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Results for “Orphan Diseases”

70 trials

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Showing 20 of 70 results

Not applicableWithdrawnNCT04007562
What this trial is testing

Acute Rhabdomyolysis and Muscle Pain Associated With Mutations in the LPIN1 Gene - A Retrospective Study Describing the Safety and Efficacy of Hydroxychloroquine Sulfate Given on a Compassionate Basis to Patients Suffering From Lipin-1 Deficiency

Who this might be right for
LIPIN1 Deficiency
Assistance Publique - Hôpitaux de Paris
Testing effectiveness (Phase 2)Study completedNCT03088878
What this trial is testing

Cirmtuzumab and Ibrutinib in Patients With B-Cell Lymphoid Malignancies

Who this might be right for
B-cell Chronic Lymphocytic LeukemiaSmall Lymphocytic LymphomaMantle Cell Lymphoma+1 more
Oncternal Therapeutics, Inc 95
Early research (Phase 1)Looking for participantsNCT05607498
What this trial is testing

First in Human Study of EMB-07 in Locally Advanced/Metastatic Solid Tumors or Relapse/Refractory Lymphoma

Who this might be right for
Advanced/Metastatic Solid TumorsRelapse/Refractory Lymphoma
EpimAb Biotherapeutics (Suzhou)Co., Ltd. 150
Testing effectiveness (Phase 2)UnknownNCT04069533
What this trial is testing

Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A

Who this might be right for
Fanconi Anemia Complementation Group A
Rocket Pharmaceuticals Inc. 7
Large-scale testing (Phase 3)Study completedNCT01000961
What this trial is testing

Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis

Who this might be right for
Cystinosis
Amgen 43
Large-scale testing (Phase 3)Looking for participantsNCT04220190
What this trial is testing

RAPA-501 Therapy for ALS

Who this might be right for
Amyotrophic Lateral Sclerosis
Rapa Therapeutics LLC 41
Not applicableStudy completedNCT04114305
What this trial is testing

Evaluation of an Early Childhood Development Intervention Among Children Born to HIV-Infected Women in Eswatini

Who this might be right for
Early Childhood Development (ECD)
Johns Hopkins Bloomberg School of Public Health 431
Testing effectiveness (Phase 2)Study completedNCT01412034
What this trial is testing

Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects

Who this might be right for
Homozygous Familial Hypercholesterolemia
Cerenis Therapeutics, SA 23
Not applicableLooking for participantsNCT03538639
What this trial is testing

Vascular Disease Discovery Protocol

Who this might be right for
Vascular DysfunctionGenetic MutationsGenetic Predisposition
National Heart, Lung, and Blood Institute (NHLBI) 1,000
Not applicableStudy completedNCT05818566
What this trial is testing

Orphan Drugs for Inherited Metabolic Diseases

Who this might be right for
Inborn Errors of Metabolism
University of Lausanne 39
Early research (Phase 1)UnknownNCT00553956
What this trial is testing

Controlled Clinical Trial With Orphans and Widows Who Experienced the Rwandan Genocide

Who this might be right for
Posttraumatic Stress DisorderDepressionProlonged Grief Disorder
University of Konstanz 150
Not applicableLooking for participantsNCT06615206
What this trial is testing

A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)

Who this might be right for
MECP2 Duplication Syndrome
HuidaGene Therapeutics Co., Ltd. 6
Not applicableStudy completedNCT02759341
What this trial is testing

Brain-heart Interactions in Tako-Tsubo Cardiomyopathy and Cardiac Syndrome X:

Who this might be right for
Takotsubo CardiomyopathyCardiac X SyndromeAcute Myocardial Infarction
Ospedale San Giovanni Bellinzona 50
Not applicableStudy completedNCT06957054
What this trial is testing

Neural Biomarkers in Fragile X Syndrome

Who this might be right for
Fragile X Syndrome (FXS)Neurotypical Adults
Connecta Therapeutics, S.L. 20
Large-scale testing (Phase 3)Study completedNCT01733316
What this trial is testing

Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis

Who this might be right for
Cystinosis
Amgen 41
Not applicableStudy completedNCT04362852
What this trial is testing

Patient Forward Access to Clinical and Technological Research: Genetic Influences on Cancer and Atopic Dermatitis

Who this might be right for
Eczema/Atopic DermatitisCancer
Boston Children's Hospital 100
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
Early research (Phase 1)Study completedNCT03082937
What this trial is testing

An Open Label, Single-dose, Single Period ADME Study of A4250 in Healthy Subjects

Who this might be right for
Orphan Cholestatic Liver DiseasesProgressive Familial Intrahepatic CholestasisAlagille Syndrome+1 more
Albireo 6
Large-scale testing (Phase 3)Study completedNCT01067235
What this trial is testing

Efficacy and Safety Study of BF2.649 and BF2.649 Add on Modafinil on Cataplexy in Patients With Narcolespy

Who this might be right for
NarcolepsyCataplexyExcessive Daytime Sleepiness
Bioprojet 14
Post-approval studies (Phase 4)WithdrawnNCT01151735
What this trial is testing

C1-INH Compared to Placebo at the Time of Prodromal Symptoms for Hereditary Angioedema (HAE) Exacerbation

Who this might be right for
Hereditary Angioedema
Penn State University
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