LCH-IV
Research type
Research Study
Full title
LCH-IV - International Collaborative Treatment Protocol for Children and Adolescents with Langerhans Cell Histiocytosis
IRAS ID
76077
Contact name
Johannes Visser
Contact email
Sponsor organisation
St. Anna Kinderkrebsforschung, Children's Cancer Research Institute
Eudract number
2011-001699-20
Clinicaltrials.gov Identifier
Duration of Study in the UK
10 years, 5 months, 1 days
Research summary
Langerhans Cell Histiocytosis (LCH) is a rare disorder with highly variable clinical presentation and biological behaviours. It can affect a single system/organ (SS-LCH) or multiple systems/organs (MS-LCH). Patients with SS-LCH of the skeleton, skin or the lymph nodes have an excellent prognosis and may need no, or minimal treatment. MS-LCH is unpredictable upon diagnosis, ranging from spontaneous resolution to rapid progression and fatal outcome. Previous research has shown that combination therapy with vinblastine and prednisolone is effective for MS-LCH however more than a third of patients suffer disease reactivation. LCH patients may also suffer permanent consequences including hormone deficiencies, a neurodegenerative syndrome and lung fibrosis. This study aims to improve overall survival, reduce reactivation rates and reduce the permanent consequences. The trial is split into seven strata, designed to tailor treatment based on disease features at diagnosis and on response to treatment. Stratum I is investigating a prolongation (12 vs. 24 months) and intensification (addition of mercaptopurine) of first line therapy (vinblastine and prednisolone) via a randomisation. In stratum II, the response to a uniform initial second line therapy (prednisolone, cytarabine and vincristine) for those patients without risk organ involvement is studied following a randomised comparison of maintenance therapy with either indomethacin or mercaptopurine and methotrexate. Stratum III (cladribine/cytarabine based salvage treatment) and stratum IV (reduced intensity haemapoietic stem cell transplant) are single arm studies of second line therapy for those patients with risk organ involvement. Stratum V explores the course and treatment of Central Nervous System-LCH (CNS-LCH). Stratum VI is an observational stratum for SS-LCH which does not require systemic treatment at diagnosis. All patients on trial will automatically enter Stratum VII following completion of treatment and involves the long term follow up of all patients diagnosed with LCH.
REC name
East Midlands - Derby Research Ethics Committee
REC reference
15/EM/0323
Date of REC Opinion
15 Sep 2015
REC opinion
Further Information Favourable Opinion