Search clinical trials for: Kabuki syndrome CRIPSR/Cas9 KMT2D

Total 37540 results

University Hospital, Montpellier
Association Française contre les Myopathies Telethon

Completed

Exploiting Epigenome Editing in Kabuki Syndrome: a New Route Towards Gene Therapy for Rare Genetic Disorders (Epi-KAB)

Kabuki Syndrome 1

France
First Affiliated Hospital, Sun Yat-Sen University
Jingchu University of Technology

Unknown

A Safety and Efficacy Study of TALEN and CRISPR/Cas9 in the Treatment of HPV-related Cervical Intraepithelial NeoplasiaⅠ

Human Papillomavirus-Related Malignant Neoplasm

China
Chinese PLA General Hospital

Unknown

Study of CRISPR-Cas9 Mediated PD-1 and TCR Gene-knocked Out Mesothelin-directed CAR-T Cells in Patients With Mesothelin Positive Multiple Solid Tumors.

Solid Tumor, Adult

China
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Active, not recruiting

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia

Hematologic Diseases | Genetic Diseases, Inborn | Hemoglobinopathies | Thalassemia | Beta-Thalassemia

United States, United Kingdom, Canada, Germany, Italy
Qianfoshan Hospital

Recruiting

Drug Resistance Mechanism of Enterobacteriaceae and Its Strategies

Carbapenem-Resistant Enterobacteriaceae Infection

China
CRISPR Therapeutics AG

Active, not recruiting

A Safety and Efficacy Study Evaluating CTX120 in Subjects With Relapsed or Refractory Multiple Myeloma

Multiple Myeloma

United States, Spain, Australia, Canada
Yang Yang

Unknown

PD-1 Knockout EBV-CTLs for Advanced Stage Epstein-Barr Virus (EBV) Associated Malignancies

Stage IV Adult Hodgkin Lymphoma | Stage IV Nasopharyngeal Carcinoma | Stage IV Diffuse Large B-Cell Lymphoma | Stage IV Gastric Carcinoma | T-Cell Lymphoma Stage IV

China
German Cancer Research Center
University Hospital Heidelberg; University Hospital Dresden

Not yet recruiting

Genetic Ablation of CD33 in HSC to Broaden the Therapeutic Index of CD33-directed Immunotherapy in Patients With AML (GALAXY33)

Relapsed/Refractory Acute Myeloid Leukemia (AML)

Germany
Shanghai BDgene Co., Ltd.
Eye & ENT Hospital of Fudan University

Completed

Safety and Efficacy of CRISPR/Cas9 mRNA Instantaneous Gene Editing Therapy to Treat Refractory Viral Keratitis

Cornea | Herpes Simplex Virus Infection | Viral Keratitis | Blindness Eye

China
Editas Medicine, Inc.

Active, not recruiting

Single Ascending Dose Study in Participants With LCA10

Retinal Degeneration | Eye Diseases, Hereditary | Vision Disorders | Leber Congenital Amaurosis 10 | Inherited Retinal Dystrophies | Retinal Disease | Eye Disorders Congenital

United States
Second Affiliated Hospital of Guangzhou Medical...
Guangdong Zhaotai InVivo Biomedicine Co. Ltd.

Recruiting

Engineered TILs/CAR-TILs to Treat Advanced Solid Tumors

Breast Cancer | Lung Cancer | Brain Tumor | Colo-rectal Cancer | Liver Cancer | Solid Tumor, Adult | CTLA4 | PD1

China
CRISPR Therapeutics AG

Active, not recruiting

A Safety and Efficacy Study Evaluating CTX130 in Subjects With Relapsed or Refractory Renal Cell Carcinoma (COBALT-RCC)

Renal Cell Carcinoma

United States, Australia, Canada, Netherlands
EdiGene (GuangZhou) Inc.

Active, not recruiting

A Safety and Efficacy Study Evaluating ET-01 in Subjects With Transfusion Dependent β-Thalassaemia (ET-01)

Transfusion Dependent Beta-Thalassaemia

China
Mahmoud Ramadan mohamed Elkazzaz

Not yet recruiting

CRISPR/Cas9-modified Human T Cell ( PD-1and ACE2 Knockout Engineered T Cells ) for Inducing Long-term Immunity in COVID-19 Patients

COVID-19 Respiratory Infection
CRISPR Therapeutics AG
ViaCyte

Recruiting

An Open-Label, FIH Study Evaluating the Safety, Tolerability, and Efficacy of VCTX211 Combination Product in Subjects With T1D

Glucose Metabolism Disorders | Immune System Diseases | Autoimmune Diseases | Diabetes Mellitus | Endocrine System Diseases | Diabetes Mellitus, Type 1 | Metabolic Disease

Canada
CRISPR Therapeutics AG
ViaCyte

Completed

An Open-Label, FIH Study Evaluating the Safety and Tolerability of VCTX210A Combination Product in Subjects With T1D

Glucose Metabolism Disorders | Immune System Diseases | Autoimmune Diseases | Diabetes Mellitus | Endocrine System Diseases | Diabetes Mellitus, Type 1 | Metabolic Disease

Canada
Sanford Health
National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators

Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)

Mitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditions

United States, Australia
Hugo W. Moser Research Institute at Kennedy Krieger...

Active, not recruiting

Study of Modified Atkins Diet in Kabuki Syndrome

Kabuki Syndrome

United States
University Hospital, Montpellier

Completed

Assessment of Memory in Children With Kabuki Syndrom (MEM-K)

Kabuki Syndrome

France
Assistance Publique - Hôpitaux de Paris

Completed

French Kabuki Syndrome Network. Epidemiology, Management of Patients and Research by Array-CGH (Kabuki)

Kabuki Syndrome

France
Child Psychopharmacology Institute

Completed

Actigraphic Analysis of Treatment Response

Psychomotor Agitation | Insomnia | Sleep Disorders, Circadian Rhythm

United States
Danderyd Hospital
University of Trieste; Istituto Auxologico Italiano

Recruiting

Mechanisms for Atrial Fibrillation in Obstructive Sleep Apnea (AF-MiTriSto)

Hypertension | Atrial Fibrillation | Obstructive Sleep Apnea | Autonomic Dysfunction | Arterial Stiffness

Sweden, Italy
Hospital do Coracao
Brazilian Research in Intensive Care Network (BRICNet)

Not yet recruiting

Norepinephrine and Vasopressin for Rescue Versus Early Vasopressin for Vasopressor Dependent Sepsis (NoVa)

Septic Shock

Brazil
Merck Healthcare KGaA, Darmstadt, Germany, an affiliate...

Not yet recruiting

Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)

Generalized Myasthenia Gravis

United States, Germany
AstraZeneca

Recruiting

STEP: Phase IIIb Study of Benralizumab to Step-down Maintenance Therapy in Patients With Severe Eosinophilic Asthma (STEP)

Severe Eosinophilic Asthma

China
pablo Juan-Salvadores
Fundacin Biomedica Galicia Sur; Conselleria de Saúde Pública de Galicia; Conselleria...

Recruiting

Influence of Air Quality on the Development and Progression of Premature Coronary Artery Disease (AIRHEART)

Myocardial Infarction | Angina, Stable | Acute Coronary Syndrome | MINOCA | Coronary Artery Disease Premature

Spain
Minovia Therapeutics Ltd.

Recruiting

A Phase Ib Study to Evaluate the MNV-201 in Patients With Low Risk MDS

Myelodysplastic Syndromes

Israel
Hospital Universitario La Paz
Hospital Universitario del Henares, Madrid, Spain

Not yet recruiting

CPAP Effect on Lipid Profile and Hyperuricemia in Patients With Dyslipidemia and Moderate-severe Obstructive Sleep Apnea (CPAPLIP)

Dyslipidemias | Sleep Apnea | Hyperuricemia
Xuanwu Hospital, Beijing

Not yet recruiting

Forel's Field Electrical Stimulation for Lennox-Gastaut Syndrome (FESL)

Lennox Gastaut Syndrome

China
Oslo University Hospital
University of Oslo; Lovisenberg Diakonale Hospital

Recruiting

Laryngomalacia, Examinations and Quality of Life in Children Before and After Treatment With Follow-up After 1 Year

Quality of Life | Sleep Apnea, Obstructive | Sleep Apnea | Surgery | Pediatric Disorder | Laryngomalacia | Ear, Nose and Throat Disorder

Norway
Tr1X, Inc.

Recruiting

Prevention of GvHD in Participants With Hematological Malignancies Undergoing Hematopoietic Stem Cell Transplant (HSCT)

Myelodysplastic Syndromes | Hematologic Malignancy | Hematopoietic Stem Cell Transplant | Acute Myeloid Leukemia in Remission | GVHD, Chronic | GVHD,Acute | GvHD | Acute Lymphoblastic Leukemia, Adult B-Cell | Cancer Remission | Acute Lymphoblastic Leukemia, Adult T-Cell | Chronic Myelomonocytic Leukemia,...

United States
Emory University
University of California, San Diego; Ventrix, Inc.

Not yet recruiting

Study of Intramyocardial Injection of Ventrix Bio Extracellular Matrix (VentriGel) to Assess the Safety and Feasibility in Pediatric Patients With Hypoplastic Left Heart Syndrome (HLHS)

Hypoplastic Left Heart Syndrome

United States
Peking University People's Hospital

Recruiting

Combined PRF of the Pudendal Nerve With Ganglion Impar Block Effectively Alleviates Pudendal Neuralgia (PRF)

Pudendal Neuralgia | Pulsed Radiofrequency

China
Riphah International University

Not yet recruiting

Comparative Effects of Jandas's Approach With and Without Post Isometric Relaxation Technique

PELVIS Syndrome

Pakistan
Riphah International University

Recruiting

Effects of Sprint Interval Training Versus Arabic Exercises BMI

PCOS

Pakistan
Women's College Hospital

Not yet recruiting

Adapting and Piloting Acceptance and Commitment Therapy (ACT) for Severe Premenstrual Mood Symptoms (ACT-PM)

Premenstrual Dysphoric Disorder | Premenstrual Exacerbation of Mood Disorder
First Affiliated Hospital of Zhejiang University
The Second Hospital of Hebei Medical University; Shenzhen Second People's Hospital and other collaborators

Not yet recruiting

Dobutamine for Management of Surgical Patients With Septic Shock

General Anesthesia | Surgical Patients | Patients With Septic Shock
TC Biopharm

Not yet recruiting

ACHIEVE2 - Safety and Preliminary Efficacy of Intravenous TCB008 in Patients With Relapse or Refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)/AML (ACHIEVE2)

Acute Myeloid Leukemia | Myelodysplastic Syndromes
Aalborg University
The Research Council of Norway

Recruiting

How Does Implementing a GetUp Assist Device in Addition to a Daily Low-intensity Functional or Traditional Training Program Affect Norwegian Female Office Workers' Physical Health After 12 Weeks?

Neck, Shoulder and Arm Syndrome

Norway
Acibadem University
Boston Children's Hospital; Ege University

Recruiting

PTEN Hamartoma Tumor Syndrome Pediatric Patient Registry

PTEN Hamartoma Tumor Syndrome | Macrocephaly Autism Syndrome

Turkey
Riphah International University

Not yet recruiting

Effects of Cuevas Medek Exercises on Trunk Control and Balance in Children With Down Syndrome

Down Syndrome

Pakistan
University of Pennsylvania

Recruiting

Improving the Organizational Social Context to Address Structural Racism and Discrimination

HIV/AIDS

United States
National University Hospital, Singapore
Alexandra Hospital

Not yet recruiting

Ambulatory Blood Pressure Control and Cognition in Patients With Obstructive Sleep Apnea (AMPLE) (AMPLE)

Obstructive Sleep Apnea

Singapore
University Hospital, Rouen

Recruiting

EFFECT OF A SUBSTANCE P ANTAGONIST ON THE SECRETION OF ALDOSTERONE IN PATIENTS WITH OBSTRUCTIVE SLEEP APNEA SYNDROME AND ARTERIAL HYPERTENSION (APHOS 3)

Apnea, Obstructive

France
Riphah International University

Not yet recruiting

Effect of Group-Task-Oriented Training on GMFM and ADL in Children With DS

Downs Syndrome

Pakistan
Riphah International University

Recruiting

Combined Effects of Progressive Relaxation Exercises and Aquatic Therapy in Fibromyalgia.

Fibromyalgia

Pakistan
Istanbul University - Cerrahpasa (IUC)

Not yet recruiting

Validity and Reliability of the Inflammatory Arthritis Facilitators and Barriers to Physical Activity Questionnaire

Rheumatic Diseases | Pediatric ALL | Children, Only

Turkey
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Analysis of the Influence of Gastric By-Pass on the Pharmacokinetics of Common Drugs (ABSORGYP)

Bypass Bariatric Surgery

France
Groupe Hospitalier de la Region de Mulhouse et...
University Hospital, Strasbourg, France

Not yet recruiting

Comparison of the SUNRISE© Device With Ventilatory Polygraphy for the Diagnosis of Obstructive Sleep Apnea Hypopnea Syndrome: Collection of Preliminary Data (SUNPV)

Obstructive Sleep Apnea Hypopnea Syndrome
Riphah International University

Not yet recruiting

Effects of Bruegger's Versus Kendall Exercises in Cervical Postural Syndrome

Neck Pain

Pakistan

Search clinical trials for: Kabuki syndrome CRIPSR/Cas9 KMT2D

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