An Open Label, Observational, Prospective Registry of Participants With Sickle Cell Disease (SCD) Treated With Oxbryta® (Voxelotor)® (Voxelotor)

Study type

• Study topic:
  Disease /health condition
• Study topic, other:
  Observational study designed to evaluate the effect of Oxbryta® (Voxelotor) in individuals with SCD.
• Study type:
  Non-interventional study
• Scope of the study:
  Other
• If ‘other’, further details on the scope of the study:
  Register observational
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Any participant who is currently taking Oxbryta® (Voxelotor) or has been prescribed and will initiate treatment with Oxbryta® (Voxelotor), is eligible to participate. Eligible participants will receive treatment with Oxbryta® (Voxelotor) as prescribed by their physician, as part of their usual care. Participants will be treated and evaluated per SOC and at the physician’s discretion.

Study drug and medical condition

• Name of medicine:
  OXBRYTA
• Study drug International non-proprietary name (INN) or common name:
  VOXELOTOR
• Anatomical Therapeutic Chemical (ATC) code:
  (B06AX03) voxelotor

  voxelotor
• Medical condition to be studied:
  Sickle cell disease

Population studied

• Short description of the study population:
  Participants who meet all the following criteria will be eligible for enrollment:; 1. Willing and able to provide written informed consent (aged ≥ 18 years), parental/guardian consent and participant assent (aged ≥ 12 to <18 years) per local regulations, or pediatric participants (aged 4 to <12 years) with parental/guardian consent per Institutional Review Board (IRB) policy and requirements, consistent with ICH guidelines; 2. Male or female participants with documented diagnosis of sickle cell disease (all genotypes); 3. Undergoing treatment with Oxbryta® (Voxelotor) according to the Oxbryta® (Voxelotor) USPI
• Age groups:
  All

  Paediatric Population (< 18 years)

  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adult and elderly population (≥18 years)

  Adults (18 to < 65 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Elderly (≥ 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  500

Study design details

• Study design:
  Any participant who is currently taking Oxbryta® (Voxelotor) or has been prescribed and will initiate treatment with Oxbryta® (Voxelotor), is eligible to participate. Eligible participants will receive treatment with Oxbryta® (Voxelotor) as prescribed by their physician, as part of their usual care.
• Main study objective:
  The primary objective is to gather long term data on Oxbryta® (Voxelotor)® in a real-world setting. The following are categories of interest in participants with SCD treated with Oxbryta® (Voxelotor):; - Clinical outcomes, as assessed by clinical and laboratory assessments of hematological parameters and end organ damage, and rate of significant clinical events ; - Healthcare resource utilization Health-related quality of life (HRQoL), as assessed by participants, parents/caregivers, and clinicians ; - Assess the safety and tolerability of Oxbryta® (Voxelotor)®

Documents

Study report

C5341019 PROSPECT GBT440-4R2 Final NI Clinical Study Report_Redacted.pdf