NCT04975841

Inclusion Body Myositis Treatment With Celution Processed Adipose Derived Regenerative Cells

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: University of Kansas Medical Center · Academic / Other
• Sex: All
• Age: 45 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label, single arm study evaluating the safety for patients with Inclusion Body Myositis. A total of 9 subjects will be enrolled in the study. Subjects will be randomized to Part 1 or Part 2 of the study in blocks of 3 every 3 months. Stem cell injections will be given in the forearm and thigh on either the left or right side of the body, depending on which side meets criteria. The overall goal of this pilot study is to test the safety of adipose derived regenerative cells in patients with Inclusion Body Myositis. If determined safe, this trial could lead to larger Phase II trials. While this specific trial's primary endpoint is safety, it our ultimate hope that ADRC injections into the forearm and thigh of IBM patients will slow, stabilize, or even reverse the progression of muscle weakness in patients with IBM.

Detailed description

Inclusion Body Myositis (IBM) is the most common progressive and debilitating muscle disease beginning in persons over age 50 years, with an annual incidence estimated at 2.2 to 7.9 per million. IBM causes both proximal and distal muscle weakness, characteristically most prominent in the quadriceps and finger flexors. Over time it can lead to severe disability, including loss of hand function, falls due to quadriceps muscle weakness and foot drop, dysphagia, and eventually respiratory muscle weakness. There is no effective therapy for IBM. This study, "Inclusion Body Myositis Treatment with Celution Processed Adipose Derived Regenerative Cells" (IBM-ADRC) evaluates the safety and efficacy of the Celution System in the processing of an autologous graft consisting of adipose-derived regenerative cells (ADRCs) in the treatment of inclusion body myositis. Specifically, the overall objective is for the proposed clinical trial to serve as a safety trial for Inclusion Body Myositis. This is an open-label, single arm study evaluating the safety for patients with Inclusion Body Myositis. A total of 9 subjects will be enrolled in the study. Subjects will be randomized to Part 1 or Part 2 of the study in blocks of 3 every 3 months. Enrollment is anticipated to be staggered into 3 groups. The nine subjects will be randomized 2:1 in groups of 3 to early injections (Part 2) versus late (Part 1). Stem cell injections will occur unilaterally in the flexor digitorum profundus muscles and the quadriceps group of muscles.

Conditions

• Inclusion Body Myositis

Interventions

Type	Name	Description
DEVICE	Adipose Derived Regenerative Cells	The Cytori Celution technology is an automated version of the process and techniques used in the research laboratory to isolate regenerative cells from adipose tissue. The Cytori Celution System uses a proteolytic enzyme blend (Celase) to disrupt the adipose tissue matrix and release the entrapped adipose derived regenerative cells (ADRCs), also referred to in the literature as "stromal vascular fraction" cells. Once digested, the digestate is separated into fractions (buoyant adipocytes (fat cells) and pelleted ADRCs) by centrifugation. The non-buoyant cell pellet (ADRCs) is then washed and centrifuged through several cycles to remove residual enzyme reagent and cellular debris. Although the Cytori Celution technology replicates known laboratory techniques, it offers significant improvements to the manual laboratory technique by controlling the processing in a closed system that has been validated for safety, performance, and reproducibility.

Timeline

• Start date: 2022-03-10
• Primary completion: 2025-09-26
• Completion: 2025-09-26
• First posted: 2021-07-23
• Last updated: 2025-12-17

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated device study