Proof of Concept Study of ACH-0144471 in C3 Glomerulopathy (C3G)

• Research type

  Research Study

• Full title

  A Phase 2, Proof of Concept, Randomized, Double-Blinded, Placebo-Controlled Study of ACH-0144471 Treatment for 6 Months in Patients with C3 Glomerulopathy (C3G)

• IRAS ID

  235423

• Contact name

  Matthew Pickering

• Contact email

  matthew.pickering@imperial.ac.uk

• Sponsor organisation

  Achillion Pharmaceuticals, Inc.

• Eudract number

  2017-000663-33

• Clinicaltrials.gov Identifier

  NCT03369236

• Clinicaltrials.gov Identifier

  U1111-1203-9076, UTN

• Duration of Study in the UK

  1 years, 0 months, 30 days

• Research summary

  This study is being done to find out how safe and effective the study drug ACH-0144471 is at controlling the complement system, by inhibiting Factor D, in patients with C3 Glomerulopathy (C3G) who have not undergone a renal transplant.
  The complement system is found in the blood and helps or "complements" the ability of the body to get rid of many disease-causing organisms. It is part of the body’s defence system. Many diseases are associated with either an inefficient control of complement or too much activity of the complement system. Factor D is one of the proteins within the complement system and the sponsor (Achillion Pharmaceuticals) believes that blocking these proteins will help treat certain diseases, such as C3G, affecting the complement system.

  The study will consist of 4 parts:
  1. Screening – which consists of two visits within 75 days of the first dose of study drug. The second screening visit will include a renal biopsy.
  2. Treatment – Subjects who qualify, will be randomly assigned by chance (like the flip of a coin) to receive either ACH-0144471 or Placebo (dummy drug) and asked to take the study drug by mouth for 203 days (approximately 7 months), three times a day.
  3. Taper - Period during which the dose of study drug will be reduced over 6 days.
  4. Follow-up - Period where subjects will be followed until 4 weeks after the last dose of study drug

  Approximately 20 patients from UK, Canada and United States will be enrolled.

  Upon each subject’s completion of the study, the effectiveness and safety of ACH-0144471 in that subject will be evaluated for consideration of dosing extension in a potential separate open-label extension protocol in which all subjects would receive active treatment. Subjects enrolling in such a study would not require the dosing taper or follow-up visits described above.

• REC name

  London - City & East Research Ethics Committee

• REC reference

  18/LO/0719

• Date of REC Opinion

  22 Jun 2018

• REC opinion

  Further Information Favourable Opinion