Condition
Genetic Diseases, Inborn clinical trials and research
As of November 12, 2026, Find a Trial lists 5 Genetic Diseases, Inborn clinical trials that are recruiting, at sites in 7 countries (most in Italy (2), France (1), Japan (1)). 1 was first registered in the last 30 days. By phase: Phase 3 (2). Every study is explained in plain English. Data from ClinicalTrials.gov and NIH RePORTER, updated November 12, 2026.
- 5Clinical trials
- 1New in 30 days
- 7Countries
Newest Genetic Diseases, Inborn clinical trials
| Study | Phase | Where | Status | Registered |
|---|---|---|---|---|
| Leopard: using multi-omics to boost genetic diagnosis for neurodevelopmental disorders | — | Genova | Recruiting | Oct 20, 2026 |
| Non-Invasive Prenatal Exclusion Diagnosis for Severe Monogenic Disorders | — | Paris, Île-de-France Region | Recruiting | Mar 13, 2026 |
| Oleogel-S10 gel for healing skin wounds in Japanese patients with epidermolysis bullosa. | Phase 3 | Fukuoka and 5 other locations | Recruiting | Apr 8, 2025 |
| Evaluating Large Neutral Amino Acids for Treating Phenylketonuria | — | Copenhagen and 1 other locations | Recruiting | Mar 29, 2024 |
| Evaluating a single dose of CTX001 for treating transfusion-dependent beta-thalassemia and severe sickle cell disease | Phase 3 | New York, New York and 5 other locations | Recruiting | Jul 28, 2022 |
All recruiting trials (5)
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Leopard: using multi-omics to boost genetic diagnosis for neurodevelopmental disorders
This study tests a multi-omics approach to find genetic explanations for neurodevelopmental disorders in people with moderate-to-severe intellectual disability.
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Non-Invasive Prenatal Exclusion Diagnosis for Severe Monogenic Disorders
This study checks if a blood test using fetal DNA from mom can help identify or exclude certain severe monogenic disorders without invasive procedures, for couples at risk.
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Oleogel-S10 gel for healing skin wounds in Japanese patients with epidermolysis bullosa.
We will try Oleogel‑S10 gel to see if it closes or shrinks wounds in Japanese children and adults with junctional or dystrophic epidermolysis bullosa.
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Evaluating Large Neutral Amino Acids for Treating Phenylketonuria
This study is testing if Large Neutral Amino Acids can be a better treatment for adults with Phenylketonuria than the usual diet that limits phenylalanine.
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Evaluating a single dose of CTX001 for treating transfusion-dependent beta-thalassemia and severe sickle cell disease
This study is testing a single dose of a new treatment called CTX001 to see if it can help people with transfusion-dependent beta-thalassemia or severe sickle cell disease feel better.
Related topics
Quick answers
How many Genetic Diseases, Inborn clinical trials are recruiting right now?
5, as of November 12, 2026. 1 of them was first registered in the last 30 days. We update this list each time we refresh our data from ClinicalTrials.gov.
Where are these trials running?
In 7 countries. The most sites are in Italy (2 trials), France (1 trial), Japan (1 trial), Germany (1 trial), Saudi Arabia (1 trial). Each study page lists its sites and which ones are recruiting.
What phases are they in?
Phase 3: 2. 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.