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LION-101 for Limb-Girdle Muscular Dystrophy
Study Summary
This trial tests if a new drug is safe & tolerable for adults with a specific type of muscular dystrophy. It will be studied in sequential groups at different dose levels.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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Who is running the clinical trial?
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- I need help with breathing during the day.I am using or willing to use barrier contraception for 6 months after treatment.My steroid treatment hasn't changed in the last 3 months.I can walk or run 10 meters in less than 30 seconds.I have serious heart issues, including weak heart pumping or specific heart rhythm problems.My kidney function is reduced.I have a history of liver disease or currently have abnormal liver function.I can climb 4 stairs in 2.5 to 10 seconds.I haven't had any life-threatening diseases or cancer, except for skin cancer, in the last 5 years.I haven't taken any experimental drugs or had gene therapy recently.I am not pregnant, planning to become pregnant within a year, or breastfeeding.I am between 18 and 65 years old and have been diagnosed with LGMD2I/R9 with a confirmed FKRP gene mutation.
- Group 1: AB-1003 Cohort 1
- Group 2: AB-1003 Cohort 2
- Group 3: Placebo (Cohorts 1 and 2)
- 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.
Frequently Asked Questions
May I qualify to participate in this medical research?
"The eligibility criteria for this medical trial mandates that participants have a diagnosis of duchenne muscular dystrophy and are aged between 18 to 65 years. At present, 10 individuals may join the study."
Are there any vacancies available for volunteers in this experiment?
"Affirmative. The clinicaltrials.gov is displaying that the trial, which was first posted on March 12th 2023, is presently hunting for participants. There are 10 individuals needed to be enrolled from 1 medical centre."
How many participants are actively involved in this clinical trial?
"Affirmative. The clinicaltrials.gov website shows that this trial is currently recruiting participants, having been posted on March 12th 2023 and updated as of the 15th of March. 10 individuals must be recruited from 1 medical facility to complete the study."
Is the age limit for this trial participants in excess of 45 years?
"To be considered for enrollment in this clinical trial, patients must range between 18 and 65 years of age. Alternatively, there are 64 medical trials specifically designed for those below 18, as well as 54 studies available to individuals over the age of 65."
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