Search clinical trials for: inherited Genetic condition

Total 439727 results

Feinstein Institute for Medical Research
Diamond Blackfan Anemia Foundation, Inc.

Recruiting

Diamond Blackfan Anemia Registry (DBAR) (DBAR)

Anemia | Blood Disease

United States
National Human Genome Research Institute (NHGRI)

Recruiting

Analysis of Specimens From Individuals With Pulmonary Fibrosis

Pulmonary Fibrosis | Healthy Volunteers | Hermansky-Pudlak Syndrome (HPS)

United States
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

Genetic Basis of Immunodeficiency

Severe Combined Immunodeficiency

United States
National Cancer Institute (NCI)

Recruiting

Cancer in Inherited Bone Marrow Failure Syndromes

Fanconi Anemia | Dyskeratosis Congenita | Diamond Blackfan Anemia | Shwachman Diamond Syndrome | Inherited Bone Marrow Failure Syndrome, Aplastic Anemia

United States
National Institute of Neurological Disorders and...

Recruiting

Phenotype/Genotype Correlations in Movement Disorders

Movement Disorder

United States
National Institute of Neurological Disorders and...

Recruiting

Study of Inherited Neurological Disorders

Motor Neuron Disease | Muscular Dystrophy | Peripheral Nervous System Disease | Muscular Disease

United States, Mali
National Cancer Institute (NCI)

Recruiting

Von Hippel-Lindau (VHL): Clinical Manifestations, Diagnosis, Management and Molecular Bases of Inherited Renal and Other Urologic Malignant Disorders

Kidney Cancer | Renal Cell Carcinoma | Urologic Malignant Disorders | Familial Renal Cancer (FRC) | Clear Cell Renal Cancer

United States
National Human Genome Research Institute (NHGRI)

Recruiting

Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome

Hermansky-Pudlak Syndrome (HPS)

United States
Bayer

Active, not recruiting

Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding (HA-SAFE)

Hemophilia A

Spain, Germany, Austria, Greece, Italy, Slovenia
David Williams

Active, not recruiting

Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome

Wiskott-Aldrich Syndrome

United States
Pfizer

Active, not recruiting

A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A

Hemophilia A

United States
Baxalta now part of Shire
Baxalta Innovations GmbH, now part of Shire

Completed

ADVATE/ ADYNOVI Hemophilia A Outcome Database (AHEAD) (AHEAD)

Hemophilia A

Czechia, Switzerland, Denmark, Canada, Australia, Austria, France, United Kingdom, Italy, Sweden, Belgium, Brazil, China, Colombia, Greece, Hungary, Norway, Poland, Portugal, Russian Federation, Slovenia, Spain
Hoffmann-La Roche

Active, not recruiting

A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors (HAVEN 7)

Severe Hemophilia A

Canada, United States, United Kingdom, Belgium, Australia, Austria, Brazil, France, Germany, Israel, Italy, South Africa, Spain, Turkey
National Human Genome Research Institute (NHGRI)

Completed

Inherited Diseases, Caregiving, and Social Networks

Undiagnosed Disease | Healthy Volunteer (Adult With Typically Developing Child) | Inherited Neurodegenerative Disorders | Inherited Metabolic Disorders (Inborn Errors of Metabolism) | Alzheimer's Disease and Related Dementias

United States
Bayer

Active, not recruiting

Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A (HEM-POWR)

Hemophilia A

Canada, Japan, Spain, United States, Germany, Sweden, Belgium, Brazil, Greece, Italy, Netherlands, Taiwan, Denmark, Kuwait, Saudi Arabia, United Arab Emirates, Norway, Colombia, Switzerland, Slovenia
Bayer

Active, not recruiting

A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood (Alfa-PROTECT)

Hemophilia A | Children | Prophylaxis of Bleeding | Treatment of Bleeding

Canada, United States, Brazil, Italy, Turkey, Argentina, Norway
Bioverativ, a Sanofi company

Active, not recruiting

Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A (XTEND-ed)

Hemophilia A

United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, China, France, Germany, Greece, Hungary, Ireland, Italy, Japan, Korea, Republic of, Netherlands, Spain, Sweden, Switzerland, Taiwan, Turkey, United Kingdom
Washington University School of Medicine
Hoffmann-La Roche; Genentech, Inc.; National Institute on Aging (NIA); Alzheimer...

Suspended

A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset AD Caused by a Genetic Mutation (DIAN-TU)

Dementia | Alzheimers Disease | Alzheimers Disease, Familial

United States, Canada, Italy, France, Spain, Ireland, Argentina, Australia, Colombia, Germany, Mexico, Netherlands, Puerto Rico, United Kingdom
Octapharma

Active, not recruiting

Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease VWD) Patients <6 Years of Age

Von Willebrand Disease

United States, Moldova, Republic of, Czechia, Germany, North Macedonia, Russian Federation, Ukraine
Novo Nordisk A/S

Active, not recruiting

Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors (explorer8)

Haemophilia A Without Inhibitors | Haemophilia B Without Inhibitors

Spain, United States, Korea, Republic of, United Kingdom, Australia, Lithuania, France, Turkey, India, Estonia, Russian Federation, Ukraine, Mexico, Japan, Serbia, Canada, Croatia, Algeria, Bosnia and Herzegovina, Bulgaria, Denmark and more
Novo Nordisk A/S

Active, not recruiting

A Research Study Looking at How Safe it is to Switch From Emicizumab to Mim8 in People With Haemophilia A (FRONTIER 5) (FRONTIER 5)

Haemophilia A With Inhibitors | Haemophilia A

Spain, Korea, Republic of, Canada, United Kingdom, United States, Germany, South Africa, Italy, Austria, Belgium, France, Japan
Novo Nordisk A/S

Available

Compassionate Use of Concizumab if You Have Haemophilia

Congenital Haemophilia

United States, Bulgaria, Sweden
Bayer
Ultragenix pharmaceutical

Active, not recruiting

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle".

Hemophilia A

Netherlands, United States, Germany, United Kingdom, France, Bulgaria
Genzyme, a Sanofi Company

Completed

An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

Hemophilia A | Hemophilia B

Bulgaria, United States, United Kingdom, Russian Federation, Switzerland
Cyclo Therapeutics, Inc.

Active, not recruiting

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 (TransportNPC)

Niemann-Pick Disease, Type C1

United States, Taiwan, United Kingdom, Israel, Spain, Brazil, Italy, Australia, Turkey, Saudi Arabia, Argentina, Poland, Germany
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
Bayer

Active, not recruiting

Drug Use Investigation of Kovaltry in Hemophilia A Patients

Hemophilia A

Japan
St. Jude Children's Research Hospital
Children's Hospital of Philadelphia; National Heart, Lung, and Blood Institute... and other collaborators

Active, not recruiting

Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B

Hemophilia B

United States, United Kingdom
Universiteit Antwerpen
University Hospital, Antwerp; Cliniques universitaires Saint-Luc- Université...

Completed

Gaining Insight Into the Complexity of Pain in Patients With Haemophilia

Hemophilia A | Hemophilia B

Belgium
CSL Behring

Enrolling by invitation

Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)

Hemophilia B

United States
Bayer

Active, not recruiting

Study to Learn More About the Physical Activity Level of Patients Suffering From Hemophilia A Treated With Damoctocog Alfa Pegol (LIFE ACTIVE Study) (LIFE-ACTIVE)

Hemophilia A

Canada, Spain, United States, Belgium, Italy, Taiwan, Slovenia
Takeda

Completed

A Study on the Outcomes of Recombinant Von Willebrand Factor on Demand Treatment and Prevention and Treatment of Bleeding During and After Surgery in Adults With Inherited Von Willebrand Disease in the United Kingdom (UK)

Von Willebrand Disease (VWD)

United Kingdom
Amgen

Completed

Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia) (HAUSER-OLE)

Familial Hypercholesterolemia

Canada, Italy, Norway, Belgium, United States, Turkey, Australia, Austria, South Africa, Poland, Switzerland, Spain, Portugal, Slovenia, Brazil, Russian Federation, Hungary, United Kingdom, Colombia, Czechia, Netherlands, Greece, Ma...
Investigación en Hemofilia y Fisioterapia

Completed

Changes in Intra-articular Parameters by Doppler Ultrasound in Patients With Hemophilic Ankle Arthropathy

Hemophilia

Spain
Swedish Orphan Biovitrum
Syneos Health

Active, not recruiting

A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa (FREEDOM)

Hemophilia A, Severe

Germany, Spain, France, Czechia, Netherlands, Ireland, Austria, Croatia, Greece, Italy, Norway, Slovenia, Sweden, United Kingdom
Novo Nordisk A/S

Active, not recruiting

A Research Study Investigating Nonacog Beta Pegol (N9-GP) for Treatment and Prevention of Bleedings in Chinese People With Haemophilia B (Paradigm9)

Haemophilia B

China
Washington University School of Medicine
Hoffmann-La Roche; National Institute on Aging (NIA); Alzheimer's Association

Terminated

Dominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia: A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset Alzheimer's Disease Caused by a Genetic Mutation (DIAN-TU)

Dementia | Alzheimers Disease | Alzheimers Disease, Familial

United States, France, Spain, Ireland, Canada, Australia, Puerto Rico, United Kingdom
CSL Behring

Active, not recruiting

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

Hemophilia B

United States, Belgium, Netherlands, Denmark, Germany, Ireland, Sweden, United Kingdom
CSL Behring

Active, not recruiting

Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B

Hemophilia B

Netherlands, Germany
Medexus Pharma, Inc.

Completed

Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B

Hemophilia B

Turkey, Brazil, Georgia, Moldova, Republic of, South Africa, Ukraine
Hoffmann-La Roche

Active, not recruiting

A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis (Beyond ABR)

Severe Hemophilia A | Moderate Hemophilia A

Brazil, Germany, Italy, Spain, United States, Turkey, United Kingdom, Tunisia, Canada, Hungary, Morocco, Serbia
National Institute of Allergy and Infectious Diseases...

Active, not recruiting

Screening Protocol for Genetic Diseases of Allergic Inflammation

Immune Deficiency | Eosinophilic Disease | HaTS | Urticaria Anaphylaxis | Elevated IgE Level

United States
BioMarin Pharmaceutical

Active, not recruiting

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A (GENEr8-JPN)

Hemophilia A

Japan
Takeda

Completed

A Proof-of-concept Study to Reduce Treatment Burden in Haemophilia Participants Receiving Factor VIII and Factor IX Infusions

Hemophilia

France
Pfizer

Active, not recruiting

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B (BENEGENE-2)

Hemophilia B

Germany, United Kingdom, Canada, Australia, Taiwan, Japan, France, Turkey, Korea, Republic of, United States, Brazil, Greece, Saudi Arabia, Sweden
Sangamo Therapeutics

Active, not recruiting

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX (LTFU)

Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II

United States
University Hospital, Montpellier
Hospices Civils de Lyon; University Hospital, Bordeaux; University Hospital,... and other collaborators

Completed

Healthy-related Quality of Life and Physical Activity of Children With Cardiac Malformations (QUALIMYORYTHM)

Dilated Cardiomyopathy | Hypertrophic Cardiomyopathy | Brugada Syndrome | Long QT Syndrome | Arrhythmogenic Right Ventricular Dysplasia | Catecholaminergic Polymorphic Ventricular Tachycardia | Restrictive Cardiomyopathy

France
Pfizer

Active, not recruiting

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

Hemophilia A | Hemophilia B

United States, Canada, Spain, Australia, Korea, Republic of, Taiwan, Brazil, Italy, Japan, Turkey, Germany, United Kingdom, Saudi Arabia, France, Sweden, Belgium, Greece, Israel
Novo Nordisk A/S

Completed

Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A (pathfinder™6)

Congenital Bleeding Disorder | Haemophilia A

United States, Spain, Australia, France, Thailand, Italy, Malaysia, Austria, Greece, Japan, Taiwan, Canada, Israel, Algeria, Argentina, Bulgaria, Germany, Romania
BioMarin Pharmaceutical

Enrolling by invitation

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) (GENEr8-LTE)

Hemophilia A

United Kingdom, United States

Search clinical trials for: inherited Genetic condition

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