Phase 3B/4 Extension Study of AX 250 in MPS IIIB Patients

• Research type

  Research Study

• Full title

  A Phase 3B/4 Open-Label Multicenter Study Extension Study to Further Evaluate Safety, Tolerability and Efficacy of Intracerebroventricular AX 250 Treatment in Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Patients

• IRAS ID

  1006388

• Contact name

  Bernice Kuca

• Contact email

  bernie@allievex.com

• Sponsor organisation

  Allievex Corporation

• Eudract number

  2022-002762-33

• Clinicaltrials.gov Identifier

  NCT05492799

• Research summary

  Mucopolysaccharidosis type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B) is a disease that primarily affects the central nervous system, where progressive neurodegeneration causes a wide range of neurological symptoms including developmental delay, disorders of language development, behavioural disturbances, such as severe hyperactivity and loss of impulse control, sleep disorders, anxiety and hearing loss.
  The purpose of this research study is to learn if the study drug, called AX 250, can continue to help children with MPS IIIB disease. This study aims to find out what effects AX 250 has in children with MPS IIIB, and how and if it continues to work in the body after nearly 6 years of dosing in two previous studies. This study will enrol participants with confirmed diagnosis of MPS IIIB who have completed Study 250-202.
  People with MPS IIIB do not have enough NAGLU, an enzyme needed to break down a molecule normally found in the body called heparan sulfate, which is one specific type of a broad class of substances known as glycosaminoglycans (GAGs). When there is not enough working NAGLU enzyme, heparan sulfate GAG builds up in the cells of the body, leading to the symptoms seen with MPS IIIB. AX 250 is intended as an enzyme replacement therapy.
  Up to 15 children will be in the whole study and will participate for almost 3 years. Each study visit will last from 1 to 3 days. A number of tests and procedures will be done at the study visits, including: collection of blood samples and cerebrospinal fluid, physical examinations, ECG (electrical test of the heart rhythm), EEG (electrical test of the brain wave pattern), questionnaires completion and MRI scans. The study is sponsored by Allievex Corporation.

• REC name

  East of England - Cambridge East Research Ethics Committee

• REC reference

  22/EE/0233

• Date of REC Opinion

  17 Oct 2022

• REC opinion

  Further Information Favourable Opinion