ALXN1101 in Newborns with molybdenum cofactor deficiency

• Research type

  Research Study

• Full title

  A Phase 2/3, Multicenter, Multinational, Open-Label Study to Evaluate the Efficacy and Safety of ORGN001 (formerly ALXN1101) in Neonates, Infants, and Children with Molybdenum Cofactor Deficiency (MoCD) Type A. Project ALXN1101-MCD-202

• IRAS ID

  194884

• Contact name

  Stephanie Grunewald

• Contact email

  stephanie.grunewald@gosh.nhs.uk

• Sponsor organisation

  Alexion Pharma GmbH

• Eudract number

  2013-002702-30

• Duration of Study in the UK

  4 years, 8 months, 1 days

• Research summary

  Molybdenum Cofactor Deficiency (MoCD) is an ultra-rare genetic disorder in which the body is not able to produce “cyclic pyranopterin monophosphate” (cPMP). This causes a build‐up of a sulphite which is toxic and damages the central nervous system (brain).
  MoCD is usually diagnosed in babies once they are born and causes symptoms such as seizures, involuntary movements and difficulties in feeding.

  There is no available approved treatment for MoCD Type A. Current management strategies are symptomatic (work to treat the symptoms) and aim to provide relief from MoCD Type A clinical manifestations as well as to provide palliative care.
  Alexion Pharma GmbH (Alexion) has developed ALXN1101, a synthetic form of cPMP and a precursor molecule to MoCo, as a treatment for MoCD Type A.
  ALXN1101 provides a therapeutic approach for the treatment of MoCD Type A that is intended to restore MoCo biosynthesis (generation).
  Results from nonclinical pharmacology studies with ALXN1101 suggest that the metabolic abnormality in MoCD Type A could be corrected by administration of synthetic cPMP, resulting in restoration of enzymatic activity and correction of the metabolic pathways that lead to accumulation of toxic metabolites that cause CNS injury.

  This study is designed to evaluate the safety and efficacy of ALXN1101 in patients with paediatric MoCD.

  It will be conducted at 32- 35 sites across 14 countries, with the aim to find
  5 -10 eligible patients

• REC name

  East Midlands - Derby Research Ethics Committee

• REC reference

  16/EM/0008

• Date of REC Opinion

  26 Feb 2016

• REC opinion

  Further Information Favourable Opinion