Phase 4
N=50
A Study of ADVATE in People With Hemophilia A in India
Hemophilia A
Bottom Line
View on ClinicalTrials.gov: NCT04985682 ↗Enrolled (actual)
50
Serious AEs
0.0%
Results posted
Mar 2024
Primary outcome: Primary: Number of Participants With Serious Adverse Events (SAE) at Least Possibly Related to ADVATE — 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- ADVATE (Biological)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Baxalta now part of Shire
- Primary completion
- Feb 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Serious Adverse Events (SAE) at Least Possibly Related to ADVATE |
— | — |
| SECONDARY Number of Participants With Non-serious Adverse Events (AEs) at Least Possibly Related to ADVATE |
— | — |
| SECONDARY Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters |
— | — |
| SECONDARY Total Annualized Bleeding Rate (ABR) With Prophylactic Treatment of ADVATE |
2.65 | — |
| SECONDARY ABR With Prophylactic Treatment of ADVATE Categorized Based on Location of Bleed |
2.33; 0.04; 0.16; 0.12 | — |
| SECONDARY ABR With Prophylactic Treatment of ADVATE Categorized Based on Type of Bleed |
1.86; 0.20; 0.59 | — |
| SECONDARY Total Number of ADVATE Infusions Required During Prophylactic Treatment |
65.8 | — |
| SECONDARY Average Number of ADVATE Infusions Required Per Week During Prophylactic Treatment |
2.5 | — |
| SECONDARY Average Number of ADVATE Infusions Required Per Month During Prophylactic Treatment of Bleeding Episode |
11.0 | — |
| SECONDARY Total Body Mass Adjusted Consumption of ADVATE During Prophylactic Treatment |
1739.7 | — |
| SECONDARY Average Body Mass Adjusted Consumption of ADVATE Per Week During Prophylactic Treatment |
67.1 | — |
| SECONDARY Average Body Mass Adjusted Consumption of ADVATE Per Month During Prophylactic Treatment |
291.7 | — |
| SECONDARY Overall Hemostatic Efficacy Rating of ADVATE for Treatment of Bleeding Episodes |
21; 26; 4; 0 | — |
| SECONDARY Number of ADVATE Infusions Required to Achieve Resolution of Bleeding Episodes |
1.2 | — |
| SECONDARY Total Body Mass Adjusted Consumption of ADVATE Per Bleeding Episode |
34.8 | — |
Summary
The main aim of this study is to learn more about side effects of Advate when given as standard treatment to people with hemophilia A who have already been treated.
The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study. Participants will need to visit the study doctor 5 times in total during the study. During these visits, study data will be collected by the study doctor.
Eligibility Criteria
Inclusion Criteria
- The participant or legally authorized representative (in case of study participants less than ( =) 6 years that has been previously treated with plasma-derived and/or recombinant FVIII concentrate(s) for a minimum of 150 exposure doses (EDs).
- Participant aged less than =200 cells per cubic millimeter (mm^3), as confirmed by central laboratory at screening.
- Participant is hepatitis C virus negative (HCV-) by antibody or polymerase chain reaction (PCR) testing (if positive, anti-body titer will be confirmed by PCR), as confirmed by central laboratory at screening; or hepatitis C virus positive (HCV+) with chronic stable hepatitis.
- Participant is willing and able to comply with the requirements of the protocol.
Exclusion Criteria
- Participant has known hypersensitivity to mouse or hamster proteins or to any of the excipients of FVIII (factor VIII) concentrates.
- Participant has been diagnosed with bleeding disorder(s) other than congenital hemophilia A, such as acquired hemophilia A, von Willebrand´s disease (VWD) or thrombocytopenia (platelet count =5 times upper limit of normal alanine aminotransferase [ALT], aspartate aminotransferase [AST] or international normalized ratio [INR] >1.5 as confirmed by central laboratory at screening).
- Participant has planned or is likely to have, surgery during the study period.
- Participant has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug or alcohol use that, in the opinion of the investigator, would affect participant's safety or compliance.
- Participant currently receiving or is scheduled to receive during the course of the study, an immunomodulating drug (example, corticosteroid agents at a dose equivalent to hydrocortisone >10 milligram per day, or α-interferon) other than antiretroviral chemotherapy.
- Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
- Participant is a family member or employee of the investigator.
Data sourced from ClinicalTrials.gov (NCT04985682). 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.