Search clinical trials for: mps ii therapy

Total 77322 results

University of California, San Francisco
Duke University

Recruiting

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases (IUERT)

Wolman Disease | MPS IVA | Pompe Disease Infantile-Onset | Gaucher Disease, Type 2 | MPS VI | MPS I | Gaucher Disease, Type 3 | MPS II | Mps VII

United States
Paul Szabolcs

Recruiting

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT (HSCT+RIC)

Congenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias | Systemic Juvenile Idiopathic Arthritis (sJIA)

United States
Ultragenyx Pharmaceutical Inc
Abeona Therapeutics, Inc

Enrolling by invitation

A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102

Mucopolysaccharidosis III-A

United States, Australia, Spain
Ultragenyx Pharmaceutical Inc
Abeona Therapeutics, Inc

Active, not recruiting

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Mucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo A

United States, Australia, Spain
Shire

Completed

An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment

Hunter Syndrome

United States, United Kingdom, Canada
Sangamo Therapeutics

Active, not recruiting

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX (LTFU)

Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II

United States
REGENXBIO Inc.

Active, not recruiting

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Mucopolysaccharidosis Type II (MPS II)

United States, Brazil
REGENXBIO Inc.

Active, not recruiting

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Mucopolysaccharidosis Type II (MPS II)

United States, Canada
Ultragenyx Pharmaceutical Inc
Abeona Therapeutics, Inc

Terminated

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

Mucopolysaccharidosis III | Sanfilippo Syndrome | MPS IIIA | Sanfilippo A

United States, Spain, Australia
Sangamo Therapeutics

Terminated

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

Mucopolysaccharidosis II | MPS II

United States
REGENXBIO Inc.

Withdrawn

Mucopolysaccharidosis Type II Observational

Mucopolysaccharidosis II

United States, Canada
Fondazione Telethon

Active, not recruiting

Gene Therapy in Patients With Mucopolysaccharidosis Disease

Mucopolysaccharidosis Type VI

Italy, Turkey
LYSOGENE

Active, not recruiting

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (AAVance)

Mucopolysaccharidosis Type IIIA

Germany, United States, United Kingdom, France, Netherlands
REGENXBIO Inc.

Enrolling by invitation

Long-term Follow-Up for RGX-121

Mucopolysaccharidosis II

United States
Shire

Completed

A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®

Hunter Syndrome

United States, United Kingdom
Shire

Completed

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment (AIM-IT)

Hunter Syndrome

Spain, United States, Canada, United Kingdom, France, Australia, Mexico
Shire

Completed

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

Hunter Syndrome | Mucopolysaccharidosis II (MPS II)

United States, France, United Kingdom, Germany, Sweden, Spain, Brazil, Canada, Italy, Romania
Shire

Completed

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

Mucopolysaccharidosis II

United States, United Kingdom, Brazil, Germany
Shire

Completed

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

Sanfilippo Syndrome

United States, Spain, United Kingdom, Argentina, France, Germany, Italy, Netherlands
Shire

Completed

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

Hunter Syndrome

United Kingdom, United States, Brazil
Shire
Covance; PRA Health Sciences; PharmaNet

Completed

Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

Mucopolysaccharidosis II | Hunter Syndrome | MPS II

Brazil, Poland, Taiwan
Institut de Recherches Internationales Servier
ADIR, a Servier Group company

Completed

S 81694 Plus Paclitaxel in Metastatic Breast Cancer

Metastatic Breast Cancer | Metastatic Triple Negative Breast Cancer

Japan, Belgium, France, Netherlands
University of Chicago
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Completed

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Krabbe Disease | Mucopolysaccharidosis Type II (MPS II) | Mucopolysaccharidosis Type I (MPS I) | Mucopolysaccharidosis Type III (MPS III) | Mucopolysaccharidosis Type VI (MPS VI)

United States
Aldagen

Terminated

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases (UCBT-002)

Mucopolysaccharidosis | Inborn Errors of Metabolism | Lysosomal Storage Disorders | Inherited Metabolic Diseases | Peroxisomal Storage Diseases

United States
Guangdong Provincial People's Hospital

Not yet recruiting

Randomized Study of ABC-14 Regimen Compared With "3+7" Standard Induction Therapy or AB-14 for ND AML

Acute Myeloid Leukemia

China
Universität des Saarlandes

Recruiting

Pharmacokinetics of Bisoprolol and SGLT2i in Acutely Decompensated Heart Failure (BISO-ADHF)

Acute Decompensated Heart Failure

Germany
New Approaches to Neuroblastoma Therapy Consortium
Nationwide Children's Hospital; United Therapeutics; Children's Neuroblastoma...

Not yet recruiting

NANT 2021-01 Phase II STING (Sequential Temozolomide, Irinotecan, NK Cells and GD2 mAb) Trial

Neuroblastoma

United States
Zachary Zumsteg

Not yet recruiting

Using CircuLating Tumor DNA to Risk Adapt Post-Operative Therapy for HPV-associated Oropharyngeal Cancer

Carcinoma | Oropharyngeal Cancer

United States
Lei Liu

Not yet recruiting

A Phase II Study of Neoadjuvant Immunotherapy in Combination With Chemotherapy in Locally Advanced Head and Neck Squamous Cell Carcinoma

Head and Neck Squamous Cell Carcinoma
Virginia Commonwealth University

Not yet recruiting

CBT-I in Primary Brain Tumor Patients: Phase IIc Randomized Feasibility Pilot Trial (CBT-I in PBT)

Glioblastoma | Astrocytoma | Oligodendroglioma | Meningioma | Primary Brain Tumor | Primary Central Nervous System (CNS) Lymphoma

United States
Instituto de Investigación Biomédica de Salamanca
Carlos III Health Institute

Recruiting

Study of the Hematopoietic Niche and the Role of Inflammation in the Pathophysiology of Cytopenias After CAR-T Cell Therapy: Potential of Therapies Directed to Repair the Bone Marrow Microenvironment

Diffuse Large B Cell Lymphoma

Spain
Dana-Farber Cancer Institute
Johns Hopkins University; Translational Breast Cancer Research Consortium

Not yet recruiting

The SAPPHO Study: A Single-Arm, Phase II Study of Sequential Therapy With Curative Intent in de Novo HER2+ Metastatic Breast Cancer

Breast Cancer | HER2-positive Breast Cancer | Stage IV Breast Cancer | Breast Cancer Female | Breast Cancer Metastatic | Estrogen Receptor-positive Breast Cancer

United States
Shanghai Chest Hospital

Not yet recruiting

Neoadjuvant Therapy of SBRT Sequencial With Toripalimab and Chemotherapy in Resectable Stage II-III NSCLC Patients(neoR-TORCH) (neoR-TORCH)

Stage II-III Non-small Cell Lung Cancer
University Hospital, Ghent

Recruiting

Pediatric Antibiotic Dosing in Extracorporal Membrane Oxygenation (PADECMO) (PADECMO)

Pharmacokinetics | Vancomycin | Amoxicillin-clavulanate | Piperacillin-tazobactam | Teicoplanin | Meropenem | Ciprofloxacin | Amikacin | Cefazolin

Belgium
Universidade Federal Fluminense

Recruiting

Clinical and Microbiological Evaluation of Laser Therapy in the Treatment of Periodontal Disease in Stages III and IV

Periodontitis | Periodontitis, Adult

Brazil
Riphah International University

Recruiting

Comparison of Scapular Stabilization and Mobilizations in Sub Acromial Pain Syndrome

Subacromial Impingement Syndrome

Pakistan
Leslie Ballas

Not yet recruiting

Bladder Preservation for Patients With Muscle Invasive Bladder Cancer (MIBC) With Variant Histology

Muscle-Invasive Bladder Carcinoma | Tumor

United States
Roswell Park Cancer Institute

Recruiting

An Education and Navigation Support Tool to Improve Participation in Care Coordination Among Patients With Locally Advanced, Metastatic and Unresectable Bladder Cancer and Their Caregivers

Stage III Bladder Cancer AJCC v8 | Stage IV Bladder Cancer AJCC v8 | Metastatic Bladder Carcinoma | Locally Advanced Bladder Carcinoma | Unresectable Bladder Carcinoma

United States
University of Kansas Medical Center

Not yet recruiting

Combination of Tagraxofusp With Pacritinib in Patients With Intermediate-1 or Higher Myelofibrosis, Who Have Had Prior Therapy With the Approved JAK Inhibitors or in Which Therapy With the Approved JAK Inhibitors is Not Appropriate, Contraindicated or Declined

Myelofibrosis，MF
Sunshine Guojian Pharmaceutical (Shanghai) Co.,...

Not yet recruiting

A Phase II Study of SSGJ-707 Combination Therapy in Advanced NSCLC Patients

First-line Advanced NSCLC Patients

China
M.D. Anderson Cancer Center

Not yet recruiting

Phase II Study of REPotrectinib With or Without Fulvestrant in Patients With Hormone Receptor-positive Human Epidermal Growth Factor 2-negative Metastatic Invasive LObular Carcinoma Who Received a Prior Endocrine Therapy in Combination With Cyclin-dependent Kinase 4 and 6 Inhibitor (REPLOT Trial)

Hormone Receptor-positive Human Epidermal Growth Factor 2-negative | Metastatic Invasive LObular Carcinoma

United States
Shanghai Changzheng Hospital

Recruiting

UCMSCs Combined With Standard Therapy for the Treatment of Newly Diagnosed Type 1 Diabetes

Newly Diagnosed Type 1 Diabetes Mellitus

China
University Hospital Heidelberg

Recruiting

Predicting Esophageal Cancer Borders Using PET-Imaging (PEGASUS)

Esophagus Cancer, Stage I | Esophagus Cancer, Stage II

Germany
European Organisation for Research and Treatment...

Not yet recruiting

Improvement of Quality of Life Through Supportive Treatments for Hormone Therapy - Related Symptoms in Women With Early Breast Cancer

Musculoskeletal Pain | Drug-Related Side Effects and Adverse Reactions | Breast Cancer Stage I | Breast Cancer Stage II | Breast Cancer Stage III | HER2-negative Breast Cancer | ER+ Breast Cancer
ETS Wound Care, LLC
Professional Education and Research Institute

Recruiting

Bioresorbable Glass Fiber Matrix in the Treatment of Diabetic Foot Ulcers (BGWM)

Diabetic Foot | Diabetic Foot Ulcer | Ulcer Foot

United States
Fudan University

Not yet recruiting

QL1706 Plus Chemotherapy as Neoadjuvant Therapy in Early High-Risk ER+/HER2- Breast Cancer (QUEEN-Neo)

Neoadjuvant Therapy | Early Breast Cancer | HR+HER2- Breast Cancer
Mayo Clinic

Not yet recruiting

Enhancing Skin Appearance and Quality of Life in Breast Cancer Survivors on Aromatase Inhibitor Therapy

Anatomic Stage I Breast Cancer AJCC v8 | Anatomic Stage II Breast Cancer AJCC v8 | Anatomic Stage III Breast Cancer AJCC v8 | Anatomic Stage 0 Breast Cancer AJCC v8 | Breast Adenocarcinoma | Estrogen Receptor-Positive Breast Carcinoma | Progesterone Receptor-Positive Breast Carcinoma

United States
Children's Oncology Group

Not yet recruiting

A Study Using Risk Factors to Determine Treatment for Children With Favorable Histology Wilms Tumors (FHWT)

Stage I Kidney Wilms Tumor | Stage II Kidney Wilms Tumor | Stage III Kidney Wilms Tumor | Stage IV Kidney Wilms Tumor
Celgene
Bristol-Myers Squibb

Recruiting

A Study to Compare the Efficacy and Safety of Idecabtagene Vicleucel With Lenalidomide Maintenance Therapy Versus Lenalidomide Maintenance Therapy Alone in Adult Participants With Newly Diagnosed Multiple Myeloma Who Have Suboptimal Response After Autologous Stem Cell Transplantation (KarMMa-9)

Multiple Myeloma

Austria, United States, Spain, Australia, Japan, France, Israel, Italy, Canada, Greece, Belgium, Norway, Romania, Czechia, Denmark, Germany, Korea, Republic of, United Kingdom, Poland
West China Hospital

Not yet recruiting

Neoadjuvant Chemoimmunotherapy in Stage IIIA- N2IIIB Non-small Cell Lung Cancer

Non-small Cell Lung Cancer Stage IIIB | Non-small Cell Lung Cancer Stage ⅢA

Search clinical trials for: mps ii therapy

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