A Phase 2 study of NGM282 in Patients with PSC

• Research type

  Research Study

• Full title

  A Phase 2, Randomized, Double Blind, Placebo Controlled, Parallel Group, Multiple Center Study to Evaluate the Safety, Tolerability, and Efficacy of NGM282 Administered for 12 Weeks in Patients with Primary Sclerosing Cholangitis (PSC)

• IRAS ID

  195296

• Contact name

  Gideon Hirschfield

• Contact email

  g.hirschfield@bham.ac.uk

• Sponsor organisation

  NGM Biopharmaceuticals, Inc.

• Eudract number

  2015-003392-30

• Duration of Study in the UK

  2 years, 4 months, 29 days

• Research summary

  Primary sclerosing cholangitis (PSC) is a chronic liver disease which causes inflammation and accumulation of scar tissue in the bile ducts which leads to bile not being able to flow through them, advanced fibrosis, liver cirrhosis (in which healthy liver tissue is replaced with scar tissue, eventually preventing the liver from functioning properly) and eventually liver failure. Currently, the primary therapy for PSC is aimed at bile-ducts (a number of long tube-like structures that carry bile) and targeted at reducing or halting progression of the disease by opening up the bile ducts.

  Treatments that have been tested in PSC have not demonstrated a significant clinical benefit and liver transplantation remains the only clinically proven treatment. Therefore, a significant unmet medical need still exists for novel agents for the treatment of PSC and its subsequent complications.

  This study is using NGM282 which acts by inhibiting the production of toxic bile acids (linked to causing PSC). Studies conducted so far with NGM282 are supportive of the clinical development of NGM282 in patients with PSC.

  Approximately 60 participants will take part in this study and will be in the study for 22 weeks.
  Participants who provide written consent and meet all eligibility criteria will be randomly assigned in a 1:1:1 ratio to one of the three treatment arms (NGM282 1 mg, NGM282 3 mg, or placebo).

  The study consists of:
  Screening period of up to 6 weeks: To see if the participant is suitable.
  Treatment period of 12 weeks with participants returning for visits on week 1, 2, 4, 8 and 12.
  Follow up period of 4 weeks

  The study involves procedures including: Physical exams, 12 lead ECG’s, Blood, stool and urine samples, Colonoscopy and MRCP (magnetic resonance cholangiopancreatography- imaging to visualise the bile ducts in a non-invasive manner).

• REC name

  East Midlands - Nottingham 2 Research Ethics Committee

• REC reference

  16/EM/0030

• Date of REC Opinion

  18 Apr 2016

• REC opinion

  Further Information Favourable Opinion