AOC 1001-CS3 HARBOR
Brief description of study
This is a phase 3 trial of AOC 1001, an antibody-oligonucleotide conjugate. Protocol includes participants ages 16-65 with a clinical and genetic diagnosis of DM1 with DMPK CTG repeat length greater than or equal to 100; Penn site will enroll ages 18 to 65 years only. Participants will be randomized (1:1) to receive an IV infusion of AOC 1001 or placebo every 8 weeks for 7 doses. Participants will undergo physical/neuro exams, ECGs, blood/urine tests, function/strength tests, and questionnaires.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Medical Research
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Age: - 99 Years
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Gender: All
TBD
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