Part A of a 2-part study of DCR-PHXC in NHV and PH patients

• Research type

  Research Study

• Full title

  A Placebo-Controlled, Single-Blind, Single-Center Phase 1 Study in Normal Healthy Volunteers and Open-Label Multi-Center Study in Patients with Primary Hyperoxaluria to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of DCR-PHXC Solution for Injection (subcutaneous use)

• IRAS ID

  235286

• Contact name

  Annelize Koch

• Contact email

  annelize.koch@simbecorion.com

• Sponsor organisation

  Dicerna Pharmaceuticals Inc

• Eudract number

  2017-003534-89

• Duration of Study in the UK

  0 years, 6 months, 2 days

• Research summary

  This study is being funded by Dicerna Pharmaceuticals Inc. This study is split into two parts; part A and B. This summary relates only to Part A of the study.

  The purpose of Part A is to evaluate the safety, tolerability and pharmacokinetic parameters in healthy volunteers. There will be up to 5 cohorts in part A of the study. The dose will be increased incrementally in each cohort, but the progressive to the next cohort will only proceed after a safety review committee has reviewed available safety data. From this part of the study the dose for Part B of the study will be confirmed.

  For part A of the study up to 25 subjects are to be enrolled if the optional 5th cohort goes ahead. Some of the most important criteria are:
  - Male or female subjects aged between 18 and 55 and are considered to be healthy
  - Subjects are not taking any medication
  - Subjects are non-smokers or ex-smokers who haven't smoked within 1 months prior to first dose
  - No history of kidney stones

  Part A: This part of the study will consist of a screening visit, 3 inpatient visits and 2 outpatient visits. The first inpatient visit will be from Day -1 to Day 3 (A total of 4 nights), the next inpatient visit will be from Day 14 to Day 16, and the final inpatient visit will take place between Day 28 and Day 30. At Day 30 subjects will be discharged from the study. In addition, 2 outpatients visits will be conducted on Day 8 and Day 22 respectively.

• REC name

  Wales REC 1

• REC reference

  17/WA/0331

• Date of REC Opinion

  23 Nov 2017

• REC opinion

  Further Information Favourable Opinion