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Results for “Neonatal Patient”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Study completedNCT02615691
What this trial is testing

PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A

Who this might be right for
Hemophilia A
Baxalta now part of Shire 120
Not applicableStudy completedNCT02528968
What this trial is testing

National Study of a Pharmacokinetic-Focused Educational Package for Patients With Severe Haemophilia A

Who this might be right for
Hemophilia A
Hampshire Hospitals NHS Foundation Trust 59
Testing effectiveness (Phase 2)UnknownNCT01914913
What this trial is testing

Clinical Study to Evaluate Safety and Efficacy of BMMNC in Retinitis Pigmentosa

Who this might be right for
Retinitis Pigmentosa
Chaitanya Hospital, Pune 15
Testing effectiveness (Phase 2)Active Not RecruitingNCT05467397
What this trial is testing

Feasibility of [11C]Acetate-PET in LAM and TSC

Who this might be right for
LymphangioleiomyomatosisTuberous Sclerosis Complex
Brigham and Women's Hospital 7
Not applicableActive Not RecruitingNCT01980797
What this trial is testing

International Bicuspid Aortic Valve Consortium (BAVCon)

Who this might be right for
Bicuspid Aortic Valve DiseaseThoracic Aortic Disease in Patients With a Bicuspid Aortic Valve
Boston University 6,464
Post-approval studies (Phase 4)Study completedNCT00097890
What this trial is testing

Replagal Enzyme Replacement Therapy for Adults With Fabry Disease

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 25
Not applicableStudy completedNCT04738474
What this trial is testing

Assessing and Promoting Resilience in Patients With Adult Congenital Heart Disease

Who this might be right for
Congenital Heart Disease
University of Washington 78
Testing effectiveness (Phase 2)Study completedNCT02960997
What this trial is testing

Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study

Who this might be right for
Epidermolysis Bullosa SimplexEpidermolysis Bullosa Simplex KobnerWeber-Cockayne Syndrome
Stanford University 8
Not applicableLooking for participantsNCT07301918
What this trial is testing

Leaf Expander Versus Hyrax Expander in Mixed Dentition Patients With Posterior Crossbite.

Who this might be right for
Posterior CrossbiteMaxilla; Hypoplasia
Cairo University 30
Not applicableLooking for participantsNCT06564974
What this trial is testing

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

Who this might be right for
Duchenne Muscular Dystrophy
Catalyst Pharmaceuticals, Inc. 250
Not applicableStudy completedNCT02439710
What this trial is testing

Patient Reported Outcomes Burdens and Experiences - Phase 2

Who this might be right for
Hemophilia
McMaster University 656
Not applicableEnded earlyNCT06014541
What this trial is testing

Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

Who this might be right for
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Ionis Pharmaceuticals, Inc. 29
Not applicableLooking for participantsNCT07649031
What this trial is testing

MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients

Who this might be right for
Fanconi Anemia
University of Minnesota 80
Post-approval studies (Phase 4)Study completedNCT00365131
What this trial is testing

Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

Who this might be right for
Gaucher Disease Type ICerebroside Lipidosis SyndromeClucocerebrosidase Deficiency Disease+2 more
Genzyme, a Sanofi Company 40
Not applicableStudy completedNCT02325505
What this trial is testing

Characterization of Patients With Tuberous Sclerosis Complex, Lymphangioleiomyomatosis and Angiomyolipoma

Who this might be right for
Tuberous SclerosisLymphangioleiomyomatosisAngiomyolipoma
InCor Heart Institute 200
Not applicableWithdrawnNCT06678256
What this trial is testing

Pregnancy Outcomes in Congenital Heart Disease

Who this might be right for
Congenital Heart Disease
Mayo Clinic
Not applicableLooking for participantsNCT05689151
What this trial is testing

Learn About Abrocitinib in Adult Patients With Moderate to Severe Atopic Dermatitis.

Who this might be right for
Atopic DermatitisEczema
Pfizer 183
Not applicableLooking for participantsNCT06967831
What this trial is testing

Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells

Who this might be right for
Leigh Syndrome (Maternally Inherited, MILS)Leigh Syndrome (AR, AD, XR)
Charite University, Berlin, Germany 80
Large-scale testing (Phase 3)UnknownNCT02963129
What this trial is testing

Treatment of Nasal Staphylococcus Aureus Colonization in Patients With HHT

Who this might be right for
Hereditary Hemorrhagic TelangiectasiaEpistaxis
Hospital Italiano de Buenos Aires 40
Testing effectiveness (Phase 2)Study completedNCT00486837
What this trial is testing

Deposition of Inhaled Prolastin in Cystic Fibrosis Patients

Who this might be right for
Cystic Fibrosis
Grifols Therapeutics LLC 72
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