Gieres

The product developed by Ceres-Brain Therapeutics is a creatine-based drug called CBT101. CBT101 is a product designed to deliver creatine to brain cells. CBT101 will be administered into the nasal cavity via a nasal spray, enabling the product to reach the brain, and neurons in particular, rapidly. In the neurons, CBT101 will be converted into creatine. This product is indicated for adults and children suffering from creatine deficiency syndrome, or for neurological diseases where a supply of energy, in the form of creatine, would be useful. These include Creatine Transporter Deficiency (a rare genetic disease that affects children and manifests itself in autistic disorders, intellectual deficits, major communication and developmental disorders, particularly psychomotor disorders, and epileptic seizures) and Charcot's disease (a degenerative disease of neurons that unfortunately currently has no curative treatment).This study is divided into 2 parts and will include a total of 48 healthy male volunteers aged between 18 and 55. The primary objective is to evaluate the safety and tolerability of CBT101 after 14 days of repeated intranasal dosing at 3 ascending doses. The secondary objective is to determine the pharmacokinetic parameters (study of the fate of the drug in the body) of CBT101 or its metabolites. The first part is a Single Ascending Doses (SAD) composed of 3 cohorts at the following doses: 4.2 mg, 8.4 mg and 12.6 mg. The second part is a Multiple Ascending Doses (MAD), comprising 3 cohorts at the following doses: 4.2 mg, 8.4 mg and 12.6 mg. There will be 8 participants per cohort, of whom 6 will receive CBT101 and 2 will receive placebo.

Leucettinib-21 First-in-Human Phase 1 Study in 6 Parts: Single (Part 1 and 5) and Multiple (Part 3 and 6) Ascending Doses, and Food-Effect (Part 2) in Healthy Subjects, and Single Dose (Part 4) in People with Down Syndrome (DS) and Alzheimer's Disease (AD). For Parts 1, 3, 4, 5 and 6, safety and tolerability of an oral administration of Leucettinib-21 will be assessed as primary objectives. Pharmacokinetics and pharmacodynamic biomarkers will be investigated as secondary objectives. For Part 2, the effect of high fat meal will be evaluated on the pharmacokinetics parameters after an oral administration of Leucettinib-21.

The two parts are open-label and randomized, with a 2 periods, cross-over design for part A and a 3-period-parallel-arm design for Part B. Expected duration for part A is approximately 8 weeks and Part B is approximately 12 weeks for each participating subject.