Evaluating how the body processes satralizumab in children with NMOSD

• Research type

  Research Study

• Full title

  A PHASE III, MULTICENTER, OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE PHARMACOKINETICS, EFFICACY, SAFETY, TOLERABILITY, AND PHARMACODYNAMICS OF SATRALIZUMAB IN PEDIATRIC PATIENTS WITH AQP4 ANTIBODY POSITIVE NEUROMYELITIS OPTICA SPECTRUM DISORDER (NMOSD)

• IRAS ID

  271346

• Contact name

  Cheryl Hemingway

• Contact email

  cheryl.hemingway@gosh.nhs.uk

• Sponsor organisation

  F. Hoffmann-La Roche Ltd

• Eudract number

  2019-004092-39

• Clinicaltrials.gov Identifier

  NCT05199688

• Clinicaltrials.gov Identifier

  IND, 118183

• Duration of Study in the UK

  5 years, 1 months, 0 days

• Research summary

  Paediatric neuromyelitis optica spectrum disorder (NMOSD) is a severe inflammatory CNS disease with relapse-associated disability accumulation, with an unmet need for effective relapse prevention treatment. The majority of paediatric AQP4-IgG positive patients (median age of onset 12 years; range-4-18 years), experience relapsing disease course, resulting in neurological disability. To date, there is no approved therapy available for the treatment of children with NMOSD.
  This phase III study will primarily evaluate the pharmacokinetics of satralizumab in paediatric patients aged 2-11 years with anti-aquaporin-4 antibody seropositive NMOSD. There will be approximately 8 participants worldwide and 1 UK hospital. Efficacy, safety, tolerability, and pharmacodynamics will be evaluated in a descriptive manner, given the small number of patients who will be enrolled in this study.

  This trial is split into four parts: Screening period, Main treatment period, Optional extension, Safety follow-up.
  In the main treatment period, all participants will be given satralizumab, as a subcutaneous injection for 48 weeks. Body weight determines the dose of satralizumab received.
  ● Weight at least 10 kg and less than 20 kg: given 60 mg of satralizumab every six weeks
  ● Weight at least 20 kg and less than 40 kg: given 60 mg of satralizumab at Weeks 0, 2 and 4, then every four weeks
  ● Weight 40 kg or more: given 120 mg of satralizumab at Weeks 0, 2 and 4, then every four weeks
  Total study duration will be approximately 4.5 years from first patient in.

  Participants in the study will undergo the following assessments:
  ● Blood samples,
  ● Review of medical history
  ● Demographic information
  ● Body height and weight
  ● Questionnaires about the disease
  ● Visual function tests
  ● Vital signs: temperature, pulse, breathing rate, blood pressure
  ● Physical examination,
  ● Neurological examination, assessed for any change to participant clinical symptoms
  ● Changes in health or medications
  ● Electrocardiogram,
  ● Urine sample,
  ● Relapse assessment,
  ● Follow up call

• REC name

  London - Chelsea Research Ethics Committee

• REC reference

  22/LO/0120

• Date of REC Opinion

  12 Apr 2022

• REC opinion

  Further Information Favourable Opinion