Clinical Trials on Mitochondrial Genetic Disorders

Total 151245 results

Affiliated Hospital of Nantong University

Not yet recruiting

CYP1A2, ABCB1, CYP2C9 and Plasma Concentration of Agomelatine in Adult Patients With Depression

Depression | Polymerase Chain Reaction | Polymorphism, Genetic | CYP1A2 Polymorphism | Plasma Concentration | CYP2C9 Polymorphism
Icahn School of Medicine at Mount Sinai

Recruiting

DDX3X Syndrome -The Seaver Autism Center for Research and Treatment is Characterizing DDX3X-related Neurodevelopmental Disorders Using Genetic, Medical, and Neuropsychological Measures.

Autism Spectrum Disorder | DDX3X | Mental Retardation, X-linked 102

United States
Novo Nordisk A/S

Completed

Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B. (paradigm™5)

Congenital Bleeding Disorder | Haemophilia B

United States, Canada, United Kingdom, France, Malaysia, Brazil, Croatia, Germany, Italy, Japan, Taiwan
Novo Nordisk A/S

Completed

Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B: An Extension to Trials NN7999-3747 and NN7999-3773 (paradigm™ 4)

Congenital Bleeding Disorder | Haemophilia B

United States, Netherlands, Austria, Spain, Germany, Macedonia, The Former Yugoslav Republic of, Taiwan, United Kingdom, Italy, Malaysia, Thailand, France, Greece, Russian Federation, Turkey, Japan, South Africa, Romania
PYC Therapeutics

Not yet recruiting

Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation

Optic Atrophies, Hereditary | Autosomal Dominant Optic Atrophy | Optic Atrophy, Autosomal Dominant | Kjer Optic Atrophy

Australia
Novo Nordisk A/S

Completed

A Trial Comparing Nonacog Beta Pegol (N9-GP) and ALPROLIX® in Patients With Haemophilia B (paradigm™7)

Congenital Bleeding Disorder | Haemophilia B

United States, Switzerland, Germany
Novo Nordisk A/S

Completed

Bioequivalence of Two Somatropin Products (Norditropin® Versus Genotropin®) in Healthy Adult Volunteers

Healthy | Prader-Willi Syndrome | Genetic Disorder | Growth Disorder | Idiopathic Short Stature

United States
National Institute of Mental Health (NIMH)

Terminated

Evaluating Genetic Risk Factors for Childhood-Onset Schizophrenia

Schizophrenia | Schizoaffective Disorder | Psychosis | Childhood Onset Psychotic Disorders | Genetic Structure

United States
University of Guelph
McMaster University; Laval University

Not yet recruiting

Phe for Me? The Effects of L-Phe on PKU Carriers and Non-carriers (Phe for Me)

Autosomal Recessive Disorder (Genetic Carriers of PKU)

Canada
West Kazakhstan Medical University

Recruiting

Clinical and Genetic Markers of Bone Metabolism in Children Under One Year

Child Development | Genetic Change | Bone Remodeling Disorder

Kazakhstan
National Human Genome Research Institute (NHGRI)

Completed

Predictors of Caregiver Adaptation to Pervasive Developmental Disorders

Autism | Asperger's Syndrome | Rett's Disorder

United States
Novo Nordisk A/S

Completed

Safety of Somatropin and Induction of Puberty With 17-beta-oestradiol in Girls With Turner Syndrome

Turner Syndrome | Genetic Disorder

Netherlands
Janssen-Ortho Inc., Canada

Completed

A Study of the Effectiveness and Safety of Risperidone Versus Placebo in the Treatment of Children With Autistic Disorder and Other Pervasive Developmental Disorders (PDD)

Autistic Disorder | Developmental Disabilities | Rett Syndrome | Asperger Syndrome | DCild Development Disorders, Pervasive
Children's Hospital Medical Center, Cincinnati

Not yet recruiting

Speech-in-noise Perception in Autism and Fragile X

Autism Spectrum Disorder | Fragile X Syndrome
University of Guelph
McMaster University

Recruiting

PKU Carriers Trial (Pilot Study): Impact on Cognition, Mental Health, Blood Pressure and Metabolism

Autosomal Recessive Disorder (Genetic Carriers of PKU)

Canada
Stanford University

Completed

Me-GC: A Randomized Controlled Trial of Meditation for Genetic Counselors

Genetic Counseling Profession

United States
Novo Nordisk A/S

Completed

Induction of Puberty With 17-beta Estradiol in Girls With Turner Syndrome

Turner Syndrome | Genetic Disorder

Spain
Novo Nordisk A/S

Completed

Investigating Safety and Pharmacokinetics of 2 Different Single Doses of NNC128-0000-2011 in Haemophilia A or B Patients

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Spain, Germany, Japan
Novo Nordisk A/S

Completed

Safety and Mode of Action of a Single Dose and Multiple Doses of Long Acting Activated Recombinant Human Factor VII in Patients With Haemophilia A and B

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Spain
Novo Nordisk A/S

Completed

Investigating Safety and Pharmacokinetics of NNC 0128-0000-2011 Compared to NNC 0128-0000-2021 in Healthy Male Subjects

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

United Kingdom
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Mutational Oncology in Clinical Practice (FPG-500)

Genetic Predisposition to Disease | Gene Rearrangement | Genome Instability

Italy
Novo Nordisk A/S

Completed

Growth Response in Girls With Turner Syndrome

Turner Syndrome | Genetic Disorder

France
Novo Nordisk A/S

Completed

Use of Somatropin in Turner Syndrome

Turner Syndrome | Genetic Disorder

United Kingdom
National Institute of Allergy and Infectious Diseases...

Recruiting

Novel Genetic Disorders of the Immune System

CTLA4 | PI3KCD | STAT3GOF | MAGT1

United States
Novo Nordisk A/S

Completed

Haemophilia Patients With Inhibitors Being Treated for Acute Joint Bleeds

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

United States, Spain, Taiwan, Turkey, Poland, Croatia, Italy, Malaysia, Brazil, United Kingdom, Hungary, Israel, Thailand, Japan, South Africa, Canada, France, Argentina
Novo Nordisk A/S

Completed

Pharmacokinetics of Single Bolus Dose of NovoSeven® in Paediatric and Adult Patients With Haemophilia A or B in a Non- Bleeding State

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Spain, Italy, Greece, Germany, United Kingdom
Novo Nordisk A/S

Completed

Post-marketing Safety Surveillance of NovoSeven® in Patients With Haemophilia and Inhibitors by Means of the UK Haemophilia Database

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

United Kingdom
Novo Nordisk A/S

Completed

Intra-subject Variability Following Administrations of Activated Recombinant Human Factor VII in Haemophilia Patients in a Non-bleeding State

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

France, Israel, Germany
Novo Nordisk A/S

Completed

Observational Study Describing the Usual Clinical Practice Use of NovoSeven® in the Home Treatment of Joint Bleeds in Patients With Haemophilia A or B and Inhibitors

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Morocco, United Arab Emirates, Algeria, Oman, Saudi Arabia
RespireRx
FRAXA Research Foundation

Completed

Effects of CX516 on Functioning in Fragile X Syndrome and Autism

Fragile X Syndrome | Autism

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute...

Terminated

Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders

Genetic Disorder | Severe Aplastic Anemia | Immunodeficiency Syndrome

United States
Novo Nordisk A/S

Completed

Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B (paradigm™6)

Congenital Bleeding Disorder | Haemophilia B

Spain, United Kingdom, Australia, Taiwan, France, Malaysia, United States, Austria, Canada, Israel, Japan, Thailand, Algeria, Argentina
Novo Nordisk A/S

Completed

Safety of NNC 0172-0000-2021 in Healthy Male Subjects and Subjects With Haemophilia A or B (Explorer 1)

Healthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Austria, Sweden, Spain, Switzerland, United Kingdom, Germany, Malaysia, Denmark, South Africa, Thailand, Italy
Novo Nordisk A/S

Completed

Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients (paradigm™ 2)

Congenital Bleeding Disorder | Haemophilia B

United States, Netherlands, Germany, Macedonia, The Former Yugoslav Republic of, United Kingdom, Italy, Malaysia, Hungary, Thailand, France, Russian Federation, Turkey, Japan, South Africa, Canada
Novo Nordisk A/S

Completed

Investigation of the Pharmacokinetics of NNC172-2021, at Two Different Dose Levels, in Healthy Japanese Subjects

Healthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

United Kingdom
Novo Nordisk A/S

Completed

A Single Dose Trial Investigating Safety and Local Tolerability of Ascending Doses of Long Acting Activated Recombinant Human Factor VII in Healthy Male Volunteers

Healthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

United Kingdom
Novo Nordisk A/S

Completed

Project to Update the Study of Congenital Haemophilia in Spain (PUCHS)

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Spain
Novo Nordisk A/S

Completed

Observational Study on Safety of Room Temperature Stable NovoSeven® in Patients With Haemophilia A or B

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Belgium, Germany, France, United Kingdom, Italy, Hungary, Austria, Sweden, Iran, Islamic Republic of, Argentina, Ireland, Poland, Portugal, Slovakia
Aarne Feldheiser
Technische Universität Berlin; Humboldt-Universität zu Berlin; German Institute... and other collaborators

Completed

Perioperative Early Tiredness (Acute Fatigue) in Patients With Epithelial Ovarian Cancer (PERISCOPE)

Nutrition | Mitochondrial Dysfunction | Electrical Cardiometry | Perioperative Early Tiredness (Acute Fatigue) | Biosignals

Germany
Freeline Therapeutics

Completed

AAV Gene Therapy Screening/Observational Protocol (ECLIPSE) (ECLIPSE)

Hemophilia B | Blood Coagulation Disorders, Inherited | Blood Coagulation Disorder | Haematologic Disease

United States, United Kingdom, Australia, Netherlands, Austria, France, Ireland, Italy, Turkey, Canada, Germany, South Africa
National Institute of Allergy and Infectious Diseases...

Recruiting

Recruitment and Apheresis Collection of Peripheral Blood Hematopoietic Stem Cells, Mononuclear Cells and Granulocytes

Granuloma | Granulomatous Disease, Chronic | Leukocyte Disease | Genetic Disease, X-Linked | Genetic Disease, Inborn

United States
Massachusetts General Hospital
The Rappaport Foundation; The Claflin Distinguished Scholar Awards

Not yet recruiting

Promoting Stress Management and Resilience Among Individuals With Von Hippel- Lindau Disease

Von Hippel-Lindau Disease | Genetic Disorder

United States
Eye & ENT Hospital of Fudan University
Shanghai Rehabilitation Institute for the Exceptional Children

Recruiting

A Comparative Analysis of Speech Perception Between Cochlear Implant Patients and DFNB9 Patients Receiving Gene Therapy

Hearing Loss | Genetic Disease | Speech Perception

China
Rhythm Pharmaceuticals, Inc.

Completed

Setmelanotide Phase 2 Treatment Trial in Participants With Rare Genetic Disorders of Obesity

Obesity | Genetic Obesity | Obesity Due to Melanocortin 4 Receptor Deficiency

United States, United Kingdom, Germany, Netherlands, France, Canada, Greece, Israel, Spain
Chang Gung Memorial Hospital

Recruiting

Investigation and Diagnosis of the Chromosome Variation in Donated/Abandoned Blastocyst

Recurrent Miscarriage | Chromosome Translocation | Genetic Disorders in Pregnancy

Taiwan
Borstkanker Onderzoek Groep
Novartis Pharma B.V.; BOOG Study Center

Recruiting

SEQUence of Endocrine Therapy in Advanced Luminal Breast Cancer (SEQUEL-Breast) (SEQUEL-Breast)

Neoplasm, Breast | Congenital, Familial and Genetic Disorders

Netherlands
Randy Kardon

Recruiting

New Non-invasive Modalities for Assessing Retinal Structure and Function

Leber Hereditary Optic Neuropathy | Ischemic Optic Neuropathy | Hemianopia | Branch Retinal Artery Occlusion | Acute Zonal Occult Outer Retinopathy

United States
Nova Mentis Life Science Corp
FourthWall Testing

Recruiting

Establishing a Diagnostic and Therapeutic Index in Autism Spectrum Disorder (ASD) and Fragile X Syndrome

Autism Spectrum Disorder | Fra(X) Syndrome

United States
Novo Nordisk A/S

Completed

Non-Interventional Study of NovoSeven® Used in Patients With Haemophilia A and B With Inhibitors When Undergoing Surgery

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

Japan
Novo Nordisk A/S

Completed

Safety and Tolerability of Intravenous Doses of Activated Recombinant Human Factor VII in Healthy Volunteers

Healthy | Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

France

Clinical Trials on Mitochondrial Genetic Disorders

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Togo

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

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