Clinical Trials on Hurler-Scheie Syndrome

Total 34203 results

Astex Pharmaceuticals, Inc.

Completed

Decitabine Versus Supportive Care in Adults With Advanced-stage MDS

Myelodysplastic Syndrome

United States
University of Michigan Rogel Cancer Center
National Cancer Institute (NCI)

Completed

Azacitidine Plus Amifostine in Treating Patients With Myelodysplastic Syndrome

Myelodysplastic Syndromes

United States
European Organisation for Research and Treatment...

Completed

Infliximab in Treating Patients With Myelodysplastic Syndrome

Myelodysplastic Syndromes

France, Belgium, Netherlands, Czech Republic, Italy, Germany
Fondazione Italiana Sindromi Mielodisplastiche-ETS
Centro di Riferimento per l'Epidemiologia e la Prev. Oncologica Piemonte

Terminated

Study of Association of Arsenic Trioxide (ATO) and Ascorbic Acid in Myelodysplastic Syndromes

Myelodysplastic Syndromes

Italy
Cooperative Study Group A for Hematology

Unknown

Cyclophosphamide, Fludarabine and Antithymocyte Globulin Conditioning in Myelodysplastic Syndrome (MDS) (CyFluATG)

Myelodysplastic Syndrome

Korea, Republic of
University of Utah
National Institutes of Health (NIH)

Completed

The Role of Erythropoietin in Myelodysplastic Syndrome

Myelodysplastic Syndrome

United States
Johnson & Johnson Pharmaceutical Research & Development...

Completed

Study of Tipifarnib in Patients With High-Risk Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndrome
St. Bartholomew's Hospital

Unknown

Erythropoietin (EPO) and Granulocyte-Colony Stimulating Factor (G-CSF) for Low-Risk Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndromes

United Kingdom
PETHEMA Foundation

Completed

Antithymocyte Globulin and Cyclosporine in Treating Low Risk Patients With Myelodysplastic Syndrome

Myelodysplastic Syndrome

Spain
National Heart, Lung, and Blood Institute (NHLBI)

Completed

A Randomized Trial of Recombinant Humanized Anti-IL-2 Receptor Antibody (Daclizumab) Versus Antithymocyte Globulin (ATG) to Treat the Cytopenia of Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndromes

United States
Hadassah Medical Organization

Withdrawn

The Efficacy of Coenzyme Q10 And Curcumin in Patients With Myelodysplastic Syndromes

Myelodysplastic Syndrome

Israel
Jazz Pharmaceuticals
Evidilya S.r.l.

Not yet recruiting

Epidyolex® in Lennox Gastaut and Dravet Syndrome: an Observational Study in ITALY (EpiTALY)

Dravet Syndrome | Lennox Gastaut Syndrome

Italy
Medical University of Silesia

Completed

Comparison the Effects of TECAR With Dry Needling in the Treatment of Myofascial Trigger Points

Myofascial Trigger Point Pain | Myofascial Pain Syndrome of Neck | Myofascial Trigger Point Syndrome

Poland
Eisai Inc.

Terminated

Study of Perampanel as Adjunctive Treatment for Inadequately Controlled Seizures Associated With Lennox-Gastaut Syndrome

Lennox-Gastaut Syndrome (LGS)

Korea, Republic of, United States, Australia, Belgium, Japan, Czechia, India
Gillette Children's Specialty Healthcare
University of Minnesota

Withdrawn

The Pharmacokinetics of Cannabidiol (CBD) and Its Effects in Children With Severe Epilepsy

Dravet Syndrome | Lennox Gastaut Syndrome
Shanghai University of Traditional Chinese Medicine
Shanghai Yueyang Integrated Medicine Hospital

Recruiting

Yan Nian Jiu Zhuan Fa Improve Chronic Fatigue Syndromes (YNJZFCFS)

Chronic Fatigue Syndromes

China
dr. Laura C. G. de Graaff-Herder

Recruiting

GROWing Up With Rare GENEtic Syndromes (GROW UR GENES)

Disorders of Sex Development | Congenital Adrenal Hyperplasia | Tuberous Sclerosis | Kallmann Syndrome | Prader-Willi Syndrome | Neurofibromatosis | Rett Syndrome | 22q11 Deletion Syndrome | Turner Syndrome | Noonan Syndrome | Allan-Herndon-Dudley Syndrome | Saethre-Chotzen Syndrome | Congenital Hypopituitarism | Cornelia... and other conditions

Netherlands
Gillette Children's Specialty Healthcare

Terminated

The Effects of Cannabidiol (CBD) on Electrical and Autonomic Cardiac Function in Children With Severe Epilepsy (CBD1)

Dravet Syndrome | Lennox-Gastaut Syndrome

United States
Jazz Pharmaceuticals

Completed

An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and Adults With Dravet or Lennox-Gastaut Syndromes (GWPCARE5)

Epilepsy | Dravet Syndrome | Lennox-Gastaut Syndrome
Laboratoires Thea

Not yet recruiting

Comparison of the Performance and Safety of T2769 Versus Hylo-Forte® in the Treatment of Moderate to Severe Dry Eye Syndrome.

Dry Eye Syndrome
Lindsey Russell, MD
Napo Pharmaceuticals, Inc.

Not yet recruiting

Open-Label Pilot Study With Crofelemer in Patients With Short Bowel Syndrome

Short Bowel Syndrome

United States
AJU Pharm Co., Ltd.
GL Pharm Tech Corporation

Recruiting

Efficacy and Safety of AJU-S56 5% in Dry Eye Syndrome Patients

Dry Eye Syndromes

Korea, Republic of
Encoded Therapeutics

Recruiting

A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only)

Dravet Syndrome

Australia
Encoded Therapeutics

Recruiting

A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome (ENDEAVOR)

Dravet Syndrome

United States
Takeda

Recruiting

A Study of Teduglutide in Japanese People With Short Bowel Syndrome

Short Bowel Syndrome

Japan
Epygenix
Forest Hills Lab; Liberyx Therapeutics

Recruiting

EPX-100 (Clemizole Hydrochloride) as Add-on Therapy to Control Convulsive Seizures in Patients With Dravet Syndrome (ARGUS)

Dravet Syndrome

United States, Canada, Spain, Georgia, Hungary
Zealand Pharma

Recruiting

Evaluation of Long Term Safety and Efficacy of Glepaglutide in Treatment of SBS (EASE SBS 2)

Short Bowel Syndrome

United States, France, Belgium, Denmark, United Kingdom, Germany, Canada, Italy, Netherlands, Poland
University of Leipzig
Curis, Inc.

Terminated

Treatment of Anemia in Patients With Very Low, Low or Intermediate Risk Myelodysplastic Syndromes With CA-4948 (LUCAS)

Myelodysplastic Syndromes | Anemia

Germany
Takeda

Completed

A Study of Teduglutide in Japanese Children With Short Bowel Syndrome Aged 4 Months or Older

Short Bowel Syndrome

Japan
AJU Pharm Co., Ltd.
GL Pharm Tech Corporation

Completed

Efficacy and Safety of AJU-S56 in Dry Eye Syndrome Patients

Dry Eye Syndromes

Korea, Republic of
The First Affiliated Hospital of University of...
Jinling Hospital, China

Completed

Survival and PN Dependence in SBS Patients

Short Bowel Syndrome
Zogenix, Inc.

Approved for marketing

ZX008 Expanded Access Protocol

Dravet Syndrome
Celgene

Active, not recruiting

A Study to Evaluate the Efficacy, Drug Levels and Safety of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes in Chinese and Japanese Participants With Ring Sideroblasts Who Require Red Blood Cell Transfusions

Myelodysplastic Syndromes

China, Japan
Takeda

Completed

A Study of Teduglutide (Revestive®) in Children, Teenagers and Adults With Short Bowel Disease

Short Bowel Syndrome

Argentina
FibroGen
AstraZeneca

Completed

Efficacy and Safety of FG-4592 for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome (MDS)

Anemia | Myelodysplastic Syndrome

China
Hospices Civils de Lyon

Active, not recruiting

New Biomarkers in Auto-immune Encephalitis and Neurological Paraneoplastic Syndromes (DeNobio)

Autoimmune Encephalitis | Paraneoplastic Neurological Syndrome

France
Zogenix International Limited, Inc., a subsidiary...

Completed

A 2-Part Study to Investigate the Dose-Ranging Safety and Pharmacokinetics, Followed by the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children ≥ 2 Years Old and Young Adults With Dravet Syndrome

Dravet Syndrome

United States, France, United Kingdom, Canada, Germany, Netherlands, Spain
Jazz Pharmaceuticals

Completed

A Dose-ranging Pharmacokinetics and Safety Study of GWP42003-P in Children With Dravet Syndrome (GWPCARE1)

Epilepsy | Dravet Syndrome

United States, United Kingdom
Jazz Pharmaceuticals

Completed

GWPCARE2 A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome

Epilepsy | Dravet Syndrome

United States, Spain, Israel, Poland, Australia, Netherlands
Pharmascience Inc.

Terminated

Efficacy, Safety and Tolerability of Enteric-Coated Cholestyramine Capsules for Adult Short Bowel Syndrome

Short Bowel Syndrome

Poland
SIFI SpA

Terminated

To Demonstrate the Non-inferiority of Eyestil Protection® Compared to Vismed® in Terms of Clinical Performance

Dry Eye Syndrome

Spain, France
Samjin Pharmaceutical Co., Ltd.

Completed

Clinical Study to Investigate the Efficacy and Safety of SJP002 Ophthalmic Solution in Patients With Dry Eye Syndrome.

Dry Eye Syndromes

Korea, Republic of
Shire

Completed

Study in Japanese Pediatric Subjects With Short Bowel Syndrome (SBS) Who Are Dependent on Parenteral Support

Short Bowel Syndrome

Japan
Mitotech, SA
ORA, Inc.

Completed

Study of SkQ1 as Treatment for Dry-eye Syndrome (VISTA-1)

Dry Eye Syndrome

United States
ReGenTree, LLC

Completed

Assessment of the Safety and Efficacy of RGN-259 Ophthalmic Solutions for Dry Eye Syndrome : ARISE-2

Dry Eye Syndrome

United States
BRIM Biotechnology Inc.
ORA, Inc.

Completed

Safety and Efficacy of BRM421 for Dry Eye Syndrome Treatment

Dry Eye Syndrome

United States
Shire

Completed

Long-term Safety and Efficacy Study of Teduglutide in Pediatric Participants With Short Bowel Syndrome (SBS)

Short Bowel Syndrome

United States, Canada, Finland, Italy, United Kingdom, Belgium
Elgan Pharma Ltd.

Terminated

Safety and Efficacy Study in Infant With SBS (GIFT)

Short Bowel Syndrome

United States
Shire

Completed

Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Teduglutide in Japanese Subjects With PN-dependent Short Bowel Syndrome (SBS)

Short Bowel Syndrome

Japan
Peking University Third Hospital

Completed

Predictive Factors for Roux Stasis Syndrome (PFRSS)

Gastrostomy

China

Clinical Trials on Hurler-Scheie Syndrome

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