Clinical trials
Genetic Diseases, Inborn trials in Japan
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
21 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2005 | 1 |
| 2012 | 1 |
| 2013 | 1 |
| 2015 | 1 |
| 2016 | 1 |
| 2017 | 2 |
| 2018 | 5 |
| 2019 | 2 |
| 2021 | 2 |
| 2023 | 1 |
| 2024 | 1 |
| 2025 | 1 |
| 2026 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 12 |
| Active, not recruiting | 4 |
| Recruiting | 4 |
| Terminated | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 6 |
| Novo Nordisk A/S | 6 |
| Genzyme, a Sanofi Company | 2 |
| ModernaTX, Inc. | 2 |
| Chiesi Farmaceutici S.p.A. | 1 |
| Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 1 |
| PTC Therapeutics | 1 |
| Pfizer | 1 |
| Shire | 1 |
Top conditions
MeSH conditions the trials were registered with, not their broader categories. A condition with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Genetic Diseases, Inborn | 21 |
| Achondroplasia | 6 |
| Musculoskeletal Diseases | 6 |
| Bone Diseases | 5 |
| Osteochondrodysplasias | 5 |
| Bone Diseases, Developmental | 4 |
| Dwarfism | 4 |
| Blood Coagulation Disorders | 3 |
| Blood Coagulation Disorders, Inherited | 3 |
| Coagulation Protein Disorders | 3 |
Trials
Showing 76 to 21 of 21. Each trial links to its page on Pharma Observatory.
No trials match these filters.
Try a different keyword, or clear a filter.
Recently changed trials
Changes observed in the last 7 days, newest first, site-count changes left out. Source: ClinicalTrials.gov.
No changes to these trials in the last 7 days.
Go deeper on Pharma Observatory
Pharma Observatory connects clinical trials, sponsors, sites and investigators in one searchable platform.