| PHASE1 | 138 |
| PHASE1, PHASE2 | 77 |
| PHASE2 | 216 |
| PHASE2, PHASE3 | 49 |
| PHASE3 | 71 |
| PHASE4 | 20 |
| NA | 527 |
| Not Applicable | 440 |
| RECRUITING | 273 |
| ACTIVE_NOT_RECRUITING | 78 |
| NOT_YET_RECRUITING | 83 |
| COMPLETED | 747 |
| Databases | OMIMOrphanetClinicalTrialsPubMed |
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