Beta-thalassemia major Hemophilia A/B Sickle cell disease
Conditions
Interventions
None listed
Sponsors
Centre for Human Drug Research
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: All subjects must be able to participate and be willing to give written informed consent and to comply with the study restrictions. In addition, eligible subjects must meet the following inclusion criteria:;Healthy volunteers 1. Healthy male or female subjects aged between 18 and 75 years (inclusive); 2. Body mass index between 18-32 kg*m-2 (inclusive). Patients with target INR of 2.0 * 3.0 1. Male and female subjects aged between 18 and 75 years (inclusive), with stable (for at least a month) INR between 2.0 * 3.0; 2. Body mass index between 18-32 kg*m-2 (inclusive). Patients with target INR *3.0 1. Male and female subjects aged between 18 and 75 years (inclusive), with a stable INR*3.0; 2. Body mass index between 18-32 kg*m-2 (inclusive). Sickle cell disease patients 1. Male and female SCD patients aged between 18 and 75 years (inclusive); 2. Moderate to severe stable SCD (HbSS HbSC or HBS-* thalassemia), with stable disease defined as no significant complications such as VOC, acute chest syndrome or any complication requiring in-patient hospitalization for at least one month prior to the baseline visit, and/or no acute transfusions for at least 2 months prior to the baseline visit. 3. Body mass index between 18-32 kg*m-2 (inclusive). Haemophilia (hemophilia) patients 1. Haemophilia (hemophilia) patients aged between 18 and 75 years (inclusive); 2. Moderate to severe stable haemophilia (hemophilia) A or B, with factor activity of
Exclusion criteria
Exclusion criteria: Eligible subjects must meet none of the following exclusion criteria at baseline:;Healthy volunteers 1. History or symptoms of any significant disease including (but not limited to), neurological, psychiatric, endocrine, cardiovascular, respiratory, gastrointestinal, hepatic, or renal disorder; 2. Systolic blood pressure (SBP) greater than 140 or less than 90 mm/Hg, and diastolic blood pressure (DBP) greater than 90 or less than 50 mm/Hg; 3. Concomitant disease or condition that could interfere with the conduct of the study or the study objectives, or that would, in the opinion of the Investigator, pose an unacceptable risk to the study participant; 4. The use of any medication or vitamin/mineral/herbal/dietary supplement within less than 5 half-lives prior to study participation is prohibited, if the Investigator judges that it may interfere with the study objectives; 5. Condition of the skin that prohibits accurate mDLS or LSCI measurements, such as large tattoos, skin ulcers, scar tissue etc; 6. Unwillingness or inability to comply with the study procedures for any other reason. Patient populations 1. Any condition that in the opinion of the investigators could potentially jeopardize the health status of the patient; 2. The use of any medication other than required for patients standard treatment, within less than 5 half-lives prior to study participation is prohibited if the Investigator judges that it may interfere with the study objectives; 3. Condition of the skin that prohibits accurate mDLS or LSCI measurements, such as large tattoos, skin ulcers, scar tissue etc; 4. Unwillingness or inability to comply with the study procedures for any other reason.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| mDLS measures 1. Directly measured a. Pulse rate b. Deep respiratory rate c. Blood flow d. Blood flow velocity e. Traube-Hering waves 2. Indirectly measured a. Global coagulation status b. Rheological characteristics of blood c. Endothelial function d. Relative cardiac output e. Vascular health LSCI measures 1. Basal blood flow 2. Blood flow upon occlusion-reperfusion Laboratory measures 1. Iron, TIBC and ferritin panel 2. Bilirubin (total and fractionated) 3. Lactate dehydrogenase 4. Prothrombin time (INR)/Activated Partial Thromboplastin Time 5. Fibrinogen 6. Complete blood count with differential 7. Erythrocyte Sedimentation rate (ESR) 8. GDF 11/15 9. Endothelin 1 10. Red cell membrane PS/PE (Phosphatidylserine/phosphatidylethanolamine) 11. Haptoglobin 12. Hepcidin Collected data or samples may also be used to derive other measures, when considered to be in line with the objectives of the research protocol. | — |
Countries
Netherlands
Outcome results
None listed