Condition

Genetic Diseases clinical trials and research

• 8Clinical trials
• 12Countries

Newest Genetic Diseases clinical trials

All recruiting trials (8)

• HSCT outcomes in children at Ramathibodi Hospital

  This study tests how hematopoietic stem cell transplantation works in pediatric patients with blood diseases, cancers, and other conditions at Ramathibodi Hospital.

  Recruiting Mahidol University · Ratchathewi, Bangkok · NCT07857746

• AOC 1044 in Duchenne Muscular Dystrophy with Exon 44 Skipping

  This study tests whether intravenous AOC 1044 helps boys with Duchenne muscular dystrophy caused by mutations amenable to exon 44 skipping.

  Recruiting at 20 sites Phase 3 Avidity Biosciences, Inc. · Westmead, New South Wales and 19 other locations · NCT07587242

• Long-term monitoring of adults with type I interferon autoantibodies or genetic interferon defects

  We will follow adults who have autoantibodies to type I interferons or genetic defects that impair interferon responses to see if they develop more infections or autoimmune problems and how antibody levels change over time.

  Recruiting at 9 sites Institut National de la Santé Et de la Recherche Médicale, France · Lille and 8 other locations · NCT06762002

• Natural history of PKP2-related arrhythmogenic cardiomyopathy

  This project will collect past and ongoing health information to see how PKP2-related arrhythmogenic cardiomyopathy progresses in people managed with standard care.

  Recruiting at 2 sites Rocket Pharmaceuticals Inc. · Boston, Massachusetts and 2 other locations · NCT06644742

• Characterizing genetic diseases affecting the immune and neurological systems

  This study is trying to learn more about rare genetic diseases that affect the immune and nervous systems to help find better ways to diagnose and treat them.

  Recruiting Imagine Institute · Paris, Île-de-France Region · NCT06235580

• Collection of genomic samples for childhood genetic diseases

  This study is collecting genetic samples from children with genetic diseases to help find better ways to diagnose and treat these conditions in the future.

  Recruiting Rady Pediatric Genomics & Systems Medicine Institute · San Diego, California · NCT02917460

• Identifying genetic and metabolic diseases in children

  This study is trying to find new genetic causes of rare diseases in children by looking closely at their genes and metabolism.

  Recruiting University of Texas Southwestern Medical Center · Dallas, Texas · NCT02650622

• Exploring genetics and characteristics of familial Mediterranean fever and related disorders

  This study is trying to understand the genetics and symptoms of diseases that cause fever, like familial Mediterranean fever, by looking at patients, their families, and healthy volunteers.

  Recruiting National Institutes of Health Clinical Center (CC) · Washington D.C., District of Columbia and 4 other locations · NCT00001373

Related topics

Quick answers

How many Genetic Diseases clinical trials are recruiting right now?

8, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 12 countries. The most sites are in United States (4 trials), France (3 trials), Netherlands (2 trials), Australia (1 trial), Belgium (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 3: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

A list is a starting point. Tell us your situation in your own words and we'll rank the studies that match and explain why. It's free and needs no account.

Describe your situation →