Haematological Disorders
Conditions
Interventions
Placebo
Sponsors
Novo Nordisk
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Key inclusion criteria: ? Male or female. ? Age 12 years or above at the time of signing the informed consent. ? Confirmed diagnosis of sickle cell disease o Documentation of SCD genotype (HbSS, HbSß0-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing or screening test results from central laboratory. Molecular genotyping is not required. SCD genotype may be determined from the results of haemoglobin (Hb) electrophoresis, highperformance liquid chromatography (HPLC) or similar testing. Note that Hb electrophoresis is performed by the central laboratory at screening. ? Have 1-15 episodes of documented VOC (defined in protocol section 8.2.2) within the 12 months prior to screening. Documentation must exist in the participant’s medical record prior to randomisation. Events based solely on participant recall without supporting documentation should not be counted towards eligibility. ? Hb =5.0 and =10.0 g/dL (=50 and =100 g/L) at screening
Exclusion criteria
Exclusion criteria: ? More than 15 VOCs within the past 12 months prior to screening documented in the participant’s medical record. Events based solely on participant recall without supporting documentation should not be counted towards eligibility. ? Use of voxelotor or similar agent within 28 days prior to starting study treatment or anticipated need for this agent during the study. ? Use of a selectin antagonist (e.g., crizanlizumab, monoclonal antibody or small molecule) within 28 days or 5 half-lives (whichever is longer) prior to starting study treatment or anticipated need for such agents during the study. ? Receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or =6 transfusion events in the previous 12 months (i.e., an average of 1 transfusion event every 60 days). ? Participants who have received an RBC transfusion for any reason within 60 days of the screening period or 60 days of the randomisation day are only eligible if HbA (adult Hb) 4.0 × upper limit of normal (ULN) or o Direct bilirubin >3.0 × ULN.Participants who are not taking or are unable to take antimalarial prophylaxis at the time of consent and during the study if they live in areas of endemic malaria where prophylaxis is recommended.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Number of adjudicated VOC events with a medical contact 2. Time to onset of first adjudicated VOC 3. Change in distance travelled during the 6-minute walking test (6MWT) 4. Change in standardised T-score on the PROMIS Fatigue 7a Scale | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Change in haemoglobin (Hb) 2. Change in Hb >1 g/dL 3. Change in lactate dehydrogenase (LDH) 4. Change in absolute reticulocyte count 5. Change in indirect 6. Changes in estimated glomerular filtration rate (eGFR) 7. Change in albumin:creatinine ratio (ACR) 8. Occurrence of moderate/severe albuminuria (yes/no) 9. Change in N-terminal pro b-type natriuretic peptide (NT-pro-BNP) 10. Proportion of participants achieving the threshold for clinically meaningful change (yes/no) in 6MWT 11. Proportion of participants achieving the threshold for clinically meaningful change (yes/no) in PROMIS fatigue scale 7a | — |
Countries
Ghana, Kenya, Nigeria, Uganda
Contacts
Public ContactMichell Botha
Assc Dr Global site Activation
Outcome results
None listed