sickle cell disease (SCD)
Conditions
Brief summary
• Hb response rate at Week 24 (increase of > 1 g/dL [> 10 g/L] from baseline) during the blinded treatment period, • Annualized VOC rate during the 52-week blinded treatment period based on adjudicated VOC review
Detailed description
1. Change from baseline in Hb at Week 52 during the blinded treatment period, 2. Change in SCD-related clinical laboratory measurements from baseline at Week 24 during the blinded treatment period in: o Absolute reticulocyte count, o Indirect bilirubin, and o Lactate dehydrogenase (LDH), 3. Change from baseline in Patient-Reported Outcome Measurement Information System (PROMIS) Fatigue Scale in adult patients at Week 52 during the blinded treatment period, 4. Time to first VOC during the blinded treatment period
Interventions
Sponsors
Novo Nordisk A/S
Eligibility
Sex/Gender
All
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| • Hb response rate at Week 24 (increase of > 1 g/dL [> 10 g/L] from baseline) during the blinded treatment period, • Annualized VOC rate during the 52-week blinded treatment period based on adjudicated VOC review | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Change from baseline in Hb at Week 52 during the blinded treatment period, 2. Change in SCD-related clinical laboratory measurements from baseline at Week 24 during the blinded treatment period in: o Absolute reticulocyte count, o Indirect bilirubin, and o Lactate dehydrogenase (LDH), 3. Change from baseline in Patient-Reported Outcome Measurement Information System (PROMIS) Fatigue Scale in adult patients at Week 52 during the blinded treatment period, 4. Time to first VOC during the blinded treatment period | — |
Countries
France, Germany, Greece, Italy, Spain
Outcome results
None listed