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Results for “Rare Disorders”

1,030 trials

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Showing 20 of 1,030 results

Not applicableLooking for participantsNCT06676046
What this trial is testing

Natural History of Uncommon Dyslipidemias, Rare Lipid Disorders and Unusual Atherosclerotic Conditions

Who this might be right for
DyslipidemiaAtherosclerosis
National Heart, Lung, and Blood Institute (NHLBI) 1,500
Not applicableLooking for participantsNCT04151342
What this trial is testing

CAnadian CAncers With Rare Molecular Alterations (CARMA) - Basket Real-world Observational Study (BROS)

Who this might be right for
CancerMalignancies MultipleMalignant Solid Tumor+7 more
University Health Network, Toronto 5,500
Large-scale testing (Phase 3)Looking for participantsNCT07668856
What this trial is testing

Cannabidiol as add-on Therapy for Children With Refractory Epilepsy (CBD-uN1que), a High-quality Individualized Approach: a Series of N-of-1 Trials

Who this might be right for
Refractory Epilepsy in ChildrenRefractory EpilepsyEpilepsy
UMC Utrecht 50
Large-scale testing (Phase 3)Study completedNCT03936777
What this trial is testing

Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome

Who this might be right for
Dravet SyndromeLennox Gastaut SyndromeEpileptic Encephalopathy
Zogenix, Inc. 412
Not applicableStudy completedNCT01914172
What this trial is testing

Health Needs of Patients With Kallmann Syndrome

Who this might be right for
Kallmann SyndromeCongenital Hypogonadotropic HypogonadismIdiopathic Hypogonadotropic Hypogonadism
Centre Hospitalier Universitaire Vaudois 249
Not applicableLooking for participantsNCT05805202
What this trial is testing

Functional Implications of Rare Gene Mutations in aHUS Open the Door to Personalized Therapy

Who this might be right for
Atypical Hemolytic Uremic Syndrome
Mario Negri Institute for Pharmacological Research 112
Early research (Phase 1)Study completedNCT00004806
What this trial is testing

Phase I Study of Liposome-Mediated Gene Transfer in Patients With Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
University of Alabama at Birmingham 9
Not applicableStudy completedNCT00004773
What this trial is testing

Study of Cardiac and Paroxysmal Abnormalities in Rett Syndrome

Who this might be right for
Rett Syndrome
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 95
Testing effectiveness (Phase 2)Study completedNCT01854294
What this trial is testing

GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS)

Who this might be right for
Amyotrophic Lateral Sclerosis
Genervon Biopharmaceuticals, LLC 12
Not applicableLooking for participantsNCT07737756
What this trial is testing

A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia

Who this might be right for
Spinal Muscular Atrophy
Novartis Pharmaceuticals 6
Not applicableEnded earlyNCT03472807
What this trial is testing

EXOme Rare Cancers in Children (EXOCARE)

Who this might be right for
Predisposition, GeneticCancer ChildhoodDevelopment Delay
University Hospital, Angers 169
Not applicableStudy completedNCT02793063
What this trial is testing

Osteogenesis Imperfecta (OI) Quality of Life Survey Pilot Project 2

Who this might be right for
Osteogenesis ImperfectaBrittle Bone Disorders
University of South Florida 300
Early research (Phase 1)Study completedNCT00004471
What this trial is testing

Phase I Pilot Study of Gene Therapy for Cystic Fibrosis Using Cationic Liposome Mediated Gene Transfer

Who this might be right for
Cystic Fibrosis
University of Alabama at Birmingham 9
Not applicableLooking for participantsNCT06969209
What this trial is testing

Brain Aging in Phenylketonuria

Who this might be right for
Phenylketonuria (PKU)
Insel Gruppe AG, University Hospital Bern 90
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
Not applicableUnknownNCT05612230
What this trial is testing

A Lifecourse Map of Diseases in China

Who this might be right for
Common DiseasesRare Diseases
Shandong University 40,000,000
Not applicableActive Not RecruitingNCT04358107
What this trial is testing

Optimal Methods of Disease Progression and Survival Analysis in Children and Adults Patients With Adrenocortical Cancer (ACC)

Who this might be right for
Adrenocortical Carcinoma
National Cancer Institute (NCI) 1,000
Not applicableStudy completedNCT07102875
What this trial is testing

Ready to Sail 2: A Pilot Study of Sail-Assisted Telerehabilitation in Rare Skeletal Diseases

Who this might be right for
Rare Bone Disorders
Istituto Ortopedico Rizzoli 24
Not applicableStudy completedNCT04694053
What this trial is testing

Fournier's Gangrene in Patient With Uncontrolled Type 2 Diabetes

Who this might be right for
Fournier Gangrene
University of Catania 1
Not applicableEnrolling By InvitationNCT03836300
What this trial is testing

Parent and Infant Inter(X)Action Intervention (PIXI)

Who this might be right for
Fragile X SyndromeAngelman SyndromePrader-Willi Syndrome+11 more
RTI International 120
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