CSEG101A2301 (STAND)

A phase III, multicenter, randomized, double-blind study to assess efficacy and safety of two doses of Crizanlizumab versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult sickle cell disease patients with vaso-occlusive crises (STAND)

Study treatments

• arm 1: Crizanlizumab (SEG101) at 5.0 mg/kg 
• arm 2: Crizanlizumab (SEG101) at 7.5 mg/kg 
• arm 3: placebo 

Inclusion criteria

• Patient should be at least 12 years on the day of signing informed consent
• Confirmed diagnosis of sickle cell disease by HPLC of hemoglobin electrophoresis
• Patient experienced at least 2 VOCs that lead to a healthcare visit within 12 months prior to screening visit. Prior VOC leading to healthcare visit must include:

            - Pain crisis defined as an acuteonset of pain for which there is no other medically determined                 explanation other than vaso-occlusion

            - A visit to a medical facility and/or healthcare professional

            - And receipt of oral/parenteral opioids or parenteral nonsteroidal anti-inflammatory drug                       (NSAID) analgesia.

• If the patient is receiving Hu/HC or L-glutamine or erythropoieting stimulating agent, must have been receiving the drug for at least 6 months prior to screening visit and plan to continue taking at the same dose and schedule until the subject had reached one year of study treatment. Patients who have not been reveiving such drug must not have received it for at least 6 months prior to screening visit to be included

Exclusion criteria

• Received blood products within 30 days of week 1 day 1 dosing.
• Participating in a chronic transfusion program (pre-planned series of transfusions for prophylactic purposes) and/or planning on undergoing an exchange transfusion and/or plasmaferesis during the duration of the study; episodic transfusion in response to worsened anemia or VOC is permitted.
• Use of therapeutic anticoagulation is or antiplatelet therapy (other than aspirin or NSAIDs) within the 10 days prior to week 1 day 1 dosing. Note: prophylactic anticoagulant dose is permitted as per local guidelines.

Participating sites:

• Hôpital Erasme Brussels
• Universitair Ziekenhuis Antwerpen (UZA)