A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia
Running, not enrolling · Phase 1/Phase 2
Conditions studied: Glanzmann Thrombasthenia
In brief
The goal of this clinical trial is to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants with Glanzmann Thrombasthenia. The main questions it aims to answer are: * Parts A, B, and C: To determine the safety and tolerability of HMB-001 * Part A: To establish the dose level(s) and dosing interval(s) of HMB-001 to be investigated in Parts B and C * Parts B and C: To estimate the ability of HMB-001 to prevent the number and severity of bleeds Part A will assess differing singular doses of HMB-001 in small groups of participants. The dose administered to a newly enrolled participant (or groups of participants) may only increase if analysis of data from previous dosing shows it is safe to do so. The planned duration of participation in Part A is approximately 6 months, which consists of a Screening Period, an optional Run-in Observation Period, and a follow-up period of 8 weeks. Part B is similar to Part A as it involves testing different dose levels of HMB-001 in small groups of participants. However, in Part B, HMB-001 is given multiple times over a 3-month period, either weekly, every 2 weeks, or every 4 weeks. Part B consists of a Screening Period, a Run-in Observation Period, a 3-month Treatment Period, and a Safety Follow-up following the last dose of HMB-001. Part C is open to participants from Part B and consists of approximately a 18-month Treatment Period and a Safety Follow-up following the last dose of HMB-001.
Key facts
- Study ID
- NCT06211634
- Run by
- Hemab ApS
- People needed
- 57
- Starts
- 2022-12-13
- Expected to finish
- 2027-08-01
- Last updated by the study team
- 2025-12-24
Who can join
Age: 18 and older, up to 67. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of California, San Diego (UCSD) (Part B/C) — La Jolla, California, United States
- Tulane University Medical Center (Part B/C) — New Orleans, Louisiana, United States
- Mayo Clinic - Rochester (Part B/C) — Rochester, Minnesota, United States
- Hemophilia Center of Western Pennsylvania (HCWP) (Part B/C) — Pittsburgh, Pennsylvania, United States
- Washington Institute for Coagulation (Part B/C) — Seattle, Washington, United States
- University Hospital Leuven - Campus Gasthuisberg (Part B/C) — Leuven, Belgium
- AP-HP Hopital Bicetre (Part B/C) — Le Kremlin-Bicêtre, France
- AP-HM - Hopital de la Timone — Marseille, France
- AP-HP Hopital Necker-Enfants Malades (Part B/C) — Paris, France
- Azienda Ospedaliero-Universitaria Careggi (Part B/C) — Florence, Italy
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano (Part B/C) — Milan, Italy
- Universitair Medisch Centrum Utrecht (Part B/C) — Utrecht, Netherlands
- Queen Elizabeth Hospital Birmingham (Part B/C) — Birmingham, United Kingdom
- Leeds Teaching Hospitals NHS Trust — Leeds, United Kingdom
- Richmond Pharmacology Ltd (Part A/B/C) — London, United Kingdom
- Royal Free London NHS Foundation Trust (Part B/C) — London, United Kingdom
- The Royal London Hospital (Part B/C) — Whitechapel, United Kingdom
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.