Clinical Trials Sponsored by Genethon

Total 66 results

Genethon

Recruiting

Gene Therapy for Severe Crigler Najjar Syndrome (CareCN)

Crigler-Najjar Syndrome

Italy, France, Netherlands
Genethon

Active, not recruiting

Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD) (CGD)

X-Linked Chronic Granulomatous Disease

United Kingdom
Genethon

Active, not recruiting

Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome (WASFUP)

Wiskott-Aldrich Syndrome

United Kingdom, France
Genethon

Recruiting

Natural History of Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

France, United Kingdom
Genethon

Completed

Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C

Limb Girdle Muscular Dystrophy Type 2C | Gamma-sarcoglycanopathy

France
Genethon

Active, not recruiting

Natural History Study of Patients With Limb-Girdle Muscular Dystrophy 2I

LGMD2I

Denmark, France, United Kingdom
Genethon

Active, not recruiting

Gene Therapy for X-linked Chronic Granulomatous Disease

X-Linked Chronic Granulomatous Disease

France
Genethon

Completed

Immunity Against AAV in Crigler Najjar Patient

Crigler Najjar Syndrome

France
Genethon
Institute of Myology

Completed

Research of Biomarkers in Duchenne Muscular Dystrophy Patients (IBISD)

Duchenne Muscular Dystrophy (DMD)

France
Genethon
Institute of Myology

Unknown

Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping (pre U7-53)

Duchenne Muscular Dystrophy

United Kingdom, France
Genethon
Hôpital Necker-Enfants Malades

Completed

Gene Therapy for WAS

Wiskott-Aldrich Syndrome

France
Fondazione Telethon

Active, not recruiting

Methodology Study of Retroviral Insertion Site Analysis in Strimvelis Gene Therapy

Severe Combined Immunodeficiency Due to ADA Deficiency

Italy
Fondazione Telethon

Completed

ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID (Gene-ADA)

Immunologic Deficiency Syndromes

Italy, Israel
Fondazione Telethon

Enrolling by invitation

Strimvelis Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

Immunologic Deficiency Syndromes

Italy
Fondazione Telethon

Unknown

Natural History Study in Subjects With Usher Syndrome

Usher Syndrome, Type 1B

Italy, Netherlands, Spain
Centre d'Etude des Cellules Souches
Genethon

Recruiting

Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome (AUDIOWOLF)

Wolfram Syndrome

France, Spain
Fondazione Telethon

Withdrawn

Retroviral Insertion Site Methodology Study

Immune System Diseases
Fondazione Telethon

Completed

Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs)

Severe Combined Immunodeficiency Due to ADA Deficiency

Italy
Fondazione Telethon

Active, not recruiting

Gene Therapy in Patients With Mucopolysaccharidosis Disease

Mucopolysaccharidosis Type VI

Italy, Turkey
Fondazione Telethon

Completed

Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency. (TIGEM2-PDH)

Pyruvate Dehydrogenase Complex Deficiency

Italy
Telethon Kids Institute

Withdrawn

Finding Better Treatment of Bronchiolitis: A Clinical Trial of Oral Nitazoxanide for the Treatment of Bronchiolitis in Infants Presenting to Hospital Emergency Departments

Bronchiolitis

Australia
Telethon Kids Institute

Recruiting

A Longitudinal Study Looking at the Prevalence, Risk Factors & Consequences of Persistent Post-surgical Pain in Children (POPSICLE)

Chronic Pain

Australia
Valerion Therapeutics, LLC
Institut de Myologie, France; Genethon

Completed

Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM) (MTM)

Myotubular Myopathy

France, Belgium, United States, Canada, Germany, Italy, Spain
IRCCS San Raffaele
Fondazione Telethon

Active, not recruiting

Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant (TigetT10_MPSIH)

Mucopolysaccharidosis IH

Italy
IRCCS San Raffaele
Fondazione Telethon

Completed

Gene Therapy for ADA-SCID

Severe Combined Immunodeficiency Syndrome
IRCCS San Raffaele
Fondazione Telethon

Recruiting

Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD (XCGD-MOBI)

Chronic Granulomatous Disease X-linked (X-CGD)

Italy
Fondazione Telethon
Ospedale San Raffaele

Completed

Gene Therapy for Wiskott-Aldrich Syndrome (TIGET-WAS)

Wiskott-Aldrich Syndrome (WAS)

Italy
Fondazione Telethon
Ospedale San Raffaele

Active, not recruiting

A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome

Wiskott-Aldrich Syndrome

Italy, United States
Genervon Biopharmaceuticals, LLC
Columbia University

Completed

Phase 2A Study of GM 608 in Mild to Moderate Parkinson Disease (GAP-PD)

Parkinson's Disease

United States
Istituto Auxologico Italiano
Fondazione Telethon

Completed

Treatment of Low Bone Density in Cystic Fibrosis. (OSCYF)

Osteoporosis | Cystic Fibrosis

Italy, Holy See (Vatican City State)
Ruijin Hospital
Hangzhou Genetron Medical Laboratory Co., Ltd.

Recruiting

Urothelial Tumor Risk Genes Detection With Genetron Uro V1 and LC-WGS

Urothelial Carcinoma

China
University of British Columbia
BCCH Telethon Grant

Unknown

The Use of Dermabond Versus Standard Staple for Wound Closure in Children With Neuromuscular Scoliosis Undergoing Spinal Deformity Correction Surgery

Wounds

Canada
IRCCS San Raffaele
Orchard Therapeutics; Fondazione Telethon

Unknown

Gene Therapy for Transfusion Dependent Beta-thalassemia (TIGET-BTHAL)

Beta-Thalassemia

Italy
The University of Queensland
Telethon Kids Institute

Completed

Prevention of Bronchiectasis in Infants With Cystic Fibrosis (COMBATCF)

Bronchiectasis | Cystic Fibrosis

Australia, New Zealand
Genton Graf Laurence

Completed

Body Composition: a Predictor of Mortality in Subjects Over 65 Years

External Causes of Morbidity and Mortality

Switzerland
Telethon Kids Institute
Menzies School of Health Research

Active, not recruiting

Optimising Rotavirus Vaccine in Aboriginal Children (ORVAC)

Viral Gastroenteritis Due to Rotavirus

Australia
Catholic University of the Sacred Heart
Fondazione Telethon

Unknown

RAMYD Study - Evaluation of Arrhythmic Risk in Myotonic Dystrophy

Myotonic Dystrophy | Sudden Cardiac Death

Italy
Genton Graf Laurence

Recruiting

Microbiota and Pancreatic Cancer Cachexia (EXTRA)

Pancreatic Cancer | Cachexia | Microbiota

Switzerland
Mario Negri Institute for Pharmacological Research
Fondazione Telethon

Completed

Familiar Fatal Insomnia: Preventive Treatment With Doxycycline in Subject With Disease Risk (FFI)

Familial Fatal Insomnia
Pontificia Universidad Catolica de Chile
Telethon Kids Institute

Completed

The Containing Coronavirus Disease 19 (COVID-19) Trial (ConCorD-19)

Covid19 | SARS-CoV Infection | Interferon

Chile
Genervon Biopharmaceuticals, LLC
Massachusetts General Hospital; Columbia University

Completed

GM604 Phase 2A Randomized Double-blind Placebo Controlled Pilot Trial in Amyotrophic Lateral Disease (ALS) (GALS)

Amyotrophic Lateral Sclerosis

United States
Genethon
Institute of Child Health; Great Ormond Street Hospital for Children NHS Foundation...

Completed

Gene Therapy for Wiskott-Aldrich Syndrome (WAS)

Wiskott-Aldrich Syndrome

United Kingdom
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
Telethon Kids Institute
Papua New Guinea Institute of Medical Research

Completed

PCV13 in Non-pregnant Papua New Guinean Women

Pneumococcal Infections

Papua New Guinea
Telethon Kids Institute
Rigshospitalet, Denmark; University of Haifa; Ariel University

Unknown

Telehealth Support to Increase Physical Activity in Rett Syndrome (ActivRett)

Rett Syndrome

Denmark, Israel
IRCCS San Raffaele
Telethon Institute for Gene Therapy (OSR-TIGET)

Active, not recruiting

Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)

Beta Thalassaemia

Italy
Genetron Health
Peking University Cancer Hospital & Institute; Shanghai 10th People's Hospital and other collaborators

Completed

Clinical Evaluation of Genetron D842V PCR Kit in GIST Patients

Gastrointestinal Stromal Tumors | GIST

China
Genetron Health
Huashan Hospital; West China Hospital; The First Hospital of Jilin University; Zhejiang...

Completed

Clinical Evaluation of Genetron IDH1 PCR Kit in Glioma Patients

Glioma, Malignant | Glioma, Mixed

China
Genetron Health
Huashan Hospital; West China Hospital; The First Hospital of Jilin University; Zhejiang...

Completed

Clinical Evaluation of Genetron TERT PCR Kit in Glioma Patients

Glioma, Malignant

China
Genetron Health
Cancer Institute and Hospital, Chinese Academy of Medical Sciences; Henan Cancer... and other collaborators

Completed

Clinical Evaluation of Genetron Lung Cancer Panel in Non-small Cell Lung Cancer Patients

Non-Small Cell Lung Cancer

China

Clinical Trials Sponsored by Genethon

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Cyprus

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

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