Sickle cell disease
Conditions
Brief summary
Safety of AG-348 including the type, incidence, severity and relationship of AG-348 to AE and SAE; number of medication discontinuations due to AE; physical examination findings, vital signs and 12-lead electrocardiogram (ECG) data., Efficacy of treatment of AG-348 as evaluated by changes in sickling behaviour (Point of Sickling) as quantified by the Oxygenscan (= primary main study parameter)., Efficacy of treatment of AG-348 evaluated by percentage of responders defined as: o Anti-sickling response o Hematological response
Detailed description
Maximum change in Hb, bilirubin, HbCO, p50 (TCS HEMOX Analyzer), 2,3-DPG and ATP levels., Maximum change in RBC deformability, dehydration and cell membrane stability, ex vivo (Osmotic gradient ektacytometry (Osmoscan)., Changes in clinical characteristics: o Health related Quality of Life (HRQoL) (EQ-5D-5L, SF-36). o Movement behaviour (accelerometer Activ8) o Dyspnea (MRC dyspnea) o Fatigue (PROMIS fatigue short form), Changes in surrogate markers of organ damage or biomarkers associated with mortality at D56 compared to baseline: o Urinary albumin to creatinine ratio o NT-proBNP o CRP o LDH/HbCO ratio o Endothelial activation: D-dimer, von Willebrand factor-antigen (VWF-Ag), soluble vascular cell adhesion molecule-1 (sVCAM-1) o Change in number of crises compared to historical rate
Interventions
DRUGMITAPIVAT
Sponsors
Julius Clinical Research B.V.
Eligibility
Sex/Gender
All
Age
0 Years to 64 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety of AG-348 including the type, incidence, severity and relationship of AG-348 to AE and SAE; number of medication discontinuations due to AE; physical examination findings, vital signs and 12-lead electrocardiogram (ECG) data., Efficacy of treatment of AG-348 as evaluated by changes in sickling behaviour (Point of Sickling) as quantified by the Oxygenscan (= primary main study parameter)., Efficacy of treatment of AG-348 evaluated by percentage of responders defined as: o Anti-sickling response o Hematological response | — |
Secondary
| Measure | Time frame |
|---|---|
| Maximum change in Hb, bilirubin, HbCO, p50 (TCS HEMOX Analyzer), 2,3-DPG and ATP levels., Maximum change in RBC deformability, dehydration and cell membrane stability, ex vivo (Osmotic gradient ektacytometry (Osmoscan)., Changes in clinical characteristics: o Health related Quality of Life (HRQoL) (EQ-5D-5L, SF-36). o Movement behaviour (accelerometer Activ8) o Dyspnea (MRC dyspnea) o Fatigue (PROMIS fatigue short form), Changes in surrogate markers of organ damage or biomarkers associated with mortality at D56 compared to baseline: o Urinary albumin to creatinine ratio o NT-proBNP o CRP o LDH/HbCO ratio o Endothelial activation: D-dimer, von Willebrand factor-antigen (VWF-Ag), soluble vascular cell adhesion molecule-1 (sVCAM-1) o Change in number of crises compared to historical rate | — |
Countries
Netherlands
Outcome results
None listed