The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with Hemophilia B with inhibitors, as part of the SerpinPC registrational program.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
3
Administered as SC injection.
University of Colorado School of Medicine
Aurora, Colorado, United States
University of South Florida
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 SerpinPC Treatment
Time frame: Up to Week 48
Pharmacokinetic Concentrations of SerpinPC
Time frame: From Day 1(Pre-dose) up to Week 48(Post-dose)
Haemophilia Quality-of-Life Questionnaire for Adults (Haem-A-QoL) Physical Health Scale
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 Week 48
Number of participants with Adverse events (AEs)
Time frame: From Baseline up to Week 52
Number of Participants with Persistent High-titer Antidrug Antibodies (ADAs)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Tampa, Florida, United States
East Carolina University
Greenville, North Carolina, United States
Centre of Haematology named after prof. R. O. Yeolian/ Hemophilia center
Yerevan, Armenia
Royal Prince Alfred Hospital
Camperdown, New South Wales, Australia
The Alfred Hospital
Melbourne, Victoria, Australia
Hopital Bicetre
Le Kremlin-Bicêtre, France
CHRU de Lille Centre de biologie et pathologie
Lille, France
Hospices Civils de Lyon (HCL) - Hopital Femme-Mere-Enfant (HFME)
Lyon, France
Hopital Necker - Enfants Malades
Paris, France
...and 10 more locations
Time frame: From Baseline up to Week 48
Number of Participants with Severity of Injection-site Reactions
Time frame: Baseline up to Week 44