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Results for “RARE DISEASE”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Active Not RecruitingNCT05256290
What this trial is testing

Phase 1/2 Study of Silevertinib (BDTX-1535) in Patients With Glioblastoma or Non-Small Cell Lung Cancer With EGFR Mutations

Who this might be right for
Non-Small Cell Lung CancerAdvanced Non-Small Cell Squamous Lung CancerMetastatic Lung Non-Small Cell Carcinoma+7 more
Black Diamond Therapeutics, Inc. 200
Testing effectiveness (Phase 2)UnknownNCT00004315
What this trial is testing

Phase II Pilot Study to Compare the Bioavailability of Buffered, Enteric-Coated Ursodiol With Unmodified Ursodiol for Chronic Cholestatic Liver Disease and Cystic Fibrosis-Associated Liver Disease

Who this might be right for
Cystic FibrosisGastrointestinal DiseasesCholestasis
National Center for Research Resources (NCRR) 20
Testing effectiveness (Phase 2)Study completedNCT00004980
What this trial is testing

Repetitive Transcranial Magnetic Stimulation for "Voices"

Who this might be right for
Schizophrenia
Yale University 50
Not applicableUnknownNCT04152876
What this trial is testing

Functional Genomics of Rare Genetic Diseases: Realization of Innovative Tools With High Diagnostic Power

Who this might be right for
Rare Diseases
Neuromed IRCCS 300
Not applicableUnknownNCT04646850
What this trial is testing

Long-term Treatment With the Ketogenic Diet in Epilepsy

Who this might be right for
EpilepsyGLUT1DS1PDH Deficiency
Oslo University Hospital 30
Not applicableStudy completedNCT03067142
What this trial is testing

Proteomics of Primary Hyperoxaluria Type 1

Who this might be right for
Primary Hyperoxaluria Type 1
Ann & Robert H Lurie Children's Hospital of Chicago 93
Not applicableStudy completedNCT01250223
What this trial is testing

Study of Prognosis of Follicular Lymphoma Through a Prospective Collection of Data (F2-study)

Who this might be right for
Lymphoma, Follicular
Associazione Angela Serra per la ricerca sul cancro 1,093
Not applicableLooking for participantsNCT06037473
What this trial is testing

The Efficacy and Safety of PEGylated GH for the Treatment of Short Stature in Chinese Children-GLOBE Reg

Who this might be right for
Growth Hormone TreatmentGrowth Disorders
Beijing Children's Hospital 2,600
Not applicableEnrolling By InvitationNCT05368038
What this trial is testing

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Who this might be right for
Acid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more
Albert Einstein College of Medicine 100,000
Not applicableLooking for participantsNCT05259605
What this trial is testing

Observational Study for Assessing Treatment and Outcome of Patients With Primary Brain Tumours Using cIMPACT-NOW and 2021 WHO Classification

Who this might be right for
GliomaGlioneuronal TumorChoroid Plexus Tumor+10 more
European Organisation for Research and Treatment of Cancer - EORTC 1,650
Testing effectiveness (Phase 2)Study completedNCT00004787
What this trial is testing

Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes

Who this might be right for
Shwachman SyndromeFanconi's AnemiaDyskeratosis Congenita+1 more
National Center for Research Resources (NCRR) 20
Not applicableEnrolling By InvitationNCT07417267
What this trial is testing

Clean Air for Rare MUcociliary Clearance dIsorders

Who this might be right for
Primary Ciliary Dyskinesia (PCD)Cystic Fibrosis (CF)
University of Cyprus 62
Large-scale testing (Phase 3)Looking for participantsNCT05770544
What this trial is testing

DETERMINE Trial Treatment Arm 03: Entrectinib in Adult, Paediatric and Teenage/Young Adult Patients With ROS1 Gene Fusion-Positive Cancers.

Who this might be right for
Haematological MalignancyMalignancyMalignant Neoplasm+8 more
Cancer Research UK 30
Large-scale testing (Phase 3)Ended earlyNCT02505087
What this trial is testing

Pharmacological Treatment of a Rare Genetic Disease: N-acetylcysteine in Myopathy Associated Selenoprotein N-related Myopathy (SEPN1-RM)

Who this might be right for
Selenoprotein N-related Myopathy
Assistance Publique - Hôpitaux de Paris 7
Not applicableStudy completedNCT05422833
What this trial is testing

Effectiveness of Medical Management of Fibrous Dysplasia of Bone.

Who this might be right for
Fibrous Dysplasia of BoneMcCune Albright Syndrome
Hospices Civils de Lyon 528
Not applicableUnknownNCT06070467
What this trial is testing

Holistic Mixed Approaches to Capture the Real Life of Children With Rare Eye Diseases

Who this might be right for
Eye DiseasesSevere Loss of VisionBlindness+1 more
University Hospital, Strasbourg, France 154
Not applicableStudy completedNCT00004378
What this trial is testing

Stem Cell Transplantation (SCT) for Genetic Diseases

Who this might be right for
ThrombocytopeniaMetachromatic LeukodystrophyFanconi's Anemia+3 more
National Center for Research Resources (NCRR)
Not applicableLooking for participantsNCT06219421
What this trial is testing

Automatic Phenotyping of Patients on 2D Photography

Who this might be right for
DysmorphiaOrphan DiseasesDysmorphies Craniofaciales
Imagine Institute 22,000
Not applicableLooking for participantsNCT07336394
What this trial is testing

Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques

Who this might be right for
Danon DiseaseFabry DiseaseCardiac Amyloidosis+4 more
Chinese Academy of Medical Sciences, Fuwai Hospital 1,000
Not applicableLooking for participantsNCT03964480
What this trial is testing

Prospective Observational International Registry of Patients With Newly Diagnosed Peripheral T Cell Lymphoma.

Who this might be right for
Peripheral T-Cell Lymphoma
Associazione Angela Serra per la ricerca sul cancro 1,000
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