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Results for “RARE DISEASE”

1,033 trials

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Showing 20 of 1,033 results

Testing effectiveness (Phase 2)Active Not RecruitingNCT04969887
What this trial is testing

Combination Immunotherapy in Rare Cancers Under InvesTigation

Who this might be right for
Advanced Biliary Tract CancerNeuroendocrine TumorsFemale Reproductive System Neoplasm+1 more
Olivia Newton-John Cancer Research Institute 240
Testing effectiveness (Phase 2)Looking for participantsNCT06563999
What this trial is testing

Neoadjuvant Umbrella Trial for Patients With Unresectable Stage III NSCLC Harboring Rare Mutations.

Who this might be right for
Lung Cancer Stage IIIMutation
Sun Yat-sen University 120
Testing effectiveness (Phase 2)UnknownNCT04553887
What this trial is testing

Almonertinib as Upfront Treatment for Uncommon EGFR Mutation Harboring Non-Small-Cell Lung Cancer Patients: A Multicenter, Open-Label, Phase II Trial

Who this might be right for
Non Small Cell Lung Cancer
Tianjin Medical University Cancer Institute and Hospital 53
Not applicableLooking for participantsNCT06343558
What this trial is testing

Gait and Balance Impairment in Rare and Very Rare Neurological Diseases

Who this might be right for
Multiple SclerosisParkinson DiseasePeripheral Neuropathy+3 more
Istituto Auxologico Italiano 200
Testing effectiveness (Phase 2)UnknownNCT01824121
What this trial is testing

Clinical Trial to Evaluate Bone Marrow Stem Cell Therapy for PSP, a Rare Form of Parkinsonism

Who this might be right for
Progressive Supranuclear Palsy
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico 25
Post-approval studies (Phase 4)Looking for participantsNCT07142343
What this trial is testing

Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease

Who this might be right for
CHAPLE Disease
Regeneron Pharmaceuticals 5
Testing effectiveness (Phase 2)Study completedNCT06368726
What this trial is testing

Result of tDCS in ASD Children With Comorbidities Like PANDAS, Rare Genetic Diseases or Autoimmune Disorders

Who this might be right for
AttentionVisual Perceptual WeaknessSocial Behavior+2 more
Spanish Foundation for Neurometrics Development 180
Early research (Phase 1)Not Yet RecruitingNCT07787650
What this trial is testing

S-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 15
Early research (Phase 1)Study completedNCT00004533
What this trial is testing

Phase I Randomized Study of Adeno-Associated Virus-CFTR Vector in Patients With Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) 19
Not applicableStudy completedNCT05367115
What this trial is testing

Verification of the Epidemiology and Mortality of Rare Diseases in Taiwan With Real-world Evidence

Who this might be right for
Rare DiseasesEpidemiologyMorbidity+3 more
National Cheng-Kung University Hospital 5,000
Not applicableStudy completedNCT00004660
What this trial is testing

Randomized Study of Sequential Orbital Radiotherapy for Graves' Ophthalmopathy

Who this might be right for
Graves' Disease
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) 42
Testing effectiveness (Phase 2)UnknownNCT03424759
What this trial is testing

Uncommon EGFR AZD9291

Who this might be right for
Non Small Cell Lung Cancer
Samsung Medical Center 37
Post-approval studies (Phase 4)Looking for participantsNCT00004317
What this trial is testing

Pyrimethamine, Sulfadiazine, and Leucovorin in Treating Patients With Congenital Toxoplasmosis

Who this might be right for
Toxoplasmosis
National Institute of Allergy and Infectious Diseases (NIAID) 600
Not applicableStudy completedNCT05245123
What this trial is testing

Psychosocial Situation of Children With Rare Solid Abdominal Tumors and Their Families

Who this might be right for
NeuroblastomaNephroblastomaHepatoblastoma
Universitätsklinikum Hamburg-Eppendorf 100
Not applicableStudy completedNCT06341127
What this trial is testing

Fluoxetine in KCNC1-related Disorder

Who this might be right for
KCNC1 Related DisorderGenetic DiseaseRare Diseases
Holland Bloorview Kids Rehabilitation Hospital 1
Large-scale testing (Phase 3)Looking for participantsNCT07185997
What this trial is testing

Study to Evaluate Efficacy and Safety of Firmonertinib Compared With Investigator's Choice of EGFR Inhibitor as First-Line Treatment in Participants Who Have Locally Advanced or Metastatic NSCLC With EGFR P-Loop and Alpha C-Helix Compressing (PACC) Uncommon Mutations

Who this might be right for
Non-Small-Cell Lung CancerMetastatic Non-Small-Cell Lung CancerAdvanced Non-Small-Cell Lung Cancer+3 more
ArriVent BioPharma, Inc.
Not applicableLooking for participantsNCT06258902
What this trial is testing

Odevixibat Pregnancy and Lactation Surveillance Program: A Study to Evaluate the Safety of Odevixibat During Pregnancy and/or Lactation

Who this might be right for
Pregnancy Related
Ipsen 20
Large-scale testing (Phase 3)Study completedNCT00004939
What this trial is testing

Phase III Randomized, Double-Blind, Placebo-Controlled Study of 3,4-Diaminopyridine for Demyelinating Polyneuropathy

Who this might be right for
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
National Center for Research Resources (NCRR) 34
Large-scale testing (Phase 3)Study completedNCT00790400
What this trial is testing

Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

Who this might be right for
Tuberous Sclerosis Complex (TSC)Lymphangioleiomyomatosis (LAM)
Novartis Pharmaceuticals 118
Large-scale testing (Phase 3)Study completedNCT00014742
What this trial is testing

Phase III Randomized Study of Collagenase in Patients With Residual Stage Dupuytren's Disease

Who this might be right for
Dupuytren's Contracture
State University of New York 72
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