Clinical Trials on Usher Syndrome Type 2A in United Kingdom

Total 2211 results

Jaeb Center for Health Research
Foundation Fighting Blindness

Active, not recruiting

Rate of Progression in USH2A-related Retinal Degeneration (RUSH2A)

Usher Syndrome, Type 2A | Retinitis Pigmentosa 39

United States, Canada, France, Germany, Netherlands, United Kingdom
Neurotech Pharmaceuticals
University of California, San Francisco

Completed

Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa

Retinitis Pigmentosa | Usher Syndrome Type 2 | Usher Syndrome Type 3

United States
ProQR Therapeutics

Terminated

Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste)

Eye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf Blind

United States, United Kingdom
Imperial College London
Homerton University Hospital NHS Foundation Trust

Withdrawn

Combining Physical Therapy With Vestibular Stimulation to Improve Postural Stability in Pusher's Syndrome

Pusher Syndrome

United Kingdom
Sanofi

Active, not recruiting

A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B

Usher's Syndrome

United States, France
Sanofi

Terminated

Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B

Retinitis Pigmentosa | Usher Syndrome

United States, France
ProQR Therapeutics

Active, not recruiting

Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)

Eye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf Blind

United States, Netherlands, United Kingdom, Germany
ProQR Therapeutics

Terminated

An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (HELIA)

Retinitis Pigmentosa | Usher Syndrome Type 2

United States, Canada, France
Virginia Commonwealth University
University of Colorado, Denver; Washington University School of Medicine; University... and other collaborators

Recruiting

Trial Readiness and Endpoint Assessment in LGMD R1 (GRASP-01-003)

Limb Girdle Muscular Dystrophy | Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A | Limb Girdle Muscular Dystrophy Type R1 | LGMD2A

United States, United Kingdom
University College, London
University of Iowa

Unknown

Muscle MRI in Charcot Mary Tooth Disease: a Prospective Cohort Study

Charcot-Marie-Tooth Disease, Type IA | Charcot-Marie-Tooth Disease Type 2A | Charcot-Marie-Tooth Disease, Type IB | Charcot-Marie-Tooth Disease, Type X

United Kingdom
ProQR Therapeutics

Completed

Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Stellar)

Eye Diseases | Retinitis Pigmentosa | Eye Diseases, Hereditary | Vision Disorders | Retinal Disease | Eye Disorders Congenital | Usher Syndrome Type 2 | Deaf Blind

United States, Canada, France
Linda Pax Lowes

Recruiting

Natural History of Limb Girdle Muscular Dystrophy Type 2A and Type 2E

Limb-Girdle Muscular Dystrophy Type 2A | Limb-Girdle Muscular Dystrophy, Type 2E

United States
National Eye Institute (NEI)

Completed

Natural History and Genetic Studies of Usher Syndrome

Retinitis Pigmentosa | Usher Syndrome | Retinitis Pigmentosa Syndromic | Congenital Deafness | Retinitis Pigmentosa and Deafness

United States
Freeman-Sheldon Research Group, Inc.

Terminated

Survey Study and Records Review of Treatment Outcomes in Freeman-Sheldon Syndrome (STOP-FSS)

Craniofacial Abnormalities | Arthrogryposis | Freeman-Sheldon Syndrome | Arthrogryposis Distal Type 2A | Whistling Face Syndrome | Craniocarpotarsal Dysplasia | Craniocarpotarsal Dystrophy | Freeman-Sheldon Syndrome Variant | Sheldon-Hall Syndrome | Arthrogryposis Distal Type 2B | Gordon Syndrome | Arthrogryposis... and other conditions

United States
National Eye Institute (NEI)

Completed

Study of Usher Syndromes, Type 1 and Type 2

Retinitis Pigmentosa

United States
National Center for Research Resources (NCRR)
Oregon Health and Science University

Terminated

Study of Dietary N-3 Fatty Acids in Patients With Retinitis Pigmentosa and Usher Syndrome

Retinitis Pigmentosa | Usher Syndrome

United States
National Cancer Institute (NCI)

Completed

Vandetanib to Treat Children and Adolescents With Medullary Thyroid Cancer

Multiple Endocrine Neoplasia Type 2A | Multiple Endocrine Neoplasia Type 2B | Medullary Thyroid Carcinoma

United States
Ohio State University
National Institute on Deafness and Other Communication Disorders (NIDCD)

Recruiting

Auditory Neural Function in Implanted Patients With Usher Syndrome

Usher Syndrome | Cochlear Implantation

United States
National Center for Research Resources (NCRR)

Completed

A Genetic Analysis of Usher Syndrome in Ashkenazi Jews

Retinitis Pigmentosa | Usher Syndrome | Congenital Hearing Impairment

United States
King's College Hospital NHS Trust

Terminated

Pilot Study of Reduced Intensity Haematopoietic Stem Cell Transplantation in Patients With Poor Risk Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukaemia (AML) Utilising Conditioning With Fludarabine, Busulphan and Thymoglobulin (FB-ATG)

Myelodysplastic Syndromes | Leukemia, Myeloid, Acute

United Kingdom
King's College Hospital NHS Trust

Unknown

Prophylactic DLI for the Prevention of Relapse Post HSCT in Patients With High Risk Myeloid Malignancy (PRO-DLI)

Myelodysplastic Syndromes | Leukemia, Myeloid, Acute

United Kingdom
Medical Research Council

Completed

Combination Chemotherapy With or Without Bone Marrow Transplantation in Treating Children With Acute Myeloid Leukemia

Myelodysplastic Syndromes | Leukemia

United Kingdom
LumiraDx UK Limited

Completed

Evaluation of Precision and Accuracy of INR Measurements in a Point of Care Device (OPTIMAL) (OPTIMAL)

Atrial Fibrillation | Thrombophilia | Deep Vein Thrombosis | Antiphospholipid Syndrome | Pulmonary Embolism | Post-Myocardial Infarction Syndrome | Artificial Heart Device User

United Kingdom
King's College London
King's College Hospital NHS Trust

Unknown

Use of Decitabine in Myelodysplastic Syndrome (MDS) Following Azacitidine (AZA) Failure (DEC-MDS)

Acute Myeloid Leukemia | Chronic Myelomonocytic Leukemia | Myelodysplastic Syndrome

United Kingdom
Riverside Haematology Group

Unknown

Combination Chemotherapy in Treating Older Patients With Acute Myeloid Leukemia

Myelodysplastic Syndromes | Leukemia

United Kingdom
Celgene

Completed

Safety Study of Oral Azacitidine (CC-486) as Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) in Participants With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes (MDS).

Myelodysplastic Syndromes | Leukemia, Myeloid, Acute

United States, United Kingdom
Medical Research Council

Completed

Combination Chemotherapy With or Without Filgrastim and/or Tretinoin in Treating Patients With Acute Myeloid Leukemia

Myelodysplastic Syndromes | Leukemia | Myelodysplastic/Myeloproliferative Neoplasms

United Kingdom
King's College London

Completed

Azacytidine for the Treatment of Myelodysplastic Syndromes/Acute Myeloid Leukemia (MDS/AML) With High Risk (Chromosome 7 and or Complex) Cytogenetic Abnormalities

Myelodysplastic Syndromes | Leukemia, Myeloid, Acute

United Kingdom
King's College Hospital NHS Trust

Completed

King's Invasive Aspergillosis Study II

Myelodysplastic Syndromes | Leukemia, Myeloid, Acute | Bone Marrow Transplantation | Aplastic Anemia

United Kingdom
Royal Marsden NHS Foundation Trust

Recruiting

Inherited Cancer Early Diagnosis (ICED) Study (ICED)

Lynch Syndrome | Familial Adenomatous Polyposis | Hereditary Diffuse Gastric Cancer | Peutz-Jeghers Syndrome | Cowden Syndrome | Li Fraumeni Syndrome | MutYH-associated Polyposis | SMAD4 Gene Mutation

United Kingdom
Kyowa Kirin, Inc.

Terminated

Study of KHK2823 in Patients With Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS)

Acute Myeloid Leukemia | Myelodysplastic Syndrome

United Kingdom
PellePharm, Inc.

Completed

Trial of Patidegib Gel 2%, 4%, and Vehicle to Decrease the Number of Surgically Eligible Basal Cell Carcinomas in Gorlin Syndrome Patients

Basal Cell Nevus Syndrome

United Kingdom
Mirati Therapeutics Inc.

Terminated

Phase 2 Study of Azacitidine (Vidaza) vs MGCD0103 vs Combination in Elderly Subjects With Newly Diagnosed Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS)

Acute Myeloid Leukemia (AML) | Myelodysplastic Syndrome (MDS)

United States, Canada, United Kingdom
Cardiff University
Experimental Cancer Medicine Centres; Leukaemia & Lymphoma Research Group

Completed

A Trial to Establish the Feasibility of Combining Either the Tyrosine Kinase Inhibitor AC220,CXCR4 Inhibitor Plerixafor or HSP90 Inhibitor Ganetespib With Chemotherapy in Older Patients With Acute Myeloid Leukaemia and High Risk Myelodysplastic Syndrome. (AML18 Pilot)

High Risk Myelodysplastic Syndrome | Acute Myeloid Leukaemia

United Kingdom
RTI International
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

Early Check: Expanded Screening in Newborns

Primary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditions

United States
Lexicon Pharmaceuticals

Completed

An Open-label Study to Evaluate the Absorption, Metabolism, and Excretion of [14C]-Telotristat Etiprate in Males (LX1606-104)

Carcinoid Syndrome

United Kingdom
Cell Medica Ltd
University College, London; Cell Therapy Catapult

Completed

A Phase I/II Study of Gene-modified WT1 TCR Therapy in MDS & AML Patients

Myelodysplastic Syndromes (MDS) | Acute Myeloid Leukaemia (AML)

United Kingdom, Germany, Belgium
St. Bartholomew's Hospital

Unknown

Early Diagnosis of Aspergillosis in Patients at High Risk of Fungal Infection Caused by Treatment for Hematologic Cancer or Other Disease

Myelodysplastic Syndromes | Leukemia | Infection | Graft Versus Host Disease | Myelodysplastic/Myeloproliferative Neoplasms

United Kingdom
University of Birmingham
Jazz Pharmaceuticals; Adienne SA; IMPACT (funded by NHS Blood & Transplant, Anthony...

Active, not recruiting

A Trial of Treatments to Assess the Effects on Outcome of Adults With AML and MDS Undergoing Allogeneic SCT (COSI)

Acute Myeloid Leukaemia | High-risk Myelodysplastic Syndrome

United Kingdom
Barts & The London NHS Trust
Imperial College London; Queen Mary University of London

Terminated

Metabolic Myopathy in Endocrinopathy (LYDIA)

Cushing Syndrome | Pheochromocytoma | Conns Syndrome | Metabolic Myopathy

United Kingdom
Sheffield Teaching Hospitals NHS Foundation Trust
University of Sheffield

Completed

Effects of Serotonin Excess on Bone in Carcinoid Syndrome

Carcinoid Syndrome

United Kingdom
Leukemia Research Fund

Completed

Intensive Compared With Nonintensive Chemotherapy in Treating Older Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

Myelodysplastic Syndromes | Leukemia | Myelodysplastic/Myeloproliferative Neoplasms

United Kingdom
Inmune Bio, Inc.

Terminated

A Phase I Open-Label Dose Escalation Study of Intravenous INKmune In Patients With MDS or AML (LAUREL)

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Cancer | AML | MDS-EB

United Kingdom, Greece
UK Kidney Association

Recruiting

National Registry of Rare Kidney Diseases (RaDaR)

Vasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditions

United Kingdom
University of Aberdeen

Completed

Effect of Fish Oil on Insulin Sensitivity

Type 2 Diabetes Mellitus | Sarcopenia | Metabolic Syndrome X

United Kingdom
University of Michigan
University of California, San Francisco

Withdrawn

An Osseoanchored Percutaneous Prosthesis Study Evaluating Stable Neural Signal Transmission in Subjects With Transhumeral Amputations

Amputation | Prosthesis User | Amputation Neuroma

United States
Shire

Terminated

Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Sanfilippo Syndrome

Netherlands, United Kingdom
Food and Drug Administration (FDA)
National Eye Institute (NEI)

Recruiting

Adaptive Optics Imaging of Outer Retinal Diseases

Age-Related Macular Degeneration | Retinal Degeneration | Retinitis Pigmentosa | Usher Syndromes | Cone Dystrophy | Rod Dystrophy | Cone Rod Dystrophy | Hydroxychloroquine Retinopathy | Late-Onset Retinal Degeneration | Rod Cone Dystrophy

United States
King's College Hospital NHS Trust

Terminated

Pilot Study of Unrelated Cord Blood Transplantation

Lymphoma, Non-Hodgkin | Myelodysplastic Syndromes | Leukemia, Myeloid, Acute | Hodgkin Disease | Chronic Lymphocytic Leukemia | Leukemia, Lymphoblastic, Acute

United Kingdom
4SC AG

Completed

A Phase 1, Open-label Study of the Absorption, Metabolism, Excretion of [14C]-Resminostat

Mycosis Fungoides | Sezary Syndrome | Cutaneous T Cell Lymphoma

United Kingdom

Clinical Trials on Usher Syndrome Type 2A in United Kingdom

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