Clinical Trials on Hurler-Scheie Syndrome

Total 34284 results

Allergan

Completed

Safety and Efficacy Study of Cyclosporine Ophthalmic Emulsion in Post-LASIK Patients

Dry Eye Syndromes

United States
Allergan

Completed

Efficacy and Acceptability of Two Lubricant Eye Drops

Dry Eye Syndromes

United States
Pharmaceutical Research Network

Completed

Soothe Versus Refresh

Dry Eye Syndromes

United States
Jazz Pharmaceuticals

Active, not recruiting

Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution

Tuberous Sclerosis Complex | Dravet Syndrome | Lennox Gastaut Syndrome

United States
Minovia Therapeutics Ltd.

Recruiting

A Study to Evaluate the MNV-201 in Patients With Low Risk MDS

Myelodysplastic Syndromes

Israel
Wuhan Union Hospital, China

Not yet recruiting

A Novel Conditioning Regimen for Myelodysplastic Syndrome With Moderate High IPSS-M Score

Myelodysplastic Syndromes
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

JAK Inhibitor in Acquired Hemophagocytic synDrome in the Intensive Care Unit (JAKAHDI)

Hemophagocytic Syndromes
Otsuka Beijing Research Institute

Recruiting

PK/Efficacy Bridging Study of ASTX727 in Chinese Subjects With Myelodysplastic Syndromes

Myelodysplastic Syndromes

China
Bristol-Myers Squibb

Recruiting

A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants (MAXILUS)

Myelodysplastic Syndromes

Spain, United States, France, Italy, Belgium, Germany, Poland, Czechia, Puerto Rico
Peking Union Medical College Hospital

Not yet recruiting

Roxadustat Combined With Luspatercept Versus Luspatercept Monotherapy in the Treatment of Refractory MDS-RS

Myelodysplastic Syndromes

China
University Hospital, Grenoble

Recruiting

Flow Cytometric Analysis of Peripheral Blood Neutrophil Myeloperoxidase Expression for Ruling Out Myelodysplastic Syndromes: Protocol for a Diagnostic Accuracy Study (MPO-MDS-Valid)

Myelodysplastic Syndromes

France
Novartis Pharmaceuticals

Recruiting

Study of Efficacy and Safety of Eltrombopag in Lower-risk MDS Patients With Platelet Transfusion Dependence

Myelodysplastic Syndromes

Japan
H. Lee Moffitt Cancer Center and Research Institute
Novartis Pharmaceuticals

Recruiting

Canakinumab With Darbepoetin Alfa in PTs With Lower-Risk MDS Who Have Failed ESA

Myelodysplastic Syndromes

United States
Novartis Pharmaceuticals

Terminated

Phase Ib Study of Select Drug Combinations in Patients With Lower Risk MDS

Myelodysplastic Syndromes

United States, Spain, Italy, Singapore, Korea, Republic of, Israel, Australia
Bristol-Myers Squibb

Active, not recruiting

A Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care (BSC) Versus Placebo Plus BSC in Participants With International Prognostic Scoring System Revised (IPSS-R) Low- or Intermediate-risk Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndromes

Argentina, Canada, France, Germany, Italy, Korea, Republic of, Poland, United States, Australia, China, Czechia, Denmark, Greece, Hong Kong, Japan, Spain, Sweden
University Hospital, Grenoble

Active, not recruiting

Development and Prospective Validation of a Standardized Flow Cytometric Assay of Peripheral Blood Neutrophil Myeloperoxidase Expression for Ruling Out Myelodysplastic Syndromes. (MPO-MDS-Develp)

Myelodysplastic Syndromes

France
Asan Medical Center

Active, not recruiting

CyFluATG in Lower Risk MDS

Myelodysplastic Syndromes

Korea, Republic of
Asan Medical Center

Completed

Hematopoietic Cell Transplantation With Post-transplantation Cyclophosphamide in MDS

Myelodysplastic Syndromes

Korea, Republic of
Thomas Jefferson University

Withdrawn

Physical Activity in Transfusion Dependent Patients With Myelodysplastic Syndrome

Myelodysplastic Syndromes

United States
Sidney Kimmel Comprehensive Cancer Center at Johns...
National Cancer Institute (NCI); Alliance for Cancer Gene Therapy

Terminated

Vaccine Therapy in Treating Patients With Myelodysplastic Syndromes

Myelodysplastic Syndromes

United States
Janssen Research & Development, LLC

Completed

Study to Separately Evaluate the Activity of Talacotuzumab (JNJ-56022473) or Daratumumab in Transfusion-Dependent Participants With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) Who Are Relapsed or Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment

Myelodysplastic Syndromes

United States, Belgium, Italy, Spain, Netherlands, Russian Federation
SymBio Pharmaceuticals

Completed

Safety and Pharmacokinetics Study of SyB L-1101 in Patients With Recurrent/Relapsed or Refractory Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndrome

Japan
SymBio Pharmaceuticals

Completed

Safety and Pharmacokinetics Study of SyB C-1101 in Patients With Recurrent/Relapsed or Refractory Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndrome

Japan
SymBio Pharmaceuticals

Withdrawn

Combination Therapy of SyB C-1101 and Azacytidine in Patients With Myelodysplastic Syndrome

Myelodysplastic Syndrome

Japan
The University of Hong Kong

Active, not recruiting

Enhanced Palliative Care in MDS and AML

Myelodysplastic Syndromes

Hong Kong
Groupe Francophone des Myelodysplasies

Completed

CPX-351 in Higher Risk Myelodysplastic Syndromes

Myelodysplastic Syndromes

France
Helsinn Healthcare SA

Terminated

A Safety and Efficacy Study of Pracinostat and Azacitidine in Patients With High Risk Myelodysplastic Syndromes

Myelodysplastic Syndromes

United States
Acceleron Pharma Inc. (a wholly owned subsidiary...

Completed

ACE-536 Extension Study - Myelodysplastic Syndromes

Myelodysplastic Syndromes

Germany
Grupo Cooperativo de Estudio y Tratamiento de las...
Celgene Corporation; Germans Trias i Pujol Hospital; Hospital Son Llatzer

Unknown

Treatment of High Risk Myelodysplastic Syndromes (MDS) Not Candidates for Allogeneic Transplantation of Hematopoietic Progenitors (ALO-HSCT)

Myelodysplastic Syndromes

Spain
Samsung Medical Center

Completed

Clinical Relevance of DNMT and HDAC Gene SNP on the Response to Decitabine Therapy for Myelodysplastic Syndrome

Myelodysplastic Syndrome

Korea, Republic of
University Hospital, Toulouse
National Cancer Institute, France

Completed

PREDICTive FactOR of Overall Survival Among Geriatric Assessment Tools and Disease Related Factors in Elderly Patients With High Risk Myelodysplastic Syndromes. (PREDICTOR)

Myelodysplastic Syndromes

France, Monaco
Hoffmann-La Roche

Completed

A Study of Epoetin Beta Treatment in Anemic Participants With Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndromes

Thailand
Celgene

Terminated

A Phase I, Open-Label, Dose-Escalation Study of CC-11006 In Subjects With Low- or Intermediate-1 Risk Myelodysplastic Syndromes

Myelodysplastic Syndrome

United States
Celgene

Completed

MORE - Monitoring Revlimid - Collecting of Patient Information From Myelodysplastic Syndrome (MDS) Italian National Registry (MORE)

Myelodysplastic Syndrome

Italy
Celgene

Completed

An Extension to Study AZA PH GL 2003 CL 001 Allowing for Continuation of Azacitidine Treatment in Patients With Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes

Italy, United Kingdom, Germany, Australia, Bulgaria, France, Greece, Hungary, Netherlands, Poland, Spain
Celgene

Completed

A Phase 1 Study to Evaluate the Pharmacokinetics and Tolerability of Oral Azacitidine in Japanese Patients With Myelodysplastic Syndromes

Myelodysplastic Syndromes

Japan
Celgene

Completed

Study of the Safety and Efficacy of CC-5013 Treatment For Patients With Myelodysplastic Syndrome

Myelodysplastic Syndrome

United States
Hoffmann-La Roche

Completed

A Study of Atezolizumab Administered Alone or in Combination With Azacitidine in Participants With Myelodysplastic Syndromes

Myelodysplastic Syndromes

United States
University of Florida
Gateway for Cancer Research; Cellworks Group Inc.

Withdrawn

iCare 2: Personalized Genomic Mutation Informed Treatment of Patients With Myelodysplastic Syndromes

Myelodysplastic Syndromes

United States
Masonic Cancer Center, University of Minnesota
Mayo Clinic

Completed

Decitabine and Vorinostat Conditioning Followed by CD3-/CD19- NK Cells Infusion for High Risk Myelodysplastic Syndromes

Myelodysplastic Syndrome

United States
Lixte Biotechnology Holdings, Inc.

Unknown

A Study of LB-100 in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes

United States
Weill Medical College of Cornell University
Sunesis Pharmaceuticals

Completed

Vosaroxin for Intermediate 2 or High-risk MDS After Failure With Hypomethylating Agent-based Therapy

Myelodysplastic Syndrome

United States
Seagen Inc.

Terminated

Study of Vadastuximab Talirine (SGN-CD33A; 33A) in Combination With Azacitidine in Patients With Previously Untreated Higher Risk MDS

Myelodysplastic Syndrome

United States
Associazione Qol-one

Terminated

Efficacy of Eltrombopag Plus Lenalidomide Combination Therapy in Patients With IPSS Low and Intermediate-risk Myelodysplastic Syndrome With Isolated del5q

Myelodysplastic Syndromes

France, Italy, Greece
Helsinn Healthcare SA

Completed

Phase 2 Study of Pracinostat With Azacitidine in Patients With Previously Untreated Myelodysplastic Syndrome

Myelodysplastic Syndrome

United States
Novartis Pharmaceuticals

Terminated

A Phase II Pilot Study to Assess the Presence of Molecular Factors Predictive for Hematologic Response in Myelodysplastic Syndrome Patients Receiving Deferasirox Therapy. (EXPHAR)

Myelodysplastic Syndrome

Belgium
Boehringer Ingelheim

Terminated

Trial of Volasertib With or Without Azacitidine in Patients With Myelodysplastic Syndromes

Myelodysplastic Syndromes

Japan
Association pour la recherche sur les Affections...

Completed

Biological Predictive Factors of Response to ESA in Low Risk MDS Patients

Myelodysplastic Syndromes
Larry Cripe, MD
Celgene Corporation; Ortho Biotech Clinical Affairs, L.L.C.; Walther Cancer Institute

Terminated

Azacitidine and Erythropoietin Versus Azacitidine Alone for Patients With Low-Risk Myelodysplastic Syndromes

Myelodysplastic Syndromes

United States
Mirati Therapeutics Inc.

Completed

Safety Study of Mocetinostat in Combination With Azacitidine in Subjects With MDS

Myelodysplastic Syndrome

United States

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