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Results for “Rare Disorders”

993 trials

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Showing 20 of 993 results

Not applicableUnknownNCT03424772
What this trial is testing

Whole Genome Sequencing in the Detection of Rare Undiagnosed Genetic Diseases in Children in China

Who this might be right for
Intellectual DisabilityMultiple Congenital AnomalyRare Diseases
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 100
Large-scale testing (Phase 3)Ended earlyNCT00004465
What this trial is testing

Phase III Randomized Study of SYNSORB Pk in Children With E. Coli-Associated Hemolytic Uremic Syndrome

Who this might be right for
Hemolytic Uremic Syndrome
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) 145
Testing effectiveness (Phase 2)Active Not RecruitingNCT06303167
What this trial is testing

Testing AZD9291 as Potentially Targeted Treatment in Cancers With EGFR Genetic Changes (MATCH-Subprotocol E)

Who this might be right for
Advanced LymphomaAdvanced Malignant Solid NeoplasmHematopoietic and Lymphoid Cell Neoplasm+3 more
National Cancer Institute (NCI) 19
Not applicableUnknownNCT05092230
What this trial is testing

Frequency of Pompe Disease in Patients With Myalgia With or Without Hyper Ckemia - Data From the Reference Center (CERCA)

Who this might be right for
Pompe Disease
University Hospital Center of Martinique 100
Not applicableEnded earlyNCT03472807
What this trial is testing

EXOme Rare Cancers in Children (EXOCARE)

Who this might be right for
Predisposition, GeneticCancer ChildhoodDevelopment Delay
University Hospital, Angers 169
Testing effectiveness (Phase 2)Looking for participantsNCT06563999
What this trial is testing

Neoadjuvant Umbrella Trial for Patients With Unresectable Stage III NSCLC Harboring Rare Mutations.

Who this might be right for
Lung Cancer Stage IIIMutation
Sun Yat-sen University 120
Not applicableStudy completedNCT00004699
What this trial is testing

Dose Ranging Study of Recombinant Human Insulin-like Growth Factor I in Children With Hyperinsulinism

Who this might be right for
Hyperinsulinism
Children's Hospital of Philadelphia 8
Not applicableStudy completedNCT00004444
What this trial is testing

Pilot Randomized Study of Paromomycin (Aminosidine) vs Streptomycin for Uncomplicated Pulmonary Tuberculosis

Who this might be right for
Tuberculosis, Pulmonary
FDA Office of Orphan Products Development 24
Not applicableLooking for participantsNCT00369421
What this trial is testing

Diagnosis and Treatment of Patients With Inborn Errors of Metabolism

Who this might be right for
Arterial Calcification Due to Deficiency of CD73
National Human Genome Research Institute (NHGRI) 4,000
Not applicableStudy completedNCT00005892
What this trial is testing

Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Myelodysplastic Syndrome and Acute Leukemia Related to Fanconi's Anemia

Who this might be right for
Fanconi's AnemiaMyelodysplastic SyndromesLeukemia, Nonlymphocytic, Acute+1 more
Fairview University Medical Center
Testing effectiveness (Phase 2)Study completedNCT00004464
What this trial is testing

Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Who this might be right for
Aplastic AnemiaParoxysmal Hemoglobinuria, Nocturnal
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins 69
Not applicableLooking for participantsNCT04777175
What this trial is testing

A Retrospective Study: Evaluation of the Efficacy of Immunotherapy With Rare Mutations in Non-small Cell Lung Cancer

Who this might be right for
Non Small Cell Lung Cancer
Yongchang Zhang 186
Large-scale testing (Phase 3)UnknownNCT03453632
What this trial is testing

Injections of Botulinic Toxin in Plantar Lesions of Localized Epidermolysis Bullosa Simplex

Who this might be right for
Epidermolysis Bullosa Simplex
University Hospital, Toulouse 25
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 applicableEnrolling By InvitationNCT06041906
What this trial is testing

International Registry of Congenital Portosystemic Shunt (IRCPSS)

Who this might be right for
Congenital Portosystemic ShuntCPSS (Congenital Portosystemic Shunt)
Prof. Valérie Mc Lin 500
Not applicableStudy completedNCT00004840
What this trial is testing

Study of Antenatal Thyrotropin-Releasing Hormone in Women in Premature Labor to Prevent Lung Disease in Preterm Infants

Who this might be right for
Respiratory Distress Syndrome
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 996
Early research (Phase 1)Study completedNCT00010335
What this trial is testing

Pilot Study of Total Body Irradiation in Combination With Cyclophosphamide, Anti-thymocyte Globulin, and Autologous CD34-Selected Peripheral Blood Stem Cell Transplantation in Children With Refractory Autoimmune Disorders

Who this might be right for
Systemic SclerosisSystemic Lupus ErythematosusDermatomyositis+2 more
Fred Hutchinson Cancer Center 6
Large-scale testing (Phase 3)Looking for participantsNCT05786716
What this trial is testing

DETERMINE Trial Treatment Arm 04: Trastuzumab in Combination With Pertuzumab in Adult, Paediatric and Teenage/Young Adult Patients With Cancers With HER2 Amplification or Activating Mutations

Who this might be right for
Haematological MalignancyColorectal NeoplasmsUrinary Bladder Neoplasm+8 more
Cancer Research UK 30
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 applicableStudy completedNCT00004838
What this trial is testing

Randomized Study of Succimer (Dimercaptosuccinic Acid) on Growth of Lead-Poisoned Children

Who this might be right for
Lead Poisoning
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 175
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