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Results for “Orphan Diseases”

67 trials

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Showing 20 of 67 results

Not applicableStudy completedNCT03563677
What this trial is testing

Dual Guidance Structure for Evaluation of Patients With Unclear Diagnosis in Centers for Rare Diseases

Who this might be right for
Rare DiseasesOrphan Diseases
Wuerzburg University Hospital 1,379
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 applicableLooking for participantsNCT02852928
What this trial is testing

European Management Platform for Childhood Interstitial Lung Diseases - chILD-EU Register and Biobank

Who this might be right for
Lung Diseases, InterstitialPulmonary Alveolar ProteinosisPulmonary Eosinophilia+3 more
Matthias Griese 1,000
Not applicableStudy completedNCT03243396
What this trial is testing

Building and Sustaining Interventions for Children: Task-sharing Mental Health Care in Low-resource Settings

Who this might be right for
GriefPost Traumatic Stress DisorderDepression
Duke University 956
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)Ended earlyNCT02697136
What this trial is testing

CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases

Who this might be right for
Familial Hypoalphalipoproteinemia
Cerenis Therapeutics, SA 30
Early research (Phase 1)Study completedNCT02548052
What this trial is testing

Study of Safety and Efficacy of Topical GSK2981278 Ointment in Plaque Psoriasis

Who this might be right for
Psoriasis
GlaxoSmithKline 15
Testing effectiveness (Phase 2)Ended earlyNCT00623298
What this trial is testing

Narrative Exposure Therapy Versus Group Interpersonal Psychotherapy

Who this might be right for
Posttraumatic Stress DisorderDepressionNarrative Exposure Therapy+1 more
University of Konstanz 26
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
Large-scale testing (Phase 3)Study completedNCT02218047
What this trial is testing

AOP2014 vs. BAT in Patients With Polycythemia Vera Who Previously Participated in the PROUD-PV Study.

Who this might be right for
Polycythemia Vera
AOP Orphan Pharmaceuticals AG 170
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)Study completedNCT03474640
What this trial is testing

Safety, Tolerability and Pharmacokinetics of an Anti-PD-1 Monoclonal Antibody in Subjects With Advanced Malignancies

Who this might be right for
Advanced Malignancies
TopAlliance Biosciences 184
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
Not applicableActive Not RecruitingNCT06347146
What this trial is testing

Bridges2Scale: Testing Implementation Strategies

Who this might be right for
HIV/AIDS
Washington University School of Medicine 1,440
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 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)Looking for participantsNCT06912763
What this trial is testing

Reversing External-beam Radiotherapy-associated Fibrosis Syndrome: an Interventional Bayesian Adaptive Randomized-controlled Orphan Drug Platform Trial for Orodental Sequelae (Reverse-fibrose)

Who this might be right for
Fibrosis SyndromeLymphedemaHead &Amp; Neck Cancer+1 more
M.D. Anderson Cancer Center 250
Not applicableStudy completedNCT01002612
What this trial is testing

Reassessment of the Nutritional Status in Thai Orphans Living With HIV in a Family Style Community

Who this might be right for
HIV InfectionHIV Infections
Thai Red Cross AIDS Research Centre 73
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
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
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