Clinical Trials on Niemann-Pick Disease, Type A

Total 58678 results

Novo Nordisk A/S

Completed

A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors (explorer™5)

Haemophilia A | Haemostasis

United Kingdom, France, United States, Sweden, Spain, Turkey, Japan, Italy, Germany, Thailand, Ukraine
CSL Behring

Completed

An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A

Hemophilia A | Severe Hemophilia A

United States, Australia, Austria, Canada, Czechia, France, Georgia, Germany, Hungary, Ireland, Italy, Japan, Lebanon, Malaysia, Netherlands, Philippines, Poland, Portugal, Romania, South Africa, Spain, Switzerland, Thailand, Ukraine, United...
Octapharma

Completed

Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A

Severe Haemophilia A

France, Japan, United States, Canada, Croatia, Finland, Netherlands, North Macedonia, Slovenia
City of Hope Medical Center
CSL Behring; Grifols Therapeutics LLC; Grifols Biologicals, LLC; Charta Foundation and other collaborators

Withdrawn

Study of First TIME Immunotolerance Induction in Severe Hemophilia A Patients With Inhibitor at High Risk of Failure: Comparison With FVIII Concentrates With or Without Von Willebrand Factor - RES.I.S.T. Naive (RESIST NAIVE)

Severe Hemophilia A
Oslo University Hospital
Norwegian School of Sport Sciences

Completed

HemFitBit, an Observational Study of Physical Activity in People With Moderate and Severe Haemophilia A

Hemophilia A Without Inhibitor

Norway
University of California, Davis

Terminated

INdividualized ITI Based on Fviii(ATE) Protection by VWF (INITIATE)

Hemophilia A | Hemophilia A With Inhibitor

United States
The Hospital for Sick Children

Completed

Canadian Hemophilia Prophylaxis Study (CHPS)

Severe Hemophilia A

Canada
Octapharma

Completed

Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A

Severe Hemophilia A

Germany, Austria, United Kingdom, Bulgaria
CSL Behring

Completed

Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A

Congenital Hemophilia A

United States, Austria, Germany, Malaysia, Netherlands, Philippines, Ukraine, Romania, Spain, Australia, France, Georgia, Italy, Lebanon, Poland, Portugal, Switzerland, Thailand, Turkey
Fondazione IRCCS Ca' Granda, Ospedale Maggiore...

Unknown

rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors (ENJOIH)

Hemophilia A With Inhibitors

United States, Germany, France, Italy, Romania, Spain
Jiangsu Gensciences lnc.

Recruiting

Phase 3 Clinical Project of Pegylated Recombinant Human Coagulation Factor VIII-Fc Fusion Protein

Severe Hemophilia A

China
Octapharma

Recruiting

Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study (NuPOWER)

Severe Hemophilia A

Spain, Croatia, Italy, North Macedonia, Serbia
Institute of Hematology & Blood Diseases Hospital...
Sinocelltech Ltd.

Recruiting

ITI Using SCT800 Alone or Combining Daratumumab in Hemophilia A Adolescents and Adults With High Titer Inhibitor

Hemophilia A With Inhibitor

China
Margaret Ragni
Health Resources and Services Administration (HRSA)

Terminated

The Hemophilia Inhibitor Eradication Trial

Hemophilia A With Inhibitor

United States
Octapharma

Terminated

Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A

Severe Hemophilia A

Bulgaria
Children's Hospital Los Angeles
Takeda

Active, not recruiting

A Study to Evaluate the Safest Dose Range for FEIBA in Hemophilia A Patients With Inhibitors on Emicizumab

Hemophilia A With Inhibitor

United States
City of Hope Medical Center
CSL Behring; Grifols Therapeutics LLC; Grifols Biologicals, LLC; Charta Foundation and other collaborators

Completed

Rescue Immunotolerance Study in Induction of Immune Tolerance (ITI)-Experienced Patients (RES.I.S.T. Experienced) (RESIST EXP)

Severe Hemophilia A

United States
Octapharma

Completed

Pharmacokinetics, Efficacy, Safety, and Immunogenicity of Wilate in Previously Treated Paediatric Patients With Severe Haemophilia A

Severe Hemophilia A

Russian Federation, Ukraine
Octapharma

Completed

Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

Severe Hemophilia A

United States, France, Canada, United Kingdom, India, Georgia, Moldova, Republic of, Poland, Ukraine
Bioverativ Therapeutics Inc.

Completed

Pharmacokinetics of rFVIIIFc at Two Vial Strengths

Severe Hemophilia A

United States, United Kingdom, Australia
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Completed

Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale (Elevate)

Severe Hemophilia A

United States, Australia, New Zealand
Bayer

Terminated

Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors

Hemophilia A and B

Australia, United Kingdom, Taiwan, Japan, Korea, Republic of, Austria, Bulgaria, Hungary, Italy, France, New Zealand
CHU de Reims

Unknown

TFPI Levels in Haemophilia A and B Patients

Haemophilia A and B

France
Octapharma

Completed

Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)

Severe Hemophilia A

United States, Bulgaria
Pfizer

Completed

PF-06741086 Multiple Dose Study in Severe Hemophilia

Hemophilia A or B

United States, Chile, Croatia, Poland, South Africa, Switzerland
Margaret Ragni

Withdrawn

Hemophilia Inhibitor Clinical Trials (INHIBIT) Platform (INHIBIT)

Severe Hemophilia A

United States
Beijing Children's Hospital

Unknown

Low-dose ITI Strategy for Children in Hemophilia A With High-titer Inhibitor and Poor ITI Risk in China (HA-LD-ITI)

Hemophilia A With Inhibitor

China
Hampshire Hospitals NHS Foundation Trust
Uppsala University; University of Wales Cardiff United Kingdom (UK)

Completed

Efficacy and Cost Effectiveness of Pharmacokinetic Dosing in Haemophilia A

Severe Haemophilia A

United Kingdom
Skane University Hospital

Completed

The Malmö International Brother Study (MIBS)

Hemophilia A With Inhibitors

Sweden
U.S. Army Medical Research and Development Command

Enrolling by invitation

Continued Safety and Immunogenicity Study of a Live Francisella Tularensis Vaccine

Tularemia

United States
Novartis Pharmaceuticals

Completed

A Phase Ib Dose De-escalation Study With BYL719 in Premenopausal Patients With Locally Advanced or Metastatic Breast Cancer (B-YOND)

Pre-menopausal Breast Cancer | PI3K Pathway Inhibition

Taiwan, Hong Kong, Thailand, Korea, Republic of
Assistance Publique - Hôpitaux de Paris

Withdrawn

Robot Based Tympanic Tube Placement (ROBOTOL)

Patient Operated on From a Vestibular Schwannoma With a Middle Ear Exclusion
University of Zurich
Kliniken Valens

Completed

Sensing Physical Activity to Evaluate and Monitor a Routine Aftercare Program (SensE-M) (SensE-M)

Disease With a Risk for Chronicity

Switzerland
National Institute of Allergy and Infectious Diseases...

Completed

Searching for Persistence of Infection in Lyme Disease

Lyme Disease

United States
University of Minnesota

Terminated

Passive Prophylaxis of Lyme Disease Using Permethrin Treated Clothes

Lyme Disease

United States
MicroB-plex, Inc.
Johns Hopkins University

Unknown

Novel Diagnostics for Early Lyme Disease

Lyme Disease

United States
Advanced Laboratory Services, Inc.

Terminated

A Multicenter Study to Evaluate a Borrelia Diagnostic Test in Subjects With Early Stage or Late Stage Lyme Disease (ALSIBDT)

Lyme Disease

United States
Silence Therapeutics plc

Active, not recruiting

Evaluate SLN360 in Participants With Elevated Lipoprotein(a) at High Risk of Atherosclerotic Cardiovascular Disease Events

Cardiovascular Diseases | Atherosclerosis | Lipoprotein(a)

Australia, Czechia, Denmark, Netherlands, Slovakia, South Africa, United Kingdom
Novartis Pharmaceuticals

Active, not recruiting

Assessing the Impact of Lipoprotein (a) Lowering With Pelacarsen (TQJ230) on Major Cardiovascular Events in Patients With CVD (Lp(a)HORIZON)

Cardiovascular Disease and Lipoprotein(a)

United States, China, Germany, South Africa, Italy, Spain, Belgium, Czechia, Hungary, Japan, Taiwan, Netherlands, Australia, Austria, Canada, France, Israel, Switzerland, United Kingdom, Korea, Republic of, Argentina, Denmark, Greece, Romani... and more
DiaSorin Inc.

Enrolling by invitation

Lyme Disease Diagnostic Assay - Collection of Whole Blood

Healthy | Lyme Disease

United States
Klinikum Nürnberg

Unknown

RV Septal Versus Minimized RV Pacing in Sick Sinus Syndrome (VOTE)

Sick Sinus Syndrome

Germany
Medtronic BRC
Vitatron GmbH

Completed

Pacing of the Atria in Sick Sinus Syndrome Trial Preventive Strategies for Atrial Fibrillation

Sick Sinus Syndrome

Germany
University Hospital, Strasbourg, France

Recruiting

Description of Case of Infection With a New Species of Babesia and Its Molecular Diagnosis (BADIAG)

Babesiosis, Human

France
LivaNova

Completed

Evolution of the PR Interval in Patients Implanted With a Pacemaker Using the SafeR Mode (PRECISE)

Atrioventricular Block | Sinus Node Dysfunction

France
Urmia University of Medical Sciences

Completed

Increasing Atrial Base Rate Pacing to Reduce Atrial Fibrillation

Paroxysmal Atrial Fibrillation | Sick Sinus Syndrome

Iran, Islamic Republic of
Bayer

Completed

Comparison of the Extent to Which the Drug Given as Two Different Capsule Sizes Becomes Available to the Body

Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men

Germany
University of Nottingham

Unknown

Dysfunction of Nutritive Blood Flow as a Determinant of Anabolic Resistance With Age; the Role of Amino Acids in Modulating Muscle Metabolism (Ajinomoto1A)

Old Age (Focus; Not a Recognized Condition)

United Kingdom
University of Valencia
Baxter Healthcare Corporation

Completed

Exploratory Pilot Study of Physical Activity Monitoring in Adult Patients With Haemophilia A by Means of Accelerometry

Hemophilia A, Congenital

Spain
Fondazione I.R.C.C.S. Istituto Neurologico Carlo...

Not yet recruiting

Fluorescence, Light-microscopy, Ultrasound Integrated / Intraoperative Diagnosis to MAXimise Resection (FLUID-MAX)

Patients With Radiological, Clinical and Anamnestic Picture Compatible With a New Diagnosis of Glioblastoma
University of Kentucky

Completed

Muscle Dysfunction in Critical Illness

Critical Illness | Muscle Weakness | Intensive Care (ICU) Myopathy | PICS

United States

Clinical Trials on Niemann-Pick Disease, Type A

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