NCT07050316

Intranasal Administration of Dodecyl Creatine Ester (CBT101) in Healthy Male Subjects

A Phase 1, Randomized, Double-Blinded, Placebo-Controlled Study to Evaluate the Safety, Tolerability,and Pharmacokinetics of Single and Multiple Ascending Doses of CBT101 Given Intranasally in Healthy Male Subjects

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 48 (actual)
• Sponsor: Ceres Brain Therapeutics · Academic / Other
• Sex: Male
• Age: 18 Years – 55 Years
• Healthy volunteers: Accepted

Summary

A Phase 1, Randomized, Double-Blinded, Placebo-Controlled study to Evaluate the Safety, Tolerability,and Pharmacokinetics of Single and Multiple Ascending Doses of CBT101 given intranasally in Healthy Male Subjects.

Detailed description

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.

Conditions

• Neurologic Diseases, General

Interventions

Type	Name	Description
DRUG	CBT101	Part 1 (SAD): at the morning * Cohort 1: 1 puff in each nostril (total 2 puffs), total 4.2 mg * Cohort 2: 2 puffs in each nostril (total 4 puffs), total 8.4 mg * Cohort 3: 3 puffs in each nostril (total 6 puffs), total 12.6 mg Part 2 (MAD): * Cohort 4: 1 puff per nostril/day (total 2 puffs/day), total 4.2 mg/day (morning) * Cohort 5: 2 puffs per nostril/day (total 4 puffs/day), total 8.4 mg/day (morning and evening) * Cohort 6: 3 puffs per nostril/day (total 6 puffs/day), total 12.6 mg/day (morning, midday and evening)
DRUG	Placebo	Part 1 (SAD): at the morning * Cohort 1: 1 puff in each nostril (total 2 puffs), total 4.2 mg * Cohort 2: 2 puffs in each nostril (total 4 puffs), total 8.4 mg * Cohort 3: 3 puffs in each nostril (total 6 puffs), total 12.6 mg Part 2 (MAD): * Cohort 4: 1 puff per nostril/day (total 2 puffs/day), total 4.2 mg/day (morning) * Cohort 5: 2 puffs per nostril/day (total 4 puffs/day), total 8.4 mg/day (morning and evening) * Cohort 6: 3 puffs per nostril/day (total 6 puffs/day), total 12.6 mg/day (morning, midday and evening)

Timeline

• Start date: 2025-03-14
• Primary completion: 2025-09-26
• Completion: 2025-09-26
• First posted: 2025-07-03
• Last updated: 2026-03-27

Locations

1 site across 1 country: France