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

First listed
Feb 11, 2022
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-

Find similar trials in Scottsdale, AZ

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

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