Phase I Study on the Bioavailability and Food Effect of Nizubaglustat in Glycosphingolipid Lysosomal Storage Disorders

The primary objective of this study is to evaluate the **relative bioavailability** of Nizubaglustat capsules compared to orally dispersible tablets in healthy volunteers. This assessment is clinically relevant as it provides critical information on the absorption and systemic availability of the drug in different formulations, which can influence dosing decisions and therapeutic outcomes in patients with **glycosphingolipid lysosomal storage disorders**. Additionally, the study aims to investigate the effect of food on the bioavailability of these formulations, which is essential for optimizing administration guidelines and ensuring consistent therapeutic efficacy.

The clinical trial involves participants diagnosed with **glycosphingolipid lysosomal storage disorders**. The study population includes both male and female subjects, with an age range that encompasses children and adolescents. The trial specifically includes a vulnerable population, although the total number of participants has not been disclosed by the sponsor. Participants were selected based on criteria that are not detailed in the available data. Lifestyle considerations such as diet, physical activity, or habits have not been specified. The trial aims to gather insights from a diverse group, ensuring representation across different age groups and genders within the specified medical condition.

The clinical trial is designed as a **Phase I**, open-label, randomized, adaptive, cross-over study. The primary aim is to evaluate the relative bioavailability of Nizubaglustat capsules compared to orally dispersible tablets and assess the food effect in healthy volunteers. The study targets individuals with **glycosphingolipid lysosomal storage disorders**. The trial is expected to commence recruitment on March 13, 2024, and conclude by July 9, 2024, with the overall duration spanning approximately four months.

Participants will undergo a sequence of study visits, beginning with an inclusion (screening) visit to assess eligibility based on predefined criteria. Following successful screening, participants will be randomized to receive either the capsule or tablet formulation, with subsequent cross-over to the alternate formulation. The study will include multiple follow-up visits to monitor safety, pharmacokinetics, and any potential adverse effects. The end-of-study visit will serve to collect final data and ensure participant well-being post-trial.

The expected length of participant involvement is contingent upon the completion of all study phases, with early termination possible if safety concerns arise or if participants withdraw consent. The study's adaptive design allows for modifications based on interim results, ensuring optimal data collection and participant safety. The trial's methodology emphasizes rigorous control and randomization to minimize bias and enhance the reliability of the findings.

The clinical trial involves the administration of an **experimental medication**. However, specific details regarding the name, pharmaceutical form, dosage, route, and frequency of administration of the experimental medication are not provided in the available data. The trial documentation does not specify whether the medication is a **paediatric formulation** or if it is classified as an **orphan drug**. Additionally, there is no information on the maximum daily dose, total dose, or treatment period for the experimental medication.

In addition to the experimental medication, the trial may include the use of **non-experimental treatments** such as standard-of-care therapy, placebo, or comparator treatments. However, the data does not provide explicit details about these treatments. Information regarding the administration, dosing schedules, and participant compliance monitoring for these non-experimental treatments is also not available.

The clinical trial is designed to assess efficacy in a Phase 3 study. The trial is scheduled to begin recruitment on March 13, 2024, with an estimated end date of July 9, 2024. The efficacy assessment will be conducted through a series of planned evaluations, although specific parameters or endpoints for efficacy evaluation are not detailed in the available data. The trial will follow a structured timeline to ensure systematic data collection and analysis. The methods and tools for measuring efficacy, as well as the specific timepoints for these assessments, are not specified in the provided information. The trial's focus on efficacy is aligned with its categorization as a Phase 3 study, which typically involves a larger participant group to confirm effectiveness, monitor side effects, and collect information that will allow the treatment to be used safely.