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Results for “RARE DISEASE”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT04790409
What this trial is testing

Sintilimab Combined With Anlotinib in Advanced NSCLC With EGFR Uncommon Mutations

Who this might be right for
Lung Cancer
Zhejiang Cancer Hospital 21
Early research (Phase 1)Study completedNCT00004809
What this trial is testing

Phase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia

Who this might be right for
Familial Hypercholesterolemia
National Center for Research Resources (NCRR) 5
Not applicableLooking for participantsNCT05810181
What this trial is testing

Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)

Who this might be right for
Sickle Cell Disease
St. Jude Children's Research Hospital 145
Testing effectiveness (Phase 2)Looking for participantsNCT06010329
What this trial is testing

Evaluate the Efficacy and Safety of Sutetinib Maleate Capsule in Locally Advanced or Metastatic NSCLC

Who this might be right for
Non-small Cell Lung Cancer
Teligene US 66
Not applicableStudy completedNCT00004399
What this trial is testing

Randomized Study of Nimodipine Versus Magnesium Sulfate in the Prevention of Eclamptic Seizures in Patients With Severe Preeclampsia

Who this might be right for
Pre-eclampsia
FDA Office of Orphan Products Development 2,000
Not applicableLooking for participantsNCT04931160
What this trial is testing

B-dependant Rare AutoImmune diseaSES - Cohort of Patients With Suspected Diagnosis of Primitive Sjögren Syndrome

Who this might be right for
Sjogren's Syndrome
University Hospital, Brest 1,000
Not applicableStudy completedNCT02985710
What this trial is testing

Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan

Who this might be right for
Small Fiber NeuropathyFabry DiseaseEhlers Danlos Syndrome+1 more
Massachusetts General Hospital 102
Not applicableLooking for participantsNCT02397824
What this trial is testing

Orodental Manifestations of Rare Diseases

Who this might be right for
Rare Disease Orodontal
University Hospital, Strasbourg, France 1,300
Not applicableLooking for participantsNCT02705846
What this trial is testing

Analysing Outcomes After Prostate Cancer Diagnosis and Treatment in Carriers of Rare Germline Mutations

Who this might be right for
Prostate Cancer
Institute of Cancer Research, United Kingdom 4,260
Not applicableStudy completedNCT00007046
What this trial is testing

Genetic Study of Patients and Families With Diaphyseal Medullary Stenosis With Malignant Fibrous Histiocytoma of the Bone

Who this might be right for
StenosisHistiocytoma
National Center for Research Resources (NCRR)
Testing effectiveness (Phase 2)Study completedNCT00004359
What this trial is testing

Phase II Pilot Study of Extracorporeal Phototherapy for Epidermolysis Bullosa Acquisita

Who this might be right for
Epidermolysis Bullosa Acquisita
National Center for Research Resources (NCRR) 10
Testing effectiveness (Phase 2)Study completedNCT00004810
What this trial is testing

Phase II Placebo-Controlled Study of 4-Aminosalicylic Acid for Ulcerative Colitis

Who this might be right for
Ulcerative Colitis
National Center for Research Resources (NCRR) 30
Not applicableStudy completedNCT00004361
What this trial is testing

Study of the Relationship Between Calcium Levels and Intact Parathyroid Hormone (iPTH) in Adults With Repaired or Palliated Conotruncal Cardiac Defects

Who this might be right for
HypoparathyroidismTetralogy of FallotPulmonary Valve Stenosis+3 more
National Center for Research Resources (NCRR) 150
Not applicableLooking for participantsNCT07362225
What this trial is testing

MPN PROGRESSion Registry: Observational Study Tracking Symptoms, Treatments, and Disease Progression in People With Myeloproliferative Neoplasms (MPNs)

Who this might be right for
Polycythemia VeraET (Essential Thrombocythemia)Polycythemia Vera (PV)+52 more
MPN Research Foundation 5,000
Not applicableUnknownNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
Testing effectiveness (Phase 2)Looking for participantsNCT06638931
What this trial is testing

Agnostic Therapy in Rare Solid Tumors

Who this might be right for
Urachal CancerParathyroid CarcinomaFibrolamellar Carcinoma+38 more
Instituto do Cancer do Estado de São Paulo 28
Not applicableLooking for participantsNCT03056794
What this trial is testing

Natural History and Advanced Genetic Study of Pyruvate Dehydrogenase Complex Deficiencies

Who this might be right for
Pyruvate Dehydrogenase Complex Deficiency Disease
University of Pittsburgh 150
Not applicableStudy completedNCT00004831
What this trial is testing

Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

Who this might be right for
Erythropoietic Protoporphyria
FDA Office of Orphan Products Development 20
Not applicableLooking for participantsNCT06878469
What this trial is testing

SIMPLIFication of Care Pathways for Patients With Rare Brain Tumors Through Artificial Intelligence

Who this might be right for
Brain Tumors
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 200
Large-scale testing (Phase 3)Looking for participantsNCT05770037
What this trial is testing

DETERMINE Trial Treatment Arm 01: Alectinib in Adult, Paediatric and Teenage/Young Adult Patients With ALK Positive Cancers

Who this might be right for
Haematological MalignancyMalignant NeoplasmLymphoproliferative Disorders+8 more
Cancer Research UK 30
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