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.
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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
OTHER
Masking
NONE
Enrollment
9
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.
University of Kansas Medical Center
Kansas City, Kansas, United States
Safety (Frequency and Severity of Adverse Events)
The primary endpoint of safety will be reported as counts of subjects experiencing adverse events events (including abnormal laboratory results and vital signs). Six months after the first participant is treated, safety and efficacy will be accessed in order to make a decision about whether to continue the trial. Measures of safety will be reported for the last visit observed for each participant
Time frame: 3 months post treatment
Change in Inclusion Body Myositis Functional Rating Scale (IBMFRS) from Baseline
The IBMFRS is a quickly administered (10-minute) ordinal rating scale used to determine participants' assessment of their capability and independence. It includes 10 measures (swallowing, handwriting, cutting food and handling utensils, fine motor tasks, dressing, hygiene, turning in bed and adjusting covers, changing position from sitting to standing, walking, and climbing stairs), graded on a Likert scale from 0 (being unable to perform) to 4 (normal). The sum of the 10 items gives a value between 0 and 40, with a higher score representing less functional limitation.
Time frame: 6 months post treatment
Change in Modified Timed Up and Go (mTUG) from Baseline
We will measure the patient's ability to get up from a chair allowing participants to use their arms (since most with sIBM cannot perform the task without pushing off), walk 3 meters, turn around and walk back to the chair and sit down. The use of nearby walls, or assistance from a caregiver is not allowed. This test will be performed twice and the fastest time will be used in the data analysis.
Time frame: 6 months post treatment
Change in Health Assessment Questionnaire-Disability Index (HAQ-DI) from Baseline
This is a self-report functional status measure based on the patient-centered dimension of disability.
Time frame: 6 months post treatment
Change in Patient Global Impression of Change from Baseline
The Patient Global Impression of Change (PGIC) is an assessment by the subject of self-perceived change in ability to conduct daily activities since the start of study medication. The scale ranges from 'very much worse' to 'much worse,' 'a little worse,' 'no change,' 'a little improved,' 'much improved,' and 'very much improved'.
Time frame: 6 months post treatment
Change in Manual Muscle Testing (MMT) from Baseline
Manual muscle testing occurs when the evaluator records strength by having the subject resist while pushing on certain muscle groups with their arms and hands. MMT will include bilateral Knee Extension and Finger Flexion on the Kendall modification of the MRC scale being 0-10 scale.
Time frame: 3, 6, and 12 months post treatment
Change in Handheld Dynamometry (HHD) from Baseline
HHD will be done using a MicroFET2 hand myometer. This is a hand-held device that allows the examiner to push against a muscle while the patient resists. Unlike the manual muscle test which provides a range, this device will provide an actual number. Knee extension will be tested bilaterally.
Time frame: 6 months post treatment
Change in Grip Strength from Baseline
Grip strength is done with the use of a Martin Vigorimeter that will measure grip strength. The maximum force generated by the patient from the two trials is used for each muscle group.
Time frame: 6 months post treatment
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.