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Results for “RARE DISEASE”

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Showing 20 of 0 results

Not applicableStudy completedNCT00006142
What this trial is testing

Study of a Phenylalanine Restricted Diet During Pregnancy to Prevent Symptoms in Offspring of Patients With Phenylketonuria

Who this might be right for
Phenylketonuria
National Center for Research Resources (NCRR)
Not applicableLooking for participantsNCT01087320
What this trial is testing

Genome Medical Sequencing for Gene Discovery

Who this might be right for
Intellectual DisabilitiesCongenital AnomalyRare Disorders
National Human Genome Research Institute (NHGRI) 2,000
Not applicableStudy completedNCT00001686
What this trial is testing

Evaluation, Treatment, and Natural History of Children and Young Adults With Cancer or Rare Diseases

Who this might be right for
Ewing's SarcomaLeukemiaLymphoma+2 more
National Cancer Institute (NCI) 482
Large-scale testing (Phase 3)Study completedNCT00004402
What this trial is testing

Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis

Who this might be right for
Osteopetrosis
FDA Office of Orphan Products Development 30
Testing effectiveness (Phase 2)Study completedNCT00004357
What this trial is testing

Absorption of Corticosteroids in Children With Juvenile Dermatomyositis

Who this might be right for
Vasculitis, HypersensitivityConnective Tissue DiseasesDermatomyositis+1 more
Northwestern University 6
Not applicableLooking for participantsNCT05868629
What this trial is testing

Clinical Study To Further Evaluate The Efficacy Of Dabrafenib Plus Trametinib In Patients With Rare BRAF V600E Mutation-Positive Unresectable or Metastatic Solid Tumors

Who this might be right for
Rare Unresectable or Metastatic BRAF V600E Mutation-positive Solid Tumors
Novartis Pharmaceuticals 40
Large-scale testing (Phase 3)Study completedNCT00004422
What this trial is testing

Intravenous Pertussis Immune Globulin in Patients With Severe Childhood Pertussis Infection

Who this might be right for
PertussisWhooping Cough
IWK Health Centre 174
Not applicableEnrolling By InvitationNCT07163260
What this trial is testing

Defeat Rare Disease in Asia and Pacific (APAC) DRDA - Haemophilia

Who this might be right for
Haemophilia
Novo Nordisk A/S 244
Not applicableLooking for participantsNCT02141308
What this trial is testing

OCT in Rare Chorioretinal Diseases

Who this might be right for
Retinal Artery OcclusionsPolypoidal Choroidal VasculopathyRetinal Arterial Macroaneurysm+2 more
Oregon Health and Science University 150
Not applicableLooking for participantsNCT04463316
What this trial is testing

GROWing Up With Rare GENEtic Syndromes

Who this might be right for
Prader-Willi SyndromePWS-like SyndromeSilver Russel Syndrome+30 more
dr. Laura C. G. de Graaff-Herder 600
Early research (Phase 1)Study completedNCT01344798
What this trial is testing

Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C

Who this might be right for
Limb Girdle Muscular Dystrophy Type 2CGamma-sarcoglycanopathy
Genethon 9
Not applicableLooking for participantsNCT03169010
What this trial is testing

Registration Study for Rare Type of Pulmonary Hypertension

Who this might be right for
Pulmonary Hypertension
China National Center for Cardiovascular Diseases 2,000
Not applicableStudy completedNCT03077308
What this trial is testing

Rare Diseases Clinical Research Network: Neurophysiological Correlates

Who this might be right for
Rett Syndrome, Preserved Speech VariantMecp2 Duplication SyndromeRett-related Disorders
University of Alabama at Birmingham 185
Not applicableLooking for participantsNCT07074015
What this trial is testing

IntelliWell: An AI-Assisted Imaging Platform for Detection and Location of Ultra-Rare Testicular Sperm in Surgical Specimens

Who this might be right for
InfertilityAzoospermia
Brigham and Women's Hospital 20
Not applicableStudy completedNCT00004489
What this trial is testing

Randomized Study of Alendronate in Adult Patients With Cystic Fibrosis Related Osteoporosis

Who this might be right for
OsteoporosisCystic Fibrosis
University of North Carolina 60
Testing effectiveness (Phase 2)Study completedNCT02358538
What this trial is testing

Open-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies

Who this might be right for
Epilepsy
Marinus Pharmaceuticals 30
Testing effectiveness (Phase 2)Looking for participantsNCT06804655
What this trial is testing

Pharmacoscopy for Patients With Refractory Primary Brain Tumors

Who this might be right for
Brain (Nervous System) CancersGlioblastomaGlioma+5 more
University of Zurich 40
Not applicableStudy completedNCT05643274
What this trial is testing

Use of Long Read Genome Sequencing in Patients Suffering From Neurodevelopmental Troubles

Who this might be right for
Genetic DiseaseNeurologic DisorderDevelopmental Delay Disorder+1 more
Nantes University Hospital 10
Not applicableStudy completedNCT00010374
What this trial is testing

Electrical Activation of The Diaphragm for Ventilatory Assist

Who this might be right for
Spinal Cord InjuryQuadriplegia
Synapse Biomedical 54
Large-scale testing (Phase 3)Looking for participantsNCT04220190
What this trial is testing

RAPA-501 Therapy for ALS

Who this might be right for
Amyotrophic Lateral Sclerosis
Rapa Therapeutics LLC 41
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