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Results for “RARE DISEASE”

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Showing 20 of 0 results

Not applicableLooking for participantsNCT04758325
What this trial is testing

The Sarcoma Biology and Outcome Project

Who this might be right for
SarcomaMalignant MesenchymomaSarcoma of Bone and Connective Tissue
Prof. Dr. Richard F Schlenk 3,000
Not applicableStudy completedNCT00005650
What this trial is testing

Genetic Study of Patients With Primary Ciliary Dyskinesia

Who this might be right for
Primary Ciliary Dyskinesia
National Center for Research Resources (NCRR) 180
Not applicableLooking for participantsNCT05499091
What this trial is testing

Functional Study to Indentify Genetic Etiology of Rare Diseases - ORIGIN

Who this might be right for
Rare DiseasesGenetic Disease
University Hospital, Angers 1,200
Testing effectiveness (Phase 2)UnknownNCT04371718
What this trial is testing

Effect of JKB-122 on Prednisolone and Azathioprine Induced Remission in Autoimmune Hepatitis (AIH)

Who this might be right for
Autoimmune Hepatitis
TaiwanJ Pharmaceuticals Co., Ltd 120
Testing effectiveness (Phase 2)WithdrawnNCT03801889
What this trial is testing

SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias

Who this might be right for
Iron OverloadBeta-Thalassemia
Abfero Pharmaceuticals, Inc
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07750990
What this trial is testing

An Extension Study of S-606001 in Participants With Late-onset Pompe Disease (LOPD)

Who this might be right for
Pompe Disease
Shionogi 60
Testing effectiveness (Phase 2)Study completedNCT00004276
What this trial is testing

Phase II Placebo Controlled Study of Thalidomide in Patients With Mycobacterial and HIV Infections

Who this might be right for
HIV InfectionsMycobacterium InfectionsTuberculosis
National Center for Research Resources (NCRR) 50
Large-scale testing (Phase 3)Study completedNCT02432144
What this trial is testing

UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Who this might be right for
Sly SyndromeMPS VIIMucopolysaccharidosis+1 more
Ultragenyx Pharmaceutical Inc 12
Not applicableLooking for participantsNCT06285500
What this trial is testing

Umbrella Study for Single Patient Treatments in Oncology

Who this might be right for
Advanced CancerRare Diseases
University Health Network, Toronto 400
Not applicableNot Yet RecruitingNCT07731971
What this trial is testing

Digital Outcome Assessment Using AI Active Gaming and Motion Capture in Friedreich Ataxia

Who this might be right for
Friedreich Ataxia
University of Exeter 40
Not applicableLooking for participantsNCT04024774
What this trial is testing

Diagnostic Research in Patients With Rare Diseases -Solving the Unsolved Rare Diseases

Who this might be right for
Rare Diseases
Centre Hospitalier Universitaire Dijon 50
Not applicableUnknownNCT04979182
What this trial is testing

Clinical Characterization of Frequent Monogenic Forms of Neurodevelopmental Disorders

Who this might be right for
Neurodevelopmental Disorders
University Hospital, Strasbourg, France 30
Not applicableUnknownNCT00004374
What this trial is testing

Study of Systemic Amyloidosis Presentation and Prognosis

Who this might be right for
Amyloidosis
National Center for Research Resources (NCRR) 100
Early research (Phase 1)Study completedNCT01894165
What this trial is testing

Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers

Who this might be right for
Molybdenum Cofactor Deficiency (MoCD)Rare Autosomal Recessive DisorderDeficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
Origin Biosciences 24
Testing effectiveness (Phase 2)Study completedNCT00004811
What this trial is testing

Phase I/II Study of Itraconazole for Blastomycosis, Histoplasmosis, and Sporotrichosis

Who this might be right for
HistoplasmosisSporotrichosisBlastomycosis
National Center for Research Resources (NCRR) 7
Not applicableActive Not RecruitingNCT06832371
What this trial is testing

Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia

Who this might be right for
Homozygous Familial Hypercholesterolemia (HoFH)Major Adverse Cardiovascular Events (MACE)Dyslipidemia
Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) 73
Not applicableLooking for participantsNCT07527624
What this trial is testing

Evaluation of Socio-professional Inclusion for Young Adults Aged 15-25 Living With a Rare Genetic Disability

Who this might be right for
Rare Diseases
Imagine Institute 300
Large-scale testing (Phase 3)Study completedNCT00004690
What this trial is testing

Phase III Study of Monolaurin Cream Therapy for Patients With Congenital Ichthyosis

Who this might be right for
Ichthyosis
Cellegy Pharmaceuticals 90
Testing effectiveness (Phase 2)Active Not RecruitingNCT03074513
What this trial is testing

Atezolizumab and Bevacizumab in Treating Patients With Rare Solid Tumors

Who this might be right for
Appendix AdenocarcinomaHuman Papillomavirus-Related Anal Squamous Cell CarcinomaHuman Papillomavirus-Related Cervical Squamous Cell Carcinoma+18 more
M.D. Anderson Cancer Center 133
Not applicableUnknownNCT02006940
What this trial is testing

The Study Of Probe-Based Confocal Laser Endomicroscopy Of Distal Airways In Diagnostics Of Lung Diseases

Who this might be right for
Inflammatory, Interstitial, Rare Lung Diseases
Federal Research Clinical Center of Federal Medical & Biological Agency, Russia 150
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