Clinical Trials on Mucopolysaccharidosis Type IVA

Total 58421 results

National Cancer Institute (NCI)

Terminated

Capecitabine and Vorinostat in Treating Patients With Recurrent and/or Metastatic Head and Neck Cancer

Recurrent Hypopharyngeal Squamous Cell Carcinoma | Recurrent Laryngeal Squamous Cell Carcinoma | Recurrent Oral Cavity Squamous Cell Carcinoma | Recurrent Oropharyngeal Squamous Cell Carcinoma | Stage IVA Oral Cavity Squamous Cell Carcinoma | Stage IVA Laryngeal Squamous Cell Carcinoma | Stage... and other conditions

Canada, United States
Clínica de Hidroterapia e Recuperacao Funcional
Integrated Faculty of Recife

Terminated

Morquio's Syndrome: a Case Study

Morquio's Disease
Denali Therapeutics Inc.

Completed

A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome

Mucopolysaccharidosis II

United Kingdom, United States, Netherlands
JCR Pharmaceuticals Co., Ltd.

Completed

A Study of JR-141 in Patients With Mucopolysaccharidosis II

Mucopolysaccharidosis II

Brazil
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Withdrawn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
Green Cross Corporation

Completed

To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients

Mucopolysaccharidosis II

Korea, Republic of
Eunice Kennedy Shriver National Institute of Child...
University of Minnesota

Completed

Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)

Mucopolysaccharidosis II

United States
University Hospital, Tours

Completed

Respiratory Cathepsins, Proteases Inhibitors and Glycosaminoglycans (GAG) in Mucopolysaccharidosis (RespiGAG)

Mucopolysaccharidoses

France
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
GC Biopharma Corp

Recruiting

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ

Mucopolysaccharidosis II | Hunter Syndrome

Korea, Republic of
Homology Medicines, Inc

Withdrawn

Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

Mucopolysaccharidosis II

United States, Canada
JCR Pharmaceuticals Co., Ltd.

Active, not recruiting

An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II

Mucopolysaccharidosis II

Brazil
Shire

Completed

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

Mucopolysaccharidosis II

United States, United Kingdom, Brazil, Germany
GlaxoSmithKline

Completed

A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723

Mucopolysaccharidosis II

Japan
Green Cross Corporation

Unknown

Study of Idursulfase-beta (GC1111) in Hunter Syndrome

Mucopolysaccharidosis II
National Cancer Institute (NCI)

Completed

Temsirolimus With or Without Cetuximab in Patients With Recurrent and/or Metastatic Head and Neck Cancer Who Did Not Respond to Previous Therapy (MAESTRO HN)

Recurrent Hypopharyngeal Squamous Cell Carcinoma | Recurrent Laryngeal Squamous Cell Carcinoma | Recurrent Oropharyngeal Squamous Cell Carcinoma | Salivary Gland Squamous Cell Carcinoma | Recurrent Lip and Oral Cavity Squamous Cell Carcinoma | Recurrent Nasopharyngeal Keratinizing Squamous... and other conditions

United States, China
BioMarin Pharmaceutical

Completed

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

MPS VI

United States, Australia, France, Brazil, Germany, Portugal, United Kingdom
JCR Pharmaceuticals Co., Ltd.

Recruiting

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

Mucopolysaccharidosis II

United States, Germany, Spain, France, Brazil, Argentina, Turkey, United Kingdom, Poland, Italy
Denali Therapeutics Inc.

Active, not recruiting

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Mucopolysaccharidosis II

United States, Netherlands, Canada, United Kingdom
JCR Pharmaceuticals Co., Ltd.

Completed

A Study of JR-141 in Patients With Mucopolysaccharidosis II

Mucopolysaccharidosis II

Japan
REGENXBIO Inc.

Enrolling by invitation

Long-term Follow-Up for RGX-121

Mucopolysaccharidosis II

United States
ArmaGen, Inc

Completed

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

Mucopolysaccharidosis II

United States, Germany, Philippines
Patricia I. Dickson, M.D.
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University... and other collaborators

Completed

A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I

Mucopolysaccharidosis I | Scheie Syndrome | Lysosomal Storage Disease | Cognitive Decline | Hurler-Scheie Syndrome

United States
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
Lysosomal and Rare Disorders Research and Treatment...
Team Sanfilippo

Recruiting

The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)

Sanfilippo Syndrome | MPS3

United States
Allievex Corporation

Enrolling by invitation

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

MPS III B

United States, Germany, Turkey, United Kingdom, Colombia
Takeda

Recruiting

A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India

Hunter Syndrome

India
City of Hope Medical Center
National Cancer Institute (NCI)

Active, not recruiting

Regorafenib, Ipilimumab and Nivolumab for the Treatment of Chemotherapy Resistant Microsatellite Stable Metastatic Colorectal Cancer

Metastatic Colorectal Adenocarcinoma | Advanced Colon Adenocarcinoma | Metastatic Colon Adenocarcinoma | Metastatic Colorectal Carcinoma | Metastatic Rectal Adenocarcinoma | Stage III Colon Cancer AJCC v8 | Stage III Rectal Cancer AJCC v8 | Stage IIIA Colon Cancer AJCC v8 | Stage IIIA Rectal Cancer... and other conditions

United States
University of Minnesota
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Completed

Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network

Mucopolysaccharidoses

United States
Lundquist Institute for Biomedical Innovation at...
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Completed

Longitudinal Study of Bone Disease in Children With Mucopolysaccharidoses (MPS) I, II, and VI

Mucopolysaccharidoses

United States
Duke University

Recruiting

MPS II Immunophenotyping

Mucopolysaccharidosis II | Hunter Syndrome | MPS II

United States
Shire

Completed

Hunter Outcome Survey (HOS) (HOS)

Hunter Syndrome

United States
Green Cross Corporation

Completed

A Study of GC1111 in Hunter Syndrom Patients

Hunter Syndrome

Korea, Republic of
Green Cross Corporation

Unknown

The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients

Hunter Syndrome

Korea, Republic of
Patricia I. Dickson, M.D.
The Ryan Foundation

Terminated

Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

Mucopolysaccharidosis I | Scheie Syndrome | Lysosomal Storage Disease | Spinal Cord Compression | Hurler-Scheie Syndrome

Finland, United States
ECOG-ACRIN Cancer Research Group
National Cancer Institute (NCI)

Recruiting

Testing the Addition of Radiotherapy to the Usual Treatment (Chemotherapy) for Patients With Esophageal and Gastric Cancer That Has Spread to a Limited Number of Other Places in the Body

Clinical Stage IV Gastric Cancer AJCC v8 | Metastatic Gastric Adenocarcinoma | Clinical Stage IV Esophageal Adenocarcinoma AJCC v8 | Clinical Stage IVA Esophageal Adenocarcinoma AJCC v8 | Clinical Stage IVA Gastric Cancer AJCC v8 | Clinical Stage IVB Esophageal Adenocarcinoma AJCC v8 | Clinical... and other conditions

United States
University of Washington
National Cancer Institute (NCI); GlaxoSmithKline; National Comprehensive Cancer...

Completed

Iodine I 131 and Pazopanib Hydrochloride in Treating Patients With Recurrent and/or Metastatic Thyroid Cancer Previously Treated With Iodine I 131 That Cannot Be Removed By Surgery

Recurrent Thyroid Cancer | Stage IVA Follicular Thyroid Cancer | Stage IVA Papillary Thyroid Cancer | Stage IVB Follicular Thyroid Cancer | Stage IVB Papillary Thyroid Cancer | Stage IVC Follicular Thyroid Cancer | Stage IVC Papillary Thyroid Cancer

United States
Masonic Cancer Center, University of Minnesota
BioMarin Pharmaceutical

Terminated

Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome

Maroteaux-Lamy Syndrome

United States
Emory University
National Cancer Institute (NCI); National Institutes of Health (NIH); Exelixis

Active, not recruiting

Pembrolizumab & Cabozantinib in Patients With Head and Neck Squamous Cell Cancer

Recurrent Head and Neck Squamous Cell Carcinoma | Recurrent Hypopharyngeal Squamous Cell Carcinoma | Recurrent Laryngeal Squamous Cell Carcinoma | Recurrent Oral Cavity Squamous Cell Carcinoma | Recurrent Oropharyngeal Squamous Cell Carcinoma | Stage IV Hypopharyngeal Squamous Cell Carcinoma... and other conditions

United States
National Cancer Institute (NCI)

Completed

Decitabine in Treating Patients With Metastatic Papillary Thyroid Cancer or Follicular Thyroid Cancer Unresponsive to Iodine I 131

Recurrent Thyroid Cancer | Stage IVA Follicular Thyroid Cancer | Stage IVA Papillary Thyroid Cancer | Stage IVB Follicular Thyroid Cancer | Stage IVB Papillary Thyroid Cancer | Stage IVC Follicular Thyroid Cancer | Stage IVC Papillary Thyroid Cancer

United States
City of Hope Medical Center
National Cancer Institute (NCI)

Completed

IRX-2, Cyclophosphamide, and Pembrolizumab in Treating Participants With Recurrent or Metastatic Gastric or Gastroesophageal Junction Cancer

Clinical Stage IV Gastric Cancer AJCC v8 | Clinical Stage IV Gastroesophageal Junction Adenocarcinoma AJCC v8 | Metastatic Gastric Adenocarcinoma | Metastatic Gastroesophageal Junction Adenocarcinoma | Postneoadjuvant Therapy Stage IV Gastroesophageal Junction Adenocarcinoma AJCC v8 | Postneoadjuvant... and other conditions

United States
Alexion Pharmaceuticals

Completed

Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging

MPS IIIB (Sanfilippo B Syndrome)

United Kingdom
M.D. Anderson Cancer Center
National Cancer Institute (NCI)

Active, not recruiting

Paclitaxel for the Treatment of Gastric or Gastroesophageal Cancer

Peritoneal Carcinomatosis | Clinical Stage IV Gastric Cancer AJCC v8 | Clinical Stage IV Gastroesophageal Junction Adenocarcinoma AJCC v8 | Metastatic Gastric Adenocarcinoma | Metastatic Gastroesophageal Junction Adenocarcinoma | Postneoadjuvant Therapy Stage IV Gastroesophageal Junction Adenocarcinoma... and other conditions

United States
University of Arizona
National Cancer Institute (NCI)

Completed

Ficlatuzumab w/wo Cetuximab in Patients w/Cetuximab-Resistant, Recurrent or Metastatic Head/Neck Squamous Cell Carcinoma

Head and Neck Cancer | Recurrent Head and Neck Squamous Cell Carcinoma | Oropharyngeal Cancer | HNSCC | Recurrent Oropharyngeal Squamous Cell Carcinoma | Stage IV Squamous Cell Carcinoma of the Lip and Oral Cavity | Stage IVA Lip and Oral Cavity Squamous Cell Carcinoma | Stage IVA Nasal Cavity and... and other conditions

United States
Masonic Cancer Center, University of Minnesota

Completed

Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

Hurler Syndrome

United States
National Cancer Institute (NCI)

Active, not recruiting

Chemotherapy With or Without Bevacizumab in Treating Patients With Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma

Recurrent Hypopharyngeal Squamous Cell Carcinoma | Recurrent Laryngeal Squamous Cell Carcinoma | Recurrent Oropharyngeal Squamous Cell Carcinoma | Stage IV Hypopharyngeal Squamous Cell Carcinoma AJCC v7 | Stage IVA Laryngeal Squamous Cell Carcinoma AJCC v7 | Stage IVA Oropharyngeal Squamous... and other conditions

United States, Puerto Rico, South Africa
City of Hope Medical Center
National Cancer Institute (NCI)

Recruiting

PIPAC for the Treatment of Peritoneal Carcinomatosis in Patients With Ovarian, Uterine, Appendiceal, Colorectal, or Gastric Cancer

Peritoneal Carcinomatosis | Stage IV Uterine Corpus Cancer AJCC v8 | Stage IVA Uterine Corpus Cancer AJCC v8 | Stage IVB Uterine Corpus Cancer AJCC v8 | Metastatic Malignant Solid Neoplasm | Malignant Uterine Neoplasm | Clinical Stage IV Gastric Cancer AJCC v8 | Metastatic Colorectal Carcinoma | Stage... and other conditions

United States
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Morquio Disease (BioMorquio) (BioMorquio)

Morquio Syndrome A | Morquio Syndrome | Morquio B Disease | Accumulation of Mucopolysaccharides

Germany, India, Sri Lanka, Egypt
National Cancer Institute (NCI)

Completed

Lenalidomide and Cetuximab in Treating Patients With Advanced Colorectal Cancer or Head and Neck Cancer (ADCC)

Recurrent Colon Carcinoma | Recurrent Rectal Carcinoma | Recurrent Hypopharyngeal Squamous Cell Carcinoma | Recurrent Laryngeal Squamous Cell Carcinoma | Recurrent Oropharyngeal Squamous Cell Carcinoma | Stage IV Hypopharyngeal Squamous Cell Carcinoma AJCC v7 | Stage IVA Laryngeal Squamous Cell... and other conditions

United States

Clinical Trials on Mucopolysaccharidosis Type IVA

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