NI-0501 in paediatric patients with reactivated primary HLH

• Research type

  Research Study

• Full title

  A pilot, open-label, single arm, multicentre study to explore safety, tolerability, pharmacokinetics and efficacy of intravenous multiple administrations of NI-0501, an anti-interferon gamma (anti-IFN?) monoclonal antibody, in paediatric patients with primary Haemophagocytic Lymphohistiocytosis (HLH) which has reactivated

• IRAS ID

  115883

• Contact name

  Anupama Rao

• Sponsor organisation

  Novimmune SA

• Eudract number

  2012-003632-23

• ISRCTN Number

  n/a

• Research summary

  Primary HLH is a rare, potentially fatal genetically inherited disease with an abnormally high production and activation of some cells usually responsible for killing microbes and viruses. Urgent, aggressive treatment is required to combat these effects before bone marrow transplantation, the only current definitive cure for the disease. The estimated prevalence in Europe is 1/50,000 live births. At present, there is no specific medication approved by health authorities against HLH: a combination of drugs, normally used as chemotherapy for other severe diseases like cancers, shows some efficacy in controlling disease but not all patients respond to these drugs and they do carry known significant short and long term side effects. The study drug, NI-0501, is a ??monoclonal antibody??, a laboratory-created protein similar to other body proteins produced to fight against foreign substances or infections. It binds and neutralises one of the most potent inflammatory agents over-produced in HLH called interferon gamma (IFN?). There is good evidence to suppose that it could help fight against reactivation of the disease and stabilise the condition. Subjects to be included in this study will be up to 18 years of age at the time of original diagnosis, suffering from life-threatening reactivation of the disease after previous treatment has had at least partial response. NI-0501 will be administered intravenously in this study for the first time to patients with HLH after previously being given to healthy volunteers and found to be generally well tolerated. It will test whether or not multiple infusions of the study drug, over an eight-week period, can reverse the disease. It will be administered in association with a corticosteroid, commonly used as part of existing treatment cocktails. During the study subjects will undergo a number of physical and laboratory examinations that will allow investigators to determine the clinical outcome, the best dose and how well the drug is tolerated.

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  12/LO/1794

• Date of REC Opinion

  14 Jan 2013

• REC opinion

  Further Information Favourable Opinion