Sickle Cell Disease Clinical Trials

∙ AGE

• Aged 2 to 17 years on the day of inclusion.

∙ TYPE OF PARTICIPANT AND DISEASE CHARACTERISTICS

• Participants who have a documented diagnosis of sickle cell disease (SCD) in their medical record.

∙ SEX, CONTRACEPTIVE/BARRIER METHOD AND PREGNANCY TESTING REQUIREMENTS

• A participant is eligible to participate if the participant is not pregnant or breastfeeding and one of the following conditions applies:

‣ Is of non-childbearing potential. To be considered of non-childbearing potential, a female must be pre-menarchal or surgically sterile. OR

⁃ Is of childbearing potential and agrees to use an effective contraceptive method or abstinence from at least 4 weeks prior to study intervention administration until at least 4 weeks after study intervention administration. A participant of childbearing potential must have a negative highly sensitive pregnancy test (urine or serum as required by local regulation) within 25 hours before the study intervention.

∙ INFORMED CONSENT

• Assent form has been signed and dated by the participant (based on local regulations), and, if applicable, informed consent form has been signed and dated by the parent(s) or another legally acceptable representative (LAR) and by an independent witness, if required by local regulations.

∙ OTHER INCLUSIONS

• Participant and parent(s)/LAR are able to attend all scheduled visits and to comply with all study procedures.