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Results for “Orphan Diseases”

67 trials

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Showing 20 of 67 results

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
Testing effectiveness (Phase 2)Active Not RecruitingNCT04069533
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
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
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)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 applicableTemporarily Not AvailableNCT06169176
What this trial is testing

RAPA-501 Therapy of ALS Expanded Access Protocol

Who this might be right for
Amyotrophic Lateral Sclerosis
Rapa Therapeutics LLC
Large-scale testing (Phase 3)Study completedNCT00339157
What this trial is testing

Interleukin-1 Receptor Antagonist (IL-1RA) (ANAKINRA) IN SEVERE SYSTEMIC-ONSET JUVENILE IDIOPATHIC ARTHRITIS

Who this might be right for
Systemic-Onset Juvenile Idiopathic Arthritis
Institut National de la Santé Et de la Recherche Médicale, France 24
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 completedNCT01447615
What this trial is testing

Bridges to the Future: Economic Empowerment for AIDS-Orphaned Children in Uganda

Who this might be right for
PovertyOrphaned ChildrenAIDS Orphans
Columbia University 1,410
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 applicableStudy completedNCT00213811
What this trial is testing

Bardet-Biedl Syndrome Study: Clinical and Genetic Epidemiology Study in Adults

Who this might be right for
Bardet-Biedl SyndromeOrphan Diseases
University Hospital, Strasbourg, France 40
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
Not applicableStudy completedNCT06897956
What this trial is testing

Oral Health Educational Program on Oral Hygiene Knowledge and Practices in a Group of Orphan Children

Who this might be right for
Dental CariesKnowledge, Attitudes, Practice
Cairo University 139
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 participantsNCT03409003
What this trial is testing

Orphan Europe Carbaglu® Surveillance Protocol

Who this might be right for
N-acetylglutamate Synthase (NAGS) Deficiency
Nicholas Ah Mew 30
Testing effectiveness (Phase 2)Study completedNCT00574613
What this trial is testing

Efficacy and Safety Study of p144 to Treat Skin Fibrosis in Systemic Sclerosis

Who this might be right for
Skin Fibrosis
ISDIN 56
Large-scale testing (Phase 3)Study completedNCT01744782
What this trial is testing

Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis

Who this might be right for
Cystinosis
Amgen 17
Large-scale testing (Phase 3)Study completedNCT01197378
What this trial is testing

Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)

Who this might be right for
Cystinosis
Amgen 60
Large-scale testing (Phase 3)Study completedNCT00049803
What this trial is testing

Safety and Efficacy of Xyrem Oral Solution (Sodium Oxybate) Compared With Placebo in Narcoleptic Patients

Who this might be right for
Narcolepsy
Orphan Medical 200
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
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