A study with NI-0501 to see efficacy and safety in children with pHLH

• Research type

  Research Study

• Full title

  An Open-label, Single Arm, Multicenter Study to Broaden Access to Emapalumab, an Anti-Interferon Gamma (Anti-IFNγ) Monoclonal Antibody, and to Assess its Efficacy, Safety, Impact on Quality of Life, and Long-term Outcome in Pediatric Patients with Primary Hemophagocytic Lymphohistiocytosis

• IRAS ID

  238084

• Contact name

  Robert Wynn

• Contact email

  Robert.Wynn@mft.nhs.uk

• Sponsor organisation

  Novimmune SA

• Eudract number

  2017-003114-10

• Clinicaltrials.gov Identifier

  NCT03312751

• Clinicaltrials.gov Identifier

  111015, US IND number

• Duration of Study in the UK

  3 years, 5 months, 1 days

• Research summary

  This is an international study and will be performed in North America and in Europe.
  The study is open to enrolment to paediatric patients with confirmed or suspected primary HLH (pHLH) who are either treatment-naïve, or failed conventional HLH therapy or showed intolerance to it. It is estimated that 34 patients with pHLH will participate in this study, investigate the efficacy, safety, impact on quality of life and long-term outcome of an experimental drug in children with pHLH.
  The experimental study drug is called emapalumab and was created by the biotechnology company Novimmune. Emapalumab will be administered to the study participants by infusions. Infusions will be performed twice weekly for a period between 4 to 12 weeks, period during which preparation for transplantation is likely to be started. Treatment will be done on top of dexamethasone, which is a drug usually given for this disease. The maximum treatment duration is 6 months. The whole study duration is maximum 18 months.

  The study is composed of 3 study periods:
  1. Screening period – up to 2 weeks: During this period, the study doctor will perform initial evaluations to assess if the subject qualifies to participate in the study.
  2. Treatment period – up to 6 months: During this period emapalumab will be given twice weekly and later weekly if certain criteria are met, under close medical supervision.
  3.. Follow-up period – 1 year: During this period, study participants will have 8 to 10 visits to closely monitor their health condition.

  The following procedures will be performed throughout the study: collection of medical history, physical examination, measurement of vital signs, blood and urine tests, electrocardiogram (ECG), abdominal ultrasound, chest X-ray, brain magnetic resonance imaging (MRI), spinal fluid sampling and completion of questionnaires.

• REC name

  North East - Tyne & Wear South Research Ethics Committee

• REC reference

  18/NE/0328

• Date of REC Opinion

  30 Nov 2018

• REC opinion

  Further Information Favourable Opinion