Delta Dose Study

• Research type

  Research Study

• Full title

  A PHASE 1B/2A STUDY TO ASSESS THE TOLERABILITY AND ADVERSE EFFECT PROFILE OF CYSTEAMINE (CYSTAGON) WHEN USED WITH EPIGALLOCATECHIN-3-GALLATE (EGCG, EPINERVE) IN ADULTS AND CHILDREN WITH HOMOZYGOUS ΔF508 CYSTIC FIBROSIS

• IRAS ID

  245067

• Contact name

  Kevin Southern

• Contact email

  k.w.southern@liverpool.ac.uk

• Sponsor organisation

  University of Liverpool

• Eudract number

  2017-001259-29

• Duration of Study in the UK

  0 years, 9 months, 4 days

• Research summary

  This is an early phase study of the tolerability and adverse effect profile of combination dosing of cysteamine (Cystagon) and EGCG (epinerve) in patients with ∆F508 cystic fibrosis (CF).
  A combination of cysteamine and EGCG has been demonstrated to reduce sweat chloride concentrations in homozygous ∆F508 cystic fibrosis patients. However, the tolerability and adverse effects of this combination have not been established in adult or paediatric CF patients.
  This study will involve 20 adult and 20 paediatric patients (aged 5-17 years) who will take part in the study for 6 weeks. They will each receive 4 weeks of an escalating dose of cysteamine (which is how cysteamine dose is increased when used for other indications), along with a dose of EGCG. In the final week, the participants will continue on the final cysteamine dose, but have an increased dose of EGCG. Cysteamine is a licensed medication, and this study will use it at the licensed doses, but for an alterntive indication. EGCG is a food supplement available to purchase over the counter, and the same brand and dose that was used in the previous adult work done in Italy will be used.

  A subgroup of participants (paediatric and adult) will also provide PK samples. All patients will receive weekly blood tests for safety monitoring, and a weekly sweat test to ensure the previously established efficacy is replicated.

  The primary outcomes will be discontinuation of the study drugs, and the adverse effects of cysteamine and EGCG in the participants. Secondary outcomes are the plasma concentrations of cysteamine and EGCG, and the changes in sweat chloride in the participants

• REC name

  North West - Liverpool Central Research Ethics Committee

• REC reference

  18/NW/0500

• Date of REC Opinion

  28 Jan 2019

• REC opinion

  Further Information Favourable Opinion