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Research for Individualized Therapeutics in Rare Genetic Disease

NCT05236595 · Status: ENROLLING_BY_INVITATION · Type: OBSERVATIONAL · Enrollment: 50

Last updated 2026-01-20

No results posted yet for this study

Summary

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.

Conditions

  • Rare Genetic Disease
  • Undiagnosed Diseases

Interventions

OTHER

Individualized drug matching per genetic disease

Patient phenotype and samples will be evaluated for individualized therapeutic drug development

Sponsors & Collaborators

Principal Investigators

  • Margot A Cousin, Ph.D. · Mayo Clinic

Eligibility

Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2021-11-24
Primary Completion
2026-11-30
Completion
2026-11-30

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05236595 on ClinicalTrials.gov