CLEE011Q12101
Research type
Research Study
Full title
Phase I/II multicentre study to assess efficacy and safety of ribociclib (LEE011) in combination with topotecan and temozolomide (TOTEM) in paediatric patients with relapsed or refractory neuroblastoma and other solid tumours
IRAS ID
1005150
Contact name
Livie Dorwling-Carter
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2021-005617-14
Clinicaltrials.gov Identifier
Research summary
The purpose of this study is to find out if ribociclib (LEE011) is safe and if it helps children and young people who have neuroblastoma and certain other solid tumours, including medulloblastoma, high grade glioma, malignant rhabdoid tumours and rhabdomyosarcoma.
The study is expected to start at the end of 2022 and finish in 2028. Participants will be on the study receiving drug for up to a year, or longer if their disease remains stable, or improves, and they can tolerate study treatment well. Participants will need to visit the study doctor about 29 times during the study. This study is split into 3 parts: Phase 1a, Phase 1b and Phase 2. The study will not move on to the next phase until the previous phase is completed and the data is checked.
All participants in phase 1a and phase 1b will receive ribociclib in combination with topotecan and temozolomide (TOTEM). About 18 participants in 9 countries will be in Phase 1a. The purpose of this phase is to determine the right dose of ribociclib+TOTEM in participants aged between 12 months and 21 years old who have cancer that has come back after earlier treatment or cancer that has not responded adequately to previous treatment. This is for participants with neuroblastoma or certain other solid tumours as listed above.
Phase 1b will be split into 5 groups (1 tumour type per group), to determine the safety and anti-tumour activity of ribociclib+TOTEM. Each group will enroll around 28 participants. For each group, an analysis will be conducted after 10 participants are enrolled to determine if that group should continue enrolling. After phase 1B the data will be analyzed to see if the study should continue to phase 2.
Phase 2 is a double-blind, randomized and placebo-controlled study in Neuroblastoma only. This will be comparing ribociclib+TOTEM with placebo+TOTEM. This is to see if ribociclib works better in combination with TOTEM than TOTEM alone. Currently phase 2 is planned to include about 97 patients.REC name
London - Westminster Research Ethics Committee
REC reference
22/LO/0737
Date of REC Opinion
16 May 2023
REC opinion
Further Information Favourable Opinion