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Condition: Rare Disorders Synced from ClinicalTrials.gov · July 28, 2026

Recruiting studies: Rare Disorders

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 →

Novel Genetic Disorders of the Immune System
Phase N/A · 1 location(s) · National Institute of Allergy and Infectious Diseases (NIAID · NCT02257892Modulation of THC Effects by CBD: a Dose-ranging Study
Phase 1/Phase 2 · 1 location(s) · Centre hospitalier de l'Université de Montréal (CHUM) · NCT06099379MPN PROGRESSion Registry: Observational Study Tracking Symptoms, Treatments, and Disease Progression in People With Myel
Phase N/A · 2 location(s) · MPN Research Foundation · NCT07362225Evaluation of Socio-professional Inclusion for Young Adults Aged 15-25 Living With a Rare Genetic Disability
Phase N/A · 1 location(s) · Imagine Institute · NCT07527624An International Study on Pediatric Patients With Rare Tumors.
Phase N/A · 1 location(s) · Azienda Ospedaliera di Padova · NCT07072143Protocol N. 2014 - Observational Study on the Quality of Life of Outpatients
Phase N/A · 1 location(s) · IRCCS San Raffaele · NCT06193850Epidural Spinal Cord Stimulation for Lower-limb Impairment in Adrenomyeloneuropathy
NA · 4 location(s) · Third Military Medical University · NCT06796920Diagnostic Research in Patients With Rare Diseases -Solving the Unsolved Rare Diseases
Phase N/A · 1 location(s) · Centre Hospitalier Universitaire Dijon · NCT04024774Personalized Training for People With Rare Neuromuscular Disorders
NA · 5 location(s) · Oslo University Hospital · NCT06708468Genetic Newborn Screening for Rare Diseases Within the Screen4Care Project
NA · 8 location(s) · University Hospital Freiburg · NCT06549218Analysing Outcomes After Prostate Cancer Diagnosis and Treatment in Carriers of Rare Germline Mutations
Phase N/A · 1 location(s) · Institute of Cancer Research, United Kingdom · NCT02705846Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network
Phase N/A · 33 location(s) · National Human Genome Research Institute (NHGRI) · NCT02450851Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center
Phase N/A · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT07205861Gait and Balance Impairment in Rare and Very Rare Neurological Diseases
Phase N/A · 2 location(s) · Istituto Auxologico Italiano · NCT06343558DETERMINE Trial Treatment Arm 02: Atezolizumab in Adult, Paediatric and Teenage/Young Adult Patients With Cancers With H
Phase 2/Phase 3 · 27 location(s) · Cancer Research UK · NCT05770102Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)
Phase 2 · 28 location(s) · Shionogi · NCT07123155Functional and Morphological Characterization of Multiple Osteochondromas Disorder
Phase N/A · 1 location(s) · Istituto Ortopedico Rizzoli · NCT06703736Orodental Manifestations of Rare Diseases
Phase N/A · 1 location(s) · University Hospital, Strasbourg, France · NCT02397824Marfan Syndrome (MFS) and Facial Dysmorphism: Non-invasive 3D Assessment
Phase N/A · 1 location(s) · IRCCS Policlinico S. Donato · NCT05702476Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
Phase N/A · 3 location(s) · Columbia University · NCT06839469Study to Evaluate Sutetinib Maleate Capsule in Locally Advanced or Metastatic Non-small Cell Lung Cancer
Phase 2 · 26 location(s) · Teligene US · NCT05168566Group CBT in Adolescents With Fragile X Syndrome and in Adolescents With Autism Spectrum Disorder
NA · 1 location(s) · Bambino Gesù Hospital and Research Institute · NCT06677866Collection of Biological Samples From Patients With Rare Neurological Diseases
Phase N/A · 1 location(s) · University Hospital, Toulouse · NCT04698421Genetics of Inherited Eye Disease
Phase N/A · 1 location(s) · National Eye Institute (NEI) · NCT02471287Natural History, Pathogenesis and Outcome of Melorheostosis A Rare Osteosclerotic Disease
Phase N/A · 1 location(s) · National Institute of Arthritis and Musculoskeletal and Skin · NCT02504879Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Cor
Phase N/A · 13 location(s) · Fondazione SISA (Societa Italiana per lo Studio della Arteri · NCT07447648A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
Phase N/A · 9 location(s) · ITF Therapeutics LLC · NCT07127978Delineating the Molecular Spectrum and the Clinical, Imaging and Neuronal Phenotype of Chopra-Amiel-Gordon Syndrome
Phase N/A · 1 location(s) · Boston Children's Hospital · NCT05528744FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
Phase N/A · 1 location(s) · xCures · NCT06539169Holistic Mixed Approaches to Capture the Real Life of Children With Rare Eye Diseases
Phase N/A · 1 location(s) · University Hospital, Strasbourg, France · NCT06070467
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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.