Your session is about to expire
← Back to Search
Regenerative Medicine
Stem Cell Therapy for Inclusion Body Myositis
N/A
Waitlist Available
Led By Mazen Dimachkie
Research Sponsored by University of Kansas Medical Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Demonstrate being able to arise from a chair without support from another person or device. Subjects may use their arms to push up
Age at onset of weakness > 45 years
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3, 6, and 12 months post treatment
Awards & highlights
Study Summary
This trial is testing the safety of injecting stem cells into the arms and legs of people with Inclusion Body Myositis, in hopes that it will slow, stabilize, or reverse the progression of muscle weakness in patients with IBM.
Who is the study for?
This trial is for individuals with Inclusion Body Myositis who can get up from a chair unaided, walk at least 20 ft, meet specific diagnostic criteria, and have muscle strength scores of 6-9 in certain areas. Participants must be over 45 years old and able to consent. Exclusions include recent drug study participants, cannabis users, current smokers, those with certain medical conditions or infections, pregnant or breastfeeding women, and people not using effective birth control.Check my eligibility
What is being tested?
The trial is testing the safety of injecting adipose derived regenerative cells into the forearm and thigh muscles of patients with Inclusion Body Myositis. It's an early-stage study involving nine subjects to see if this treatment might slow down or reverse muscle weakness.See study design
What are the potential side effects?
While the primary focus is on safety rather than side effects, potential risks may include reactions at injection sites such as pain or infection; however detailed side effect profiles will be determined during this pilot study.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I can get up from a chair without help, but I can use my arms to assist.
Select...
I started experiencing weakness after turning 45.
Select...
I can walk at least 20 feet on my own with a walking aid but without someone's help.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 3, 6, and 12 months post treatment
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3, 6, and 12 months post treatment
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Safety (Frequency and Severity of Adverse Events)
Secondary outcome measures
Change in Grip Strength from Baseline
Familial benign pemphigus
Change in Health Assessment Questionnaire-Disability Index (HAQ-DI) from Baseline
+4 moreTrial Design
2Treatment groups
Active Control
Group I: Standard of CareActive Control1 Intervention
Standard of Care (SOC) Study: The 6 subjects in the late injection group will start on Part 1. - The Part 1 study subject participation is 12 months. Two subjects will be enrolled at each of Months 0, 3 and 6. This will include an initial assessment and SOC follow-up. Subjects will continue standard of care treatment. Part 1 study duration (with staggering included) will be 18 months. After Part 1, the late injection subjects may proceed to Part 2 depending on safety data from the early injection group (see 3. below).
Group II: Stem Cell InjectionActive Control1 Intervention
Stem Cell Injection: The three subjects randomized to early injections will proceed directly to Part 2 with staggered enrollment of 1 subject every 3 months. Once the safety data of the first subject at Month 3 is assessed, the second subject will be enrolled. Once the safety data of the first 2 subject (Subject 1 at Month 6 and Subject 2 at Month 3) are assessed, the third early injection subject will be enrolled in Part 2.
Who is running the clinical trial?
University of Kansas Medical CenterLead Sponsor
460 Previous Clinical Trials
169,106 Total Patients Enrolled
Mazen DimachkiePrincipal InvestigatorUniversity of Kansas Medical Center
1 Previous Clinical Trials
140 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I haven't taken systemic steroids or immunosuppressants recently, except for local or specific minor uses.I have taken blood thinners within 1 hour before liposuction.My blood test shows high alkaline phosphatase, suggesting I might have Paget's disease.I have not used drugs known to affect muscles or have used them safely for over a year.I am not using any other drugs for myositis or undergoing cell or gene therapy.I am not pregnant or breastfeeding and will use effective birth control during the trial.I have a history of chronic infection, such as HIV or Hepatitis B/C.I can get up from a chair without help, but I can use my arms to assist.I have not had any cancer other than basal cell or serious illnesses in the last 5 years.I have followed all my previous treatment plans.I use testosterone only in small doses for a diagnosed hormone deficiency.I do not have any major health issues that could affect my study results.I am a current smoker.I have taken blood thinners within 1 hour before my stem cell procedure.I cannot stop my strong blood thinner medication two weeks before the fat collection procedure.My IBM (a muscle disease) started before I was 45.I have an active skin infection where they plan to remove fat.I started experiencing weakness after turning 45.My latest creatine kinase levels were very high due to IBM.My muscle strength in my fingers and knee on one side is moderately strong.My blood counts are low, and I have kidney, liver, or blood issues.I can walk at least 20 feet on my own with a walking aid but without someone's help.
Research Study Groups:
This trial has the following groups:- Group 1: Standard of Care
- Group 2: Stem Cell Injection
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Inclusion Body Myositis Patient Testimony for trial: Trial Name: NCT04975841 — N/A
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are there any open enrollment opportunities for this clinical research?
"Information available on clinicaltrials.gov suggests that the posting for this trial has expired, with an initial post date of 3rd October 2022 and a final update made 12th October 2022. Nevertheless, 23 other studies are still actively recruiting participants at present."
Answered by AI
Who else is applying?
What state do they live in?
Idaho
Pennsylvania
What portion of applicants met pre-screening criteria?
Did not meet criteria
Met criteria
How many prior treatments have patients received?
0
1
What site did they apply to?
University of Kansas Medical Center
Why did patients apply to this trial?
I have been dealing with the effects of Ibm for 10 years. It has progressed to where I am now bed ridden. I had a heart transplant in 2003 and have been taking Sirolimus as one of my transplant drugs for 4 years. I think the Sirolimus has slowed my progression, but my left side was very week when.
PatientReceived no prior treatments
Recent research and studies
Other Trials to Consider
Popular Categories
Share this study with friends
Copy Link
Messenger