A first-in-human study of HMB-001 in patients with Glanzmann thrombasthenia
- Conditions
- Glanzmann thrombastheniaNot Applicable
- Registration Number
- ISRCTN66310879
- Lead Sponsor
- Richmond Pharmacology Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Ongoing
- Sex
- All
- Target Recruitment
- 57
1. Male or female, aged 18 to 65 years, inclusive, at the time of signing informed consent
2. Glanzmann thrombocythemia:
2.1. Documented abnormal, diagnostic platelet aggregometry plus deficiency of the aIIbß3 (GPIIb/GPIIIa) receptor via flow cytometry
2.2. Genetic diagnosis
3. Has not received a COVID-19 vaccine dose in the last 28 days. Has not received any live vaccine within 4 weeks of enrollment and is not planning to have a live vaccine during the study period.
4. Agrees to not receive COVID-19 vaccination throughout the dosing period and for 4 weeks after the final dose.
5. Has the ability to provide written, personally signed and dated informed consent to participate in the trial, in accordance with the International Council for Harmonisation (ICH) Good Clinical Practice (GCP) Guideline E6 (R2) (2016) and applicable regulations, before completing any trial-related procedures.
6. Has an understanding, ability, and willingness to fully comply with trial procedures and restrictions.
7. Women of child-bearing potential have a negative serum pregnancy test within 72 hours prior to the first dose of HMB-001.
8. Women of child-bearing potential agree to use highly effective contraceptive methods (excluding estrogen containing combined oral contraceptive pill as per exclusion criteria) and avoid egg donation for 14 days prior to Day 1, during the study treatment, and for 6 months after the last dose of HMB-001. A woman is considered to be of child-bearing potential unless she:
8.1. Has had a hysterectomy, bilateral tubal ligation, or bilateral oophorectomy;
8.2. Is aged 50 years old and over and has been amenorrhoeic for = 12 months (including no irregular menses or spotting) in the absence of any medication which induces a menopausal state and has documented ovarian failure by serum estradiol and follicle-stimulating hormone levels within the institutional laboratory postmenopausal range).
9. Men of child-producing potential agree to use highly effective contraceptive methods and avoid sperm donation for 14 days prior to Day 1, during the study treatment, and for 6 months after the last dose of HMB-001. A man is considered to be of child-producing potential unless he has had a bilateral vasectomy with documented aspermia or a bilateral orchiectomy.
1. Severe infection or inflammation at the time of Screening
2. History of clinically significant hypersensitivity associated with monoclonal antibody therapies
3. Personal history of venous or arterial thrombosis or thromboembolic disease
4. Known severe congenital or acquired thrombophilia
5. Has vital signs outside of the following normal range at Screening:
5.1 Supine heart rate <100 bpm (after at least 5 minutes of supine rest)
5.2. Blood pressure (BP): Supine BP (after at least 5 minutes of supine rest):
5.3. Systolic blood pressure: 90 - 140 mmHg
5.4. Diastolic blood pressure: 40 - 90 mmHg
6. Has a positive test for Hepatitis B surface antigen (HBsAg), Hepatitis C antibody (HCV Ab), or human immunodeficiency virus antibody (HIV Ab) at Screening
7. Is currently positive for COVID-19 based on lateral flow
8. Is within 6 weeks of COVID-19 infection
9. Other conditions that substantially increase risk of thrombosis by the discretion of the investigator including, but not limited to: significant family history, BMI >30 kg/m2 (moderately obese, adjusted for ethnicity), reduced mobility, active malignancy, major surgery within 6 weeks preceding first dose of study drug, post-partum within 12 weeks preceding first dose of study drug
10. Women who are using estrogen-containing medication or hormone modulators (within 8 weeks pre dose to 8 weeks post dose of study drug) including:
10.1. Combined oral contraception pill
10.2. Hormone replacement therapy (excluding transdermal patches)
10.3. Oestrogen receptor modulators (eg, Tamoxifen)
10.4. Gonadotropin releasing hormone receptor (GnRH receptor) agonist
11. Clinically significant cardiovascular disease including, but not limited to: New York Heart Association Class III or IV heart failure, coronary artery disease, uncontrolled arrythmia, moderate to severe valvular heart disease, peripheral vascular disease, and ischaemic stroke.
12. Other conditions that substantially increase risk of cardiovascular events by the discretion of the Investigator including, but not limited to: smoking, cocaine use, and uncontrolled hypertension.
13. Congenital or acquired bleeding disorders other than Glanzmann thrombocythemia.
14. Concurrent disease, treatment, medication, or abnormality in clinical laboratory tests that may pose additional risk in the opinion of the investigator and preclude the participant’s safe participation in and completion of the study.
15. Addiction or other diseases that prevent the participant from appropriately assessing the nature and scope of the clinical study or participating in study procedures by the discretion of the Investigator.
16. Received investigational medication in another clinical study within 5 half-lives before administration of the study drug.
17. Female participants who are pregnant (including a positive serum pregnancy test at Screening) or breastfeeding.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method