Clinical Trials Sponsored by Foundation for Rare Diseases

Total 495 results

The Hospital for Sick Children
Rare Disease Foundation

Completed

Comparing Body Composition Assessment Methods

Body Composition | Pediatric ALL | Gastrointestinal Failure

Canada
Imagine Institute
Reference center for rare diseases (Rare Gynecologic Diseases)

Recruiting

Molecular Genetic Study of Mayer-Rokitansky-Kuster-Hauser Syndrome (MRKH)

Mayer Rokitansky Kuster Hauser Syndrome

France
Assistance Publique - Hôpitaux de Paris
French rare diseases Healthcare Network; The French Foundation for Rare Diseases

Active, not recruiting

Non-syndromic Inherited Anomalies of Mineralized Tooth Tissues: a Whole Exome Study to Identify New Pathogenic Variants (EXODENT)

Amelogenesis Imperfecta | Dentinogenesis Imperfecta | Dentin Anomalies

France
The Hospital for Sick Children
Rare Disease Foundation, Vancouver, Canada

Completed

Markers of Bone Disease in Children on Parenteral Nutrition

Metabolic Bone Disease

Canada
The Hospital for Sick Children
Rare Disease Foundation Microgrant competition

Completed

Chromium Contamination of Parenteral Nutrition

Trace Element Excess

Canada
Université de Sherbrooke
Rare Disease Foundation

Completed

Effects of a 12-week Strength Training Program in Women With Myotonic Dystrophy Type 1

Myotonic Dystrophy 1

Canada
Foundation For Rare Disease Research
Institute for Asthma & Allergy; Virant Diagnostics, Inc.; MedBio Reference Laboratories...

Not yet recruiting

Angioedema Biomarker Research Study

Angioedema | Urticaria | C1 Inhibitor Deficiency | Angioedemas, Hereditary | Mastocytosis | Indolent Systemic Mastocytosis | Systemic Mastocytoses | ACE Inhibitor-Induced Angioedema
Centre for Human Drug Research, Netherlands
Recordati Rare Diseases

Completed

Deep Phenotyping of Cutaneous T Cell Lymphoma, Type Mycosis Fungoides

Mycosis Fungoides

Netherlands
University of British Columbia
Rare Disease Foundation, Vancouver, Canada

Completed

Simple Breath Test to Examine Phenylalanine Metabolism

Phenylketonuria (PKU)

Canada
University of British Columbia
Rare Disease Foundation, Vancouver, Canada

Completed

Energy Requirements in Mitochondrial Disease

Mitochondrial Disease | Chronic Metabolic Disorder

Canada
Colombian Foundation for Epilepsy and Neurological...

Unknown

Efficacy of Tranexamic Acid in Brain Tumor Resections (COLFIRE)

Neoplasms | Brain Tumors | Gliomas | Astrocytomas | Meningiomas

Colombia
Assistance Publique - Hôpitaux de Paris
Foundation for Rare Diseases; Necker Imagine Foundation; Laboratory of Clinical...

Completed

Evolution of the Quality of Life and Experience of Young Women With Utero-vaginal Aplasia (MRKHPSY) (MRKHPSY)

Mrkh Syndrome

France
University of Rochester
National Center for Research Resources (NCRR); Rare Diseases Clinical Research... and other collaborators

Completed

Characteristics of Andersen-Tawil Syndrome

Andersen-Tawil Syndrome | Andersen Syndrome

United States, Canada, Italy, United Kingdom
European Foundation for the Care of Newborn Infants

Not yet recruiting

Self-assessment Tool for Implementing the ESCNH

Self-Assessment | Development of a Tool to Assess the Level of Implementing the European Standards of Care for Newborn Health | Testing of the Developed Self-assessment Tool in European Hospitals

Germany
National Institute for Communicable Diseases, South...
Bill and Melinda Gates Foundation; Centers for Disease Control and Prevention

Unknown

Effectiveness of Trivalent Inactivated Influenza Maternal Vaccination Among Pregnant Women and Their Newborns in South Africa

Influenza Viral Infections

South Africa
University Hospital for Infectious Diseases, Croatia
Croatian Science Foundation

Completed

The Role of Immune Semaphorins in NAFLD (SepsisFAT)

Immune Response | Biomarkers | Liver Fibrosis | Non-Alcoholic Fatty Liver Disease | Non Alcoholic Steatohepatitis

Croatia
University Hospital for Infectious Diseases, Croatia
Croatian Science Foundation

Recruiting

The Role of Immune Semaphorins in Steatotic Liver Disease and Sepsis (SepsisFAT)

Sepsis | Immune Response | Steatosis of Liver

Croatia
German Center for Neurodegenerative Diseases (DZNE)
German Research Foundation

Completed

The Effects of Attentional Filter Training on Working Memory (DIIN)

Healthy | Cognitive Decline | Age-related Cognitive Decline

Germany
University Hospital, Grenoble
French Society for Intensive Care; Act For Chronic Diseases

Unknown

Prone Position and Renal Resistive Index (PRO-KIDNEY)

Acute Kidney Injury | Acute Respiratory Distress Syndrome | Intra-Abdominal Hypertension | Prone Position
National Institute of Allergy and Infectious Diseases...
IPPOX Foundation; EuroVacc Foundation; Global Solutions for Infectious Diseases

Withdrawn

Evaluating the Safety and Immune Response to Five Different Combinations of HIV Vaccines in Healthy, HIV-Uninfected Adults

HIV Infections
Aplastic Anemia and MDS International Foundation
National Organization for Rare Disorders

Recruiting

Global PNH Patient Registry

Paroxysmal Nocturnal Hemoglobinuria

United States
Mario Negri Institute for Pharmacological Research
Heart Care Foundation

Completed

CYCLosporinE A in Reperfused Acute Myocardial Infarction (CYCLE)

Acute Myocardial Infarction

Italy
Andrea Gropman
National Center for Research Resources (NCRR); Rare Diseases Clinical Research... and other collaborators

Completed

Neurologic Injuries in Adults With Urea Cycle Disorders

Brain Diseases, Metabolic, Inborn | Ornithine Transcarbamylase Deficiency | Urea Cycle Disorder

United States
Rambam Health Care Campus
Israeli association for lung disease

Completed

Prospective Study of the Impact of Systemic Corticosteroid Use on Measures of Sleep Disordered Breathing

Sleep Apnea

Israel
China National Center for Cardiovascular Diseases
China International Medical Foundation

Recruiting

The Effectiveness and Safety of Levosimendan in Patients With Severe Aortic Stenosis and Heart Failure Undergoing Transcatheter Aortic Valve Replacement

Heart Failure | Aortic Valve Stenosis | Transcatheter Aortic Valve Replacement | Cardiac Event | Safety Issues | Effect of Drug | Inotropic Agents

China
Manuel Monreal

Completed

Prevalence of Severe Bleeding in COVID-19 Patients Treated With Higher Than Recommended Thromboprophylaxis Doses (BLEEDING)

COVID-19

Spain
Drugs for Neglected Diseases
Bayer; Bill and Melinda Gates Foundation

Completed

First in Man Clinical Trial of Emodepside (BAY 44-4400)

Healthy Volunteers

United Kingdom
Drugs for Neglected Diseases
Bayer; Bill and Melinda Gates Foundation

Completed

Relative Bioavailability Study of Emodepside IR-tablets and Solution

Filariasis

United Kingdom
Manuel Monreal

Completed

Prevalence of DVT or PE in Patients Infected With COVID-19 Admitted to Hospital (SCREENING)

COVID-19

Spain
Manuel Monreal

Recruiting

Computerized Registry of Patients With Venous Thromboembolism (RIETE) (RIETE)

Venous Thromboembolism

United States, France, Spain, Israel, Italy, Czechia, Vietnam, Austria, Switzerland, Latvia, Argentina, Belgium, Brazil, China, Colombia, Ecuador, Egypt, Germany, Greece, Iran, Islamic Republic of, Ireland, Japan, Morocco, North Macedonia and more
Drugs for Neglected Diseases
Bayer; Bill and Melinda Gates Foundation

Completed

Safety, Tolerability and PK of Multiple-ascending Doses of Emodepside

Filariasis

United Kingdom
ASST Gaetano Pini-CTO
Grisons Foundation for Parkinson's Disease

Completed

Mucuna Pruriens Therapy in Parkinson's Disease

Parkinson's Disease

Bolivia
International Centre for Diarrhoeal Disease Research...
Nestlé Foundation

Completed

Oral Rehydration SolutionContaining Amylase Resistant Starch in Severely Malnourished Children.

Diarrhoea | Severe Malnutrition

Bangladesh
National Center for Research Resources (NCRR)
Mayo Clinic

Completed

Phase III Randomized, Double-Blind, Placebo-Controlled Study of 3,4-Diaminopyridine for Demyelinating Polyneuropathy

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
National Center for Research Resources (NCRR)
Northwestern University

Completed

Phase II Pilot Study of Extracorporeal Phototherapy for Epidermolysis Bullosa Acquisita

Epidermolysis Bullosa Acquisita
National Center for Research Resources (NCRR)
Yale University

Completed

Phase III Randomized, Placebo-Controlled, Crossover Study of Soy Protein Isolate for Hereditary Hemorrhagic Telangiectasia

Hereditary Hemorrhagic Telangiectasia
National Center for Research Resources (NCRR)
Northwestern University

Completed

Randomized Placebo-Controlled Study of Aerobic Exercise and Resistance Training Plus Megestrol Acetate for HIV-Wasting

HIV Infections | Acquired Immunodeficiency Syndrome | HIV Wasting Syndrome
National Center for Research Resources (NCRR)
Northwestern University

Completed

Phase II Study of Lactulose and Circadian Rhythms in Patients With Cirrhosis

Hepatic Encephalopathy | Cirrhosis | Portal Hypertension
National Center for Research Resources (NCRR)
Emory University

Completed

Phase II Study of Pallidotomy for Parkinson Disease

Parkinson Disease
National Center for Research Resources (NCRR)
Northwestern University

Unknown

Study of Novel Types of Familial Diabetes Insipidus

Diabetes Insipidus
National Center for Research Resources (NCRR)
Stanford University

Completed

Pilot Study of Enalapril and Renal Function in Patients With IgA Nephropathy

IGA Glomerulonephritis
National Center for Research Resources (NCRR)
Yale University

Completed

Phase II Double Blind Placebo Controlled Trial of Risperidone in Tourette Syndrome

Tourette Syndrome
National Center for Research Resources (NCRR)
Boston University

Completed

Study of Topical Calcitriol in Children With Psoriasis

Psoriasis
National Center for Research Resources (NCRR)
Northwestern University

Completed

Phase II Randomized Study of Cidofovir for Peripheral Cytomegalovirus Retinitis

Acquired Immunodeficiency Syndrome | Cytomegalovirus Retinitis
National Center for Research Resources (NCRR)
Northwestern University

Completed

Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dehydroepiandrosterone in Women With Active Systemic Lupus Erythematosus

Systemic Lupus Erythematosus
National Center for Research Resources (NCRR)
Yale University

Completed

Phase III Randomized, Double-Blind, Placebo-Controlled Study of Guanfacine for Tourette Syndrome and Attention Deficit Hyperactivity Disorder

Tourette Syndrome
National Center for Research Resources (NCRR)
Northwestern University

Completed

Phase II Study of Long-Term Dehydroepiandrosterone for Systemic Lupus Erythematosus

Systemic Lupus Erythematosus
National Center for Research Resources (NCRR)
Stanford University

Completed

A Maintenance Extension of Phase I Pilot Study of Chimeric Anti-CD4 Antibody M-T412 in Patients With Multiple Sclerosis

Multiple Sclerosis
National Center for Research Resources (NCRR)
Yale University

Completed

Phase II Pilot Study of Octreotide, a Somatostatin Octapeptide Analog, for Gastrointestinal Hemorrhage in Hormone-Refractory Hereditary Hemorrhagic Telangiectasia and Senile Ectasia

Hereditary Hemorrhagic Telangiectasia | Ectasia
National Center for Research Resources (NCRR)
Northwestern University

Completed

Phase II/III Randomized, Double-Blind, Placebo-Controlled Study of Dehydroepiandrosterone in Women With Mild to Moderate Systemic Lupus Erythematosus

Systemic Lupus Erythematosus

Clinical Trials Sponsored by Foundation for Rare Diseases

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Rare Diseases

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