Search clinical trials for: Inborn Genetic Disease

Total 384042 results

GeneCradle Inc

Recruiting

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

Pompe Disease (Late-onset)

China
University College, London
London School of Hygiene and Tropical Medicine; King's College London; University... and other collaborators

Recruiting

Deep Brain Stimulation in Tourette Syndrome (Op-TICS)

Tourette Syndrome | Deep Brain Stimulation

United Kingdom
Laval University

Recruiting

Unravelling the Impact of Diet on Cardiovascular Health in Treated Heterozygous Familial Hypercholesterolemia.

Familial Hypercholesterolemia | Cholesterol, Elevated | Nutrition, Healthy

Canada
Ocugen

Recruiting

A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa (liMeliGhT)

Retinitis Pigmentosa

United States
Clinuvel Europe Limited

Recruiting

Pharmacokinetics of Afamelanotide in EPP Patients

Erythropoietic Protoporphyria

Belgium, Netherlands
Belite Bio, Inc

Not yet recruiting

A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease (DRAGON II)

Stargardt Disease 1 | STGD1

United States
University of Mississippi Medical Center
University of Alabama at Birmingham

Recruiting

Low Systemic/High Local Exercise Load in Peds SCD

Sickle Cell Anemia in Children

United States
Beam Therapeutics Inc.

Not yet recruiting

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)

Alpha 1-Antitrypsin Deficiency

United Kingdom
AbbVie

Recruiting

A Study of Upadacitinib in Adult Participants With Moderate-to-Severe Atopic Dermatitis and Inadequate Response to Dupilumab (Switch-Up)

Atopic Dermatitis

United States
Soligenix

Not yet recruiting

Dusquetide for the Treatment of Behcet's Disease

Behçet Disease

Turkey
RTI International

Recruiting

Recruitment and Engagement in Care to Impact Practice Enhancement (RECIPE) for Sickle Cell Disease (RECIPE)

Sickle Cell Disease

United States
University of Utah

Recruiting

Clinical Characteristics and Temporal Properties of Individual Tics in Persistent Tic Disorder (PTD)

Tourette Syndrome | Tic Disorders | Tics | Tourette Syndrome in Children | Tourette Syndrome in Adolescence | Vocal Tic | Motor Tic | Tics/Tremor

United States
Yonsei University

Recruiting

Intestinal Ultrasound, Magnetic Resonance Enterography, and Conventional Assessment Tools Before and After Treatment of Intestinal Behcet's Disease

Intestinal Behcet Disease

Korea, Republic of
Akeso

Recruiting

A Phase III Study to Evaluate the Efficacy and Safety of AK120 in Patients With Moderate to Severe Atopic Dermatitis

Atopic Dermatitis

China
Viridian Therapeutics, Inc.

Recruiting

A Randomized, Active Controlled, Safety and Tolerability Study of VRDN-001 in Participants With Thyroid Eye Disease (TED)

Thyroid Eye Disease

United States
BioMarin Pharmaceutical

Not yet recruiting

A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

Idiopathic Short Stature
AstraZeneca
Parexel

Recruiting

A Study to Investigate Safety, Tolerability, and Pharmacokinetics of Oral AZD0233 Compared With Placebo in Healthy Adult Participants.

Dilated Cardiomyopathy

United States
Shanghai Ninth People's Hospital Affiliated to...

Recruiting

A Retrospective Study on Epidemiological Characteristics of Chinese NF1 Patients in Real World (PROMISE)

Neurofibromatosis 1 | Plexiform Neurofibroma

China
The First Affiliated Hospital of Zhejiang Chinese...

Recruiting

Peripheral Mechanisms of Humoral Immune Effector-mediated Tourette's Syndrome and the Role of Tuina Intervention

Inflammatory Response | Tourette Syndrome in Children | Food Intolerance

China
Regeneron Pharmaceuticals
Intellia Therapeutics

Not yet recruiting

A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Pediatric, Adolescent and Adult Patients With Hemophilia B (BEYOND-9)

Hemophilia B
IRCCS Eugenio Medea

Recruiting

Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe] (MDCJ-NeuBeRe)

Myotonic Dystrophy 1

Italy
IRCCS Eugenio Medea

Recruiting

Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project [RIMUDI] (RIMUDI)

Muscular Dystrophies | Becker Muscular Dystrophy | Limb Girdle Muscular Dystrophy | Facio-Scapulo-Humeral Dystrophy

Italy
Assiut University

Not yet recruiting

Thyroid Function , HbA1c in Relation to Ferritin Level in Adullt Patient e B Thalassemia

B Thalassemia
Ray Therapeutics, Inc.
The Vision Research and Assessment Institute (VRAI)

Recruiting

Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Retinitis Pigmentosa Patients

Retinitis Pigmentosa

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

Not yet recruiting

Peer Support for Adolescents and Emerging Adults With Sickle Cell Pain (PRESENCE)

Pain | Sickle Cell Disease
Apollo Therapeutics Ltd

Not yet recruiting

A Study to Investigate APL-4098 Alone and/or in Combination With Azacitidine in R/R AML and High-Risk MDS

Myelodysplastic Syndrome With Excess Blasts | Acute Myeloid Leukemia, in Relapse | Acute Myeloid Leukemia Refractory | Myelodysplastic Syndrome Acute Myeloid Leukemia
Enzyre B.V.
Indiana Hemophilia & Thrombosis Center

Not yet recruiting

Case and Use Scenario Study to Gain Knowledge on the User Needs for a Medical Device for Hemophilia A Monitoring

Hemophilia A

United States
Chugai Pharmaceutical

Not yet recruiting

Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)

Antiphospholipid Syndrome (APS) | Immune Thrombocytopenia (ITP) | Dermatomyositis (DM) | Bullous Pemphigoid (BP) | Behçet's Syndrome (BS) | Immune-mediated Necrotizing Myopathy (IMNM)

United States, Japan
Hospices Civils de Lyon

Recruiting

Circadian Rhythm Disorders in Children With Cystic Fibrosis Under CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) Modulators (CHRONO-MUCO)

Cystic Fibrosis

France
Centre Hospitalier Universitaire de la Réunion

Recruiting

Pathogenicity of Species of the Achromobacter Genus in Patients From Reunion Island With Cystic Fibrosis (ACHROMO-MUCO)

Cystic Fibrosis

Réunion
Massachusetts General Hospital
n-Lorem Foundation

Not yet recruiting

Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

Genetic Disease
Medical University of Warsaw

Recruiting

Tocilizumab in Corticosteroid-Resistant Graves' Orbitopathy (Thyroid Eye Disease)

Thyroid Eye Disease | Ophthalmopathy | Orbitopathy, Graves

Poland
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

phenotypeS in Non Ambulant Duchenne Muscular Dystrophy (GUP21003)

Duchenne Muscular Dystrophy | Natural History | Motor Function; Retardation

Italy
Harmony Biosciences, LLC

Recruiting

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Prader-Willi Syndrome

United States
Instituto de Investigación Hospital Universitario...

Recruiting

Optimization of Atopic Dermatitis Treatment That Requires Second-line Systemic Therapy Through Predictive Models

Atopic Dermatitis

Spain
St. Jude Children's Research Hospital
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

School Readiness Intervention for Preschool Children With Sickle Cell Disease

Sickle Cell Disease

United States
University of Kansas Medical Center

Recruiting

Targeting Inflammation With Losartan to Improve Response to Modulator Therapy in Cystic Fibrosis.

Cystic Fibrosis

United States
Children's Hospital of Philadelphia

Not yet recruiting

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Beta-Thalassemia

United States
AstraZeneca

Recruiting

hATTR Polyneuropathy in Russia (PRIMER)

Hereditary Transthyretin Amyloidosis

Russian Federation
Editas Medicine, Inc.

Not yet recruiting

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

Sickle Cell Disease | Hemoglobinopathies | Transfusion-dependent Beta-Thalassemia
Octapharma

Recruiting

Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks

Acute Hereditary Angio Edema

Romania
University Hospital, Toulouse

Not yet recruiting

Efficacy of Injectable Gentamicin in Hereditary Ichthyosis (GENTIC)

Ichthyosis

France
West China Hospital

Not yet recruiting

AI App for Management of Atopic Dermatitis

Artificial Intelligence | Atopic Dermatitis
Chang Gung University

Recruiting

Electrical Stimulation Cycling Training Effects on SCA

Spinocerebellar Ataxia (SCA)

Taiwan
Technoderma Medicines Inc.
Therapeutics, Inc.

Recruiting

Study of TDM-180935 in Atopic Dermatitis Patients

Atopic Dermatitis

United States
University of Malaga

Not yet recruiting

Effectiveness of 5-week Digital Respiratory Practice in a Group of Children With Duchenne Muscular Dystrophy and Becker Muscular Dystrophy. (DMDrespy2024)

Muscular Dystrophy | Duchenne Muscular Dystrophy | Becker Muscular Dystrophy

Spain
Fondazione SISA (Societa Italiana per lo Studio...

Recruiting

Lipid Transport Disorder Italian Genetic Record (LIPIGEN) (LIPIGEN)

Familial Hypercholesterolemia | Genetic Disorder

Italy
Seoul National University Hospital

Not yet recruiting

The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements of the Upper Limb in Patients With Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne | Upper Extremity | Orthotic Devices

Korea, Republic of
AstraZeneca

Not yet recruiting

Real-World Treatment Study of Koselugo (Selumetinib)

Neurofibromatosis 1 | Neurofibroma, Plexiform
Cliniques universitaires Saint-Luc- Université...

Recruiting

Blood Spot and Urine Metabolomic Screening Applied to Rare Diseases (BUSARD)

Rare Diseases | Inborn Errors of Metabolism

Belgium

Search clinical trials for: Inborn Genetic Disease

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