Clinical Trials on Griscellis syndrom

Total 34145 results

Fondazione Italiana Sindromi Mielodisplastiche-ETS
Centro di Riferimento per l'Epidemiologia e la Prev. Oncologica Piemonte

Terminated

Multicentric Study Comparison Between Erythropoietin and Erythropoietin Associated to Differentiating Therapy With Acid 13-cis-retinoic and Dihydroxyvitamin D3 in Myelodysplastic Syndromes Without Excess of Blasts

Myelodysplastic Syndromes

Italy
Basque Health Service

Unknown

Treatment of Myelodysplastic Syndromes Comparing Hydralazine/Ac. Valproic and Supportive Care in Patients Not Candidates, Refractory and/or Intolerant to Intensive Chemotherapy

Myelodysplastic Syndrome

Spain
Cooperative Study Group A for Hematology

Unknown

Reduced-intensity Conditioning Allogeneic Hematopoietic Cell Transplantation (RICandDLI)

Myelodysplastic Syndrome

Korea, Republic of
Amgen

Completed

A Study of Darbepoetin Alfa in Anemic Subjects With Low Risk Myelodysplastic Syndrome

Myelodysplastic Syndromes
Tel-Aviv Sourasky Medical Center

Unknown

Safety and Efficacy of Azacitidine, and Thalidomide in Higher Risk MDS (Myelodysplastic Syndrome) (IMDS001)

Myelodysplastic Syndrome

Israel
Groupe Francophone des Myelodysplasies

Unknown

Lenalidomide in Subjects With intermediate2 or High MDS Associated With a Deletion (DEL) 5q [31]

Myelodysplastic Syndromes

France
Novacea

Unknown

Study of High-Dose Pulse Administration DN-101 (Calcitriol) in Patients With Myelodysplastic Syndrome (MDS)

Myelodysplastic Syndrome

United States
SIFI SpA

Completed

Investigation of Preservative-Free Ophthalmic Solution in Ocular Dryness

Dry Eye Syndromes

Spain
Otsuka Pharmaceutical Co., Ltd.

Completed

Safety Study of OPC-12759 Ophthalmic Solution

Dry Eye Syndromes

Japan
Allergan

Completed

Investigation of Maintaining Visual Performance Achieved With Cyclosporine Therapy (IMPACT)

Dry Eye Syndromes

United States
Sight Sciences, Inc.

Unknown

Pilot Study of TearCare System - Long-Term Extension

Dry Eye Syndromes

United States
Allergan

Completed

Evaluation of Maintaining Visual Performance in Participants Who Engage in Electronic Visual Tasking While Using Restasis® (EMPOWER)

Dry Eye Syndromes

United States
Novaliq GmbH

Completed

NovaTears® Eye Drops Observational Study NT-002

Dry Eye Syndromes

Germany
Meir Medical Center

Unknown

Estrogen and the Outer Eye in Women During Fertility Treatment

Dry Eye Syndromes
Alcon Research

Completed

Systane Clinical Experience Study

Dry Eye 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
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
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
Peking University Third Hospital

Completed

Predictive Factors for Roux Stasis Syndrome (PFRSS)

Gastrostomy

China

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