Evaluation of the Pharmacokinetics and Safety of NT 0150 in Patients with Cryopyrin-Associated Periodic Syndrome

The primary objective of this study is to evaluate the **safety**, absorption, and elimination of NT 0150, a novel compound with potential therapeutic applications in the treatment of brain disorders such as Alzheimer's disease and Parkinson's disease. Understanding the pharmacokinetics and safety profile of NT 0150 is clinically relevant as it may inform future therapeutic strategies for these neurodegenerative conditions. No secondary objectives are specified for this study.

The clinical trial involves participants diagnosed with **Cryopyrin associated periodic syndrome**. The study population includes both male and female subjects, with an age range spanning from 18 to 64 years. The trial does not focus on a vulnerable population. The sponsor has not provided information regarding the total number of participants. Selection criteria and lifestyle considerations such as diet, physical activity, or habits have not been specified. Key inclusion or exclusion criteria are not detailed in the available data.

The clinical trial is designed to evaluate the safety, absorption, and elimination of a new compound, NT 0150, which may be used in the treatment of brain disorders such as **Alzheimer's disease** and **Parkinson's disease**. The study is a Phase 1 trial, which is typically the first stage of testing in human subjects and focuses on assessing the safety profile of the investigational compound. The trial is structured as a randomized, double-blind, controlled study, ensuring that neither the participants nor the researchers know who is receiving the treatment or placebo, thus minimizing bias and ensuring the reliability of the results.

The trial is expected to commence recruitment on September 1, 2025, and is estimated to conclude by July 3, 2026. Participants will be involved in the study for a duration that aligns with the trial's timeline, with specific involvement periods determined by the study protocol. The sequence of study visits includes an initial inclusion (screening) visit, where eligibility is assessed based on predefined criteria. This is followed by a series of follow-up visits, which are scheduled to monitor the participants' health, collect data on the compound's pharmacokinetics, and assess any adverse effects. The end-of-study visit marks the conclusion of the participant's involvement, where final assessments are conducted, and data is collected for analysis.

Participants may be withdrawn from the study early if they experience significant adverse effects, fail to comply with the study protocol, or if the study is terminated for any reason. The trial specifically targets individuals with **Cryopyrin associated periodic syndrome**, a condition that is not classified as a rare disease. The study's design and procedures are meticulously planned to ensure the collection of high-quality data while prioritizing participant safety and adherence to ethical standards.

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.

Due to the lack of specific information in the provided data, further details about the **experimental medication** and any **non-experimental treatments** used in the study cannot be elaborated upon. The trial documentation should be consulted for comprehensive information regarding the treatments involved in this clinical trial.

The clinical trial is designed to assess efficacy through a structured evaluation process. The trial is categorized as a Phase 1 study, indicating an early stage of clinical research primarily focused on safety and dosage. The estimated recruitment start date is September 1, 2025, with an anticipated end date of July 3, 2026. Although specific efficacy parameters such as primary and secondary endpoints are not detailed, typical Phase 1 trials often involve preliminary assessments of efficacy alongside safety evaluations. These assessments may include the use of validated scales, laboratory tests, or patient-reported outcomes to measure changes in relevant clinical markers or symptoms. The schedule for measuring and collecting data is typically aligned with the trial's protocol, ensuring systematic and consistent data collection throughout the study duration. The analysis of efficacy data will be conducted using appropriate statistical methods to determine the potential therapeutic effects of the investigational product. The trial's design and methodology adhere to regulatory standards to ensure the reliability and validity of the findings.