Clinical Trials on Hermansky-Pudlak-Syndrom (HPS)

Total 80 results

National Human Genome Research Institute (NHGRI)

Recruiting

Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome

Hermansky-Pudlak Syndrome (HPS)

United States
National Human Genome Research Institute (NHGRI)

Recruiting

Analysis of Specimens From Individuals With Pulmonary Fibrosis

Pulmonary Fibrosis | Healthy Volunteers | Hermansky-Pudlak Syndrome (HPS)

United States
National Human Genome Research Institute (NHGRI)

Terminated

Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome

Pulmonary Fibrosis | Metabolic Disease | Oculocutaneous Albinism | Hermansky-Pudlak Syndrome (HPS) | Platelet Storage Pool Deficiency

United States
Vanderbilt University
National Heart, Lung, and Blood Institute (NHLBI); University of South Florida and other collaborators

Completed

A Longitudinal Study of Hermansky-Pudlak Syndrome Pulmonary Fibrosis

Hermansky Pudlak Syndrome

United States
Jesse Roman
Genentech, Inc.

Unknown

Efficacy and Safety of Pirfenidone Treatment in HPS-ILD (PEARL)

Interstitial Lung Disease | Hermansky Pudlak Syndrome

United States, Puerto Rico
National Human Genome Research Institute (NHGRI)

Completed

Clinical and Pathophysiological Investigations Into Erdheim Chester Disease

Cancer | Myelofibrosis | Pulmonary Fibrosis | Gaucher Disease | Hermansky-Pudlak Syndrome (HPS)

United States
William Gahl, M.D.
National Human Genome Research Institute (NHGRI)

Completed

Oral Pirfenidone for the Pulmonary Fibrosis of Hermansky-Pudlak Syndrome

Pulmonary Fibrosis | Inborn Errors of Metabolism | Albinism | Oculocutaneous Albinism | Platelet Storage Pool Deficiency

United States
National Institute of Allergy and Infectious Diseases...

Withdrawn

Medical Treatment of Colitis in Patients With Hermansky-Pudlak Syndrome

Colitis | Cytokines | Hermanski-Pudlak Syndrome | Lymphocytes | Drug Evaluation

United States
University of Michigan Rogel Cancer Center

Completed

A Pilot Study of Ruxolitinib in Secondary Hemophagocytic Syndrome

Hemophagocytic Syndrome (HPS)

United States
Mayo Clinic

Completed

Midodrine in Hepatopulmonary Syndrome

Hepatopulmonary Syndrome (HPS)

United States
Sun Jie

Recruiting

Analysis of Clinical Features of Chronic Liver Disease Complicated With Hepatopulmonary Syndrome

Hepatopulmonary Syndrome (HPS)

China
Ruijin Hospital

Recruiting

Effect of Telitacicept on Antibody Titers in Primary APS Patients

Antiphospholipid Syndrome (APS)

China
University Hospital, Rouen

Unknown

Study of the Efficiency of Hydroxychloroquine on the Endothelial Dysfunction and Its Vascular Consequences During the Antiphospholipid Syndrome (APLAQUINE)

Antiphospholipid Syndrome (APS)

France
Yale University
National Institute of Allergy and Infectious Diseases (NIAID); Hospital for...

Active, not recruiting

Pilot Study of Gut Commensals in Antiphospholipid Syndrome

Antiphospholipid Syndrome (APS)

United States
Novartis Pharmaceuticals

No longer available

MAP to Provide Access to Nilotinib, for Patients With HES

Hypereosinophilic Syndrome (HES)
Dilşad Sindel

Completed

Investigation of the Effectiveness of Muscle Inhibition and Space Correction Techniques of Kinesiotaping Method in Female Patients With Myofascial Pain Syndrome Related to Upper Trapezius Active Trigger Points

Female Patients With Myofascial Pain Syndrome (MPS) Related to Upper Trapezius Active Trigger Points (TP)
AstraZeneca

Recruiting

TrinetX Study of Hypereosinophilic Syndrome (HES) Without an Identifiable Non-haematological Secondary Cause

Hypereosinophilic Syndrome (HES)

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
Masonic Cancer Center, University of Minnesota

Withdrawn

MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT

Mucopolysaccharidosis Type IH | Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome) | MPS IH, Hurler Syndrome
Greenwood Genetic Center
Shriners Hospitals for Children; BioMarin Pharmaceutical

Completed

Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome

MPS IVA | Maroteaux Lamy Syndrome | MPS VI | Morquio Syndrome A

United States
Assistance Publique - Hôpitaux de Paris

Recruiting

Register of Patients With Anti-Phospholipids Syndrome (APS) and/or Systemic Lupus Erythematosus (SLE) (SAPL-LUPUS)

Anti-Phospholipids Syndrome (APS) | Systemic Lupus Erythematosus (SLE)

France
Postgraduate Institute of Medical Education and...

Completed

Terlipressin Infusion Alone Vs Terlipressin With Noradrenaline Infusion In The Treatment of Hepatorenal Syndrome Type 1

Type 1 HEPATO RENAL SYNDROME(HRS)

India
Swedish Orphan Biovitrum

Completed

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

Sanfilippo Syndrome Type A (MPS IIIA)

United States, Turkey
Swedish Orphan Biovitrum

Completed

A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

Sanfilippo Syndrome Type A (MPS IIIA)

United States, Germany, Turkey
Ultragenyx Pharmaceutical Inc
PRA Health Sciences

Recruiting

Mucopolysaccharidosis VII Disease Monitoring Program

Sly Syndrome | Mucopolysaccharidosis VII | MPS VII | MPS 7

United States, Germany, Brazil, Argentina, France, Netherlands, Portugal, Spain, Turkey
Ultragenyx Pharmaceutical Inc

Completed

A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Mucopolysaccharidosis | Sly Syndrome | MPS VII | MPS 7

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
Calliditas Therapeutics AB

Completed

Efficacy and Safety of Xepol (Human Immunoglobulin) in Subjects With Post-Polio Syndrome (PPS)

Post Polio Syndrome, PPS

Sweden
Allergan

Recruiting

Study of BOTOX Injections to Assess Change in Disease Activity and Adverse Events in Adult Female Participants With Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS)

Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS)

United States, Canada
REGENXBIO Inc.

Active, not recruiting

RGX-111 Gene Therapy in Patients With MPS I

Hurler Syndrome | Hurler-Scheie Syndrome | Mucopolysaccharidosis Type I (MPS I)

United States, Brazil, Israel
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
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
Ultragenyx Pharmaceutical Inc

Completed

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Mucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VII

United States, Brazil, Mexico, Portugal
Tanta University

Recruiting

Possible Protective Effect of Celecoxib Against Capecitabine Induced Hand and Foot Syndrome in Patients With Colorectal Cancer (HFS)

Colorectal Cancer | Hand and Foot Syndrome | Erythrodysesthesia Syndrome | HFS

Egypt
Ultragenyx Pharmaceutical Inc

Completed

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

Mucopolysaccharidosis | Sly Syndrome | Mucopolysaccharidosis VII | MPS VII

Spain, United States, Portugal
University of California, San Francisco
National Institutes of Health (NIH); Fogarty International Center of the National...

Completed

Thermocoagulation for Treatment of Precancerous Cervical Lesions

HPV Infection | Human Immunodeficiency Virus | CIN 2/3

Kenya
Masonic Cancer Center, University of Minnesota

Terminated

Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome

Hurler Syndrome | Mucopolysaccharidosis Type IH | MPS I

United States
BioMarin Pharmaceutical

Terminated

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

Morquio A Syndrome | MPS IV A | Mucopolysaccharidosis IVA

France, United Kingdom, Taiwan, United States, Argentina, Netherlands, Canada, Brazil, Germany, Italy
Postgraduate Institute of Medical Education and...

Completed

TYPE 2 HEPATORENAL SYNDROME (Type2 HRS)

Safety and Efficacy of Terlipressin and Noradrenaline and Predictive Factors of Response in Type 2 HRS

India
Alexion Pharmaceuticals

Completed

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

MPS IIIB (Sanfilippo Syndrome)

Spain, Netherlands, United Kingdom, United States, Brazil
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
University of Alabama at Birmingham

Completed

The Role of Endothelium Dysfunction in Progression of CKD (Chronic Kidney Disease) After AKI (Acute Kidney Injury)

Acute Renal Failure | Endothelial Dysfunction | Chronic Kidney Failure | Hemolytic-uremic Syndrome (HUS)

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
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
University of British Columbia

Recruiting

A Study of Reduced Dosing of the Nonavalent HPV Vaccine in Women Living With HIV (NOVA-HIV)

Human Immunodeficiency Virus | HPV | Human Papilloma Virus

Canada
Talaris Therapeutics Inc.
Duke University

Terminated

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Niemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...

United States
BioMarin Pharmaceutical

Terminated

Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IVA

United Kingdom, United States, Germany
BioMarin Pharmaceutical

Completed

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

Morquio A Syndrome | MPS IVA | Mucopolysaccharidosis IV A

United States, Canada, France, Taiwan, Argentina, Colombia, Spain, Turkey, Japan, Saudi Arabia, Netherlands, Denmark, Korea, Republic of, Brazil, United Kingdom, Germany, Norway, Portugal, Italy, Qatar
Merck Sharp & Dohme LLC

Active, not recruiting

A Study of the Safety and Tolerability in Participants With PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated With Miransertib (MK-7075) in Other Studies (MK-7075-006)

PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)

United States, Australia, Brazil, Italy, United Kingdom
Montefiore Medical Center
National Cancer Institute (NCI); University of Rwanda; Rwanda Military Hospital

Recruiting

HPV Vaccine Effectiveness Study in Rwandan Women Living With HIV

Cervical Cancer | Human Immunodeficiency Virus | HPV-Related Carcinoma | Human Papilloma Virus

Rwanda

Clinical Trials on Hermansky-Pudlak-Syndrom (HPS)

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