Safety, Tolerability, PK, PD, and Efficacy of HMB-001 in Participants With Glanzmann Thrombasthenia
Research type
Research Study
Full title
A Phase 1/2, First-in-Human, Single and Multiple Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants With Glanzmann Thrombasthenia
IRAS ID
1006088
Contact name
Catherine Rea
Contact email
Sponsor organisation
Hemab Therapeutics ApS
Eudract number
2022-001583-10
ISRCTN Number
ISRCTN66310879
Research summary
Hemab Therapeutics ApS is funding this research to evaluate the ability of a new drug, HMB-001, to help patients with Glanzmann thrombasthenia. HMB-001 is a medicine intended to prevent and reduce bleeding events in patients with Glanzmann thrombasthenia.
Glanzmann thrombasthenia is a rare bleeding disorder, in which patients’ bodies cannot properly form blood clots to stop bleeding. This leads to frequent bleeding, with many patients experiencing a bleed every day. While there are drugs currently available to help stop bleeding once it has occurred, there are no drugs currently available to effectively prevent bleeding from occurring. This will be the first time HMB-001 will be given to humans, and the main goal of this trial is to look at the safety of the drug. In addition to determining how safe and well tolerated it is in humans, this clinical trial will also look at how long the drug will remain in the body (pharmacokinetics), how it effects the body (pharmacodynamics), and whether it works for preventing bleeding (efficacy).
This is a multicenter clinical trial conducted in the UK and can have up to 15 research sites. This trial plans to enrol a maximum of 57 male and female participants with Glanzmann thrombasthenia. The trial is comprised of two parts (Part A and Part B). Part A will include up to 21 participants and will evaluate a single dose of HMB-001. Part B will include up to 36 participants and will evaluate multiple doses of HMB- 001. If a participant is recruited into Part A, they may also be able to enter into Part B of the study once they have completed Part A. Participants will be in the trial for approximately one and half years.
REC name
London - London Bridge Research Ethics Committee
REC reference
22/LO/0615
Date of REC Opinion
3 Nov 2022
REC opinion
Further Information Favourable Opinion