CSL222_3004
This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately severe hemophilia B.
- Investigator
- Tung T Wynn
- Status
- Accepting Candidates
- Ages
- 138 Months - 206 Months
- Sexes
- Male
WP45338
This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been diagnosed with Type 3 von…
- Investigator
- Tung T Wynn
- Status
- Accepting Candidates
- Ages
- 1 Month - N/A
- Sexes
- All
R131L1265-HEMB-2318; BEYOND-9
Participants in this study have a genetic mutation, specifically in the coagulation (blood clotting) Factor 9 gene that causes severe or moderately severe hemophilia B. This study is researching an experimental gene insertion therapy (the adding of…
- Investigator
- Tung T Wynn
- Status
- Accepting Candidates
- Ages
- 2 Years - N/A
- Sexes
- Male