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Results for “RARE DISEASE”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT00872729
What this trial is testing

Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis

Who this might be right for
Cystinosis
Amgen 9
Not applicableStudy completedNCT01380054
What this trial is testing

Pulmonary Hypertension, Quality of Life and Psychosocial Factors

Who this might be right for
Pulmonary Hypertension
University Hospital, Montpellier 55
Not applicableLooking for participantsNCT05031507
What this trial is testing

Study of Skeletal Disorders

Who this might be right for
Skeletal Disorders
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 100
Not applicableStudy completedNCT00006137
What this trial is testing

Pilot Study of Enalapril and Renal Function in Patients With IgA Nephropathy

Who this might be right for
IGA Glomerulonephritis
National Center for Research Resources (NCRR) 43
Not applicableLooking for participantsNCT07665307
What this trial is testing

Delayed Toxicities Post-CAR-T

Who this might be right for
Multiple Myeloma
Icahn School of Medicine at Mount Sinai 30
Large-scale testing (Phase 3)Ended earlyNCT04221451
What this trial is testing

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

Who this might be right for
Tay-Sachs DiseaseSandhoff Disease
Genzyme, a Sanofi Company 75
Not applicableLooking for participantsNCT06856902
What this trial is testing

BEhavioral and Adherence Model for Improving Quality, Health Outcomes and Cost-Effectiveness of healthcaRe

Who this might be right for
Cardiovascular DiseasesEndocrinologyInmunology+3 more
Technical University of Madrid 3,100
Testing effectiveness (Phase 2)UnknownNCT02859792
What this trial is testing

Riluzole in the Treatment of Spasticity in the Traumatic Chronic Spinal Cord Injury Condition

Who this might be right for
Spinal Cord Injury
Assistance Publique Hopitaux De Marseille 90
Testing effectiveness (Phase 2)Looking for participantsNCT07314216
What this trial is testing

Firmonertinib Combined With Definitive Radiotherapy in Stage III Unresectable EGFR Uncommon Mutant Pulmonary Adenocarcinoma

Who this might be right for
NSCLC (Non-small Cell Lung Carcinoma)Stage IIIEGFR Uncommon Mutations
Hunan Cancer Hospital 15
Not applicableUnknownNCT04702295
What this trial is testing

ERN ReCONNET Study on COVID-19 Vaccination in Rare and Complex Connective Tissue Disease (VACCINATE)

Who this might be right for
Connective Tissue DiseasesRare DiseasesRheumatologic Disorder
University of Pisa 250
Not applicableWithdrawnNCT06227429
What this trial is testing

A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients

Who this might be right for
Hereditary Tyrosinemia, Type I
Swedish Orphan Biovitrum
Early research (Phase 1)UnknownNCT00004307
What this trial is testing

Study of Treatment and Metabolism in Patients With Urea Cycle Disorders

Who this might be right for
Amino Acid Metabolism, Inborn Errors
National Center for Research Resources (NCRR) 66
Not applicableStudy completedNCT02464644
What this trial is testing

Frequency of Common Medical Conditions in People With and Without HHT

Who this might be right for
Hereditary Hemorrhagic Telangiectasia (HHT)
Imperial College London 2,174
Not applicableStudy completedNCT01938014
What this trial is testing

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Who this might be right for
Mucopolysaccharidosis Type I (MPS I)Mucopolysaccharidosis Type II (MPS II)Mucopolysaccharidosis Type III (MPS III)+2 more
University of Chicago 19
Testing effectiveness (Phase 2)Study completedNCT00004688
What this trial is testing

Phase II Study of Carmustine, Streptozocin, and Mercaptopurine for Refractory or Recurrent Brain Neoplasms

Who this might be right for
Anaplastic AstrocytomaAnaplastic OligodendrogliomaMixed Gliomas+2 more
Emory University 35
Large-scale testing (Phase 3)Study completedNCT04966741
What this trial is testing

Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity

Who this might be right for
Bardet-Biedl SyndromePOMC Deficiency ObesityPCSK1 Deficiency Obesity+1 more
Rhythm Pharmaceuticals, Inc. 12
Not applicableLooking for participantsNCT06855901
What this trial is testing

Home Ultra-long Term EEG Monitoring for Rare Epilepsies and Developmental and Epileptic Encephalopathies

Who this might be right for
EpilepsiesEpilepsies, FocalEpileptic Encephalopathy
Azienda Ospedaliero-Universitaria di Modena 30
Not applicableLooking for participantsNCT02471287
What this trial is testing

Genetics of Inherited Eye Disease

Who this might be right for
Genetic Eye Disease
National Eye Institute (NEI) 1,500
Not applicableStudy completedNCT00004793
What this trial is testing

Pilot Study of the Effect of Baclofen and Bromocriptine on Luteinizing Hormone Secretion in Pubertal Children

Who this might be right for
Growth Disorders
National Center for Research Resources (NCRR) 4
Early research (Phase 1)Study completedNCT01046487
What this trial is testing

Imatinib Mesylate And Cyclophosphamide In Metronomic Administration: Dose Escalation Study Of Imatinib Mesylate

Who this might be right for
Cancer
Centre Oscar Lambret 26
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