Compassionate Use of Baricitinib to Treat Auto-inflammatory Syndromes
Research type
Research Study
Full title
Compassionate Use Treatment Protocol: Treatment of Conditions Expected to Benefit from JAK 1/2 Inhibition: CANDLE, CANDLE-Related Conditions, SAVI, Severe Juvenile Dermatomyositis and Aicardi-Goutières Syndrome.
IRAS ID
208555
Contact name
Debbie Froud
Contact email
Sponsor organisation
Eli Lilly and Company
Eudract number
2015-003424-31
Clinicaltrials.gov Identifier
Duration of Study in the UK
0 years, 11 months, 13 days
Research summary
The immune system is the body’s natural defence system that uses blood cells and inflammation to attack pathogens, for example virus. In autoinflammatory diseases, genetic mutations in the cells trigger the immune system to react without cause or control, leading to excessive inflammation throughout the body. These diseases often present with episodes of organ-specific inflammation and symptoms include severe rash, joint pain, headaches and frequent fever. In this protocol we will be studying a group of rare autoinflammatory diseases including: CANDLE, CANDLE-related conditions, SAVI, Severe Juvenile Dermatomyositis and Aicardi-Goutières Syndrome. These conditions are serious and life-threatening and there are currently very few treatment options available.
The JAK/STAT pathway is a cell signalling pathway that is involved in producing proteins which trigger the inflammatory response. It is thought that in these patients, genetic mutations cause this pathway to be overstimulated. The study medicine, Baricitinib is currently being developed for treatment of Rheumatoid Arthritis and other inflammatory conditions in which inhibition of the JAK/STAT pathway is beneficial. It is hoped that treatment of the patients in this study with Baricitinib will also help improve and manage their disease.
This study is an open-label, compassionate use, treatment protocol to provide the study medicine, Baricitinib to patients with specific autoinflammatory diseases who are not responsive to biologic therapies. These patients usually require unsustainable chronic doses of steroids to control their symptoms which can further contribute to the life-threatening nature of their disease.
During the study, patient’s will receive an initial dose of Baricitinib based on their disease type and weight. The dose may then be increased to determine a tolerable level. Disease severity will be recorded in a daily diary and average diary scores will be assessed by their doctor to trigger additional dose increases or steroid weaning as appropriate for each patient.
REC name
London - Central Research Ethics Committee
REC reference
16/LO/1485
Date of REC Opinion
4 Nov 2016
REC opinion
Further Information Favourable Opinion