Clinical Trials on Inborn Errors of Metabolism in United States

Total 3229 results

Shire

Completed

Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks (CHANGE 2)

Hereditary Angioedema

United States
Pharming Technologies B.V.

Completed

Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks

Hereditary Angioedema

United States, Bulgaria, Canada, Hungary, Israel, Italy, Macedonia, The Former Yugoslav Republic of, Poland, Romania, Serbia, South Africa
Baylor College of Medicine
University of California, San Diego; University of Alabama at Birmingham; Boston... and other collaborators

Completed

Sleep Abnormalities in Rare Genetic Disorders: AS, RTT, and PW (RDCRN)

Prader-Willi Syndrome | Rett Syndrome | Angelman Syndrome | Sleep Problems

United States
Transposon Therapeutics, Inc.

Active, not recruiting

A Phase 2a Study of TPN-101 in Patients With C9ORF72 ALS/FTD

Amyotrophic Lateral Sclerosis | Frontotemporal Dementia

Spain, United States, Belgium, France, Germany
ACADIA Pharmaceuticals Inc.

Completed

Safety and Efficacy of Pimavanserin in Adults With Parkinson's Disease and Depression

Treatment of Depression in Adults With Parkinson's Disease (PD)

United States
BioCryst Pharmaceuticals

Completed

Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE (APeX-2)

Hereditary Angioedema | HAE

United States, Austria, Canada, Czechia, France, Germany, Hungary, North Macedonia, Romania, Spain, United Kingdom
DePuy International

Terminated

A Two Centre Study to Assess the Stability and Long-term Performance of the C-Stem™ AMT in a Total Primary Hip Replacement

Osteoarthritis | Rheumatoid Arthritis | Avascular Necrosis | Post-traumatic Arthritis | Congenital Hip Dysplasia | Slipped Capital Femoral Epiphysis | Collagen Disorders | Traumatic Femoral Fractures | Nonunion of Femoral Fractures

United Kingdom
University of Arkansas

Withdrawn

Pancreatic Cancer Screening of High-Risk Individuals in Arkansas

Pancreatic Neoplasms | Colorectal Neoplasms, Hereditary Nonpolyposis | BRCA1 Gene Mutation | BRCA2 Gene Mutation | Hereditary Pancreatitis | Ataxia Telangiectasia | Peutz-Jegher's Syndrome | Familial Atypical Mole-Malignant Melanoma Syndrome

United States
CSL Behring

Completed

A Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema

Hereditary Angioedema Types I and II

United States, Spain, Germany, Australia, Canada, Czechia, Hungary, Israel, Italy, Romania, United Kingdom
Pfizer

Completed

Study to Compare the Effects of Repeated Doses of an Investigational New Drug and a Placebo on Appetite in Advanced Cancer and Anorexia

Anorexia | Breast Cancer | Fatigue | Colorectal Cancer | Pancreatic Cancer | Ovarian Cancer | Prostate Cancer | Cachexia | Non-small Cell Lung Cancer | Loss of Appetite

United States, Canada
Reata, a wholly owned subsidiary of Biogen

Completed

A Phase 2 Trial of the Safety and Efficacy of Bardoxolone Methyl in Patients With Rare Chronic Kidney Diseases - PHOENIX (PHOENIX)

Focal Segmental Glomerulosclerosis | IgA Nephropathy | Autosomal Dominant Polycystic Kidney | CKD Associated With Type 1 Diabetes

United States
Royal Free Hospital NHS Foundation Trust
Fisher and Paykel Healthcare; Royal Free Charity

Active, not recruiting

Domiciliary Nasal High Flow and Patient Outcomes in Chronic Hypercapnic Respiratory Failure in the United Kingdom

Chronic Obstructive Pulmonary Disease | Chronic Hypercapnic Respiratory Failure | Health-related Quality of Life | Obesity Hypoventilation Syndrome (OHS) | Nasal High Flow

United Kingdom
Novo Nordisk A/S

Recruiting

A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow (REAL 8)

SGA, Turner Syndrome, Noonan Syndrome, ISS

Korea, Republic of, United States, Ireland, United Kingdom, Finland, France, Netherlands, Japan, Thailand, Belgium, China, Israel, Brazil, India, Portugal, Austria, Bulgaria, Canada, Croatia, Germany, Greece, Malaysia, Poland, Saudi Arabia and more
University of Pennsylvania
University of South Florida; Duke University

Recruiting

Vasculitis Pregnancy Registry (V-PREG)

Vasculitis | Wegener's Granulomatosis | Systemic Vasculitis | Eosinophilic Granulomatosis With Polyangiitis (EGPA) | Churg-Strauss Syndrome (CSS) | Cryoglobulinemic Vasculitis | Behcet's Disease | CNS Vasculitis | IgA Vasculitis | Granulomatosis With Polyangiitis (GPA) | Microscopic Polyangiitis (MPA) | Polyarteritis... and other conditions

United States
DePuy International

Terminated

A Two Centre Study to Assess the Long-term Performance of the Pinnacle™ Cup With a Metal-on-Metal Bearing in Primary Total Hip Replacement

Osteoarthritis | Rheumatoid Arthritis | Avascular Necrosis | Post-traumatic Arthritis | Congenital Hip Dysplasia | Slipped Capital Femoral Epiphysis | Collagen Disorders | Traumatic Femoral Fractures | Nonunion of Femoral Fractures

United Kingdom, Italy
Denali Therapeutics Inc.
Takeda

Active, not recruiting

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL593 in Healthy Participants and Participants With Frontotemporal Dementia (FTD-GRN)

Frontotemporal Dementia

Spain, Turkey, France, Belgium, Netherlands, Portugal, Italy, Colombia, Czechia, Brazil, United Kingdom, Serbia
Mayo Clinic

Terminated

COVID-19 Vaccination in Rheumatic Disease Patients

Osteoarthritis | Systemic Lupus Erythematosus | Psoriatic Arthritis | Sjogren's Syndrome | Gout | Ankylosing Spondylitis | Inflammatory Myositis | Arthritis of Multiple Sites Associated With Inflammatory Bowel Disease (Diagnosis)

United States
University of Pennsylvania

Completed

Journey of Patients With Vasculitis From First Symptom to Diagnosis

Vasculitis | Giant Cell Arteritis | Microscopic Polyangiitis | Polyarteritis Nodosa | Systemic Vasculitis | Granulomatosis With Polyangiitis | Takayasu Arteritis | Cryoglobulinemic Vasculitis | Behcet's Disease | CNS Vasculitis | Temporal Arteritis | IgA Vasculitis | Urticarial Vasculitis | Wegener Granulomatosis | Eosinophilic... and other conditions

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
Dyax Corp.

Completed

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

Hereditary Angioedema (HAE)

United States, Canada, Germany, Italy, Jordan, Puerto Rico, United Kingdom
Presage Biosciences
Merck Sharp & Dohme LLC

Completed

PBI-MST-01 (NCT04541108) Substudy MSD-03: Intratumoral Microdosing of Pembrolizumab Alone and With MK-0482 or MK-4830 in HNSCC or STS

Soft Tissue Sarcoma | Head and Neck Squamous Cell Carcinoma

United States
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
KalVista Pharmaceuticals, Ltd.

Recruiting

PK Subtrial in Adolescent Patients With HAE Type I or II Participating in the KVD900-302 Trial

Hereditary Angioedema

United States, Bulgaria, France, Greece, Israel, Netherlands, Spain, United Kingdom, Australia, Germany, South Africa, Slovakia, Austria, New Zealand, Romania, Canada, Japan
Shire
Takeda Development Center Americas, Inc.

Completed

A Study of Long-Term Safety and Efficacy of Lanadelumab for Prevention of Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor

Angioedema

United States, France, Netherlands, Spain, Hungary, Italy, Canada, Poland, Japan, Germany
Shire

Completed

C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks

Hereditary Angioedema

United States
Novartis

Completed

Effect of Treatment With Fingolimod on the Immune Response Following Seasonal Flu Vaccination and Tetanus Booster Injection in Patients With Relapsing Multiple Sclerosis (MS)

Relapsing Multiple Sclerosis

Belgium, Poland, Spain, France, United Kingdom, Guatemala, Canada, Finland, Switzerland
Arkansas Colleges of Health Education

Recruiting

High Intensity Functional Training for Individuals With Neurologic Diagnoses and Their Care Partners (SHIFT-AR)

Stroke | Multiple Sclerosis | Brain Injuries | Parkinson Disease | Spinal Cord Injuries | Neurologic Disorder | Poliomyelitis | Spina Bifida

United States
Jazz Pharmaceuticals

Completed

A 4-Week Study of the Safety, Efficacy, and Pharmacokinetics of JZP-110 [(R)-2-amino-3-phenylpropylcarbamate Hydrochloride] in Subjects With Parkinson's Disease and Excessive Sleepiness

Parkinson Disease | Excessive Sleepiness

United States
BioCryst Pharmaceuticals

Active, not recruiting

Berotralstat Treatment in Children With Hereditary Angioedema (APeX-P)

Hereditary Angioedema | Pediatric

Spain, Austria, Canada, France, Germany, Israel, Italy, Poland, Romania, United Kingdom
CSL Behring

Completed

A Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema

Hereditary Angioedema Types I and II

United States, Spain, Australia, Canada, Czechia, Hungary, Israel, Italy, Romania, United Kingdom
TauRx Therapeutics Ltd

Completed

Safety and Efficacy Study Evaluating TRx0237 in Subjects With Behavioral Variant Frontotemporal Dementia (bvFTD)

Behavioral Variant Frontotemporal Dementia (bvFTD)

United States, Australia, Canada, Croatia, Poland, Netherlands, United Kingdom, Spain, Germany, Italy, Romania, Singapore
KalVista Pharmaceuticals, Ltd.

Completed

A Phase III, Crossover Trial Evaluating the Efficacy and Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)

Hereditary Angioedema

United States, Australia, Bulgaria, Canada, France, Germany, Greece, Hungary, Israel, Italy, Japan, Netherlands, New Zealand, North Macedonia, Poland, Portugal, Puerto Rico, Romania, Slovakia, Spain, United Kingdom
DePuy International

Terminated

A Multi-centre Study to Assess the Long-term Performance of the DePuy ASR™ System in Primary Hip Resurfacing Surgery

Osteoarthritis | Rheumatoid Arthritis | Avascular Necrosis | Post-traumatic Arthritis | Congenital Hip Dysplasia | Slipped Capital Femoral Epiphysis | Collagen Disorders | Nonunion of Femoral Fractures

Australia, Germany, United Kingdom
Takeda
Takeda Development Center Americas, Inc.

Completed

A Study of Lanadelumab in Teenagers and Adults to Prevent Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor (C1-INH)

Angioedema

United States, Spain, Netherlands, Hungary, Italy, Canada, Germany, France, Japan, Poland
Shire
Takeda Development Center Americas, Inc.

Completed

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

Hereditary Angioedema (HAE)

Canada, United States, Puerto Rico
Massachusetts General Hospital

Recruiting

Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders (BMT)

Autoimmune Diseases | Multiple Myeloma | Hodgkin Disease | AL Amyloidosis | Myelofibrosis | Chronic Kidney Disease | Sickle Cell Anemia | Thalassemia | Aplastic Anemia | Chronic Lymphocytic Leukemia (CLL) | Acute Myeloid Leukemia (AML) | Acute Lymphoblastic Leukemia (ALL) | Myelodysplastic Syndrome (MDS) | Non-Hodgkin... and other conditions

United States
University of Miami
National Institute of Neurological Disorders and Stroke (NINDS); National Institutes... and other collaborators

Recruiting

Clinical Procedures to Support Research in ALS (CAPTURE-ALS)

Amyotrophic Lateral Sclerosis | Primary Lateral Sclerosis | Progressive Muscular Atrophy | ALS-Frontotemporal Dementia

United States
KalVista Pharmaceuticals, Ltd.

Recruiting

An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)

Hereditary Angioedema

United States, Bulgaria, France, Greece, Hungary, Israel, Italy, Netherlands, New Zealand, North Macedonia, Poland, Spain, United Kingdom, Australia, Germany, South Africa, Austria, Portugal, Slovakia, Romania, Canada, Saudi Arabia, Japa...
Gynecologic Oncology Group
National Cancer Institute (NCI)

Completed

Zoledronate or Observation in Maintaining Bone Mineral Density in Patients Who Are Undergoing Surgery to Remove Both Ovaries

Osteoporosis | Ovarian Carcinoma | Hereditary Breast and Ovarian Cancer Syndrome

United States
Nido Biosciences, Inc.

Recruiting

A Study of NIDO-361 in Patients With SBMA (PIONEER KD)

Spinal and Bulbar Muscular Atrophy | Kennedy's Disease

Korea, Republic of, Denmark, Italy, United Kingdom
Shire

Completed

Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks (CHANGE 3)

Hereditary Angioedema

United States
Rhythm Pharmaceuticals, Inc.

Completed

Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity

Bardet Biedl Syndrome (BBS) | Alström Syndrome (AS)

United States, United Kingdom, France, Canada, Puerto Rico, Spain
MD Stem Cells

Enrolling by invitation

Alzheimer's Autism and Cognitive Impairment Stem Cell Treatment Study (ACIST)

Alzheimer Disease | Traumatic Brain Injury | Autism Spectrum Disorder | Lewy Body Disease | Vascular Dementia | Autism | Dementia, Mixed | Alzheimer Dementia | Dementia, Multi-Infarct | Cadasil | Parkinson-Dementia Syndrome | Chronic Traumatic Encephalopathy | Autistic Behavior | Lewy Body Dementia With Behavioral... and other conditions

United States, United Arab Emirates
Pfizer

Active, not recruiting

Study of the Efficacy and Safety of Ponsegromab in Patients With Cancer, Cachexia and Elevated GDF-15 (PROACC-1)

Fatigue | Colorectal Cancer | Pancreatic Cancer | Cachexia | Non-small Cell Lung Cancer | Loss of Appetite

China, United States, Canada, Hungary, Australia, Slovakia, Japan, Taiwan, Bulgaria, Poland, Spain, Czechia
Shire

Completed

A European Post-Authorisation Observational Study Of Patients With Hereditary Angioedema

Hereditary Angioedema (HAE)

Germany, Spain, United Kingdom, France, Italy, Belgium
University of Pennsylvania
Michael J. Fox Foundation for Parkinson's Research

Completed

Biomarkers in Neural Disorders

Parkinson's Disease | Myasthenia Gravis | Spinal Cord Injuries | Multiple System Atrophy | Progressive Supranuclear Palsy | Essential Tremor | Alzheimer's Disease | Diffuse Lewy Body Disease | Drug Induced Parkinson's Disease

United States
CSL Behring

Active, not recruiting

Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks

Hereditary Angioedema

Australia, Canada, United States, New Zealand, Germany, Hong Kong, Netherlands, Taiwan, Czechia, Israel, Hungary, Japan, Russian Federation, Spain
CSL Behring
Chiltern International Inc.

Completed

Patient Registry Study of Berinert® in Normal Clinical Practice

Includes: Hereditary Angioedema

United States, Denmark, Germany, Switzerland
KalVista Pharmaceuticals, Ltd.

Terminated

A Trial to Evaluate the Efficacy and Safety of Different Doses of KVD824 for Prophylactic Treatment of HAE Type I or II (KVD824-201)

Angioedema, Hereditary, Types I and II

United States, Czechia, Germany, Hungary, Italy, North Macedonia, United Kingdom, Australia, Bulgaria, Canada, France, New Zealand, Puerto Rico

Clinical Trials on Inborn Errors of Metabolism in United States

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Bahamas

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations