This is a first-in-human (FIH), Phase 1/2, open-label, dose escalation, safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and efficacy study of HMB-002 in participants with VWD. Part A of the study involves a single ascending dose (SAD) design to establish safety, tolerability, PK, and PD effect. In Part B of the study, the safety and tolerability of repeat dosing will be established prior to cohort expansion to explore efficacy.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
108
HMB-002 will be administered subcutaneously. Part A will utilize sentinel dosing. The planned duration of study participants in Part A is approximately 12 weeks.
HMB-002 will be administered subcutaneously. Part B dosing intervals will be determined following evaluation of Part A results. The planned duration of study participants in Part B will be approximately 21 weeks.
Fiona Stanley Hospital
Murdoch, Perth, Australia
NOT_YET_RECRUITINGRoyal Prince Alfred Hospital
Camperdown, Sydney, Australia
RECRUITINGThe Alfred Hospital
Melbourne, Victoria, Australia
RECRUITINGRichmond Pharmacology
London, United Kingdom
RECRUITINGIncidence of Treatment emergent adverse events (TEAE)
Time frame: up to Day 113
Pharmacokinetic Parameter: Maximum observed plasma concentration (Cmax)
Time frame: Day 1 to Day 113
Pharmacokinetic Parameter: Area under the curve from time zero to last quantifiable concentration (AUClast)
Time frame: Day 1 to Day 113
Pharmacokinetic Parameter: Area under the curve from time zero to extrapolated infinite time (AUCinf)
Time frame: Day 1 to Day 113
Pharmacokinetic Parameter: Time to reach maximum observed plasma concentration (Tmax)
Time frame: Day 1 to Day 113
Pharmacodynamics Parameters: Assessment of VWF antigen (VWF:Ag)
Time frame: Day 1 to Day 113
Pharmacodynamics Parameters: Assessment of VWF activity
Time frame: Day 1 to Day 113
Pharmacodynamics Parameters: Assessment of FVIII activity
Time frame: Day 1 to Day 113
Annualized Bleeding Rate Assessments
Time frame: Day 1 to Day 113
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