Clinical Trials on x Linked Combined Immunodeficiency in France

Total 260 results

Assistance Publique - Hôpitaux de Paris

Completed

Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID2)

X-linked Severe Combined Immunodeficiency

France
Assistance Publique - Hôpitaux de Paris

Recruiting

Safety and Efficacy Study of Transplantation of Autologous CD34+ Cells Transduced With the G2ARTE Lentiviral Vector Expressing the DCLRE1C cDNA in Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency Patients (ARTEGENE) (ARTEGENE)

Artemis (DCLRE1C ) Deficient Severe Combined Immunodeficiency

France
Assistance Publique - Hôpitaux de Paris

Terminated

Hematopoietic Stem Cells Transplantation in Children With Combined Immunodeficiency (CID) (CD45RA)

Combined Immunodeficiencies

France
Assistance Publique - Hôpitaux de Paris

Recruiting

Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients (HTLP Necker)

Pediatric Patients | Any Type of Severe Combined Immunodeficiency (SCID) | Partial HLA Incompatible Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

France
Hospices Civils de Lyon

Unknown

Clinical Phenotyping and Characterization of Neural Networks and Cognitive Processes Involved in Mental Retardation X-linked (XLMR)

X-linked Mental Retardation

France
Nantes University Hospital

Completed

Generalized Neonatal Screening of Severe Combined Immunodeficiencies (DEPISTREC)

Severe Combined Immunodeficiency, Atypical

France
Astellas Gene Therapies

Completed

A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects (INCEPTUS)

X-linked Myotubular Myopathy

United States, France, United Kingdom, Canada, Germany
Association Pour l'Aide a la Recherche en Immunologie...

Completed

Study Through Imaging of Visceral Lymphoid Organs in Patients With SCID Who Have Recieved Bone Marrow Allograft (LymphImaging)

Severe Combined Immunodeficiency

France
CSL Behring

Completed

Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy

Common Variable Immunodeficiency | X-linked Agammaglobulinemia | Autosomal Recessive Agammaglobulinemia

Germany, United Kingdom, France, Italy, Poland, Romania, Spain, Sweden, Switzerland
Assistance Publique Hopitaux De Marseille

Completed

Pilot Study of the Effects of the Desipramine on the Neurovegetative Parameters of the Child With Rett Syndrome

Rett Syndrome

France
Assistance Publique Hopitaux De Marseille

Unknown

Patients With RETT Syndrome (RETT)

Rett Syndrome

France
Edimer Pharmaceuticals

Active, not recruiting

Extension Study of XLHED-Affected Male Subjects Treated With EDI200 in Protocol ECP-002 (ECP-002e)

X-linked Hypohidrotic Ectodermal Dysplasia

United States, United Kingdom, France, Germany, Italy
University Hospital, Tours

Completed

Glutaminergic Transmission in Autism : Molecular Imaging Exploration (TANGAU)

Healthy Volunteers | Autism | Fragile-X Syndrome (FXS)

France
EspeRare Foundation
Iqvia Pty Ltd; Pierre Fabre Medicament

Recruiting

Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia (EDELIFE)

X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)

United States, Spain, France, Germany, Italy, United Kingdom
Novartis

Completed

Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients

Fragile X Syndrome

France, Italy, Switzerland
Genethon

Active, not recruiting

Gene Therapy for X-linked Chronic Granulomatous Disease

X-Linked Chronic Granulomatous Disease

France
Viking Therapeutics, Inc.

Recruiting

A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN

Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)

United States, France, Germany, Italy, United Kingdom
Pitié-Salpêtrière Hospital

Recruiting

Observatoire Des Patients Atteints de Laminopathies et Emerinopathies (Observatory for PAtients With Laminopathies and Emerinopathies) (OPALE)

Laminopathies | Emerinopathies

France
Central Hospital, Nancy, France

Completed

New Genes Involved in Molecular Etiology of Rett Syndrome Through DNA Microarray Comparative Genomic Hybridization (RETT)

Rett Syndrome

France
Mitsubishi Tanabe Pharma America Inc.

Recruiting

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP (INSPIRE)

Erythropoietic Protoporphyria (EPP) | X-Linked Protoporphyria (XLP)

United States, Spain, France, Japan, United Kingdom, Italy, Australia, Poland, Bulgaria, Czechia, Netherlands
Minoryx Therapeutics, S.L.

Recruiting

Disease Progression in Women With X-linked Adrenoleukodystrophy

X-linked Adrenoleukodystrophy

France
Genethon
Hôpital Necker-Enfants Malades

Completed

Gene Therapy for WAS

Wiskott-Aldrich Syndrome

France
NightstaRx Ltd, a Biogen Company

Enrolling by invitation

Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa (SOLSTICE)

Choroideremia | X-Linked Retinitis Pigmentosa

United States, United Kingdom, Netherlands, Germany, France, Brazil, Canada, Denmark, Finland
Assistance Publique - Hôpitaux de Paris

Completed

Osteoporosis in RETT Syndrome (OSRETT)

RETT Syndrome With Proven MECP2 Mutation

France
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
Tetra Discovery Partners

Recruiting

A Study of BPN14770 in Male Adults (Aged 18 to 45) With Fragile X Syndrome

Fragile X Syndrome

United States
Assistance Publique - Hôpitaux de Paris
Institut National de la Santé Et de la Recherche Médicale, France; Association...

Recruiting

Cell Signaling, Reinnervation and Metabolism in Kennedy Disease and Amyotrophic Lateral Sclerosis (ALS) (CERMALS)

Amyotrophic Lateral Sclerosis | Kennedy Disease

France
Novartis Pharmaceuticals

Terminated

Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome

Fragile X Syndrome

Germany, United States, Switzerland, Australia, Canada, Denmark, France, Italy, United Kingdom, Spain
Novartis Pharmaceuticals

Completed

Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

Fragile X Syndrome

United States, Switzerland, Germany, Australia, Canada, Denmark, France, Italy, Spain, United Kingdom
University Hospital, Montpellier
Association Xtraordinaire sub-group DDX3X; Genida

Unknown

Better Delineation of DDX3X Related Phenotype and Epigenetic Signature.

Intellectual Developmental Disorder | X-LINKED

France
University Hospital, Brest

Unknown

Dysautonomic Phenotype in Male Patients With MECP2 Mutation (MECP2BOYS)

Dysautonomia | Masculinity | MECP2-Related Severe Neonatal Encephalopathy

France
Tetra Discovery Partners

Recruiting

A Randomized Study of BPN14770 in Male Adolescents (Aged 9 to < 18 Years) With Fragile X Syndrome

Fragile X Syndrome

United States
Tetra Discovery Partners

Enrolling by invitation

An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

Fragile X Syndrome

United States
Novartis Pharmaceuticals

Terminated

Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)

Fragile X Syndrome

United States, Belgium, Australia, Israel, Switzerland, Germany, Denmark, Italy, Netherlands, Spain, Sweden, United Kingdom, France
Zynerba Pharmaceuticals, Inc.

Recruiting

Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome (RECONNECT)

Fragile X Syndrome

United States, Australia, United Kingdom, Ireland
Seaside Therapeutics, Inc.

Completed

Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome (Harbor-C)

Fragile X Syndrome

United States
Astellas Gene Therapies

Active, not recruiting

Gene Transfer Clinical Study in X-Linked Myotubular Myopathy (ASPIRO)

X-Linked Myotubular Myopathy

United States, Canada, France, Germany
University Hospital, Montpellier
Agence de La Biomédecine

Active, not recruiting

NIPD on cffDNA for Triplet Repeat Diseases

Fragile X Syndrome | Huntington Disease | Myotonic Dystrophy 1

France
Hoffmann-La Roche

Completed

A Study of RO4917523 in Patients With Fragile X Syndrome

Fragile X Syndrome

France, Spain, United States, Peru, Canada, United Kingdom, Chile, Argentina, Mexico, Sweden
NeuroVia, Inc.

Withdrawn

Safety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With Adrenoleukodystrophy

X-Linked Adrenoleukodystrophy

Australia, Argentina, United Kingdom, Colombia, Chile, France, Russian Federation, Ukraine
bluebird bio

Active, not recruiting

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

Cerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)

United States, Argentina, Australia, Brazil, France, Germany, Netherlands, United Kingdom, Italy
Edimer Pharmaceuticals

Completed

Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) (ECP-002)

X-Linked Hypohidrotic Ectodermal Dysplasia

United States, United Kingdom, France, Germany, Italy
Novartis Pharmaceuticals

Completed

Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome

Fragile X Syndrome

United States, Belgium, Australia, Israel, Switzerland, Germany, France, Canada, Denmark, Indonesia, Italy, Netherlands, Spain, Sweden, Turkey, United Kingdom
Seaside Therapeutics, Inc.

Completed

Efficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X Syndrome (Harbor-A)

Fragile X Syndrome

United States
Seaside Therapeutics, Inc.

Terminated

An Open Label Extension Study in Subjects With Fragile X Syndrome (209FX303)

Fragile X Syndrome

United States
Edimer Pharmaceuticals

Completed

Natural History and Outcomes in X-Linked Hypohidrotic Ectodermal Dysplasia (ECP-015)

X-Linked Hypohidrotic Ectodermal Dysplasia

United States, United Kingdom, France, Germany, Italy
Biogen

Completed

A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111 (GEMINI)

Choroideremia

United States, Germany, France
Centre Hospitalier Universitaire de Saint Etienne

Completed

Study of Sensorimotor Compatibility Effects in Bipolar Affective Disorder.

Bipolar Affective Disorder

France
Assistance Publique - Hôpitaux de Paris
Centre National de la Recherche Scientifique, France

Completed

Acoustic and Perceptual Markers of Dysarthria in Amyotrophic Lateral Sclerosis (ALS) (SPEECH-ALS)

Amyotrophic Lateral Sclerosis | Primary Lateral Sclerosis | Kennedy's Disease

France
Centre Leon Berard

Recruiting

Feasibility Study to Assess an Adapted Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation (EVAADE)

Myelodysplastic Syndromes | Severe Combined Immunodeficiency | Acute Leukemia | Haematological Malignancy | Hemoglobinopathy in Children | Bone Marrow Aplasia

France

Clinical Trials on x Linked Combined Immunodeficiency in France

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