Clinical Trials on X Linked Hypohidrotic Ectodermal Dysplasia in United Kingdom

Total 946 results

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
Edimer Pharmaceuticals

Completed

A Phase 1, Open-label, Multicenter, Safety and Pharmacokinetic Study of EDI200

X-linked Hypohidrotic Ectodermal Dysplasia

United States
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
Edimer Pharmaceuticals

Completed

Phenotypic Properties in Individuals Affected With XLHED (ECP-014)

X Linked Hypohidrotic Ectodermal Dysplasia

United States
Edimer Pharmaceuticals

Completed

Phenotypic and Genetic Properties in Males at Risk for X-linked Hypohidrotic Ectodermal Dysplasia: Evaluation of an Early Diagnosis Technology and Tests to Assess Nutritional Status (ECP-013)

X-linked Hypohidrotic Ectodermal Dysplasia | Hypohidrotic Ectodermal Dysplasia

United States
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
Edimer Pharmaceuticals

Completed

Sweat Duct Imaging in Mother/Newborn Dyads

X-Linked Hypohidrotic Ectodermal Dysplasia

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
Edimer Pharmaceuticals

Completed

X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) Carrier Outlook Toward Reproduction Survey (X-CORS)

Hypohidrotic Ectodermal Dysplasia | X-Linked Hypohidrotic Ectodermal Dysplasia

United States
Edimer Pharmaceuticals
National Foundation of Ectodermal Dysplasia

Completed

Medical Record Review of Hypohidrotic Ectodermal Dysplasia Clinical Phenotype (ECP-006)

Hypohidrotic Ectodermal Dysplasia | X-Linked Hypohidrotic Ectodermal Dysplasia

United States
Edimer Pharmaceuticals

Completed

Evaluation of Phenotypic and Genetic Properties in Male Subjects Affected By Hypohidrotic Ectodermal Dysplasia

Ectodermal Dysplasia

United States
Edimer Pharmaceuticals

Completed

Male Subjects Affected By Hypohidrotic Ectodermal Dysplasia: Intrafamilial Variation

Hypohidrotic Ectodermal Dysplasia

United States
Edimer Pharmaceuticals

Completed

Evaluation of Phenotypic and Genetic Properties in Male Subjects Affected by Hypohidrotic Ectodermal Dysplasia - A

Hypohidrotic Ectodermal Dysplasia

United States
Great Ormond Street Hospital for Children NHS Foundation...

Unknown

Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)

X-linked Severe Combined Immunodeficiency

United Kingdom
Newcastle-upon-Tyne Hospitals NHS Trust

Recruiting

Myotubular and Centronuclear Myopathy Patient Registry

X-linked Myotubular Myopathy | Centronuclear Myopathy | Myotubular Myopathy | Myotubular Myopathy 1 | Myotubular (Centronuclear) Myopathy | Centronuclear Myopathy, X-Linked

United Kingdom
James Dowling
Canadian Institutes of Health Research (CIHR); Cures Within Reach; The Joshua... and other collaborators

Recruiting

Tamoxifen Therapy for Myotubular Myopathy (TAM4MTM)

X Linked Myotubular Myopathy

United Kingdom, Canada, United States
Great Ormond Street Hospital for Children NHS Foundation...

Recruiting

Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency

Severe Combined Immunodeficiency, X-Linked

United Kingdom
Baylor College of Medicine

Active, not recruiting

Study of Selected X-linked Disorders: Goltz Syndrome

Focal Dermal Hypoplasia (FDH) | Goltz Syndrome

United States
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
VegaVect, Inc.
National Eye Institute (NEI)

Active, not recruiting

Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis

Retinoschisis | X-Linked

United States
Astellas Gene Therapies

Completed

A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)

Males With X-linked Myotubular Myopathy (XLMTM)

United States, United Kingdom
National Eye Institute (NEI)

Completed

Mothers Experiences With X-linked Retinoschisis Compared to Fathers Experiences

Retinoschisis | X-Linked

United States
David Williams

Active, not recruiting

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

Gene Therapy | Severe Combined Immunodeficiency, X Linked

United States, United Kingdom
University of Miami
Jackson Health System

No longer available

cliniMACs HUD for T Cell Depletion

x Linked Combined Immunodeficiency

United States
Genethon

Active, not recruiting

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

X-Linked Chronic Granulomatous Disease

United Kingdom
Retina Foundation of the Southwest
DSM Nutritional Products, Inc.; Foundation Fighting Blindness

Completed

DHA and X-Linked Retinitis Pigmentosa

Retinitis Pigmentosa | X-linked Genetic Diseases

United States
National Institute of Allergy and Infectious Diseases...

Terminated

Studies of Disorders in Antibody Production and Related Primary Immunodeficiency States

Ectodermal Dysplasia | Hyper-IgM Syndrome

United States
Great Ormond Street Hospital for Children NHS Foundation...

Withdrawn

Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease (X-CGD)

Granulomatous Disease, Chronic, X-linked, Variant

United Kingdom
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
Rare Thyroid Therapeutics International AB
Vitaccess Ltd

Completed

MCT8 Deficiency Caregiver Study

Allan-Herndon-Dudley Syndrome | Monocarboxylate Transporter 8 (MCT8) Deficiency

United Kingdom
University of Florida
Audentes Therapeutics

Completed

Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM)

X-linked Myotubular Myopathy

United States
National Institute of Allergy and Infectious Diseases...

Recruiting

Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)

X-linked Severe Combined Immunodeficiency (XSCID)

United States
St. Jude Children's Research Hospital
National Heart, Lung, and Blood Institute (NHLBI); Assisi Foundation; California...

Suspended

Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants (LVXSCID-ND)

Severe Combined Immunodeficiency Disease, X-linked

United States
University of Oxford
University College, London; Moorfields Eye Hospital NHS Foundation Trust

Completed

REP1 Gene Replacement Therapy for Choroideremia (REGENERATE)

Choroideremia

United Kingdom
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, United Kingdom, Japan, Italy, Australia, Bulgaria, Czechia, Netherlands, Poland
National Eye Institute (NEI)

Completed

People s Expectations When Enrolling in a Phase I/II RS1 Ocular Gene Transfer Clinical Trial

Retinoschisis | X-Linked

United States
National Institute of Allergy and Infectious Diseases...

Terminated

Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1

Growth Failure | X-linked Severe Combined Immunodeficiency (XSCID) | Growth Hormone Resistence

United States
Dermelix Biotherapeutics, LLC.

Withdrawn

A Cross-Sectional Natural History Study to Evaluate Sweat Volume and Other Phenotypic and Genetic Characteristics in Patients Affected by XLHED

XLHED

United States
Cure CMD
University of Michigan; Congenital Muscle Disease International Registr

Completed

Myotubular Myopathy Event Study (MTMES)

X-linked Myotubular Myopathy

United States
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
University of Manchester
Manchester University NHS Foundation Trust; Great Ormond Street Hospital for... and other collaborators

Recruiting

Autologous Ex-vivo Gene Modified HSCT in MPSII

Mucopolysaccharidosis II

United Kingdom
Anavex Life Sciences Corp.

Completed

ANAVEX2-73 Study in Patients With Rett Syndrome (AVATAR)

Rett Syndrome

Australia, United Kingdom
Anavex Life Sciences Corp.
Anavex Australia Pty Ltd.; Anavex Germany GmbH

Completed

ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome (EXCELLENCE)

Rett Syndrome

Canada, Australia, United Kingdom
University of California, Davis
University of Alberta; St. Justine's Hospital

Recruiting

A Trial of Metformin in Individuals With Fragile X Syndrome (Met)

Neurobehavioral Manifestations | Genetic Diseases, X-Linked | Intellectual Disability | Fragile X Syndrome | Sex Chromosome Disorders | Fragile X Mental Retardation Syndrome | Trinucleotide Repeat Expansion | Fra(X) Syndrome | FXS | Mental Retardation, X Linked

United States, Canada
Palvella Therapeutics, Inc.

Recruiting

A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita (VAPAUS)

Pachyonychia Congenita

United States, United Kingdom
National Institute of Allergy and Infectious Diseases...

Unknown

Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency (LVXSCID-OC)

X-Linked Combined Immunodeficiency Diseases

United States
National Institute of Allergy and Infectious Diseases...

Completed

Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID)

Severe Combined Immunodeficiency

United States
Taysha Gene Therapies, Inc.

Recruiting

Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study)

Rett Syndrome

United States, United Kingdom
National Institute of Allergy and Infectious Diseases...

Completed

Genetic Studies of X-linked Lymphoproliferative Disease

Genetic Diseases, X-Linked | Lymphoproliferative Disease | X-Linked Lymphoproliferative Disease

United States
Rare Thyroid Therapeutics International AB
Premier Research Group plc; Egetis Therapeutics

Recruiting

Withdrawal of Tiratricol Treatment in Males With Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency) (ReTRIACt)

Monocarboxylate Transporter 8 Deficiency | Allan-Herndon-Dudley Syndrome

United States, United Kingdom, Netherlands

Clinical Trials on X Linked Hypohidrotic Ectodermal Dysplasia in United Kingdom

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