Clinical Trials on FXS

Total 32 results

Sirnaomics

Recruiting

Single Ascending Dose of FXI-GalNAc-siRNA in Healthy Subjects

FXI

United States, Canada
Hospital de Base

Not yet recruiting

Diagnostic Accuracy Study of a Point-of-Care Test for Coagulation Function

CoaguChek® XS System | Coagulation Function
Novartis Pharmaceuticals

Completed

A Pilot Study to Assess the Efficacy and Safety of LCQ908 Alone and in Combination With Fenofibrate or Lovaza® in Patients With Severe Hypertriglyceridemia

Non Familial Chylocmicronemia Syndrome (Non-FCS)

United States, Canada, Russian Federation
Centre Georges Francois Leclerc

Recruiting

Evaluation of the Unconformity of the Hormonal Status of Metastatic Lesions During a First Relapse of Breast Cancer Initially Expressing Estrogen Receptors: a Pilot Study on the Interest of PET With FES (EstroTEPCompar)

FES TEP in Detection of Estrogen Receptors

France
RTI International
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Completed

Decisional Capacity and Informed Consent in Fragile X Syndrome

Fragile X Syndrome (FXS)

United States
Taipei Veterans General Hospital, Taiwan
InSightec

Recruiting

Focused Ultrasound for Drug-resistant Epilepsy

Drug Resistant Epilepsy | Focused Ultrasound | Drug Refractory Epilepsy | Medication Resistant Epilepsy | MR-guided FUS

Taiwan
Ovid Therapeutics Inc.

Completed

A Study of OV101 in Individuals With Fragile X Syndrome (ROCKET)

Fragile X Syndrome (FXS)

United States
Guido A. Davidzon, MD, SM

Withdrawn

Novel Clinical Target in Fragile X Syndrome

Fragile X Syndrome (FXS)

United States
Tetra Discovery Partners

Completed

A 2-Period Crossover Study of BPN14770 in Adults Males With Fragile X Syndrome

Fragile X Syndrome | Fra(X) Syndrome | FXS

United States
University of Michigan
National Institute of Nursing Research (NINR)

Completed

Light Treatment to Improve Symptom Management of Fibromyalgia Syndrome

Fibromyalgia | FMS

United States
University Hospital, Tours

Completed

Glutaminergic Transmission in Autism : Molecular Imaging Exploration (TANGAU)

Healthy Volunteers | Autism | Fragile-X Syndrome (FXS)

France
Astellas Pharma Korea, Inc.

Recruiting

A Study to Assess the Safety of Xospata in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FMS-Like Tyrosine Kinase 3 (FLT3) Mutation

Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation

Korea, Republic of
Astellas Pharma Global Development, Inc.

Completed

A Study of ASP2215 (Gilteritinib) Combined With Atezolizumab in Patients With Relapsed or Treatment Refractory FMS-like Tyrosine Kinase (FLT3) Mutated Acute Myeloid Leukemia (AML)

Acute Myeloid Leukemia (AML) | Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation

United States
University of Alberta
St. Justine's Hospital

Recruiting

A Trial of Metformin in Individuals With Fragile X Syndrome (Met)

Neurobehavioral Manifestations | Genetic Diseases, X-Linked | Mental Retardation, X-Linked | Intellectual Disability | Fragile X Syndrome | Sex Chromosome Disorders | Fragile X Mental Retardation Syndrome | Trinucleotide Repeat Expansion | Fra(X) Syndrome | FXS

Canada
Stanford University

Terminated

Neuroimaging GABA Physiology in Fragile X Syndrome

Fragile X Syndrome (FXS) | Idiopathic Intellectual Developmental Disorder (IDD)

United States
Kasr El Aini Hospital

Not yet recruiting

Contribution of SA and Coma to The Total Ocular HOA Before and After FS-LASIK Using An Aspheric Ablation Profile

FS-LASIK Surgery Effect on Higher Order Aberrations

Egypt
Astellas Pharma Inc

Active, not recruiting

A Study of ASP2215 Versus Salvage Chemotherapy In Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) With FLT3 Mutation

Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation

China, Malaysia, Russian Federation, Singapore, Thailand
University of California, Davis
University of Alberta; St. Justine's Hospital

Recruiting

A Trial of Metformin in Individuals With Fragile X Syndrome (Met)

Neurobehavioral Manifestations | Genetic Diseases, X-Linked | Intellectual Disability | Fragile X Syndrome | Sex Chromosome Disorders | Fragile X Mental Retardation Syndrome | Trinucleotide Repeat Expansion | Fra(X) Syndrome | FXS | Mental Retardation, X Linked

United States, Canada
Astellas Pharma Global Development, Inc.

Active, not recruiting

A Study of ASP2215 (Gilteritinib) by Itself, ASP2215 Combined With Azacitidine or Azacitidine by Itself to Treat Adult Patients Who Have Recently Been Diagnosed With Acute Myeloid Leukemia With a FLT3 Gene Mutation and Who Cannot Receive Standard Chemotherapy

Acute Myeloid Leukemia (AML) | Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) Mutation

United States, Japan, Taiwan, Spain, Australia, Belgium, Canada, France, Germany, Italy, Korea, Republic of, Poland, United Kingdom
Astellas Pharma Global Development, Inc.

Completed

A Study of ASP2215 (Gilteritinib), Administered as Maintenance Therapy Following Induction/Consolidation Therapy for Subjects With FMS-like Tyrosine Kinase 3 (FLT3/ITD) Acute Myeloid Leukemia (AML) in First Complete Remission

Acute Myeloid Leukemia (AML) | Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) / Internal Tandem Duplication (ITD) Mutation

Taiwan, Poland, Serbia, United States, Brazil, Canada, Czechia, Denmark, France, Germany, Greece, Hungary, Israel, Italy, Japan, Korea, Republic of, Portugal, Romania, Spain, Sweden, United Kingdom
Astellas Pharma Global Development, Inc.

No longer available

Early Access Program (EAP) of Gilteritinib (ASP2215) in Patients With FMS-like Tyrosine Kinase 3 (FLT3) Mutated Relapsed or Refractory Acute Myeloid Leukemia (AML) or With FLT3-Mutated AML in Complete Remission (CR) With Minimal Residual Disease (MRD)

Acute Myeloid Leukemia (AML) | FMS-like Tyrosine Kinase-3 (FLT3) Mutations

Australia, Italy, United Kingdom
Astellas Pharma Global Development, Inc.

Recruiting

A Study of Gilteritinib (ASP2215) Combined With Chemotherapy in Children, Adolescents and Young Adults With FMS-like Tyrosine Kinase 3 (FLT3)/Internal Tandem Duplication (ITD) Positive Relapsed or Refractory Acute Myeloid Leukemia (AML)

Acute Myeloid Leukemia (AML) | Acute Myeloid Leukemia With FMS-like Tyrosine Kinase 3 (FLT3) Mutation / Internal Tandem Duplication (ITD)

United States, Canada, Germany, Italy, Spain, United Kingdom, France
Novartis Pharmaceuticals

Completed

A Randomized, Double-blind, Placebo Controlled Study to Assess Efficacy, Safety and Tolerability of LCQ908 in Subjects With Familial Chylomicronemia Syndrome

Familial Chylomicronemia Syndrome (FCS)

South Africa, Spain, France, Germany, United Kingdom, United States, Canada, Netherlands
Parc de Salut Mar
Fundación Mutua Madrileña

Completed

Epigallocatechin Gallate (EGCG) to Improve Cognitive Performance in Foetal Alcohol Syndrome (FAS) Children (Neuro-SAF)

Fetal Alcohol Syndrome (FAS)

Spain
Novartis Pharmaceuticals

Terminated

Extension to a Randomized, Double-blind, Placebo Controlled Study of LCQ908 in Subjects With Familial Chylomicronemia Syndrome.

Familial Chylomicronemia Syndrome (FCS) (HLP Type I)

South Africa, Germany, United Kingdom, France, United States, Canada, Netherlands
CENTOGENE GmbH Rostock

Enrolling by invitation

Biomarker for Friedreich's Ataxia (BioFridA) (BioFridA)

Hereditary Diseases | FXN Gene | FRDA

Lebanon
Astellas Pharma Global Development, Inc.

Approved for marketing

Expanded Access Study of Gilteritinib (ASP2215) in Patients With FMS-like Tyrosine Kinase 3 (FLT3) Mutated Relapsed or Refractory Acute Myeloid Leukemia (AML) or FLT3-Mutated AML in Complete Remission (CR) With Minimal Residual Disease (MRD)

Acute Myeloid Leukemia (AML) | FMS-like Tyrosine Kinase-3 (FLT3) Mutations

United States, Canada, Japan
University of Oklahoma
National Institute on Alcohol Abuse and Alcoholism (NIAAA); Fogarty International... and other collaborators

Completed

Preventing FAS/ARND in Russian Children

Fetal Alcohol Spectrum Disorders | Fetal Alcohol Syndrome (FAS) | Alcohol Abuse in Pregnancy

Russian Federation
Daping Hospital and the Research Institute of Surgery...

Recruiting

Role and Molecular Mechanism of Farnesoid X Receptor(FXR) and RIPK3 in the Formation of Acute Respiratory Distress Syndrome in Neonates

Acute Respiratory Distress Syndrome | Acute Lung Injury | FXR | RIPK3

China
University of Oklahoma
Centers for Disease Control and Prevention

Completed

Development of Education Materials for Prevention of FAS in Russia

Fetal Alcohol Spectrum Disorders | Fetal Alcohol Syndrome (FAS) | Alcohol Related Neurodevelopmental Disorder | Alcohol Use Complicating Pregnancy, Unspecified Trimester

United States, Russian Federation
Novartis Pharmaceuticals

Available

Managed Access Programs for PKC412, Midostaurin

Acute Myeloid Leukemia | Mast Cell Leukemia | Aggressive Systemic Mastocytosis | Systemic Mastocytosis With an Associated Hematologic Neoplasm | FMS-Like Tyrosine Kinase 3 (FLT3)-Mutated Acute Myeloid Leukemia
Assistance Publique - Hôpitaux de Paris

Completed

Association Corticosteroid/Azathioprine in Microscopic Polyangiitis/ Polyarteritis Nodosa or Eosinophilic Granulomatosis With Polyangiitis (Churg Strauss Syndrome) (CHUSPAN2)

MPA | PAN or EGPA With FFS=0 | At Diagnosis or Within the First 15 Days Following Initiation of Corticosteroids

France

Clinical Trials on FXS

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Togo

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations