Inclusion Body Myositis Treatment With Celution Processed Adipose Derived Regenerative Cells (NCT04975841) | Clinical Trial Compass
CompletedNot Applicable
Inclusion Body Myositis Treatment With Celution Processed Adipose Derived Regenerative Cells
United States9 participantsStarted 2022-03-10
Plain-language 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.
Who can participate
Age range45 Years
SexALL
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Inclusion criteria
✓. Meet any of the European Neuromuscular Centre Inclusion Body Myositis research diagnostic criteria 2011 categories for IBM. (see Appendix 3)
✓. Demonstrate being able to arise from a chair without support from another person or device. Subjects may use their arms to push up.
✓. Able to ambulate at least 20 ft/6 meters with or without assistive device. Once arisen from the chair, participant may use any walking device, i.e. walker/frame, cane, crutches, or braces. They cannot be supported by another person and cannot use furniture or wall for support.
✓. Age at onset of weakness \> 45 years
✓. Able to give informed consent
✓. Muscle strength graded between 6 and 9 for finger flexion and knee extension unilaterally on one side of the body (right or left) using the Kandell 0-10 scale. A subject may meet this criterion bilaterally and still be included in the study.
Exclusion criteria
✕. History of any of the following excludes subject participation in the study: chronic infection particularly HIV or Hepatitis B or C; cancer other than basal cell cancer less than five years prior, or other chronic serious medical illnesses.
✕. Presence of any of the following on routine blood screening: WBC \< 3000; platelets \< 100,000; hematocrit \< 30%; BUN \> 30 mg/dL; creatinine \> 1.5 x upper limit of normal; symptomatic liver disease with serum albumin \< 3 g/dl
✕. History of most recent creatine kinase \>15x the upper limit of normal without any other explanation besides IBM.
✕. History of non-compliance with other therapies.
✕. Use of testosterone except for physiologic replacement doses in case of androgen deficiency. Participants must have documented proof of the androgen deficiency.
✕. Coexistence of any other neurological, cardiac, pulmonary, psychiatric, or rheumatologic disease that would likely to affect outcome measures.
✕. Drug or alcohol abuse within past three months. Patient has recent history (within 6 months before Screening) of chronic alcohol or drug abuse which may compromise the patient's safety or ability to participate in study activities.