participants
Study Design & Population
- Study type
- Observational
- Phase
- N/A
- Interventions
- —
- Age
- Pediatric, Adult, Older Adult
- Sex
- Male
- Sponsor
- Octapharma
- Primary completion
- Aug 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients With FVIII Inhibitors |
0; 0; 0 | — |
| PRIMARY Number of Patients With Adverse Drug Reactions |
— | — |
| SECONDARY Annualized Rate of Breakthrough Bleeds to Assess Efficacy in Prophylactic Treatment |
2.39; 0.00; 0.00 | — |
| SECONDARY Assessment of the Efficacy of On-demand Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale |
50; 4; 1; 0 | — |
| SECONDARY Overall Assessment of the Effectiveness of Surgical Prophylaxis by the Treating Physicians |
5; 0; 0; 0 | — |
| SECONDARY Assessment of the Efficacy of Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale |
167; 50; 26; 3 | — |
Summary
Prospective, multinational, non-interventional post-authorisation study to collect additional clinical data and to ensure consistency in the long-term between the outcome from pre-authorisation clinical studies (in 135 previously treated paediatric and adult patients) and routine clinical practice. Besides aspects such as general product safety and efficacy, there will be a focus on immunogenicity, particularly on inhibitor development. The diagnosis of FVIII inhibitor will be based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
Eligibility Criteria
Inclusion Criteria
- Haemophilia A (FVIII: C ≤ 2%) based on medical history; at least 100 patients should have severe haemophilia A (FVIII:C 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.
Data sourced from ClinicalTrials.gov (NCT02962765). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.