A Study to Evaluate Cinacalcet Use and Safety in Children with sHPT

  • Research type

    Research Study

  • Full title

    An Observational Registry Study To Evaluate The Use And Safety Of Cinacalcet Among Paediatric Patients With Secondary Hyperparathyroidism

  • IRAS ID

    333878

  • Contact name

    Ben Reynolds

  • Contact email

    ben.reynolds@ggc.scot.nhs.uk

  • Sponsor organisation

    Amgen Ltd

  • Clinicaltrials.gov Identifier

    EUPAS24954, EU Post Authorisation Study (PAS) Register No

  • Duration of Study in the UK

    0 years, 4 months, 9 days

  • Research summary

    Secondary hyperparathyroidism (sHPT) is a condition in which small pea-sized glands, located in the neck, become enlarged and hyperactive) in both children and adults with chronic kidney disease and is a complication of end-stage kidney disease requiring dialysis (the process of removing excess water, solutes, and toxins from the blood in people whose kidneys can no longer perform these functions naturally).

    sHPT is characterised by persistently higher levels of parathyroid hormone ([PTH] (a hormone secreted by the parathyroid glands that controls the serum calcium concentration through its effects on bone, kidney, and intestines) concentrations in serum or plasma, and it represents a response that primarily maintains calcium homeostasis (maintaining a blood calcium level critical for normal body functions) as kidney function declines and prevents debilitating skeletal complications and helps achieve normal growth in children. The goal of treatment for sHPT in children is to achieve optimal control of PTH concentrations but there is also an aim to improving their growth and final height. Cinacalcet is a calcimimetic agent that mimics what calcium is doing in the body and was approved in Europe for children undergoing maintenance dialysis. Clinical Practice Guidelines now include calcimimetics as a therapeutic option for managing sHPT. However following approval of paediatric cinacalcet for treatment of sHPT in Europe, there is a lack of data on real-world use. There are uncertainties about the incidence of, risk factors for, and management of hypocalcaemia (low blood calcium levels) among children in routine clinical practice.

    As part of the agreement with the European Medicines Agency (EMA) following marketing authorisation, an observational registry is being conducted to describe real-world use of cinacalcet and to describe the incidence of, risk factors for, and management of hypocalcaemia in a group of paediatric sHPT patients receiving maintenance dialysis.

  • REC name

    North West - Haydock Research Ethics Committee

  • REC reference

    23/NW/0328

  • Date of REC Opinion

    20 Feb 2024

  • REC opinion

    Further Information Favourable Opinion