Search clinical trials for: Atypical CLN2 Disease

Total 383393 results

Jessica Scherr
BioMarin Pharmaceutical

Enrolling by invitation

Examining Developmental Outcomes of Children Diagnosed With CLN2 Disease

Batten Disease | Neuronal Ceroid-Lipofuscinoses | CLN2

United States
BioMarin Pharmaceutical

Approved for marketing

A Multicenter, Multi-national Open-label Program to Provide BMN 190 to Patients Diagnosed With CLN2 Disease

CLN2 Disease

United Kingdom, United States, Germany, Italy
REGENXBIO Inc.

Withdrawn

An Observational Study in Children With CLN2 Batten Disease

Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
BioMarin Pharmaceutical

Completed

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

Batten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky Disease

United States, Italy, Germany, United Kingdom
REGENXBIO Inc.

Withdrawn

A Retrospective, Natural History Study in Children With CLN2

Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
REGENXBIO Inc.

Active, not recruiting

A First-in-Human Study in Pediatric Patients With Ocular CLN2 Disease

Neuronal Ceroid Lipofuscinosis Type 2

United Kingdom
BioMarin Pharmaceutical

Active, not recruiting

Cerliponase Alfa Observational Study in the US

Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2

United States
BioMarin Pharmaceutical

Completed

An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

Batten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky Disease | CLN2 Disorder

United States, Germany, Italy, United Kingdom
Nadir Hastalıkları Araştırma Derneği

Recruiting

A Multicenter Selective Screening Study to Investigate the Frequency of Neuronal Ceroid Lipofuxinosis Type 2 (CLN2) (ENIGMA)

Neuronal Ceroid Lipofuxinosis Type2 (CLN2)

Turkey
Children's Hospital of Orange County

Active, not recruiting

Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

Neuronal Ceroid-Lipofuscinoses | Neuronal Ceroid Lipofuscinosis CLN2 | Spinocerebellar Ataxia, Autosomal Recessive 7

United States
BioMarin Pharmaceutical

Completed

A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

Batten Disease | CLN2 Disease | Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 | Jansky-Bielschowsky Disease | CLN2 Disorder

United States, Germany, Italy, United Kingdom
David L Rogers, MD

Active, not recruiting

Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

Neuronal Ceroid Lipofuscinosis Type 2

United States
Esanex Inc.

Terminated

Study of SNX-5422 in TP53 Null Cancers

Cancer

United States
Jazz Pharmaceuticals

Completed

CX-8998 for Absence Seizures (T-WAVE)

Epilepsy

United States
Jazz Pharmaceuticals

Completed

A Phase 2 RCT Study of CX-8998 for Essential Tremor

Essential Tremor

United States
Jazz Pharmaceuticals

Withdrawn

A Phase 2 Study of CX-8998 in Adults With Tremor Associated With Parkinson's Disease

Parkinson's Disease | Tremor
Mucosal Therapeutics

Completed

Efficacy of SNX-1012 in the Treatment of Oral Mucositis

Stomatitis | Oral Mucositis

United States, India
innoVactiv Inc.

Completed

Safety and Efficacy of a Single Dose of a Polyphenol-enriched Brown Seaweed Powder in Human (ICAST)

Glycemic Index

Canada
University of Rochester

Recruiting

Clinical and Neuropsychological Investigations in Batten Disease

Batten Disease | Neuronal Ceroid Lipofuscinosis | Neuronal Ceroid Lipofuscinosis CLN6 | Neuronal Ceroid Lipofuscinosis CLN3 | Neuronal Ceroid Lipofuscinosis CLN5 | Neuronal Ceroid Lipofuscinosis CLN1 | Neuronal Ceroid Lipofuscinosis CLN2 | Neuronal Ceroid Lipofuscinosis CLN7 | Neuronal Ceroid Lipofuscinosis... and other conditions

United States
Universitätsklinikum Hamburg-Eppendorf

Recruiting

Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database

Batten Disease | Neuronal Ceroid Lipofuscinosis | CLN1 Disease | CLN2 Disease | CLN3 Disease | CLN4 Disease | CLN5 Disease | CLN6 Disease | CLN7 Disease | CLN8 Disease | CLN10 Disease | CLN11 Disease | CLN12 Disease | CLN13 Disease | CLN14 Disease

Germany
Xiaojun Chen

Recruiting

GnRHa + Letrozole in Obese Progestin-insensitive Endometrial Atypical Hyperplasia Patients

Obesity | Atypical Endometrial Hyperplasia | Progesterone Resistance

China
Xiaojun Chen

Recruiting

GnRHa + Letrozole in Obese Progestin-insensitive Endometrial Cancer Patients

Obesity | Atypical Endometrial Hyperplasia | Endometrial Neoplasms | Progesterone Resistance

China
Mayo Clinic

Recruiting

Non-Invasive Identification of Endometrial Cancer/Endometrial Atypical Hyperplasia With an AI-Based Classifier Applied to Transvaginal Ultrasound in Patients With Post-Menopausal Bleeding

Endometrial Carcinoma

United States
Alexion Pharmaceuticals, Inc.

Not yet recruiting

Study of Ultomiris® (Ravulizumab) Safety in Pregnancy

Pregnancy | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Atypical Hemolytic Uremic Syndrome (aHUS) | Ultomiris-exposed Pregnant/ Postpartum | Generalized Myasthenia Gravis (gMG) | Neuromyelitis Optica Spectrum Disorder (NMOSD)
All India Institute of Medical Sciences, Bhubaneswar

Recruiting

Atypical Antipsychotic-induced Mitochondrial Dysfunction in Patients With Schizophrenia

Schizophrenia

India
National Cancer Institute (NCI)

Not yet recruiting

Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)

Neurofibromatosis 1 | Nerve Sheath Neoplasms

United States
National Cancer Institute (NCI)

Not yet recruiting

Low Dose Tamoxifen With or Without Omega-3 Fatty Acids for Breast Cancer Risk Reduction

Breast Carcinoma | Breast Ductal Carcinoma In Situ | Breast Lobular Carcinoma In Situ | Breast Atypical Hyperplasia

United States
Children's National Research Institute

Recruiting

Immunotherapy for Malignant Pediatric Brain Tumors Employing Adoptive Cellular Therapy (IMPACT) (IMPACT)

Medulloblastoma, Childhood | Embryonal Tumor With Multilayered Rosettes | Pineoblastoma | Embryonal Tumor of CNS | Atypical Teratoid/Rhabdoid Tumor of CNS

United States
Beijing Obstetrics and Gynecology Hospital

Not yet recruiting

Conservative Treatment for Atypical Endometrial Hyperplasia and Early Endometrial Cancer

Atypical Endometrial Hyperplasia and Endometrial Cancer
Haukeland University Hospital
Nordlandssykehuset HF; Helse Møre og Romsdal HF; Helse Fonna HF; Oslo universitetssykehus... and other collaborators

Not yet recruiting

The NADAPT Study: a Randomized Double-blind Trial of NAD Replenishment Therapy for Atypical Parkinsonism (NADAPT)

Multiple System Atrophy | Progressive Supranuclear Palsy | Corticobasal Syndrome

Norway
Peking University People's Hospital

Not yet recruiting

Endometrial Cancer Screening in High-risk Populations

Cancer Screening
Amylyx Pharmaceuticals Inc.

Recruiting

AMX0035 and Progressive Supranuclear Palsy (ORION)

Neurodegenerative Diseases | Progressive Supranuclear Palsy | Atypical Parkinsonism | PSP

United States
Zhujiang Hospital

Recruiting

Clinical Study of Stellate Ganglion Block in Treatment of Patients With Advanced Primary Parkinson's Disease

Parkinson Disease | Stellate Ganglion

China
University Hospital, Strasbourg, France

Recruiting

Hematological Effects of Atypical Antipsychotics (AAPs) in a Geriatric Population: What is the Role of 5-HT2 Receptors (AAPs)

Neutropenia

France
Peking University People's Hospital

Recruiting

Progesterone Therapeutic Regimen Plus Statins in Young Women With Early Endometrial Carcinoma and Atypical Endometrial Hyperplasia

Atypical Endometrial Hyperplasia and Endometrial Carcinoma Stage I

China
AstraZeneca

Recruiting

A Prospective, Non-interventional, Observational Study of Presentation, Treatment Patterns and Outcomes in Atypical Hemolytic Uremic Syndrome Patients

Atypical Hemolytic Uremic Syndrome(aHUS)

China
University Health Network, Toronto

Not yet recruiting

Weight-loss Drug for Fertility-Sparing Treatment of Atypical Hyperplasia and Grade 1 Cancer of the Endometrium (WE-FiERCE)

Endometrial Cancer | Atypical Hyperplasia
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
Mario Negri Institute for Pharmacological Research

Recruiting

Developing a Pipeline to Employ RNA-Seq as a Complementary Diagnostic Tool in Rare Diseases (ANTHEM)

Healthy | Autosomal Dominant Polycystic Kidney | Atypical Hemolytic Uremic Syndrome | Membranoproliferative Glomerulonephritis

Italy
National Taiwan University Hospital

Recruiting

Treatment Responses of Early Syphilis to Ceftriaxone Plus Doxycycline

Early Syphilis

Taiwan
National Cancer Institute (NCI)

Not yet recruiting

Acolbifene Versus Low Dose Tamoxifen for the Prevention of Breast Cancer in Premenopausal Women at High Risk for Development of Breast Cancer

Breast Carcinoma | Breast Ductal Carcinoma In Situ | Breast Lobular Carcinoma In Situ | Breast Atypical Hyperplasia

United States
Novartis Pharmaceuticals

Recruiting

Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)

Atypical Hemolytic Uremic Syndrome

Japan, Turkey
Children's Hospital of Philadelphia
National Institute of Neurological Disorders and Stroke (NINDS); Vanderbilt...

Recruiting

Neurophysiologic Biomarkers in Rett Syndrome

Rett Syndrome | Rett Syndrome, Atypical | RTT

United States
European Institute of Oncology
Ministero della Salute, Italy

Recruiting

Excisional Vacuum-Assisted Breast Biopsy

Ductal Carcinoma in Situ | Atypical Ductal Hyperplasia

Italy
Alexion Pharmaceuticals, Inc.
AstraZeneca

Recruiting

Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China (Soliris)

Atypical Hemolytic Uremic

China
Hospital Universitario 12 de Octubre

Recruiting

Substrate abLatIon as Concomitant trEatment for Left Atrial Macroreentrant Tachycardia (SLICE-LAMRT)

Atrial Fibrillation

Spain
Gruppo Italiano Malattie EMatologiche dell'Adulto

Recruiting

Novel Approaches to Target MECOM/EVI1 in AML

AML, Adult

Italy
St. Jude Children's Research Hospital

Recruiting

Loc3CAR: Locoregional Delivery of B7-H3-CAR T Cells for Pediatric Patients With Primary CNS Tumors

Glioblastoma | Central Nervous System Neoplasms | High Grade Glioma | Ependymoma | Medulloblastoma | Diffuse Midline Glioma, H3 K27M-Mutant | Atypical Teratoid/Rhabdoid Tumor

United States
Columbia University
National Institute on Minority Health and Health Disparities (NIMHD)

Recruiting

FHIR-Enhanced RealRisks to Improve Accuracy of Breast Cancer Risk Assessments (FHIR)

Breast Cancer

United States
Mario Negri Institute for Pharmacological Research

Recruiting

Functional Implications of Rare Gene Mutations in aHUS Open the Door to Personalized Therapy (aHUS-iPSC-EC)

Atypical Hemolytic Uremic Syndrome

Italy

Search clinical trials for: Atypical CLN2 Disease

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