The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of prophylactic SerpinPC administered subcutaneously (SC) to participants with severe hemophilia A (HemA) (with or without inhibitors) or moderately severe to severe hemophilia B (HemB) (without inhibitors) as part of the SerpinPC registrational program. This study consists of 3 parts: Part 1: dose-justification phase, Part 2: dose-confirmatory phase, Part 3: extension phase for participants who complete either Part 1 or Part 2. This adaptive design study has a randomized dose-justification component to investigate the efficacy and safety of SerpinPC as a therapeutic option, principally for participants with HemB without inhibitors. SerpinPC has a novel mechanism of action compared with marketed treatments and those that are in development.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
60
Administered as SC injection.
University of Colorado School of Medicine
Aurora, Colorado, United States
University of South Florida
Tampa, Florida, United States
Indiana Hemophilia and Thrombosis Center, Inc.
Indianapolis, Indiana, United States
University of Iowa Healthcare
Iowa City, Iowa, United States
University of Michigan
Ann Arbor, Michigan, United States
Annualized Bleeding Rate (ABR) for Treated Bleeds up to Week 24
Time frame: Up to Week 24
Annualized Bleeding Rate (ABR) for Treated Bleeds Up to Week 48
Time frame: Up to Week 48
Annualized Bleeding Rate (ABR) for Treated Spontaneous Bleeds
Time frame: Up to Week 48
Annualized Bleeding Rate (ABR) for Treated Spontaneous Joint Bleeds
Time frame: Up to Week 48
Total Coagulation Factor and/or Bypass Product Consumption During Parts 2 and 3
Time frame: Up to Week 48
Pharmacokinetic Plasma Concentrations of SerpinPC
Time frame: From Day 1 up to 24 weeks
Haemophilia-specific QoL Instrument for Adults (Haem-A-QoL) Physical Health scale in participants aged 17 to ≤65 years with hemophilia
The Haem-A-QoL instrument contains 44 items across 10 domains relevant to HRQoL in adults (physical health, feelings, view of participant's self, sports and leisure, work and school, dealing with hemophilia, treatment, future, family planning, partnership, and sexuality). Each item is rated on a 5-point scale (1=never, 2=rarely, 3=sometimes, 4=often, 5=all the time). Higher scores are indicative of greater impairment in HRQoL.
Time frame: From Baseline up to 24 weeks
Number of Participants With Adverse Events (AEs)
Time frame: From Baseline up to Week 48
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
University of Minnesota Medical Center
Minneapolis, Minnesota, United States
East Carolina Univeristy
Greenville, North Carolina, United States
Penn State Hershey Medical Center
Hershey, Pennsylvania, United States
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, United States
University of Texas Health Science Center at Houston-Gulf States HTC
Houston, Texas, United States
...and 49 more locations