Research for Individualized Therapeutics in Rare Genetic Disease

Part of paid clinical trials in Scottsdale, Arizona.

Sponsor
Mayo Clinic
Study ID
NCT05236595
Status
Enrolling By Invitation

Conditions

  • Rare Genetic Disease
  • Undiagnosed Diseases

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Accepted

Interventions

  • Individualized drug matching per genetic disease — OTHER
    Patient phenotype and samples will be evaluated for individualized therapeutic drug development

Study Details

The purpose of this research study is to identify individuals that have a rare genetic disease without an adequate therapeutic strategy that might be treatable with drug developed to target the disease-causing genetic alteration.

Key Dates

Start date
Nov 24, 2021
Status verified
Jan 2026
Primary completion
Nov 30, 2026
Completion
Nov 30, 2026

Study Design

Enrollment
50 participants (estimated)

Arms

  • Arm: Rare genetic disease individualized drug development screening candidate
    Patients with targetable disease-causing genetic alterations will be evaluated on a case by case basis. The research study will utilize biospecimens to determine if an individualized therapeutic may be developed as a possible treatment option. If an individualized therapeutic drug can be developed, a future IND FDA application (n=1) will be filed.

Primary Outcome Measure

Enrollment of study participants [ Time Frame: 5 years ]

Locations (3)

FacilityCityStateZIPSite coordinators
Mayo Clinic in ArizonaScottsdaleArizona85259-
Mayo Clinic FloridaJacksonvilleFlorida32224-
Mayo Clinic RochesterMinneotaMinnesota55905-

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By research site
Mayo Clinic in Arizona· Scottsdale, AZMayo Clinic Florida· Jacksonville, FLMayo Clinic Rochester· Minneota, MN

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