Part B 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

  235287

• 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 Ltd. This study is split into two parts; part A and B.

  The purpose of Part B is to evaluate the safety, tolerability and pharmacokinetic parameters and pharmacodynamic parameters in patients with primary hyperoxaluria. This means we’ll be looking at how the body handles the drug and also what effect the drug has on the body, and whether it has the desired effect. The levels of oxalate, glyoxylate and glycolate (and other biochemical markers) will be measured to see if they change during the study.
  There will be up to 4 cohorts in part B of the study. The dose will be increased incrementally in each cohort, but the progression to the next cohort will only proceed after a safety review committee has reviewed available safety data. The dose for this part of the study will be decided based on the results from part A.
  For part B of the study up to 16 patients are to be enrolled if the optional 4th cohort goes ahead. Some of the most important criteria are:
  - Male or female subjects at least 6 years old at the time of consent
  - Patients diagnosed with primary hyperoxaluria 1 or primary hyperoxaluria 2.
  -Patient has never had a kidney or live transplant

  Part B: This part of the study will consist of a screening visit, 2 inpatient visits and 4 outpatient visits. The first inpatient visit will be from Day 0 to Day 3 (A total of 3 nights), the next inpatient visit will be from Day 57 to 58. At Day 58 subjects will be discharged from the study provided their levels of oxalate and glycolate have returned to at least 80% of the original value. In addition, 4 outpatient visits will be conducted during the study on Day 8, Day 15, Day 22 and Day 43 respectively.

• REC name

  Wales REC 1

• REC reference

  17/WA/0332

• Date of REC Opinion

  23 Nov 2017

• REC opinion

  Further Information Favourable Opinion