Rare Disease clinical trials
Data updated daily from ClinicalTrials.gov, last refresh 2026-11-14
What changed in the last 30 days
5 trials changed in the last 30 days: 0 stopped, 2 with notable changes, 3 with minor changes only.
- Notable: FACE · Marfan Syndrome (MFS) and Facial Dysmorphism: Non-invasive 3D Assessment Primary completion pushed from Jun 2026 to Jun 2027
- Minor: IPaNeMA · Neurovegetative Pattern · Thoracic Aortic Aneurysms (TAA) Study completion pushed from Jan 2026 to Jan 2027
- Notable: PULSEWAVE · Aortic Stiffness · Genetic Aortopathies Primary completion pushed from Nov 30, 2025 to Feb 28, 2027
- Minor: BEAMER · BEhavioral and Adherence Model for Improving Quality, Health Outcomes and Cost-Effectiveness of healthcaRe Trial completed
- Minor: MUSCLE · Analysis of Muscular Properties · MFS and EDS Trial completed
- Stopped: terminated, suspended or withdrawn
- Notable: the plan changed
- Minor: routine updates
New sponsor entrants, last 90 days sponsors with their first trial in this area
| First posted | Trial | Sponsor | Phase |
|---|---|---|---|
| 2026-09-24 | NCT07839754 EpiSign International: Expanding Epigenomic Testing in Rare Disease | University of Manchester new |
Active trials by phase
| Phase 2 | 1 | |
| Phase 2/3 | 1 |
Not charted: 58 active trials with no phase listed on ClinicalTrials.gov (typically behavioural, device, procedure and observational studies).
Top sponsors by active trials in this area
IRCCS Policlinico S. Donato (7) · University Hospital Tuebingen (4) · Centre Hospitalier Universitaire Dijon (3) · Assistance Publique - Hôpitaux de Paris (2) · Fondazione Policlinico Universitario Agostino Gemelli IRCCS (2) · Imagine Institute (2) · Mayo Clinic (2) · National Human Genome Research Institute (NHGRI) (2) · TESS Research Foundation (2) · University Hospital, Lille (2)
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