PK/PD of RO7234292 in CSF and plasma and safety and tolerability in HD

• Research type

  Research Study

• Full title

  An Open-Label, Adaptive Multiple-Dose Study To Investigate The Pharmacokinetics And Pharmacodynamics Of RO7234292 In CSF And Plasma, And Safety And Tolerability Following Intrathecal Administration In Patients With Huntington’s Disease

• IRAS ID

  253287

• Contact name

  Edward Wild

• Contact email

  E.Wild@ucl.ac.uk

• Sponsor organisation

  F.Hoffmann-La Roche Ltd.

• Eudract number

  2018-003010-40

• Duration of Study in the UK

  0 years, 8 months, 15 days

• Research summary

  This is an open-label, adaptive multiple-dose study designed to assess the pharmacokinetics (PK), pharmacodynamics (PD) and the PK/PD relationship after administration of 2 doses of RO7234292 in patients with Huntington's Disease (HD). All patients will receive 2 intrathecally administered doses of RO7234202 at one the following dose levels:
  - Dose level 1: 120 mg (No. of Patients minimum 4)
  - Dose level 2: 60 mg (No. of Patients minimum 4)
  - Dose level 3: 90 mg (No. of Patients minimum4)

  It is expected up to 20 patients with HD will be enrolled in this study. Patients will undergo a 4-week screening period, a 4-months treatment period, and a follow up visit 6-months after the last study drug administration. Patients will undergo safety and tolerability assessments that include, but are not limited to: physical and neurological examinations, laboratory tests, collection of CSF via lumbar punctures, Intrathecal catheter insertion and questionnaires throughout the study. At the end of the study Patients who complete the study may be given the opportunity to continue receiving the study drug on an Open-label extension (OLE) study.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  19/LO/0359

• Date of REC Opinion

  10 Jun 2019

• REC opinion

  Further Information Favourable Opinion