Phase I Study of Autologous Uncultured Adipose-Derived Stromal Vascular Fraction for Muscle Injection in Inclusion Body Myositis

The primary objective of this study is to evaluate the safety and feasibility of **cell therapy** for **Inclusion Body Myositis** (IBM) through muscle injection of autologous uncultured Adipose-Derived Stromal Vascular Fraction (ADSVF). This is a phase I trial aimed at determining the potential of ADSVF in addressing the progressive muscle weakness characteristic of IBM, a condition with limited treatment options. The study seeks to provide insights into the therapeutic potential of ADSVF, which could lead to advancements in the management of IBM.

The clinical trial involves participants diagnosed with **Inclusion Body Myositis**, a progressive muscle disorder. The study population includes both male and female subjects, with an age range encompassing adults and older adults. The trial does not focus on a vulnerable population, and the sponsor has not provided information regarding the total number of participants. Selection criteria for the trial population have not been disclosed, and there are no specific lifestyle considerations such as diet or physical activity mentioned. The sponsor has not provided detailed inclusion or exclusion criteria for this study.

The clinical trial is designed to evaluate the safety and efficacy of a **cell therapy** for patients with **Inclusion Body Myositis**. This is a phase I trial involving the muscle injection of autologous uncultured Adipose-Derived Stromal Vascular Fraction (ADSVF). 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. The estimated duration of the trial spans from February 10, 2021, to December 14, 2026, allowing for comprehensive data collection and analysis over this period.

Participants will undergo a series of study visits, beginning with an inclusion visit, which serves as a screening to determine eligibility based on predefined criteria. Following successful inclusion, participants will attend regular follow-up visits to monitor their health status, assess the treatment's effects, and ensure adherence to the study protocol. The end-of-study visit will conclude the participant's involvement, where final assessments will be conducted to gather data on the primary and secondary endpoints of the trial.

The expected length of participant involvement is contingent upon the trial's timeline, with each participant's duration depending on their enrollment date. Conditions that may lead to early termination from the study include adverse reactions to the treatment, non-compliance with study procedures, or withdrawal of consent by the participant. The trial's design and procedures are meticulously crafted to ensure the collection of reliable and valid data, contributing to the understanding of the potential therapeutic benefits of ADSVF in treating Inclusion Body Myositis.

The clinical trial documentation does not provide specific details regarding the **experimental medication** used in the study. Information such as the name, pharmaceutical form, dosage, route, and frequency of administration is not available. Additionally, there is no data on whether the medication is a **paediatric formulation** or if it is classified as an **orphan drug**. The maximum daily dose, total dose, and treatment period are also unspecified.

Details about any **non-experimental treatments** used in the study, such as standard-of-care therapy, placebo, or comparator treatment, are not provided. There is no information on additional relevant aspects of drug administration, dosing schedules, or participant compliance monitoring. The absence of these details limits the ability to describe the treatment regimen comprehensively.

The clinical trial is designed to assess efficacy through a structured evaluation process. The trial is set to conclude on December 14, 2026, with recruitment having commenced on February 10, 2021. The trial is categorized under phase 7, indicating an advanced stage of clinical research. Although specific efficacy parameters such as primary and secondary endpoints are not detailed, the trial will likely employ standard methodologies for measuring and analyzing efficacy outcomes. These may include validated scales, laboratory tests, or patient-reported outcomes, assessed at predetermined timepoints throughout the trial duration. The absence of explicit endpoints suggests a focus on comprehensive data collection to evaluate the therapeutic impact of the intervention under investigation.