Clinical Trials on Lipid Metabolism, Inborn Errors

Total 7154 results

National Neuroscience Institute
Nanyang Technological University; Institute for Infocomm Research; Institute... and other collaborators

Active, not recruiting

[BrainConnexion] - Neurodevice Phase I Trial

Motor Neuron Disease | Amyotrophic Lateral Sclerosis | Muscular Dystrophies | Spinal Cord Injuries | Tetraplegia | Tetraplegia/Tetraparesis | Locked-in Syndrome

Singapore
National Institute of Neurological Disorders and...

Completed

Study of Hepatic Function in Patients With Spinal and Bulbar Muscular Atrophy

Motor Neuron Disease | Spinal and Bulbar Muscular Atrophy (SBMA)

United States
Sheba Medical Center

Completed

Amantadine for Improving Neurologic Symptoms in Ataxia-Telangiectasia

Fatigue | Ataxia | Dystonia | Parkinsonism | Chorea

Israel
University Hospital, Grenoble

Recruiting

Status of Dental Care Practices in Patients With Hereditary Angioedema (PRADA)

Angio Edema Hereditary

France
Takeda

Recruiting

A Survey of Icatibant in Pediatric Participants With Hereditary Angioedema

Hereditary Angioedema (HAE)

Japan
Sidney Kimmel Comprehensive Cancer Center at Johns...

Recruiting

Reduced Intensity BMT for Immune Dysregulatory and Bone Marrow Failure Syndromes Using Post-Transplant Cyclophosphamide

Primary Immune Deficiency Disorder | Bone Marrow Failure | Immune Deficiency Disease

United States
Seoul National University Hospital

Recruiting

Prevention of Scoliosis in Patients With Duchenne Muscular Dystrophy Using Portable Seat Device

Duchenne Muscular Dystrophy | Scoliosis Neuromuscular | Lordosis Lumbar

Korea, Republic of
Hoffmann-La Roche

Active, not recruiting

A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of Emicizumab in Participants With Mild or Moderate Hemophilia A Without FVIII Inhibitors (HAVEN 6)

Hemophilia A | Mild Hereditary Factor VIII Deficiency Disease Without Inhibitor | Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor

United States, Netherlands, Belgium, France, Germany, South Africa, Spain, Canada, United Kingdom, Poland
Shire
Takeda Development Center Americas, Inc.

Active, not recruiting

A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II (ENABLE)

Hereditary Angioedema (HAE)

Germany, Switzerland, Spain, Israel, Italy, Austria, Portugal, Kuwait
Shire
Takeda

Completed

FIRAZYR General Drug Use-Results Survey (Japan)

Hereditary Angioedema (HAE)

Japan
Takeda

Completed

A Study of Lanadelumab (SHP643) in Chinese Participants With Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

China
University of Colorado, Denver
Cystic Fibrosis Foundation

Terminated

Sensor Augmented Pump (SAP) Therapy for Inpatient CFRD Management

Cystic Fibrosis-related Diabetes | Cystic Fibrosis Pulmonary Exacerbation | Cystic Fibrosis in Children

United States
BioCryst Pharmaceuticals

Completed

Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan (APeX-J)

Hereditary Angioedema, HAE

Japan
University of British Columbia

Suspended

Gene Mutations and Orthopaedic Symptoms Correlation of Multiple Hereditary Exostoses: Multicentre Project

Exostoses, Multiple Hereditary

Canada
Shire

Completed

Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

United States
Shire

Completed

Long-term Safety and Efficacy Study of DX-2930 (SHP643) to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE

Hereditary Angioedema (HAE)

United States, United Kingdom, Germany, Canada, Italy, Jordan, Puerto Rico
Shire

Completed

Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1 Esterase Inhibitor for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

Hereditary Angioedema (HAE)

Spain, Israel, United States, Hungary, Germany, Canada, Romania
Shire

Completed

CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12

Hereditary Angioedema (HAE)

United States, Germany, Hungary
Shire

Completed

Subcutaneous CINRYZE With Recombinant Human Hyaluronidase for Prevention of Angioedema Attacks

Hereditary Angioedema (HAE)

United States, Germany, Spain, Sweden
Shire

Completed

Open-label, Single-arm Study to Assess the Pharmacokinetics, Safety, and Tolerability of a Single Subcutaneous Dose of Icatibant in Healthy Japanese Volunteers

Hereditary Angioedema (HAE)

United States
Shire

Completed

Safety and Efficacy Study of CINRYZE for Prevention of Angioedema Attacks in Children Ages 6-11 With Hereditary Angioedema

Hereditary Angioedema (HAE)

Israel, Germany, Mexico, United States, Romania
Shire

Completed

A Study of Icatibant for Acute Attacks of Hereditary Angioedema in Japanese Participants

Hereditary Angioedema (HAE)

Japan
Shire

Completed

A Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Participants

Hereditary Angioedema (HAE)

United States, Jordan, Italy
BioCryst Pharmaceuticals

Completed

Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema (APeX-1)

Hereditary Angioedema (HAE)

Canada, United Kingdom, Germany, Spain, Australia, Hungary, Italy, Austria, North Macedonia, Denmark, Switzerland
Stanley Jordan, MD
CSL Behring

Completed

Safety & Tolerability of Berinert® (C1 Inhibitor) Therapy to Prevent Rejection

Kidney Transplantation

United States
Second Affiliated Hospital, School of Medicine,...

Unknown

The Pathophysiology of Tourette Syndrome: a Multimodal Study (PTS)

Tourette Syndrome

China
Penn State University

Withdrawn

C1-INH Compared to Placebo at the Time of Prodromal Symptoms for Hereditary Angioedema (HAE) Exacerbation

Hereditary Angioedema
University Hospital Muenster

Completed

Epigenetics and Metabolic Disorders in Men With the Klinefelter Syndrome (IZKF-CRA03-09)

Klinefelter Syndrome, Hypogonadism
Pharming Technologies B.V.
University of Wisconsin, Madison

Withdrawn

Recombinant Human C1 Inhibitor for the Treatment of Early Antibody-Mediated Rejection in Renal Transplantation

Kidney Transplantation | Graft Rejection

United States
Centre Hospitalier Sud Francilien

Not yet recruiting

Infectious and Non-infectious Lower Respiratory Diseases in Children With Down Syndrome Followed in Pediatric Pulmonology Consultations in Ile-de-France (ARBT21)

Down Syndrome

France
University of Kansas Medical Center

Not yet recruiting

Brain Outcomes With Lifestyle Change in Down Syndrome (BOLD)

Obesity | Alzheimer Disease | Down Syndrome

United States
Takeda

Recruiting

A Study to Review the Treatment and Outcomes of Teenagers and Adults With Non-histaminergic Angioedema With Normal C1 Inhibitor in Canada (PROSPECT)

Angioedema | Hereditary Angioedema (HAE)

Canada
Takeda

Recruiting

A Study in Adults With Hereditary Angioedema (HAE) Who Currently Receive Icatibant at Home

Hereditary Angioedema (HAE)

United Kingdom
Shire
Takeda Development Center Americas, Inc.

No longer available

Expanded Access for the Prevention of Acute Attacks of 1) Hereditary Angioedema (HAE) in Children and 2) Non-histaminergic Angioedema With Normal C1-Inhibitor (C1-INH) in Teenagers and Adults

Angioedema | Hereditary Angioedema (HAE)

United States, Canada, Germany, Hungary, Spain
Istituto Ortopedico Rizzoli

Completed

Height, Ulnar Length and Forearm Function in Multiple Hereditary Exostoses

Exostoses, Multiple Hereditary

Italy
BioCryst Pharmaceuticals

Completed

A Long Term Safety Study of BCX7353 in Hereditary Angioedema (APeX-S)

Hereditary Angioedema | Prophylaxis | HAE

Austria, Denmark, France, Germany, Hungary, Israel, Italy, North Macedonia, Poland, Switzerland, United Kingdom, United States, Australia, Hong Kong, Korea, Republic of, New Zealand, Serbia, Slovakia, South Africa, Spain
Shire

Completed

Efficacy and Safety of Lanadelumab (SHP643) in Japanese Participants With Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

Japan
Shire

Completed

A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema

Hereditary Angioedema (HAE)

United States, Canada, Australia, Austria, Colombia, Germany, Hungary, Israel, Italy, Spain
Shire

Completed

Safety and Efficacy Study of Repeated Doses of DX-88 (Ecallantide) to Treat Attacks of Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

United States, Canada, Jordan
Shire

Completed

Observational Safety Study for KALBITOR (Ecallantide) in the Treatment of Acute Attacks of Hereditary Angioedema

Hereditary Angioedema (HAE)

United States
Shire

Completed

Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of Recombinant Human C1 Esterase Inhibitor in Healthy Adult Subjects

Hereditary Angioedema (HAE)

United States
Shire

Completed

Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

Japan
Shire

Completed

EDEMA2: Evaluation of DX-88's Effect in Mitigating Angioedema

Hereditary Angioedema (HAE)
Shire
Dyax Corp.

Completed

A Single Increasing Dose Study to Assess Safety and Tolerability of DX-2930 in Healthy Subjects

Hereditary Angioedema (HAE)

United States
Novo Nordisk A/S

Completed

Awareness, Care, and Treatment In Obesity Management of Patients With Hemophilia (ACTION) to Inform Hemophilia Obesity Patient Empowerment (HOPE) (ACTION to HOPE)

Haemophilia A, Haemophilia B, Obesity

United States
Pharming Technologies B.V.

Completed

A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration (OPERA)

Hereditary Angioedema | Genetic Disorders

Netherlands, Romania
Duke University
Foundation for Prader-Willi Research

Completed

Prader-Willi Syndrome Macronutrient Study

Childhood Obesity | Prader Willi Syndrome | Syndromic Obesity

United States, Canada
Pharming Technologies B.V.

Completed

Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

Genetic Disorders

Netherlands
Pharming Technologies B.V.

Completed

Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

Hereditary Angioedema | Angioneurotic Edema

Netherlands
Pharming Technologies B.V.

Completed

Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

Hereditary Angioedema | Genetic Disorders | Angioneurotic Edema

Netherlands, Romania

Clinical Trials on Lipid Metabolism, Inborn Errors

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Lithuania

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations