Clinical Trials on SCID

Total 1201 results

ReWalk Robotics, Inc.

Completed

Safety and Performance Evaluation of ReWalk Reciprocating Gait Orthosis (RGO)

Paraplegia | Spinal Cord Injuries (SCI)

United States, Italy
BioMarin Pharmaceutical

Recruiting

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) (PALace)

Phenylketonuria (PKU)

United States, Germany, Italy
Institut Pasteur
University of Copenhagen; Institut Cochin

Completed

Study on the Association Between Vitamin C Deficiency and Diarrhea in Children (VITAL)

Diarrhea | Infantile

France, New Caledonia, Central African Republic
University of Aarhus

Completed

Modulation of Circulating Levels of the Ketone Body 3-hydroxybutyrate in Patients With Type 2 Diabetes and Heart Failure With Preserved Ejection Fraction: Cardiovascular Effects. (KETO-HFpEF)

Diabetes Mellitus, Type 2 | Heart Failure With Preserved Ejection Fraction | Ketonemia

Denmark
Nutricia Research

Completed

PANDA: PKU Amino Acid Evaluation (PANDA)

Phenylketonuria (PKU)

United Kingdom, Belgium
University of Aarhus
Aarhus University Hospital

Completed

Metabolic Effects of Exogenous 3-hydroxybutyrate in Patients With Type 1 Diabetes and Healthy Controls

Type 1 Diabetes Mellitus | Diabetic Ketoacidosis

Denmark
BioMarin Pharmaceutical

Active, not recruiting

AAV Gene Therapy Study for Subjects With PKU

Phenylketonuria (PKU)

United States, United Kingdom
BioMarin Pharmaceutical

Completed

A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults (165-901)

Phenylketonuria (PKU)

United States, Turkey, Canada, France, Germany, Italy, United Kingdom
Sutphin Drugs

Unknown

Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine (Orfadin)

Hereditary Tyrosinemia, Type I

India
The Cleveland Clinic

Withdrawn

Thiamine Supplementation in Patients With Septic Shock

Septic Shock | Shock, Septic | Lactic Acidosis | Thiamine Deficiency

United States
London School of Hygiene and Tropical Medicine

Completed

Effectiveness of a Micronutrient Supplement to Lower Plasma Homocysteine MDEG2 Pilot Supplementation Trial (MDEG2-PST)

Hyperhomocysteinemia | Micronutrient Deficiencies

Gambia
Assiut University

Unknown

Incidence of Renal Tubular Acidosis in Nephrology Unit in Assiut University Childern Hospital

Renal Tubule Acidosis
National Institute of Cardiology, Laranjeiras,...

Completed

Effects of Oral Supplementation With Creatine on Systemic Microvascular Endothelial Function in Vegetarian Individuals

Endothelial Dysfunction | Hyperhomocysteinemia

Brazil
Charite University, Berlin, Germany
Johannes Gutenberg University Mainz

Completed

Observational Study of Endothelial Dysfunction in Phenylketonuria

Phenylketonuria | Endothelial Dysfunction
Washington University School of Medicine
University of Missouri-Columbia; Northwestern University; Oregon Health and Science... and other collaborators

Terminated

Behavioral Effects of Kuvan in Children With Mild Phenylketonuria

Phenylketonuria

United States
University of California, Davis
National Institute of Neurological Disorders and Stroke (NINDS)

Completed

Cerebral Edema in Pediatric Diabetic Ketoacidosis

Diabetic Ketoacidosis

United States
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Exploring Near Infrared Spectroscopy (NIRS) Technologies for Assessment of Muscle Physiology, Tissue Oxygenation, and Blood Flow in Patients With Sickle Cell Disease (SCD)

SCD

United States
BioMarin Pharmaceutical

Recruiting

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

Phenylketonuria (PKU)

United States
Shanghai Vitalgen BioPharma Co., Ltd.

Recruiting

A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b

Glutaric Acidemia Type I | Glutaric Aciduria Type I

China
Rajavithi Hospital

Recruiting

Comparative Study of Tranexamic Acid, Estrogen for Treatment AUB in DMPA Users

Abnormal Uterine Bleeding | Tranexamic Acid | Estrogen | Depot Medroxyprogesterone Acetate

Thailand
PTC Therapeutics

Completed

A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia

BH4 Deficiency | Hyperphenylalaninemia

United States
Homology Medicines, Inc

Terminated

Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

Phenylketonuria | Phenylketonurias | PAH Deficiency

United States
Homology Medicines, Inc

Terminated

Gene Therapy Clinical Study in Adult PKU (pheNIX)

Phenylketonurias | PAH Deficiency

United States
Beth Israel Deaconess Medical Center
National Heart, Lung, and Blood Institute (NHLBI)

Terminated

Thiamine vs. Placebo to Increase Oxygen Consumption After Cardiac Arrest

Shock | Cardiac Arrest | Lactic Acidosis | Thiamin Deficiency

United States
Boehringer Ingelheim
Eli Lilly and Company

Completed

Post-authorisation Safety Study in Patients With Type 2 Diabetes to Assess the Risk of Liver Injury, Kidney Injury, Urinary Tract and Genital Infections, and Diabetic Ketoacidosis in Patients Treated With Empagliflozin, Compared to DPP-4 Inhibitors

Diabetes Mellitus, Type 2

United Kingdom
Guy's and St Thomas' NHS Foundation Trust

Unknown

A Study Into the Future Cardiovascular Disease (CVD) Risk of Phenylketonuria (PKU) Patients on a Low Phenylalanine Treatment Diet (LPD).

Cardiovascular Diseases | Phenylketonurias

United Kingdom
Taipei Veterans General Hospital, Taiwan
BioMarin Pharmaceutical

Unknown

BH4 Responsiveness in PAH Deficiency PKU Patients

PAH Deficiency | Pku Phenylketonuria
Albert Einstein College of Medicine

Completed

Comparison of High Versus Low Bicarbonate Hemodialysis

Chronic Kidney Disease | End-stage Renal Disease | Metabolic Acidosis

United States
Nutricia UK Ltd

Completed

Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia

Phenylketonurias | Hyperphenylalaninaemia, Type I

United Kingdom
University of Novi Sad, Faculty of Sport and Physical...

Completed

Guanidinoacetic Acid With Creatine Compared With Creatine Alone for Tissue Bioenergetics, Hyperhomocysteinemia and Exercise Performance

Energy Metabolism
Medical University of Vienna

Completed

Homocysteine After Laparoscopic Roux-enY Gastric Bypass

Obesity, Morbid | Bariatric Surgery Candidate | Vitamin B 12 Deficiency | Folate Deficiency | Homocystine; Metabolic Disorder

Austria
University of Southern California
BioMarin Pharmaceutical

Completed

Effects of Kuvan on Melatonin Secretion

Phenylketonuria (PKU)

United States
University of Aarhus
Rehaler

Completed

Normalizing CO2 in Chronic Hyperventilation by a Novel Breathing Mask: A Pilot Study (HVMASKE)

Hyperventilation | Dysfunctional Breathing | Chronic Idiopathic Hyperventilation | Respiratory Alkalosis

Denmark
Queen Sirikit National Institute of Child Health
Chulalongkorn University

Completed

Effect of Folic Acid Supplementation on Plasma Homocysteine Level in Obese Children

Obesity | Hyperhomocysteinemia

Thailand
Stanford University
BioMarin Pharmaceutical

Withdrawn

The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKU

Classical Phenylketonuria(PKU)
Hospital Universitario Dr. Jose E. Gonzalez

Unknown

Autologous Stem Cells in Newborns With Oxygen Deprivation

Multiple Organ Failure | Hypoxia, Brain | Metabolic Acidosis | Apgar; 0-3 at 1 Minute

Mexico
Kissei Pharmaceutical Co., Ltd.

Completed

A Phase II Double Blind Comparative Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD)

SCD

Japan
Rigshospitalet, Denmark

Not yet recruiting

Evaluating the Efficacy and Safety of Large Neutral Amino Acids in the Treatment of Classical Phenylketonuria

Brain Diseases | Genetic Diseases, Inborn | Metabolism, Inborn Errors | Brain Diseases, Metabolic | Phenylketonurias | Brain Diseases, Metabolic, Inborn | Amino Acid Metabolism, Inborn Errors | Metabolic Disease

Denmark
Cairo University

Recruiting

The Effect of Vitamin C on GCF Total Oxidant Capacity in Smoker Patients With Periodontitis

Vitamin C Deficiency

Egypt
Vitaflo International, Ltd
Birmingham Women's and Children's NHS Foundation Trust

Completed

Evaluation of PKU Explore (PKU Explore)

Phenylketonuria | Inborn Errors of Metabolism

United Kingdom
Vitaflo International, Ltd

Active, not recruiting

Evaluation of TYR Sphere in France (TYR sphere Fra)

Tyrosinemia | AKU

France
University of California, Davis
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Completed

Randomized Control Trial of Fluid Therapy for Pediatric Diabetic Ketoacidosis

Diabetic Ketoacidosis | Cerebral Edema

United States
University of Minnesota

Completed

Trial of L-DOPA as a Treatment to Improve Vision in Albinism

Albinism

United States
University of South Florida
National Institutes of Health (NIH)

Completed

Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment

Urea Cycle Disorders

United States
Aalborg University
North Denmark Regional Hospital

Unknown

Is Venous to Arterial Conversion (v-TAC) of Blood Gas Reliable in Critical Ill Patients in the ICU?

Sepsis | Respiratory Insufficiency | Alkalosis | Acidosis | Abdomen, Acute | Metabolic Disease

Denmark
Cycle Pharmaceuticals Ltd.
Parexel

Completed

Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin

Hereditary Tyrosinemia, Type I

South Africa
Swedish Orphan Biovitrum

Completed

Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 (HT-1)

Hereditary Tyrosinemia, Type I

Belgium, Denmark, France, Germany, Sweden
Medical University of Vienna

Completed

Renal Tubular Acidosis is Highly Prevalent in Critically Ill Patients

Acidosis, Renal Tubular
Cytonet GmbH & Co. KG

Completed

Study to Evaluate 13 C Isotope Ratio Measurement for Urea Cycle Capacity Assessment

Urea Cycle Disorders

Germany
Novartis Pharmaceuticals

Completed

Pharmacokinetics and Pharmacodynamics Study of SEG101 (Crizanlizumab) in Sickle Cell Disease (SCD) Patients With Vaso- Occlusive Crisis (VOC)

Sickle Cell Disease (SCD)

United States

Clinical Trials on SCID

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Medical Conditions

Drug Interventions

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CROs in Tanzania

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