N/A
Completed N=106
Post-Marketing Surveillance To Observe Safety And Efficacy Of Xyntha Solofuse Prefilled Syringe
Factor VIII Deficiency, Congenital · Factor 8 Deficiency, Congenital · Autosomal Hemophilia A · Classic Hemophilia
Source: ClinicalTrials.gov NCT03034044 ↗
Enrolled (actual)
106
Serious AEs
0.0%
Results posted
Apr 2019
Primary outcomePrimary: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) — 15; 0 Participants
Summary
This study aims to observe the safety and efficacy of the Xyntha Solofuse prefilled syringe in the setting of routine practice. The primary objective is to detect medically significant events (factor VIII inhibitor). The secondary objective is to observe the overall efficacy and safety of the Xyntha Solofuse prefilled syringe including serious adverse events. In this open-label, non-comparative, observational, non-interventional, retrospective and multi-center study, post-marketing surveillance data will be collected retrospectively for up to 6 months from the initial administration day of the Xyntha Solofuse prefilled syringe injected into patients who have been administered the Xyntha Solofuse prefilled syringe.
As specified in the product approval issued by the Ministry of Food and Drug Safety, the study will be conducted for 4 years from the approval date. At least 600 study subjects will be enrolled in this study to meet the MFDS requirements. Although 600 is the assigned number of study subjects, the number of cases will be adjusted considering the actual number of enrolled subjects after the study start day.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
15; 0 | — |
| PRIMARY Number of Participants With Adverse Events (AEs) by Severity |
10; 5; 0 | — |
| PRIMARY Number of Participants Who Discontinued Due to Adverse Events |
— | — |
| PRIMARY Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events |
2; 0 | — |
| PRIMARY Number of Participants Who Died Due to Adverse Events |
— | — |
| PRIMARY Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Surgery |
6; 0; 0; 0 | — |
| PRIMARY Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Bleeding |
28; 3; 0; 0 | — |
| PRIMARY Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Surgery |
— | — |
| PRIMARY Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Bleeding |
— | — |
| PRIMARY Number of Infusions Required to Treat Each New Bleeding Episode |
1.22 | — |
| PRIMARY Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Surgery |
2619.29 | — |
| PRIMARY Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Bleeding |
2140.08 | — |
| PRIMARY Percentage of Participants With Bleeding Event |
6.67 | — |
| PRIMARY Annualized Bleeding Rates (ABRs) |
13.33 | — |
| PRIMARY Number of Participants With Less Than Expected Therapeutic Effect (LETE): Prophylactic Therapy |
6 | — |
| PRIMARY Average Dose of Infusions Per Bleeding Event: Prophylactic Therapy |
1881.33 | — |
| PRIMARY Total Factor VIII Consumption |
106116.16 | — |
Eligibility Criteria
-. Inclusion criteria
To be eligible to enroll in this study, the study subjects will have to meet all the following inclusion criteria:
- Hemophilia A (congenital factor VIII deficiency) patients who have been administered according to the indication of the product 1) Control and prevention of bleeding episodes and for routine and surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency) 2) This drug does not contain von Willebrand factor and, therefore, is not indicated in von Willebrand's disease
- Those who have been administered the Xyntha Solofuse prefilled syringe at least once - Exclusion criteria
Patients who satisfy the following criteria are not included in the study according to the local labeling:
- Patients who have a history of hypersensitivity to the Xyntha Solofuse prefilled syringe or the ingredients of this drug.
- Patients who have a history of hypersensitivity to hamster proteins.
- Patients who have bleeding disorders other than hemophilia A.
- Patients who have a history of FVIII inhibitors, or currently have or are suspected of having FVIII inhibitors. In case inhibitor titers quantified in Bethesda Units in the laboratory test results are within the normal laboratory range or at least 0.6 BU/mL. If laboratory tests cannot be performed, the investigator will determine whether or not inhibitors exist based on the clinical assessment results that show a decrease in efficacy of the replacement of FVIII (e.g. bleeding at least once, if the replacement of anti-bleeding agents is needed to be administered, and if frequency or dosage of replacement FVIII therapy needs to be increased).
- Use of immunomodulatory therapy. (e.g. intravenous injection of immunoglobulin, use of regular systemic corticosteroids, cyclosporine, and mediators of anti-TNF-α)
Data sourced from ClinicalTrials.gov (NCT03034044). 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. Informational only — not medical advice.