Clinical Trials on Wiskott-Aldrich Syndrome

Total 34112 results

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
Shire

Completed

A Pharmacokinetic, Safety, and Pharmacodynamic Study of Teduglutide in Pediatric Subjects With Short Bowel Syndrome

Short Bowel Syndrome

United States, United Kingdom
Brighton and Sussex University Hospitals NHS Trust
Medtronic Vascular

Completed

Revascularisation or Medical Therapy in Elderly Patients With Acute Anginal Syndromes (RINCAL)

Acute Coronary Syndromes

United Kingdom
Astex Pharmaceuticals, Inc.

Completed

Pharmacokinetic Guided Dose Escalation and Dose Confirmation With Oral Decitabine and Oral Cytidine Deaminase Inhibitor (CDAi) in Patients With Myelodysplastic Syndromes (MDS)

Myelodysplastic Syndrome | MDS

United States, Canada
Assistance Publique - Hôpitaux de Paris
Imagine Institute

Completed

Characterization of the Long-term Safety, Efficacy, and Pharmacodynamics Revestive® in the Management of Short Bowel Syndrome Pediatric Patients (REVE)

Short Bowel Syndrome

France
GlyPharma Therapeutics
VectivBio AG

Completed

Safety, Efficacy, PD of FE203799 in Short Bowel Syndrome on Parenteral Support

Short Bowel Syndrome

Denmark
Bausch & Lomb Incorporated

Completed

Study to Assess the Safety and Efficacy of BOL-303242-X Ophthalmic Suspension in Dry Eye Syndrome

Dry Eye Syndrome

United States
Huons Co., Ltd.

Completed

Efficacy and Safety of HU007 Eye Drops in Patients With Dry Eye Syndrome

Dry Eye Syndrome

Korea, Republic of
Stanley Dudrick's Memorial Hospital

Terminated

The Use of DPP-4 Inhibitors in Short Bowel Syndrome (DPP-4)

Short Bowel Syndrome

Poland
Boston Children's Hospital
Entrinsic Bioscience Inc.

Terminated

Amino Acid-based Oral Rehydration Solution in Children With Short Bowel Syndrome

Short Bowel Syndrome

United States
Instituto Mexicano del Seguro Social

Completed

Multicenter Study to Develop a Risk Model for Early Major Cardiovascular Events (RENASCA-IMSS)

Acute Coronary Syndromes

Mexico
Le Bonheur Children's Hospital
University of Tennessee Health Science Center

Unknown

Pectin Use in Pediatric Intestinal Rehabilitation (PUPI)

Short Bowel Syndrome

United States
H. Lee Moffitt Cancer Center and Research Institute
Celgene Corporation

Completed

Lenalidomide and Prednisone in Low and Int-1 Myelodysplastic Syndrome (MDS) Non 5q MDS

Myelodysplastic Syndrome | MDS

United States
University of Colorado, Denver

No longer available

Compassionate Use of Stiripentol in Dravet Syndrome

Dravet Syndrome

United States
Medical University of Vienna

Completed

Difference in the Frequency of Use of Lachrymal Substitutes in Patients With Moderate to Severe Dry Eye Syndrome

Dry Eye Syndromes

Austria
Children's Hospital Medical Center, Cincinnati

Approved for marketing

Treatment Plan to Provide Expanded Access to Stiripentol for Patients With Dravet Syndrome

Dravet Syndrome

United States
British Columbia Children's Hospital

Completed

Probiotics in Short Bowel Syndrome

Short Bowel Syndrome
St. Louis University
Epharmix, Inc.

Terminated

Effect of a Short Message Service Intervention on Patients With Short Bowel Syndrome

Short Bowel Syndrome

United States
Dong-A University

Completed

PRAsugrel or clopIdogrel In Acute Coronary SyndromE With CYP2C19 GENEtic Variants (PRAISE-GENE)

Acute Coronary Syndromes

Korea, Republic of
Laboratorios Sophia S.A de C.V.

Completed

Efficacy and Safety of PRO-148 Ophthalmic Solution Versus SYSTANE® in the Treatment of Patients With Dry Eye Syndrome (PRO-148)

Dry Eye Syndrome

Mexico
Mikkael Sekeres MD
National Cancer Institute (NCI)

Completed

Lenalidomide and Azacitidine in Treating Patients With Advanced Myelodysplastic Syndromes

Myelodysplastic Syndromes | Leukemia

United States

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