Rare Disease Trial, By Invitation
voxsanity.com.au · Eligibility summary from public government registries · 18 August 2026 · not medical advice
Who may and may not be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who may be able to join
- The person must have a Mayo Clinic, another health system ID, or some other unique identifying number.
- The person must be able to give their informed consent to participate.
- The person must have a confirmed or strongly suspected genetic disorder, identified by a doctor or genetic counsellor, with a specific genetic change found through laboratory testing.
- The genetic change must be one that researchers believe could potentially be targeted by a type of drug called an antisense oligonucleotide (a treatment designed to interact with genetic material in specific ways to correct how genes work).
- The person must be able to travel to a Mayo Clinic location for ongoing treatment if a therapy is developed.
- Based on current medical understanding, treatment at the person's current stage of illness would likely be beneficial.
- Alternatively, a biological family member of someone already enrolled in the trial may also be eligible, provided they can give informed consent — or have a legally authorised representative who can do so on their behalf.
Who may not be able to join
Each point below is a reason the trial team may not be able to accept someone. It is not a list of requirements to meet.
- People whose personal circumstances would make it difficult to follow the study's requirements.
- People who are currently held in an institution such as a federal medical prison.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Enrollment of study participants; Collection of biospecimens; Partnered research with external entities; Future IND applications; Determine natural history and clinical baseline; Determine individualized therapeutic efficacy; Publish findings
Can't join this trial?
Data last synced from ClinicalTrials.gov: 13 August 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.