Clinical Trials on Joubert Syndrome

Total 33973 results

Hadassah Medical Organization

Withdrawn

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

Myelodysplastic Syndrome

Israel
Jazz Pharmaceuticals

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
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Withdrawn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
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
Sohag University
Assiut University; Kyoto University

Recruiting

Mutations and Phenotypes of Unclassifiable Inherited Bone Marrow Failure Syndromes

Inherited BMF Syndrome

Egypt
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
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
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
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
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

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