Clinical Trials on Sanfilippo Disease Type A

Total 58073 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Withdrawn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
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
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
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
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
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
Novo Nordisk A/S

Recruiting

A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors

Haemophilia A With or Without Inhibitors

Korea, Republic of, India, China, Canada, Spain, Netherlands, South Africa, United States, Israel, Japan, Poland, United Kingdom, Italy, Lithuania, Portugal, Switzerland, Taiwan
Zhang Lei, MD
Henan Cancer Hospital; Qilu Hospital of Shandong University; Tianjin First Central... and other collaborators

Completed

Trial of Acquired Haemophilia With Steroid Combined With Cyclophosphamide Versus Steroid Combined With Rituximab

Acquired Hemophilia A

China
Baxalta now part of Shire
Baxalta Innovations GmbH, now part of Shire

Completed

FEIBA Reconstitution Volume Reduction and Faster Infusion Study (FEIBA STAR)

Hemophilia A or B With Inhibitors

Croatia, North Macedonia, Ukraine
AryoGen Pharmed Co.

Completed

Clinical Trial Comparing a Biosimilar Eptacog Alfa With Novoseven, in Patients With Hemophilia With Inhibitors

Hemophilia A or B With Inhibitor

Iran, Islamic Republic of, Turkey
Grifols Italia S.p.A
Thrombinoscope

Unknown

Thrombin Generation Assay (TGA) as Predictive Test for Haemostatic. Effectiveness of FVIII Concentrates in Haemophiliac A With Inhibitors (PredicTGA)

Severe Hemophilia A With Inhibitor

Italy
Novartis Pharmaceuticals

Recruiting

An Open Label Extension (OLE) Study to Evaluate Long-term Safety and Tolerability of Pelacarsen (TQJ230) (OLE)

Hyperlipoproteinemia (a)

Germany
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
Leiden University Medical Center
Andaluz Health Service

Completed

"POWER2DM Evaluation Campaign" (POWER2DMEC)

Diabetes Mellitus | Health Behavior | Self Efficacy | Type2 Diabetes Mellitus | Type1 Diabetes Mellitus

Netherlands, Spain
UPECLIN HC FM Botucatu Unesp

Completed

Factors Associated to Success of Hepatitis C Therapy

Hepatitis, Viral, Non-A, Non-B, Parenterally-Transmitted

Brazil
Merck Sharp & Dohme LLC

Completed

Concomitant Use of Hepatitis A Vaccine, Inactivated With Haemophilus b Conjugate Vaccine (Meningococcal Protein Conjugate) and Diphtheria and Tetanus Toxoids and Acellular Pertussis Vaccine Adsorbed Given to Healthy Children 15 Months of Age (V251-068)

Hepatitis A Virus
University Hospital, Montpellier

Completed

Medico-economic Evaluation of a Project of Wounds by a Remote Network (DOMOPLAIES)

Resident of Languedoc-roussillon | Carrying a Chronic Wound

France
North-West University, South Africa
Medical Research Council, South Africa; National Research Foundation of South... and other collaborators

Completed

Effect of African Leafy Vegetables on Nutritional Status of South African School Children. (ALV)

Malnutrition | Vitamin A Status | Iron Status | Zinc Status

South Africa
Assistance Publique Hopitaux De Marseille

Completed

Assessment of the Mutation of Pig-A Gene as Biomarker of Genotoxic Exposure in Humans (PIG-A)

Patient With Breast Cancer

France
Novo Nordisk A/S

Completed

Efficacy and Safety of Turoctocog Alfa Pegol (N8-GP) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A (pathfinder10) (Pathfinder10)

Haemophilia A

China
Baxalta now part of Shire
Baxalta Innovations GmbH, now part of Shire

Completed

Prospective and Retrospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice

Acquired Hemophilia A

United States, Italy, United Kingdom, Germany, Austria, Netherlands, France
Baxalta now part of Shire

Completed

Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Acquired Hemophilia A

Acquired Hemophilia A

United States, Canada, India, United Kingdom
OPKO Health, Inc.

Completed

A Single-dose, Dose-escalation Study of a Long-acting MOD-5014 in Healthy Adult Male

Hemophilia A or B With Inhibitors

Israel
Peking Union Medical College Hospital

Unknown

Study to Evaluate Rituximab and Bortezomib in Patients With Newly Diagnosed Acquired Hemophilia A

Acquired Hemophilia A

China
Georgetown University
Genentech, Inc.

Terminated

Trial of Rituximab Versus Oral Cyclophosphamide to Eradicate or Suppress Autoimmune Anti-Factor VIII Antibodies in Acquired Hemophilia A

Hemophilia A
Hospital Universitario La Paz

Unknown

Case-control Pilot Study of the Immune Modulating Effect of FEIBA on Patients With Haemophilia A and Inhibitors

Severe Haemophilia A With Inhibitors

Spain
Recoly N.V.

Completed

Study of the Efficacy of Human Recombinant Factor VIII (Kogenate FS) Reconstituted in Pegylated Liposomes.

Haemophilia A

Russian Federation
Skane University Hospital

Completed

The FEIBA NovoSeven Comparative Study

Severe Hemophilia A With an Inhibitor

Sweden
Tri-Service General Hospital

Recruiting

A Study on the Bone-health Effectiveness of Applying Recombinant Factor VIII Fc (rFVIIIFc) to Patients With Hemophilia A (Prototype A)

Severe Hemophilia A Without Inhibitor

Taiwan
Institute of Hematology & Blood Diseases Hospital

Recruiting

An Exploratory Study of Cluster of Differentiation 38 (CD38) Monoclonal Antibody Combined Corticosteroid in Acquired Hemophilia A (AHA2023)

Acquired Hemophilia

China
Groupe Maladies hémorragiques de Bretagne

Recruiting

Genetic Influence of Genetic Factors Influencing the Desmopressin's Efficacy in Mild/Moderate Hemophilia A (GIDEMHA)

Desmopressin | Hemophilia A, Mild | Factor VIII

France
University of Washington
Genentech, Inc.

Recruiting

Emicizumab in Patients With Acquired Hemophilia A (AHAEmi)

Acquired Hemophilia A

United States
Novo Nordisk A/S

Enrolling by invitation

A Study Following Males With Haemophilia A on Prophylaxis With Esperoct® (pathfinder9)

Haemophilia A

Austria, Spain, Bulgaria, Croatia, Estonia, Hungary, Italy, Lithuania, Portugal, Greece, Germany, Czechia, Slovenia, Switzerland, Slovakia
Chulalongkorn University

Completed

Clinical Outcomes of Low Dose PK-guided EHL FVIII Concentrates Versus Standard Prophylaxis in Severe Haemophilia A

Severe Hemophilia A Without Inhibitor

Thailand
Baxalta now part of Shire

No longer available

Expanded Access to B-Domain Deleted Recombinant Porcine Factor VIII (OBI-1) in the Treatment of Acquired Hemophilia A Due to Factor VIII Inhibitory Auto-antibodies

Acquired Hemophilia A

United States
The Hospital for Sick Children
Unity Health Toronto; Nationwide Children's Hospital; Canadian Hemophilia Society

Unknown

DDAVP vs. Exercise in Patients With Mild Hemophilia A

Mild Haemophilia A Without Inhibitor

Canada
China Medical University Hospital

Unknown

Inherited Myokymia: A Clinical and Genetic Study of a Family

A Study of Clinical and Genetic Mutations in a Family of Neuromyotonia, so Called Isaac's Syndrome

Taiwan
Odense University Hospital

Not yet recruiting

Extended Cross-sectoral Nurse Follow-up After Discharge From a Geriatric Ward - Benefits and Challenges. A Mixed-method Study

Extended Cross-sectoral Nurse Follow-up After Discharge From a Geriatric Ward
Brigham and Women's Hospital

Completed

Post-Acute Physician Home Visit Program

A Patient Discharged From an Acute-care Hospital Who Had an Acute Illness

United States

Clinical Trials on Sanfilippo Disease Type A

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