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Results for “Genetic Rare Disease”

449 trials

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Showing 20 of 449 results

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 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)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
Not applicableStudy completedNCT00004274
What this trial is testing

Effect of Estrogen on Mental and Social Functioning in Girls With Turner's Syndrome

Who this might be right for
Turner's Syndrome
National Institute of Neurological Disorders and Stroke (NINDS) 260
Early research (Phase 1)Active Not RecruitingNCT03478462
What this trial is testing

Dose Escalation Study of CLR 131 in Pediatric Relapsed/Refractory Malignant Tumors Including Neuroblastoma and Sarcomas

Who this might be right for
Pediatric Solid TumorPediatric LymphomaPediatric Brain Tumor+5 more
Cellectar Biosciences, Inc. 30
Not applicableStudy completedNCT00004495
What this trial is testing

Randomized Study of Folic Acid Therapy for Hyperhomocysteinemia in Patients With End Stage Renal Disease Receiving Hemodialysis

Who this might be right for
End Stage Renal DiseaseHyperhomocysteinemia
Georgetown University 84
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
Testing effectiveness (Phase 2)Study completedNCT00004488
What this trial is testing

Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease

Who this might be right for
Gaucher's DiseaseOsteopenia
Children's Hospital Medical Center, Cincinnati 82
Not applicableLooking for participantsNCT06213402
What this trial is testing

RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)

Who this might be right for
Sickle Cell DiseaseThalassemiaHemolytic; Anemia, Hereditary, Due to Enzyme Disorder+6 more
Hospital Universitari Vall d'Hebron Research Institute 32,564
Not applicableStudy completedNCT04916977
What this trial is testing

Global Burden and Treatment Trajectiories in Italian Patients With Fabry Disease

Who this might be right for
Fabry Disease
Chiesi Italia 200
Not applicableLooking for participantsNCT06595940
What this trial is testing

Genetic Analysis of Uncommon Disease Presentations in Non-US Populations

Who this might be right for
Undiagnosed DiseasesRare Diseases
National Human Genome Research Institute (NHGRI) 400
Testing effectiveness (Phase 2)Looking for participantsNCT06525636
What this trial is testing

A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia

Who this might be right for
X-linked Hypophosphatemia
Kyowa Kirin Co., Ltd. 39
Early research (Phase 1)UnknownNCT00005896
What this trial is testing

Phase I Pilot Study of CD34 Enriched, Fanconi's Anemia Complementation Group C Gene Transduced Autologous Peripheral Blood Stem Cell Transplantation in Patients With Fanconi's Anemia

Who this might be right for
Fanconi's Anemia
University of Minnesota 6
Not applicableStudy completedNCT06397443
What this trial is testing

Ready to Sail: Evaluating Sailing's Feasibility as Ergotherapy

Who this might be right for
Multiple OsteochondromaOllier DiseaseOsteogenesis Imperfecta
Istituto Ortopedico Rizzoli 8
Not applicableStudy completedNCT04795206
What this trial is testing

Natural Disease Progression in Participants With Choroideremia

Who this might be right for
Choroideremia
Biogen 1,178
Not applicableStudy completedNCT00004441
What this trial is testing

Study of Tauroursodeoxycholic Acid for Hepatobiliary Disease in Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
Children's Hospital Medical Center, Cincinnati 39
Not applicableStudy completedNCT02706639
What this trial is testing

Williams Syndrome (WS) and Supravalvar Aortic Stenosis (SVAS) DNA and Tissue Bank

Who this might be right for
Williams SyndromeSupravalvular Aortic StenosisCardiovascular Disease
National Heart, Lung, and Blood Institute (NHLBI) 305
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