Paroxysmal Nocturnal Hemoglobinuria Clinical Trial
Official title:
A Phase 1 Dose-ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of BCX9930 in Healthy Subjects and in Subjects With Paroxysmal Nocturnal Hemoglobinuria
This is a 3-part Phase 1 dose-ranging study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single (Part 1) and multiple (Part 2) ascending doses of BCX9930 in healthy subjects and in subjects with paroxysmal nocturnal hemoglobinuria (PNH; Part 3). Pharmacokinetics is an analysis of how the body handles the study drug BCX9930 and pharmacodynamics is an analysis of the activity that the study drug BCX9930 may have in the body.
Up to 6 sequential ascending dose cohorts are planned to be dosed in a sequential manner in Part 1 of the study. Eight subjects will be treated with a single dose of the study drug per dose cohort (6 subjects per cohort will receive BCX9930 and 2 subjects per cohort will receive matching placebo). Escalation to the next higher dose level will occur only after completion of a review of clinical safety and pharmacokinetics by the Sponsor and PI. Up to 7 ascending, multiple dose cohorts will be enrolled in a sequential manner in Part 2 of the study. In Cohorts 1 through 3, twelve subjects will be treated with either a 7-day or 14-day course of study drug (10 subjects per cohort will receive BCX9930 and 2 subjects per cohort will receive matching placebo) administered orally. In Cohorts 4 through 7, twelve subjects will be treated with a 3-day course of study drug (10 subjects per cohort will receive BCX9930 and 2 subjects per cohort will receive matching placebo) administered orally. The daily dose may be split into 2 times daily (BID) or 3 times daily (TID) dosing for the multiple ascending dose part as needed. Escalation to the next higher dose level in Part 2 will occur only after completion of a review of clinical safety and pharmacokinetics by the Sponsor and PI. Part 3 of the study consists of up to 2 sequential ascending multiple dose cohorts of up to 8 subjects; each cohort may enroll up to 4 subjects with PNH who are naïve to both eculizumab and ravulizumab and up to 4 subjects with PNH who are currently being treated with either eculizumab or ravulizumab. In each cohort, subjects will receive one daily dose of BCX9930 on Days 1 to 14 and a higher daily dose on Days 15 to 28. Cohort 2 will start after independent data monitoring committee (DMC) review of Cohort 1 data and communication of their evaluation to Part 3 investigators. In South Africa, subjects that have clinical benefit from BCX9930 will be allowed to continue dosing for up to 48 weeks. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT04432584 -
A Study Evaluating The Safety, Pharmacokinetics, and Efficacy Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
|
Phase 3 | |
Completed |
NCT05828485 -
Effect of Food on Pharmacokinetics of MY008211A Tablets in Healthy Adult Subjects
|
Phase 1 | |
Recruiting |
NCT02179359 -
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
|
N/A | |
Active, not recruiting |
NCT04434092 -
A Phase III Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors.
|
Phase 3 | |
Terminated |
NCT05131204 -
Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
|
Phase 3 | |
Recruiting |
NCT01374360 -
Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
|
||
Active, not recruiting |
NCT05389449 -
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
|
Phase 3 | |
Recruiting |
NCT06100900 -
Dose Escalation of BCX10013 in Subjects With Paroxysmal Nocturnal Hemoglobinuria
|
Phase 1 | |
Completed |
NCT01272817 -
Nonmyeloablative Allogeneic Transplant
|
N/A | |
Completed |
NCT06326814 -
A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults
|
Phase 1 | |
Completed |
NCT04463056 -
Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH
|
Phase 3 | |
Recruiting |
NCT05476887 -
To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104
|
Phase 2 | |
Completed |
NCT01192399 -
Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
|
Phase 2 | |
Active, not recruiting |
NCT06051357 -
Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
|
Phase 2 | |
Recruiting |
NCT06154512 -
A Real-world, Multi-center, Prospective, Observational Study for PNH in China
|
||
Completed |
NCT04128943 -
Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
|
||
Active, not recruiting |
NCT03329365 -
Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS
|
||
Recruiting |
NCT05755867 -
Global PNH Patient Registry
|
||
Completed |
NCT04679103 -
A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation)
|
Phase 3 | |
Completed |
NCT05642585 -
A Study of Single-dose MY008211A in Healthy Adults
|
Phase 1 |