Search clinical trials for: Axenfeld-Rieger syndrom, genetic findings

Total 19018 results

Bioray Laboratories
Xiangya Hospital of Central South University; The 923rd Hospital of Joint Logistics...

Recruiting

Long-term Follow-up Study of BHC001 for TDT

Thalassemia, Beta

China
Noema Pharma AG

Not yet recruiting

Efficacy and Safety of Gemlapodect in Adults and Adolescents With Tourette Syndrome (Allevia2)

Tourette Syndrome
Societa Italiana di Dermatologia Medica, Chirurgica...

Recruiting

National Register of Moderate and Severe Adult Atopic Dermatitis (ATOPYREG)

Atopic Dermatitis

Italy
Fondazione IRCCS Ca' Granda, Ospedale Maggiore...

Recruiting

Development of a Neuronal Microscope

Hepatic Carcinoma

Italy
Maximilian Stahl, MD
Syndax Pharmaceuticals

Not yet recruiting

Revumenib in Combination With 7+3 + Midostaurin in AML

Acute Myeloid Leukemia | Leukemia | AML | AML, Adult | AML With Gene Mutations

United States
Daniel J. Weiner

Recruiting

Intrapulmonary Percussive Ventilation for Sputum Induction in Adults With Cystic Fibrosis

Cystic Fibrosis

United States
LEO Pharma

Recruiting

A Trial to Evaluate the Efficacy and Safety of Tralokinumab in Combination With Topical Corticosteroids in Children and Infants With Moderate-to-severe Atopic Dermatitis (TRAPEDS 2)

Atopic Dermatitis

United States
Niguarda Hospital
Istituto di Fisiologia Clinica CNR

Recruiting

Vascular Senescence and Atherosclerotic Plaque Vulnerability (VICTORIA)

Coronary Artery Disease

Italy
Suzhou Alphamab Co., Ltd.

Recruiting

Efficacy and Safety of KN057 Prophylaxis in Patients With Haemophilia A or B With Inhibitors

Hemophilia A With Inhibitor | Hemophilia B With Inhibitor

China
National Heart, Lung, and Blood Institute (NHLBI)
Children's National Research Institute

Recruiting

Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling

Sickle Cell Disease | Thalassemia | Hemoglobinopathy

United States
University of California, Los Angeles

Recruiting

Remote Assessment and Intervention for Behavior Problems in Kids With TSC (RAINBOW)

Tuberous Sclerosis

United States
University of Valencia
Asociación de Fibrosis Quística - Comunidad Valenciana

Not yet recruiting

e-Health Program to Prevent Exacerbations in the Cystic Fibrosis Population

Cystic Fibrosis

Spain
Tenaya Therapeutics
Medical University of South Carolina; Johns Hopkins University; University of... and other collaborators

Recruiting

Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) (RIDGE)

Arrhythmogenic Right Ventricular Cardiomyopathy

United States, Italy, United Kingdom, France, Sweden, Germany
University Hospital, Toulouse

Not yet recruiting

Safety Study for the Use of Rapamycin in Children With Familial Adenomatous Polyposis (RAPA-4-PAF)

Familial Adenomatous Polyposis

France
Lantu Biopharma

Not yet recruiting

An Open-label Study of a Gene Therapy Product in Beta-Thalassemia

Beta-Thalassemia
M.D. Anderson Cancer Center
Novartis Pharmaceuticals

Not yet recruiting

Phase II Study of the Combination of Blinatumomab and Asciminib in Patients With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia

Acute Lymphoblastic Leukemia | Philadelphia Chromosome-Positive

United States
argenx

Recruiting

A Study of Efgartigimod PH20 SC Given by Prefilled Syringe in Adults With Thyroid Eye Disease (UplighTED)

Thyroid Eye Disease

United States
BioMarin Pharmaceutical

Recruiting

A Study to Assess Growth in Children With Idiopathic Short Stature

Idiopathic Short Stature

United States
Hangzhou Yirui Pharmaceutical Technology Co., Ltd

Not yet recruiting

Topical YR001 Ointment in Adult With Mild to Moderate Atopic Dermatitis

Atopic Dermatitis

United States
Forge Biologics, Inc

Not yet recruiting

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients

Krabbe Disease

United States
argenx

Recruiting

A Study of Efgartigimod PH20 SC Given by Prefilled Syringe in Adults With Thyroid Eye Disease. (UplighTED)

Thyroid Eye Disease

United States
BioMarin Pharmaceutical

Recruiting

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

Phenylketonuria (PKU)

United States
Rady Pediatric Genomics & Systems Medicine Institute

Recruiting

An Adaptive Clinical Trial of BeginNGS Newborn Screening for Hundreds of Genetic Diseases by Genome Sequencing (BeginNGS)

Genetic Disease

United States
Halic University

Recruiting

Effect of Parental Attitude on Functional and Physical Level of Children With Cystic Fibrosis

Cystic Fibrosis | Cystic Fibrosis in Children

Turkey
Peking University Cancer Hospital & Institute

Not yet recruiting

Effectiveness of Risk-based Sequential Screening for Esophageal and Gastric Cancer (ERSS-EGC)

Gastric Cancer | Esophageal Cancer | Risk Stratification | Precision Screening | Sequential Screening

China
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Biomarkers in Retinitis Pigmentosa (RPMARKER)

Retinitis Pigmentosa

Italy
Shriners Hospitals for Children

Recruiting

Defining Treatment Outcomes and Genetic Architecture in Idiopathic Toe Walking* (ITW)

Idiopathic Toe Walking

United States
Massachusetts General Hospital
Johns Hopkins University

Not yet recruiting

Assessing the Efficacy of Repeat, Monthly Treatments of Cutaneous Neurofibromas (cNFs)

Neurofibromatosis 1 | Neurofibromas, Cutaneous

United States
Inozyme Pharma
GACI Global

Not yet recruiting

PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency

ATP-Binding Cassette Subfamily C Member 6 Deficiency | Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 Deficiency
Bioray Laboratories
First Affiliated Hospital of Guangxi Medical University

Not yet recruiting

Clinical Study of BRL-101 in Severe SCD

Sickle Cell Disease

China
Children's Hospital Medical Center, Cincinnati
Makerere University; Ministry of Health, Uganda; Mulago Hospital, Uganda

Recruiting

Uganda Sickle Surveillance Study (US-3)

Sickle Cell Disease

Uganda
University of Michigan

Recruiting

Michigan Food and Atopic Dermatitis (M-FAD) Program - Molecular Analytics Project

Healthy | Atopic Dermatitis | Food Allergy

United States
Marco Capogrosso
Roche-Genentech

Not yet recruiting

Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb

Spinal Muscular Atrophy | Spinal Muscular Atrophy Type 3 | SMA | Spinal Muscular Atrophy Type II | Spinal Muscular Atrophy 4

United States
Kind Pharmaceuticals LLC

Not yet recruiting

A Study of Safety and Efficiency of AND017 in Patients With Transfusion Dependent and Non-transfusion Dependent β-thalassemia

β -Thalassemia
Hôpital Necker-Enfants Malades

Not yet recruiting

Assessment of Patients Not Concluded After Neonatal Screening of Cystic Fibrosis.

CFTR-RD
Bangabandhu Sheikh Mujib Medical University, Dhaka...

Recruiting

Efficacy of Combination of Hdroxyurea and Thalidomide Over Either Hydroxyurea or Thalidomide Alone in the Treatment of Transfusion Dependent Thalassemia in Children: A Quasi-Randomised Clinical Trial

Transfusion-dependent Thalassemia

Bangladesh
Region Stockholm
Karolinska Institutet

Recruiting

CFTR Modulators in Pregnancy and Postpartum

Cystic Fibrosis

Sweden
PTC Therapeutics

Recruiting

A Study of Sepiapterin in Participants With Phenylketonuria (PKU) (EPIPHENY)

Phenylketonuria

United States
ARTHEx Biotech S.L.

Recruiting

Study of ATX-01 in Participants With DM1 (ArthemiR)

Myotonic Dystrophy 1

United States
Shanghai Institute Of Biological Products
The First Affiliated Hospital of Henan University of Science and Technology; First Affiliated Hospital Bengbu Medical College and other collaborators

Recruiting

A Phase Ib Clinical Trial of Peguricase for Injection With Methotrexate in Patients With Uncontrolled Gout.

Gout

China
metaX Institut fuer Diatetik GmbH
Great Ormond Street Hospital for Children NHS Foundation Trust; Birmingham...

Not yet recruiting

Acceptability/Tolerance of Protein Substitutes in Tablet Form for the Dietary Management of Rare Aminoacidopathies (ZeroMinisMR)

Alkaptonuria | Homocystine; Metabolic Disorder | Tyrosinemia, Type I | Tyrosinemia, Type II | Tyrosinemia, Type III | MSUD (Maple Syrup Urine Disease)
Chia Tai Tianqing Pharmaceutical Group Nanjing...

Not yet recruiting

A Clinical Study of Recombinant Human Coagulation Factor VIII for Injection in Patients With Severe Hemophilia A

Hemophilia A

China
Bioray Laboratories
Xiangya Hospital of Central South University; Sun Yat-Sen Memorial Hospital... and other collaborators

Not yet recruiting

Long-term Follow-up Study of BRL-101 for TDT

Thalassemia, Beta

China
GENGE

Recruiting

GENETIC VARIABILITY TO GLP1 TREATMENT (GLP1 treatment)

Obesity | Type 2 Diabetes

Israel
Spark Therapeutics, Inc.

Recruiting

Study of a Gene Therapy Treatment for Hemophilia A (KEYSTONE 1)

Hemophilia A

United States
John Elfar

Not yet recruiting

4AP to Delay Carpal Tunnel Release (CTR) (CTR)

Carpal Tunnel Syndrome | Compression Neuropathy
Lise Aksglæde

Not yet recruiting

Klinefelter Syndrome and Testosterone Treatment in Puberty (TiPY)

Klinefelter Syndrome
University of Pittsburgh
National Heart, Lung, and Blood Institute (NHLBI); Cystic Fibrosis Foundation

Recruiting

Health Outcomes of Parents With Cystic Fibrosis-Aim 2 (HOPeCF)

Cystic Fibrosis | Parenthood Status

United States
Istituto Auxologico Italiano

Recruiting

Short Stature and Psychological Well-being (PSICOSHORT)

Short Stature | Short; Stature, Psychosocial

Italy
RenJi Hospital

Recruiting

fMRI for BPS: A Descriptive Study of Findings and Symptoms

Bladder Pain Syndrome

China

Search clinical trials for: Axenfeld-Rieger syndrom, genetic findings

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Trinidad & Tobago

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations