Clinical Trials on Mucopolysaccharidosis Type IIIA

Total 57669 results

Joyce Fox
Ultragenyx Pharmaceutical Inc

No longer available

An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

Mucopolysaccharidosis Type 7

United States
Orchard Therapeutics

Recruiting

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT (HURCULES)

MPS-IH (Hurler Syndrome)

Netherlands, United States, Italy, United Kingdom
Children's Hospitals and Clinics of Minnesota
BioMarin Pharmaceutical; Gillette Children's Specialty Healthcare; Greenwood...

Completed

Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease

Mucopolysaccharidosis VI | Mucopolysaccharidosis IV A

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
Ohio State University Comprehensive Cancer Center

Completed

Aerobic and Strength Training Exercise in Improving Fitness and Arm Health During and After Radiation Therapy in Patients With Stage II-III Breast Cancer

Stage II Breast Cancer | Stage IIIA Breast Cancer | Stage IIIB Breast Cancer | Stage IIA Breast Cancer | Stage IIB Breast Cancer | Stage IIIC Breast Cancer | Stage III Breast Cancer

United States
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
BioMarin Pharmaceutical
ICON plc

Completed

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) (MARS)

Mucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVA

United States, United Kingdom, Australia, Taiwan, Belgium, Malaysia, Austria, Canada, Portugal, France, Ireland, Czechia, Denmark, Italy, Netherlands, Poland, Puerto Rico
Takeda

Completed

A Study to Improve the Awareness of Mucopolysaccharidosis Type II in Brazil

Mucopolysaccharidosis (MPS)

Brazil
Genzyme, a Sanofi Company

Recruiting

Mucopolysaccharidosis I (MPS I) Registry

Mucopolysaccharidosis I (MPS I)

Croatia, United States, Argentina, Australia, Belgium, Brazil, Canada, Denmark, France, Hong Kong, India, Indonesia, Korea, Republic of, Kuwait, Lebanon, Malaysia, Netherlands, Pakistan, Philippines, Poland, Saudi Arabia, Singapore, Slovaki... and more
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
BioMarin Pharmaceutical

Completed

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)

United States, France, Australia, Germany, Ireland, Austria, Belgium, Italy, Lithuania, Netherlands, Portugal, Sweden, United Kingdom
JCR Pharmaceuticals Co., Ltd.

Active, not recruiting

An Extension Study of JR-171-101 Study in Patients With MPS I

Mucopolysaccharidosis I

United States, Brazil, Japan
Institut National de la Santé Et de la Recherche...

Recruiting

MPS (RaDiCo Cohort) (RaDiCo-MPS)

Mucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Mucopolysaccharidosis III | Mucopolysaccharidosis IV | Mucopolysaccharidosis VII | Multiple Sulfatase Deficiency Disease | Mucopolysaccharidosis IX

France
University of Washington

Terminated

Occupational Therapist-Led Work Intervention in Facilitating Work Maintenance or Re-entry to the Workforce in Patients With Stage I to III Breast Cancer Undergoing Chemotherapy

Breast Cancer | Breast Cancer Stage I | Breast Cancer Stage II | Breast Cancer Stage III | Breast Cancer Stage IIB | Breast Cancer Stage IIA | Breast Cancer Stage IIIA | Breast Cancer Stage IIIB | Breast Cancer Stage IIIc

United States
Genzyme, a Sanofi Company

Completed

China Post-marketing Surveillance (PMS) Study of Aldurazyme®

Mucopolysaccharidosis I

China
JCR Pharmaceuticals Co., Ltd.

Completed

A Study of JR-171 in Patients With Mucopolysaccharidosis I

Mucopolysaccharidosis I

United States, Brazil, Japan
Inventiva Pharma

Completed

A Study in MPS VI to Assess Safety and Efficacy of Odiparcil (iMProveS)

Mucopolysaccharidosis VI

United Kingdom, France, Germany, Portugal
BioMarin Pharmaceutical

Completed

Study of rhASB in Patients With Mucopolysaccharidosis VI

Mucopolysaccharidosis VI

United States
BioMarin Pharmaceutical

Completed

Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

Mucopolysaccharidosis VI

United States
BioMarin Pharmaceutical

Completed

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

Mucopolysaccharidosis VI

United States
BioMarin Pharmaceutical

Completed

Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

Mucopolysaccharidosis VI

United States
Kibo Nam
National Cancer Institute (NCI)

Recruiting

Monitoring Neoadjuvant Chemotherapy of Breast Cancer Using 3D Subharmonic Aided Pressure Estimation

Anatomic Stage III Breast Cancer AJCC v8 | Anatomic Stage IIIA Breast Cancer AJCC v8 | Anatomic Stage IIIB Breast Cancer AJCC v8 | Anatomic Stage IIIC Breast Cancer AJCC v8 | Prognostic Stage III Breast Cancer AJCC v8 | Prognostic Stage IIIA Breast Cancer AJCC v8 | Prognostic Stage IIIB Breast... and other conditions

United States
GOIZET
BioMarin Pharmaceutical

Recruiting

Natural History of Atypical Morquio A Disease

Mucopolysaccharidosis IV A

France
Nemours Children's Clinic
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Recruiting

Non-invasive Functional Assessment and Pathogenesis of Morquio A (NIFAMA)

Mucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVA

United States
Hospital de Clinicas de Porto Alegre
The Isaac Foundation

Active, not recruiting

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

Mucopolysaccharidoses | Mucopolysaccharidosis VI | Morquio A Syndrome | Mucopolysaccharidosis IV A | MPS IV A | MPS VI | MPS - Mucopolysaccharidosis | Morquio Syndrome A | Morquio Syndrome

Brazil
BioMarin Pharmaceutical

Approved for marketing

BMN 110 US Expanded Access Program

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVA

United States, Puerto Rico
JCR Pharmaceuticals Co., Ltd.

Active, not recruiting

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

Mucopolysaccharidosis II

Japan
JCR Pharmaceuticals Co., Ltd.

Completed

A Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Mucopolysaccharidosis II

Japan
M.D. Anderson Cancer Center
National Cancer Institute (NCI)

Active, not recruiting

M7824 in Treating Patients With Stage II-III HER2 Positive Breast Cancer

Anatomic Stage II Breast Cancer AJCC v8 | Anatomic Stage IIA Breast Cancer AJCC v8 | Anatomic Stage IIB Breast Cancer AJCC v8 | Anatomic Stage III Breast Cancer AJCC v8 | Anatomic Stage IIIA Breast Cancer AJCC v8 | Anatomic Stage IIIB Breast Cancer AJCC v8 | Anatomic Stage IIIC Breast Cancer AJCC... and other conditions

United States
ArmaGen, Inc

Completed

Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I

Mucopolysaccharidosis I

Brazil
Emory University
BioMarin Pharmaceutical

Completed

Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them? (MorqPreg)

MPS - Mucopolysaccharidosis | Morquio Disease

United States
BioMarin Pharmaceutical

Terminated

A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Morquio A Syndrome | MPS IV A | Mucopolysaccharidosis IVA

United Kingdom
Phoenix Nest
National Institute of Neurological Disorders and Stroke (NINDS)

Enrolling by invitation

A Natural History Study of Sanfilippo Syndrome Type D (MPSIIID)

Sanfilippo Syndrome Type D

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
Denali Therapeutics Inc.

Recruiting

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) (COMPASS)

Mucopolysaccharidosis II

United States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Brazil, Colombia
AO GENERIUM

Completed

A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers

Metabolic Diseases | Mucopolysaccharidosis Type II

Russian Federation
University of Washington
National Cancer Institute (NCI)

Terminated

IGFBP-2 Vaccine and Combination Chemotherapy in Treating Patients With Stage III-IV Ovarian, Fallopian Tube, or Primary Peritoneal Cancer Undergoing Surgery

Stage IIIA Fallopian Tube Cancer | Stage IIIA Ovarian Cancer | Stage IIIA Primary Peritoneal Cancer | Stage IIIB Fallopian Tube Cancer | Stage IIIB Ovarian Cancer | Stage IIIB Primary Peritoneal Cancer | Stage IIIC Fallopian Tube Cancer | Stage IIIC Ovarian Cancer | Stage IIIC Primary Peritoneal Cancer and other conditions

United States
Sidney Kimmel Cancer Center at Thomas Jefferson...

Completed

Carevive Survivor Care Planning System in Improving Quality of Life in Breast Cancer Survivors

Stage I Breast Cancer | Stage I Uterine Corpus Cancer | Stage II Uterine Corpus Cancer | Stage III Uterine Corpus Cancer | Stage II Breast Cancer | Stage IIIA Breast Cancer | Stage IIIB Breast Cancer | Stage IA Breast Cancer | Stage IB Breast Cancer | Stage IIA Breast Cancer | Stage IIB Breast Cancer | Stage... and other conditions

United States
ArmaGen, Inc

Completed

Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181

Mucopolysaccharidosis I
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC

Completed

Immune Tolerance Study With Aldurazyme® (Laronidase)

Mucopolysaccharidosis I

Brazil, Russian Federation
AO GENERIUM

Recruiting

A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II

Metabolic Diseases | Mucopolysaccharidosis Type II

Russian Federation
Denali Therapeutics Inc.

Enrolling by invitation

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

Mucopolysaccharidosis II

United States, Canada, Belgium, Czechia, Netherlands
JCR Pharmaceuticals Co., Ltd.

Enrolling by invitation

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Subjects

Mucopolysaccharidosis II

United States
REGENXBIO Inc.

Completed

Mucopolysaccharidosis Type II Natural History

Mucopolysaccharidosis II

Brazil, United States, United Kingdom
Shire

Completed

A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Sanfilippo Syndrome Type A

United States
BioMarin Pharmaceutical

Completed

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVA

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

Enrolling by invitation

Long Term Follow-Up for RGX-111

Mucopolysaccharidosis I

United States, Brazil
ArmaGen, Inc

Completed

Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I

Mucopolysaccharidosis I

United States
National MPS Society
MediResource Inc.

Terminated

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

Mucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidoses | Mucopolysaccharidosis VI | Mucopolysaccharidosis IV

United States
BioMarin Pharmaceutical

Terminated

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVA

United States, United Kingdom, Canada, Germany

Clinical Trials on Mucopolysaccharidosis Type IIIA

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