This is a first-in-human (FIH), Phase 1/2, 3-part open-label, dose escalation, safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and efficacy study evaluating HMB-002 in participants with VWD. Part A of the study involves a single ascending dose (SAD) regimen 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. Part C will evaluate the safety, PK, and PD of a single concomitant dose of HMB-002 and factor concentrate with Type 3 VWD or Type 1 VWD with low residual VWF and FVIII who use factor concentrate as prophylaxis.
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.
HMB-002 will be administered as a single dose with a concomitant single dose of factor concentrate. The planned duration of study participants in Part C will be approximately 17 weeks.
Phoenix Children's Hospital
Phoenix, Arizona, United States
NOT_YET_RECRUITINGArkansas Children's Hospital
Little Rock, Arkansas, United States
NOT_YET_RECRUITINGChildren's Hospital of Los Angeles
Los Angeles, California, United States
NOT_YET_RECRUITINGUniversity of Miami Hospital and Clinics, Sylvester Comprehensive Cancer Center
Miami, Florida, United States
Incidence 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
Pharmacokinetic Parameter: Terminal elimination half-life (t1/2)
Time frame: Day 1 to Day 113
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Emory Children's Center
Atlanta, Georgia, United States
NOT_YET_RECRUITINGInnovative Hematology, Inc./Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, United States
RECRUITINGTulane University School of Medicine
New Orleans, Louisiana, United States
NOT_YET_RECRUITINGUniversity of Michigan Hospitals, Department of Hemophilia and Coagulation Disorders
Ann Arbor, Michigan, United States
NOT_YET_RECRUITINGMayo Clinic - Rochester
Rochester, Minnesota, United States
NOT_YET_RECRUITINGOregon Health & Science University
Portland, Oregon, United States
NOT_YET_RECRUITING...and 15 more locations