NI-0501 in Primary HLH
Research type
Research Study
Full title
A Phase 2/3 , Open-label, Single Arm, Multicentre Study to Assess Safety, Tolerability, Pharmacokinetics and Efficacy of Intravenous Multiple Administrations of NI-0501, an Anti-interferon Gamma (Anti-IFNγ) Monoclonal Antibody, in Paediatric Patients with Primary Haemophagocytic Lymphohistiocytosis (HLH)
IRAS ID
191535
Contact name
Anupama Rao
Contact email
Sponsor organisation
NovImmune SA
Eudract number
2012-003632-23
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 0 months, 31 days
Research summary
Haemophagocytic lymphohistiocytosis (HLH) results from a defective immune system and is often characterised by a severe inflammation that the body cannot switch off. The overstimulated immune system produces an excessive number of certain immune cells which accumulate in tissues and organs causing damage and also attack healthy blood cells, consuming and killing them. HLH can be inherited (primary HLH) which is extremely rare and is normally seen in early childhood; or acquired HLH (secondary HLH) which can occur at any age. Currently, there is no specific treatment approved for HLH. Primary HLH is managed by giving a combination of medications that can control the disease and help patients achieve the stable condition required for stem cell transplantation, which may potentially cure HLH. Once stable condition is achieved, a mixture of medications is given to suppress the immune system before transplantation. Transplantation replaces the ill-functioning immune system with cells that can develop into healthy blood cells and a healthy immune system.
This study will investigate how safe, effective and well-tolerated NI-0501 (an experimental medication) is in controlling the disease and achieving a stable condition required for transplantation. Participants of both sexes, aged 18 years and below at the time of HLH diagnosis (predominantly children) will be enrolled until ten evaluable participants have completed the study. Participants diagnosed with primary HLH will either have failed conventional HLH therapy or will not have received previous HLH treatment. Participants will receive NI-0501 for maximum 8 weeks and will be monitored afterwards for 4 weeks. After completion of 8 weeks of treatment and if an appropriate donor has not been identified, or there is a delay in transplant, upon request of Investigator and providing a favourable benefit/risk was established, treatment with NI-0501 may continue in a separate long-term safety study which the participant could join.
REC name
London - Bloomsbury Research Ethics Committee
REC reference
15/LO/1959
Date of REC Opinion
24 Feb 2016
REC opinion
Further Information Favourable Opinion