Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03370172 | A Study of BAX 888 in Male Adults With Severe Hemophilia A | PHASE1 | COMPLETED | 4 | — | — | Feb 27, 2018 | Jul 9, 2024 | Sep 11, 2025 | 26 | United States, Austria +4 |
An AE is defined as any untoward medical occurrence in a participant administered an investigational product (IP) that does not necessarily have a causal relationship with the treatment. A Serious adverse event (SAE) is an AE resulting in any of the following outcomes: death; life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. AEs include both serious and non-serious adverse events including development of FVIII inhibitory antibodies, clinically significant changes in standard laboratory parameters, physical exam, and vital signs.
| Arm | Type | Description |
|---|---|---|
| Cohort 1: BAX 888 2.0*10^12 cp/kg | EXPERIMENTAL | Cohort 1 participants received a single peripheral intravenous (IV) infusion of BAX 888 at a dose of 2.0\*10\^12 capsid particles per kilogram (cp/kg) on the day of dosing (Day 0). |
| Cohort 2: BAX 888 6.0*10^12 cp/kg | EXPERIMENTAL | Cohort 2 participants received a single peripheral IV infusion of BAX 888 at a dose of 6.0\*10\^12 cp/kg on the day of dosing (Day 0). |
| Name | Type | Description |
|---|---|---|
| BAX 888 | DRUG | Participants will receive a single peripheral IV infusion of BAX 888 in Cohort 1 and 2 Day 0. |
Inclusion Criteria: * Male, aged 18 to 75 years at the time of screening. * Established severe hemophilia A (FVIII:C \<1%, measured following \>=5 days without FVIII treatment) and/or documented intron 1 inversion or intron 22 inversion mutation in the F8 gene, consistent with severe hemophilia A ,...