Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-24 @ 1:39 PM
Ignite Modification Date:
2025-12-24 @ 1:39 PM
NCT ID:
NCT05236595
Eligibility Criteria:
Inclusion Criteria:
* Has Mayo Clinic or other medical health system ID, or another unique identifier.
* Able to provide informed consent.
* Individual must have evidence of a genetic disorder as determined by a provider or genetic counselor with causative or likely causative genetic variants identified by molecular testing.
* Genetic variants must be hypothesized to be targetable using antisense oligonucleotide drugs (such as: knockdown gain of function alterations, increase protein production for reduced function alterations, or modulate mRNA splicing to correct abnormal splicing, promote normal splicing, or return reading frame to an out-of-frame transcript to restore function, etc.) based on current acceptable understanding of ASO mechanisms of action and tissue/organ targeting efficiency.
* Biological family member of an enrolled individual.
* Would be able to travel to a Mayo Clinic site for ongoing treatment should a therapeutic be developed.
* Treatment at the individual's current disease state would likely provide benefit based on current clinical data and understanding of the progression of the disease.
-Or-
* Biological family member of an enrolled individual
* Able to provide informed consent or has a LAR available to provide informed consent
Exclusion Criteria
* Individuals who have situations that would limit compliance with the study requirements.
* Institutionalized (i.e. Federal Medical Prison).
Healthy Volunteers:
True
Sex:
ALL
Study:
NCT05236595
Study Brief:
Protocol Section:
NCT05236595