Clinical Trials on Oculocerebrorenal+Syndrome

Total 33752 results

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

Recruiting

Methylprednisolone, Horse Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, and/or Pegfilgrastim or Pegfilgrastim Biosimilar in Treating Patients With Aplastic Anemia or Low or Intermediate-Risk Myelodysplastic Syndrome

Previously Treated Myelodysplastic Syndrome | Myelodysplastic Syndrome | Aplastic Anemia | de Novo Myelodysplastic Syndrome

United States
Zogenix International Limited, Inc., a subsidiary...

Completed

A Study to Assess the Safety and Tolerability of ZX008 in Children and Young Adults With Dravet Syndrome or Lennox Gastaut Syndrome Currently Taking Cannabidiol

Dravet Syndrome | Lennox Gastaut Syndrome

United States
Yonsei University

Unknown

Research on Cognitive Effect of Cannabidiol on Dravet Syndrome and Lennox-Gastaut SyndromeGastaut Syndrome

Dravet Syndrome | Lennox Gastaut Syndrome

Korea, Republic of
Kars State Hospital

Completed

Online Information on Myofascial Pain Syndrome

Myofascial Pain Syndrome, Diffuse

Turkey
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Withdrawn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
Sohag University
Assiut University; Kyoto University

Recruiting

Mutations and Phenotypes of Unclassifiable Inherited Bone Marrow Failure Syndromes

Inherited BMF Syndrome

Egypt
Franziska Wachter
Harvard Clinical and Translational Science Center (Harvard Catalyst)

Recruiting

A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies (MORE)

Acute Myeloid Leukemia | Myelodysplastic Syndromes | MDS | Aml | Myeloid Neoplasm | Myeloid Malignancies | Inherited Bone Marrow Failure Syndrome

United States
Dana-Farber Cancer Institute
Charles H. Hood Foundation

Recruiting

Protocol For Genomically Profiling, Collecting, Archiving And Distributing Blood And Bone Marrow Specimens From Children And Young Adults With Hematologic Malignancy

Myelodysplastic Syndromes | Leukemia | Myeloproliferative Syndrome

United States
375th Medical Group, Scott Air Force Base

Terminated

Botulinum for Chronic Exertional Compartment Syndrome (Botox)

Chronic Exertional Compartment Syndrome

United States
Catalyst Pharmaceuticals, Inc.

No longer available

Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome (EAP-001)

Lambert-Eaton Myasthenic Syndrome | Congenital Myasthenic Syndrome | Nystagmus, Acquired

United States
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
Walter Reed National Military Medical Center
Uniformed Services University of the Health Sciences

Active, not recruiting

Comparison of Non-Surgical Treatment Options for Chronic Exertional Compartment Syndrome (CECS)

Chronic Exertional Compartment Syndrome

United States
Thomas Jefferson University
Rothman Institute Orthopaedics; Lantheus Medical Imaging

Completed

Diagnosing Compartment Syndrome With SHAPE vs Elastography

Chronic Exertional Compartment Syndrome

United States
Huons Co., Ltd.

Not yet recruiting

To Evaluate the Efficacy and Safety of HUC3-053 in Patients With Dry Eye Syndrome

Dry Eye Syndromes
UCB BIOSCIENCES, Inc.

Recruiting

A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome (ORCHID)

Dravet Syndrome

United States
Bristol-Myers Squibb

Recruiting

ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions (ELEMENT-MDS)

Myelodysplastic Syndromes

Canada, United States, China, Australia, France, Greece, Hungary, Germany, Italy, Czechia, Poland, Spain, Brazil, Colombia, India, Mexico, Argentina, Puerto Rico
University Hospital, Bordeaux

Not yet recruiting

Ferroptosis Study in SF3B1-mutant Myelodysplastic Syndromes (FerMDS) (FerMDS)

Myelodysplastic Syndromes | Ferroptosis

France
Cook Children's Health Care System
Encoded Therapeutics

Recruiting

GABA Biomarkers in Dravet Syndrome

Dravet Syndrome

United States
Hospices Civils de Lyon

Recruiting

Neurodevelopmental Impact of Epilepsy on Autonomic Function in Dravet Syndrome (AUTONOMIC)

Epilepsy | Dravet Syndrome

Belgium, France, Germany
University of Leipzig
Novartis Pharmaceuticals

Recruiting

Efficacy and Safety of Canakinumab for the Treatment of Anemia in LR-MDS Patients

Myelodysplastic Syndromes | Anemia

Germany
Hanmi Pharmaceutical Company Limited

Recruiting

Phase 2 Study to Assess the Safety, PK, and PD in SBS-IF Subjects

Short Bowel Syndrome

Belgium, Korea, Republic of, United States, Germany, Denmark, France, Poland
University of Aberdeen
NHS Grampian; Softcell Medical Ltd

Not yet recruiting

Real-time Soft Tissue Assessment Using a Technologically Advanced pH Monitoring System (SoftpH)

Acute Compartment Syndrome

United Kingdom
Fondation Ophtalmologique Adolphe de Rothschild

Recruiting

Three-dimensional Analysis of EMMPRIN on Conjunctival Epithelial Cells Surface in Severe Dry Eye Syndrome (ALTESSE) (ALTESSE)

Dry Eye Syndromes | Conjunctival Diseases

France
Agios Pharmaceuticals, Inc.

Active, not recruiting

A Study of AG-946 in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes (LR-MDS)

Myelodysplastic Syndromes

Israel, United States, Korea, Republic of, Australia, France, Germany, Spain, United Kingdom, Czechia, Austria, Italy, Greece, Poland
Alexandria University

Completed

Effect of an Educational Program on Health-related Outcomes of Patients With Dry Eye Syndrome

Dry Eye Syndrome

Egypt
University of Cincinnati
National Heart, Lung, and Blood Institute (NHLBI); Rare Diseases Clinical Research...

Completed

Assessment of Safety of Air Travel in Patients With Birt-Hogg-Dube Syndrome

Birt-Hogg-Dube Syndrome

United States
VectivBio AG

Active, not recruiting

Open-label Extension Trial to Evaluate the Long-term Safety of Apraglutide in Short Bowel Syndrome. (STARS extend)

Short Bowel Syndrome

United States, Belgium, United Kingdom, Korea, Republic of, Japan, Taiwan, Germany, France, Czechia, Hungary, Spain, Italy, Poland, Argentina, Denmark, Israel, Norway, Sweden
Celgene
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway...

Active, not recruiting

A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve (COMMANDS)

Myelodysplastic Syndromes

United States, Australia, Austria, Belgium, Canada, Czechia, France, Germany, Greece, Hungary, Israel, Italy, Japan, Korea, Republic of, Lithuania, Netherlands, Poland, Portugal, Russian Federation, Spain, Sweden, Switzerland, Taiwan, Turke... and more
Stoke Therapeutics, Inc

Enrolling by invitation

An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome

Dravet Syndrome

United States
Zealand Pharma

Completed

The Long-term Effect on Intestinal Absorption and Safety of Treatment With Glepaglutide in Patients With Short Bowel Syndrome (EASE SBS 4)

Short Bowel Syndrome

Denmark
University of Nebraska
Eli Lilly and Company

Terminated

Evaluate the Efficacy and Safety Growth Hormone, Glutamine and Diet in Patients With Short Bowel Syndrome (SBS)

Short Bowel Syndrome

United States
Shire
Takeda Development Center Americas, Inc.

Active, not recruiting

Registry for Participants With Short Bowel Syndrome

Short Bowel Syndrome

United States
Celgene

Completed

An Efficacy and Safety Study of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in Japanese Subjects Who Are Not Requiring Red Blood Cell Transfusion

Myelodysplastic Syndromes

Japan
Zealand Pharma

Completed

Efficacy And Safety Evaluation of Glepaglutide in Treatment of Short Bowel Syndrome (SBS) (EASE SBS 1)

Short Bowel Syndrome

United States, Denmark, Belgium, Germany, France, United Kingdom, Canada, Netherlands, Poland
Fondation Ophtalmologique Adolphe de Rothschild

Completed

Neuronal Excitability of HCN1 Channel Mutations in Dravet Syndrome (EXCIDRAH)

Dravet Syndrome

France
Jazz Pharmaceuticals

Completed

Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1)

Epilepsy | Dravet Syndrome

France, United States, United Kingdom, Poland
Samjin Pharmaceutical Co., Ltd.

Completed

Clinical Study to Investigate the Efficacy and Safety of Orally Administered SA001 in Patients With Dry Eye Syndrome.

Dry Eye Syndrome

Korea, Republic of
Laboratoires Thea

Completed

Efficacy, Safety of T2769 in Dry Eye Disease

Dry Eye Syndrome

Tunisia
Mitotech, SA
ORA, Inc.

Completed

Vehicle-Controlled Study of SkQ1 as Treatment for Dry-eye Syndrome (VISTA-2)

Dry Eye Syndrome

United States
La Fundacion Favaloro para la Investigacion y la...
Fundación de Ciencias Exactas - UNLP

Enrolling by invitation

ImpRovE underSTanding of Short bOwel syndRome in Latin-amErica (RESTORE)

Short Bowel Syndrome

Argentina
Shire
Nycomed Germany GmbH

Completed

Open-Label Study of Teduglutide for Subjects With PN-Dependent Short Bowel Syndrome (SBS)

Short Bowel Syndrome

Spain, United States, Canada, France, Denmark, Poland, Germany, Italy, United Kingdom
Shire

Completed

Safety and Efficacy Study of Teduglutide in Subjects With Short Bowel Syndrome

Short Bowel Syndrome

United States, France, Germany, Canada, Netherlands, Poland, Denmark, Belgium, United Kingdom
Shire

Completed

Safety and Efficacy Study of Teduglutide in Subjects With Short Bowel Syndrome Who Completed Protocol CL0600-004 (NCT00081458)

Short Bowel Syndrome

United States, Denmark, France, Canada, Belgium, Netherlands, Poland, United Kingdom
Peking University Third Hospital

Unknown

CDSS of Traditional Chinese Medicine Intervention for Dry Eye Syndrome

Dry Eye Syndromes

China
Heart Care Foundation
AstraZeneca; Centro Servizi ANMCO S.r.l.

Completed

EYESHOT (EmploYEd Antithrombotic Therapies in Patients With Acute Coronary Syndromes HOspitalized in iTalian Coronary Care Units) (EYESHOT)

Acute Coronary Syndromes

Italy

Clinical Trials on Oculocerebrorenal+Syndrome

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Costa Rica

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

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