Search clinical trials for: Inherited mitochondrial disease

Total 383628 results

I.R.C.C.S. Fondazione Santa Lucia
Università Foro Italico Roma; CNR Pisa

Not yet recruiting

Motor Function Efficacy of Pharmacological Treatments Targeting Energy Metabolism, in Parkinson's Patients

Parkinson Disease | Metabolic Disease | Gait Analysis | Therapy, Directly Observed
Institute of Hematology & Blood Diseases Hospital

Recruiting

An Exploratory Study of Cluster of Differentiation 38 (CD38) Monoclonal Antibody Combined Corticosteroid in Acquired Hemophilia A (AHA2023)

Acquired Hemophilia

China
University of California, San Diego
New York University; Heinrich-Heine University, Duesseldorf; Universität Tübingen

Recruiting

Natural History Study of Patients With HPDL Mutations

Genetic Disease | Mitochondrial Encephalomyopathies | Neonatal Encephalopathy | Mutation | Hereditary Spastic Paraplegia | White Matter Disease | Spastic Paraplegia

United States
Centro di Riferimento Oncologico - Aviano

Recruiting

InheriteD brEast caNcer iTalian regIsTrY A Retrospective-prospective Observational Cohort Study to Evaluate Cancer Prevention Strategies in Women With a Deleterious Mutation in BRCA1-2 (IDENTITY)

Breast Cancer

Italy
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Study of the Selective GlyT1 Inhibitor Bitopertin for Steroid-Refractory Diamond-Blackfan Anemia

Steroid-refractory Diamond-Blackfan Anemia (DBA)

United States
Neurophth Therapeutics Inc

Recruiting

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations

Leber Hereditary Optic Neuropathy (LHON)

China, United States
University of Lausanne

Recruiting

Orphan Drugs for Inherited Metabolic Diseases

Inborn Errors of Metabolism

Switzerland
Institute for Atherosclerosis Research, Russia

Not yet recruiting

The Effects of Allicor on Patients After Revascularization Treatment During a Year (TEA-PARTY)

Peripheral Arterial Disease | Atherosclerosis

Russian Federation
SWOG Cancer Research Network
National Cancer Institute (NCI)

Recruiting

Carboplatin Chemotherapy Before Surgery for People With High-Risk Prostate Cancer and an Inherited BRCA1 or BRCA2 Gene Mutation

Prostate Adenocarcinoma | Stage IIIB Prostate Cancer AJCC v8 | Stage IIIC Prostate Cancer AJCC v8

United States
Institute for Atherosclerosis Research, Russia

Recruiting

The Effects of Allicor on Patients After Coronary Arteria Revascularization Treatment (TEA-CART)

Myocardial Ischemia | Coronary Arteriosclerosis | Coronary Artery Bypass Grafting | Percutaneous Coronary Revascularization

Russian Federation
Christoph Königs
Roche Pharma AG; Chugai Pharma Germany GmbH

Recruiting

Dynamics of the Anti-factor VIII Antibody Signature During Treatment With Emicizumab (NAVIGATE)

Severe Hemophilia A | Severe Hemophilia A With Inhibitor | Severe Hemophilia A Without Inhibitor

Germany
Franziska Wachter
Harvard Clinical and Translational Science Center (Harvard Catalyst)

Recruiting

A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies (MORE)

Acute Myeloid Leukemia | Myelodysplastic Syndromes | MDS | Aml | Myeloid Neoplasm | Myeloid Malignancies | Inherited Bone Marrow Failure Syndrome

United States
Istituto Superiore di Sanità
Fondazione G.B. Bietti, IRCCS; Ospedale Pediatrico Bambin Gesù

Recruiting

Mechanisms of Inherited Retinal Dystrophies Using Whole Genome Sequencing and in Vitro and in Vivo Models

Retinitis Pigmentosa | Macular Dystrophy | Inherited Retinal Dystrophy

Italy
ApcinteX Ltd
Centessa Pharmaceuticals plc

Recruiting

The Efficacy and Safety of SerpinPC in Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B (PRESent-2)

Hemophilia A | Hemophilia B

United States, Italy, Spain, Taiwan, Australia, Belgium, United Kingdom, France, Germany, India, South Africa, Canada, Poland, Armenia, Turkey
ApcinteX Ltd
Centessa Pharmaceuticals plc

Recruiting

A Study of SerpinPC in Participants With Hemophilia B (HemB) With Inhibitors (PRESent-3)

Hemophilia B | Hemophilia B With Inhibitor

United States, Spain, Australia, Taiwan, France, Armenia, Germany
Vega Therapeutics, Inc

Recruiting

Study of VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease

Von Willebrand Diseases

United States, Austria, South Africa, United Kingdom
Tomsk National Research Medical Center of the Russian...
Russian Science Foundation

Recruiting

Ultrastructural Characteristics of Mitochondria in Cardiomyocytes in Heart Failure (MITOCH-HF)

Coronary Artery Disease | Heart Failure

Russian Federation
Children's Hospital of Fudan University
First Affiliated Hospital, Sun Yat-Sen University; Shandong Provincial Hospital and other collaborators

Recruiting

Effects of Traditional Chinese Medicine (GSJYF) in Children With Inherited Proteinuric Kidney Disease

Kidney Diseases | Proteinuria | Hereditary Nephropathy

China
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Emotions in the Communication and Relationship Styles of Parents With Hemophilic Children.

Hemophilia A

Italy
National University Hospital, Singapore
National University of Singapore

Recruiting

In Vivo Investigation on Mitochondrial Dysfunction in Post-COVID Fatigue and Cancer Fatigue.

COVID-19 | Fatigue | Mitochondrial Dysfunction

Singapore
Istituto Auxologico Italiano

Recruiting

Oxidative Stress and Mitochondrial TERT in Papillary Thyroid Cancer. (TEMPEST)

Papillary Thyroid Cancer

Italy
UMC Utrecht
Julius Clinical

Not yet recruiting

Mitochondrial dysfUnction: a Key Player in Doxorubicin-induced Skeletal and Cardiac muscLE Damage (MUSCLE)

B-cell Lymphoma | Chemotherapeutic Toxicity | T-cell Lymphoma | Doxorubicin Induced Cardiomyopathy

Netherlands
Institute of Hematology & Blood Diseases Hospital

Not yet recruiting

Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old

Hemophilia B
Laboratoire français de Fractionnement et de Biotechnologies

Recruiting

A Phase 3 Study of the Safety and Efficacy of Coagulation Factor VIIa (Recombinant) for the Prevention of Excessive Bleeding in Patients With Congenital Hemophilia A or B With Inhibitors to Factor VIII or IX Undergoing Elective Major Surgical Procedures SCOPE HIM (SCOPE HIM)

Hemophilia

United States, Thailand, Mexico, Turkey, South Africa, Malaysia
National Cancer Institute (NCI)

Recruiting

Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia

Fanconi Anemia | Inherited Bone Marrow Failure Syndrome

United States
Novo Nordisk A/S

Recruiting

A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A (FRONTIER 4) (FRONTIER4)

Haemophilia A With Inhibitors | Haemophilia A

Taiwan, Canada, Spain, Netherlands, Korea, Republic of, India, United States, United Kingdom, China, Portugal, Lithuania, Turkey, South Africa, Ireland, Italy, France, Japan, Belgium, Malaysia, Poland, Austria, Bulgaria, Denmark, Germany and more
Sohag University

Recruiting

Mitochondrial DNA Copy Number and Leukocyte Telomere Length as Biomarkers in Type 2 Diabetic Patients

Type 2 Diabetes Mellitus

Egypt
Sanofi

Recruiting

A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia (ATLAS-NEO)

Hemophilia

Greece, Korea, Republic of, France, Taiwan, Italy, Spain, United States, Canada, China, Germany, India, Japan, Mexico, South Africa, Turkey, Poland, Saudi Arabia
Hospital Universitari Vall d'Hebron Research Institute

Recruiting

Digestive Dysmotility in Mitochondrial Neurogastrointestinal Encephalomyopathy

MNGIE | Dysmotility

Spain
Cristina Domínguez González

Recruiting

Natural History in Primary Mitochondrial Myopathies (NHPMM)

Primary Mitochondrial Myopathies

Spain
Heinrich-Heine University, Duesseldorf

Recruiting

Reference Values of Intraepidermal Nerve Fiber Density in Children and Small Fiber Neuropathy in Neurometabolic and Neurodevelopmental Disorders in Children (SFN_children)

Cerebral Palsy | Mitochondrial Diseases | Small Fiber Neuropathy | Developmental Delay Disorder

Germany
Medical University Innsbruck

Recruiting

Mitochondrial Function in Peripheral Arterial Disease (MIVA)

Cardiovascular Diseases | Peripheral Arterial Disease

Austria
Bayer

Recruiting

An Observational Study Called JOIHA to Learn More About How Well the Treatment With Jivi Works to Prevent Problems With Joints in Adults With Hemophilia A (JOIHA)

Hemophilia A | Prophylaxis of Bleeding

Italy
Institute of Hematology & Blood Diseases Hospital

Not yet recruiting

A Study to Evaluate the Safety and Efficacy of ZS801 in Adult Hemophilia B Patients

Hemophilia B

China
Qianfoshan Hospital

Not yet recruiting

Neutrophil-to-lymphocyte and Platelet-to-lymphocyte Ratio in Predicting the Incidence of Nausea and Vomiting

Hemophilia A

China
Institute of Hematology & Blood Diseases Hospital

Not yet recruiting

The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.

Hemophilia B

China
Groupe Maladies hémorragiques de Bretagne

Recruiting

Genetic Influence of Genetic Factors Influencing the Desmopressin's Efficacy in Mild/Moderate Hemophilia A (GIDEMHA)

Desmopressin | Hemophilia A, Mild | Factor VIII

France
University of Washington

Recruiting

Disulfiram for Treatment of Retinal Degeneration

Inherited Retinal Dystrophy Primarily Involving Sensory Retina

United States
Novo Nordisk A/S

Recruiting

An Observational Research Study of the Health of Joints in People With Haemophilia Taking the Medicine Esperoct

Haemophilia A

Canada
VA Office of Research and Development
University of Pittsburgh

Recruiting

Nitrite Supplementation in Long COVID Patients

Long COVID | Cardiorespiratory Fitness

United States
Centre Hospitalier Universitaire de Saint Etienne

Recruiting

In Vitro Correction of Thrombin Generation by Concizumab (Anti-TFPI) for Severe Hemophilia Patients

Hemophilia

France
Opus Genetics, Inc

Recruiting

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD)

LCA5

United States
Pfizer

Recruiting

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B (BASIS KIDS)

Hemophilia A | Hemophilia B

Denmark, Taiwan, Canada, Turkey, Spain, China, United Kingdom, South Africa, Italy, Saudi Arabia, Israel, Korea, Republic of, Austria, Germany, Japan, Australia, India, Slovakia, France, United States, Brazil, Argentina
Virginia Commonwealth University
American Heart Association

Recruiting

Mitochondrial Derived Reactive Oxygen Species on Cardiovascular Health in Chronic Obstructive Pulmonary Disease (COPD)

Chronic Obstructive Pulmonary Disease

United States
ApcinteX Ltd
Centessa Pharmaceuticals plc

Recruiting

Hemophilia Non-Interventional Study Prior to SerpinPC Intervention (PRESent-5)

Hemophilia A | Hemophilia B

Spain, Taiwan, United Kingdom, Italy, Germany, United States, South Africa, Australia, Poland, Armenia, France, India, Canada, Brazil, Turkey, Egypt
Investigación en Hemofilia y Fisioterapia

Not yet recruiting

Clinical, Functional and Musculoskeletal Differences Between Adult Patients With Hemophilia and Their Healthy Peers

Hemophilia Arthropathy
Eduardo N. Chini

Recruiting

A Study to Evaluate Vitamin B3 Derivative to Treat Mitochondrial Myopathy

Mitochondrial Myopathy

United States
Jaeb Center for Health Research
Foundation Fighting Blindness

Recruiting

Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants (Uni-Rare)

Retinitis Pigmentosa | Inherited Retinal Degeneration

United States, United Kingdom, France, Finland, Israel, Netherlands, Switzerland, Australia, Canada, Brazil, Mexico
Takeda

Not yet recruiting

A Study of Recombinant Von Willebrand Factor (rVWF) (TAK-577) in Children With Severe Von Willebrand Disease (vWD)

Von Willebrand Disease (VWD)
BioMarin Pharmaceutical

Recruiting

A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)

Hemophilia A

Argentina

Search clinical trials for: Inherited mitochondrial disease

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