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Results for “RARE DISEASE”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Looking for participantsNCT04998396
What this trial is testing

AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

Who this might be right for
Canavan Disease
Aspa Therapeutics 26
Not applicableLooking for participantsNCT07737756
What this trial is testing

A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia

Who this might be right for
Spinal Muscular Atrophy
Novartis Pharmaceuticals 6
Not applicableNot Yet RecruitingNCT07125105
What this trial is testing

Integrating Art Therapy and Adapted Physical Activity: Toward Improved Quality of Life in Patients With Myasthenia Gravis

Who this might be right for
Myasthenia Gravis
Universite du Littoral Cote d'Opale 102
Not applicableLooking for participantsNCT04880356
What this trial is testing

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Who this might be right for
Inherited DiseaseRare DiseasesMetabolic Disease+3 more
Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 100
Testing effectiveness (Phase 2)UnknownNCT02034630
What this trial is testing

Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in GRD

Who this might be right for
Genetic Rare Disease
Seoul National University Hospital 5
Testing effectiveness (Phase 2)Study completedNCT00004328
What this trial is testing

Phase II Study of the Pathophysiology and Treatment With Enalapril and Polystyrene Sulfonate for Pseudohypoaldosteronism, Type I

Who this might be right for
Pseudohypoaldosteronism
National Center for Research Resources (NCRR) 1
Not applicableEnrolling By InvitationNCT04681781
What this trial is testing

SLC13A5 Deficiency Natural History Study - Remote Only

Who this might be right for
Citrate Transporter DeficiencyEpilepsyRare Diseases+7 more
TESS Research Foundation 20
Testing effectiveness (Phase 2)Not Yet RecruitingNCT05320367
What this trial is testing

Inhaled Cannabidiol in Healthy Occasional Cannabis Users

Who this might be right for
Cannabis
Centre hospitalier de l'Université de Montréal (CHUM) 80
Not applicableStudy completedNCT02327364
What this trial is testing

Natural History of Pearson Syndrome

Who this might be right for
Pearson Syndrome
Sumit Parikh 11
Large-scale testing (Phase 3)Study completedNCT00004467
What this trial is testing

Randomized Study of Acetylcysteine in Patients With Acute Liver Failure Not Caused by Acetaminophen

Who this might be right for
Acute Liver Failure
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) 173
Not applicableUnknownNCT00006056
What this trial is testing

Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders

Who this might be right for
Chediak-Higashi SyndromeGraft Versus Host DiseaseX-Linked Lymphoproliferative Syndrome+3 more
Fairview University Medical Center 40
Testing effectiveness (Phase 2)Study completedNCT00010413
What this trial is testing

Phase II Study of High-Dose Cyclophosphamide in Patients With Refractory Pemphigus

Who this might be right for
Pemphigus
Johns Hopkins University 35
Not applicableEnrolling By InvitationNCT06629623
What this trial is testing

Current Status of Diagnosis, Treatment and Quality of Life for Patients with Idiopathic Pulmonary Fibrosis

Who this might be right for
Idiopathic Pulmonary Fibrosis
Chu Hongling 310
Not applicableEnded earlyNCT00014768
What this trial is testing

Study of Metabolic Effects of Pregnancy in Women With Cystic Fibrosis

Who this might be right for
Cystic Fibrosis
National Center for Research Resources (NCRR) 36
Testing effectiveness (Phase 2)Study completedNCT00004323
What this trial is testing

Phase II Study of Bone Marrow Transplantation Using Related Donors in Patients With Aplastic Anemia

Who this might be right for
Aplastic Anemia
National Center for Research Resources (NCRR) 10
Not applicableLooking for participantsNCT05974696
What this trial is testing

A Research Registry on Aggressive PitNETs

Who this might be right for
Endocrine Tumor
Hospices Civils de Lyon 200
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Not applicableUnknownNCT02588638
What this trial is testing

Next Generation Sequencing Diagnostics - On the Road to Rapid Diagnostics for Rare Diseases

Who this might be right for
Movement DisorderCognitive Decline
University Hospital Tuebingen 100
Testing effectiveness (Phase 2)Study completedNCT00004333
What this trial is testing

Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I

Who this might be right for
Tyrosinemia, Type I
National Center for Research Resources (NCRR)
Testing effectiveness (Phase 2)Temporarily pausedNCT05337735
What this trial is testing

A Phase II Clinical Trial to Evaluate Safety and Efficacy of XmAb20717 in Advanced Rare Cancers

Who this might be right for
Microsatellite High CancersPeritoneal MesotheliomaExtrapulmonary High Grade+5 more
M.D. Anderson Cancer Center 140
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