Recruiting studies: Orphan Diseases

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

Automatic Phenotyping of Patients on 2D Photography
Phase N/A · 1 location(s) · Imagine Institute · NCT06219421 Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome
Phase 2 · 2 location(s) · Connecta Therapeutics, S.L. · NCT07654114 Gene Discovery Core, The Manton Center
Phase N/A · 1 location(s) · Boston Children's Hospital · NCT02743845 RAPA-501 Therapy for ALS
Phase 2/Phase 3 · 1 location(s) · Rapa Therapeutics LLC · NCT04220190 European Management Platform for Childhood Interstitial Lung Diseases - chILD-EU Register and Biobank
Phase N/A · 3 location(s) · Matthias Griese · NCT02852928 Orphan Europe Carbaglu® Surveillance Protocol
Phase N/A · 3 location(s) · Nicholas Ah Mew · NCT03409003 Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Phase N/A · 2 location(s) · Sanford Health · NCT01793168 A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy
NA · 1 location(s) · HuidaGene Therapeutics Co., Ltd. · NCT06615206 First in Human Study of EMB-07 in Locally Advanced/Metastatic Solid Tumors or Relapse/Refractory Lymphoma
Phase 1 · 10 location(s) · EpimAb Biotherapeutics (Suzhou)Co., Ltd. · NCT05607498 Reversing External-beam Radiotherapy-associated Fibrosis Syndrome: an Interventional Bayesian Adaptive Randomized-contro
Phase 2 · 1 location(s) · M.D. Anderson Cancer Center · NCT06912763 Vascular Disease Discovery Protocol
Phase N/A · 1 location(s) · National Heart, Lung, and Blood Institute (NHLBI) · NCT03538639 Education & Care in RARE: Efficacy of Targeted Psychoeducational Intervention Among Pediatric Rare Disease Patients
NA · 7 location(s) · Medical University of Vienna · NCT06729554

Look up another condition

Patient organization or foundation? You can brand this page for your community — your logo, your resources, same honest registry data, kept current daily. Ask about branding →

This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.