Inclisiran in adolescents with heterozygous familial hypercholesterolemia

• Research type

  Research Study

• Full title

  Two part (double-blind inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2]) randomized multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with heterozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-16)

• IRAS ID

  1003480

• Contact name

  Ian McDowell

• Contact email

  Mcdowell@cardiff.ac.uk

• Sponsor organisation

  Novartis Pharma AG

• Eudract number

  2020-002757-18

• Clinicaltrials.gov Identifier

  NCT04652726

• Research summary

  CKJX839C12301 (ORION-16) is a pivotal phase III study designed to evaluate safety,
  tolerability, and efficacy of inclisiran in 6 adolescents (aged 12 to <18 years) with
  heterozygous familial hypercholesterolemia (HeFH) and elevated LDL-cholesterol (LDLC)
  across 4 research sites. The use of inclisiran (as an addition to stable, optimal
  background lipid-lowering therapy) for the treatment of HeFH in adolescent patients
  who require additional lipid-lowering will be investigated in order to obtain needed
  pediatric information on inclisiran. The follow-up period (Part 2/Year 2) serves to collect
  longer-term data on inclisiran and also allows access of study participants to a
  potentially effective treatment.

• REC name

  North West - Haydock Research Ethics Committee

• REC reference

  20/NW/0459

• Date of REC Opinion

  22 Jan 2021

• REC opinion

  Further Information Favourable Opinion