Clinical Trials on Pompe's Disease Juvenile Onset

Total 1333 results

Aristotle University Of Thessaloniki
NHS Pulmonary Clinic G. Papanikolaou Hospital, Thessaloniki, Greece; Pulmonary... and other collaborators

Active, not recruiting

Efficacy of Mepolizumab in Severe Asthmatics on a Long Term (MESILICO) (MESILICO)

Severe Asthma | Asthma; Eosinophilic | Late-Onset Asthma

Greece
Groupe Francophone des Myelodysplasies
Astex Pharmaceuticals, Inc.

Completed

A Phase II Trial of SGI-110 in Patients With IPSS High and Int 2 Myelodysplastic Syndrome, Acute Myeloid Leukemia With 20-30% Marrow Blasts or Chronic Myelomonocytic Leukemia Type 2 Not Responding to Azacitidine or Decitabine After at Least 6 Courses or Relapsing After a Response

MDS

France
University of Utah
Genzyme, a Sanofi Company

Terminated

Low-dose Oral Clofarabine for the Treatment of IPSS INT-1, INT-2 or HIGH Myelodysplastic Syndromes and Chronic Myelomonocytic Leukemia

Cancer | Chronic Myelomonocytic Leukemia

United States
Rigshospitalet, Denmark
Odense University Hospital; University of Copenhagen; Imperial College London; University... and other collaborators

Active, not recruiting

Epigenetics, Vitamin C, and Abnormal Blood Cell Formation - Vitamin C in Patients With Low-Risk Myeloid Malignancies (EVITA)

Myelodysplastic Syndromes | Cytopenia | Chronic Myelomonocytic Leukemia-1

Denmark, United States
M.D. Anderson Cancer Center
Genentech, Inc.; Astex Pharmaceuticals, Inc.

Recruiting

Venetoclax in Combination With ASTX727 for the Treatment of Treatment-Naive High-Risk Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia

Chronic Myelomonocytic Leukemia | Myelodysplastic Syndrome

United States
National Cancer Institute (NCI)

Active, not recruiting

Lenalidomide With or Without Epoetin Alfa in Treating Patients With Myelodysplastic Syndrome and Anemia

Anemia | Chronic Myelomonocytic Leukemia | Myelodysplastic Syndrome | de Novo Myelodysplastic Syndrome

United States
Emma Marie Caroline Slack
University of Zurich; University Children's Hospital, Zurich

Completed

Characterization of Intestinal Microbiota Stability in Preterm Born Neonates (NEC)

Late-Onset Neonatal Sepsis | Necrotising Enterocolitis

Switzerland
H. Lee Moffitt Cancer Center and Research Institute
Pfizer

Completed

Phase II Hedgehog Inhibitor for Myelodysplastic Syndrome (MDS)

Chronic Myelomonocytic Leukemia (CMML) | Myelodysplastic Syndrome (MDS)

United States
Groupe Francophone des Myelodysplasies
AbbVie

Recruiting

Azacitidine Combined With Venetoclax in Patients With Higher-risk Chronic Myelomonocytic Leukemia (AVENHIR) (AVENHIR)

Chronic Myeloid Leukemia

France
Kirby Institute
Celgene

Completed

Evaluating in Vivo AZA Incorporation in Mononuclear Cells Following Vidaza or CC486

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Chronic Myelomonocytic Leukemia

Australia
Pfizer

Completed

Continuation Study of B1371019(NCT03416179) and B1371012(NCT02367456) Evaluating Azacitidine With Or Without Glasdegib In Patients With Previously Untreated AML, MDS or CMML

Acute Myeloid Leukemia | Chronic Myelomonocytic Leukemia | Myelodysplastic Syndrome

Spain, Canada, Austria, Czechia, France, Hungary, Italy, Japan, Mexico, Poland, United Kingdom
University College Dublin
Wellcome Trust; Health Research Board, Ireland; National Maternity Hospital,... and other collaborators

Recruiting

Markers of Platelet Activation foR Identification of Late Onset Sepsis in Preterm Infants (PARENT)

Late-Onset Neonatal Sepsis | Necrotising Enterocolitis | Culture Negative Neonatal Sepsis

Ireland
Stemline Therapeutics, Inc.

Completed

Tagraxofusp (SL-401) in Patients With CMML or MF

Myelofibrosis | Chronic Myelomonocytic Leukemia

United States, Canada
Karen Ballen, MD

Recruiting

Study of Tagraxofusp for Post-Transplant Maintenance for Patients With CD 123+ AML, MF and CMML (HSCT 002)

Acute Myeloid Leukemia | Myelofibrosis | Chronic Myelomonocytic Leukemia

United States
Polaryx Therapeutics, Inc.

Not yet recruiting

Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3

Juvenile Neuronal Ceroid Lipofuscinosis
University of Rochester
Batten Disease Support and Research Assocation (BDSRA)

Completed

Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JUMP)

Juvenile Neuronal Ceroid Lipofuscinosis

United States
Heinrich-Heine University, Duesseldorf
Koordinierungszentrum für Klinische Studien - Duesseldorf

Recruiting

Venetoclax in Addition to Sequential Conditioning With Fludarabine / Amsacrine / Ara-C (FLAMSA) + Treosulfan for Allogeneic Blood Stem Cell Transplantation in Patients With MDS, CMML or sAML

Myelodysplastic Syndromes | Chronic Myelomonocytic Leukemia | Secondary Acute Myeloid Leukemia

Germany
Mayo Clinic

Recruiting

Onvansertib for the Treatment of Recurrent or Refractory Chronic Myelomonocytic Leukemia

Recurrent Chronic Myelomonocytic Leukemia | Refractory Chronic Myelomonocytic Leukemia

United States
Astex Pharmaceuticals, Inc.

Enrolling by invitation

A Study for Participants Who Participated in Prior Clinical Studies of ASTX727 (Standard Dose), With a Food Effect Substudy at Select Study Centers

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Chronic Myelomonocytic Leukemia

United States, Spain, Canada, Austria, Slovakia, Bulgaria, Hungary
Arbeitsgemeinschaft medikamentoese Tumortherapie

Completed

Registry on Hypomethylating Agents in Myeloid Neoplasms

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Chronic Myelomonocytic Leukemia

Austria, Croatia
Pfizer

Completed

A Combination Study of PF-04449913 (Glasdegib) and Azacitidine In Untreated MDS, AML and CMML Patients (BRIGHT 1012)

Acute Myeloid Leukemia | Chronic Myelomonocytic Leukemia | Myelodysplastic Syndrome

United States, United Kingdom, Belgium, Canada, Germany, France
Heinrich-Heine University, Duesseldorf
Celgene Corporation; Koordinierungszentrum für Klinische Studien Düsseldorf

Active, not recruiting

IDH2-Post-Allo-Trial for Patients With IDH2-mut Myeloid Neoplasms After Allo-SCT

Myelodysplastic Syndromes | Leukemia, Myeloid, Acute | Chronic Myelomonocytic Leukemia | IDH2 Gene Mutation | IDH2 R172 | IDH2 R140

Germany
Seagen Inc.

Completed

A Safety Study of Lintuzumab in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndrome

Acute Myeloid Leukemia | Myeloproliferative Disorders | Chronic Myelomonocytic Leukemia | Myelodysplastic Syndrome

United States
University Hospital, Lille

Completed

Patients With Alzheimer's Disease or Related Youth Disease (PARMAAJJ)

Lewy Body Disease | Frontotemporal Lobar Degeneration | Alzheimer Disease, Early Onset

France
Odense University Hospital

Unknown

Bone Structure in Patients With Adult Onset Growth Hormone Deficiency Assessed Using High Resolution Peripheral Quantitative Computed Tomography

Adult Onset Growth Hormone Deficiency

Denmark
Clinical Hub for Interventional Research (CHOIR)
The University of New South Wales; Australian National University

Recruiting

Combined Evaluation of Epigenetic and Sensitising Therapy in AML and MDS (CELESTIAL-MDS)

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Chronic Myelomonocytic Leukemia

Australia
Ascentage Pharma Group Inc.

Recruiting

A Study of APG-115 Alone or Combined With Azacitidine in Patients With AML, CMML, or MDS

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Chronic Myelomonocytic Leukemia | AML | MDS | CMML | High-risk Myelodysplastic Syndrome

United States
Otsuka Australia Pharmaceutical Pty Ltd

Active, not recruiting

A Study Comparing Treatment Preference Between Oral Decitabine/Cedazuridine and Azacitidine in Myelodysplastic Syndrome, Low-Blast Acute Myeloid Leukemia, or Chronic Myelomonocytic Leukemia (PREFER-HMA)

Myelodysplastic Syndromes | Leukemia, Myeloid, Acute | Leukemia, Myelomonocytic, Chronic | Patient Preference

New Zealand, Australia
Takeda
Takeda Development Center Americas, Inc.

Active, not recruiting

Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML), or Low-Blast Acute Myelogenous Leukemia (AML) (PANTHER)

Leukemia, Myeloid, Acute | Leukemia, Myelomonocytic, Chronic | Myelodysplastic Syndrome

Australia, United States, Canada, Spain, Belgium, France, Czechia, Italy, Korea, Republic of, Israel, Brazil, China, Germany, Turkey, Japan, Greece, Poland, United Kingdom, Russian Federation, Mexico
Texas Cardiac Arrhythmia Research Foundation

Recruiting

Natural History of COVID-19-Related Atrial Fibrillation (unCOVer-AF)

COVID-19 | Atrial Fibrillation New Onset

Italy, United States, Belgium
Amicus Therapeutics

Recruiting

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 (ROSSELLA)

Glycogen Storage Disease Type II Infantile Onset

United States, Taiwan, France, Germany, Italy, United Kingdom
Per Torp Sangild

Completed

Bovine Colostrum as a Human Milk Fortifier for Preterm Infants (FortiColos-Ⅱ)

Necrotizing Enterocolitis | Feeding Intolerance | Postnatal Growth | Late-Onset Neonatal Sepsis

China
University of Colorado, Denver
Johns Hopkins University

Completed

Evaluation of Coffee Therapy for Improvement of Renal Oxygenation (COFFEE)

Diabetes Complications | Type1diabetes | Diabetic Kidney Disease | Diabetic Nephropathies | Type1 Diabetes Mellitus | Diabetes, Autoimmune | Juvenile Diabetes

United States
Mental Health Services in the Capital Region, Denmark
TrygFonden, Denmark; The Danish Mental Health Foundation; Defactum, Central Denmark...

Completed

Transdiagnostic, Cognitive and Behavioral Intervention for in School-aged Children With Emotional and Behavioral Disturbances (MindMyMind RCT)

Problem Behavior | Anxiety Disorders | Anxiety | Depressive Symptoms | Conduct Disorder | Oppositional Defiant Disorder | Depressive Disorders | Other Behavioral and Emotional Disorders With Onset Usually Occurring in Childhood and Adolescence

Denmark
University of Leicester
University of Oxford; University of Manchester

Recruiting

A Randomised, Controlled Trial of a Low-energy Diet for Improving Functional Status in Heart Failure With PRESERVED Ejection Fraction Preserved Ejection Fraction (AMEND)

Diabetes Mellitus, Type 2 | Heart Failure, Diastolic | Heart Failure With Preserved Ejection Fraction | Diabetes Mellitus Type 2 in Obese | Obesity Adult Onset

United Kingdom
Bristol-Myers Squibb

Completed

A Study of Azacitidine in Participants With International Prognostic Scoring System (IPSS) Intermediate-2 and High-risk Myelodysplastic Syndromes (MDS), or Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML) (DIM Vidaza)

Myelodysplastic Syndromes | Leukemia, Myeloid, Acute | Leukemia, Myelomonocytic, Chronic

China
Central Hospital, Nancy, France

Completed

C.O.R.P.U.S.: Observational Cohort of Adult and Child Inflammatory Arthritis in France

Arthritis, Rheumatoid | Arthritis, Psoriatic | Spondylitis, Ankylosing | Arthitis, Juvenile Idiopathic

France
Kahr Medical

Recruiting

A Study of Dual-SIgnaling Protein 107 (DSP107) for Patients With Hematological Malignancies

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Chronic Myelomonocytic Leukemia

United States
Onconova Therapeutics, Inc.

Completed

Phase II Part 2 Expansion of Oral Rigosertib in Combination With Azacitidine

Acute Myeloid Leukemia | Chronic Myelomonocytic Leukemia | Myelodysplastic Syndrome

United States, France
Fox Chase Cancer Center
M.D. Anderson Cancer Center; Teva Pharmaceuticals USA

Completed

Leukemia SPORE Phase II DAC Study for R/R and Elderly Acute AML and MDS

Acute Myeloid Leukemia | Chronic Myelomonocytic Leukemia | Myelodysplastic Syndrome

United States
Astex Pharmaceuticals, Inc.

Completed

Effect of Food on Blood Levels of ASTX727

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Chronic Myelomonocytic Leukemia

United States
University of Colorado, Denver
American College of Radiology

Completed

Preclinical Imaging Biomarkers of Alzheimer's Disease Neuropathology in Young Adults With Youth-onset Diabetes: a Proof-of-concept Study

Diabetes Mellitus, Type 2 | Diabetes Mellitus, Type 1 | Diabetes, Youth Onset

United States
Imperial College London
Chelsea and Westminster NHS Foundation Trust; National Heart and Lung Institute and other collaborators

Completed

Defining the Intestinal Microbiota in Premature Neonates

Necrotizing Enterocolitis | Premature Intestinal Microbiota | Late Onset Bloodstream Infection

United Kingdom
Nerviano Medical Sciences

Recruiting

Study of NMS-03592088 in Patients With Relapsed or Refractory AML or CMML

Acute Myeloid Leukemia (AML) | Chronic Myelomonocytic Leukemia (CMML)

Italy, Spain, France
M.D. Anderson Cancer Center
National Cancer Institute (NCI)

Recruiting

Venetoclax and Azacitidine for the Treatment of High-Risk Recurrent or Refractory Myelodysplastic Syndrome

Chronic Myelomonocytic Leukemia | Myelodysplastic Syndrome | Recurrent Myelodysplastic Syndrome | Refractory Myelodysplastic Syndrome | Therapy-Related Myelodysplastic Syndrome

United States
Janssen Research & Development, LLC

Active, not recruiting

A Study of JNJ-74856665 in Participants With Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) (DHODH)

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Chronic Myelomonocytic Leukemia

United Kingdom, France, Korea, Republic of, Spain
Amgen

Completed

AMG 176 With Azacitidine in Subjects With Myelodysplastic Syndrome /Chronic Myelomonocytic Leukemia

Chronic Myelomonocytic Leukemia | Higher Risk Myelodysplastic Syndrome

United States
Janssen Research & Development, LLC

Completed

A Study to Characterize the Clinical Course of Pregnant Women and Children at High Risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn

Erythroblastosis, Fetal | Early Onset Severe Hemolytic Disease of the Fetus and Newborn (EOS-HDFN)

United States, Canada, Netherlands, Belgium, Australia, Germany, Spain, Sweden, United Kingdom
LLS PedAL Initiative, LLC
National Cancer Institute (NCI); Children's Oncology Group

Recruiting

The Pediatric Acute Leukemia (PedAL) Screening Trial - A Study to Test Bone Marrow and Blood in Children With Leukemia That Has Come Back After Treatment or Is Difficult to Treat - A Leukemia & Lymphoma Society and Children's Oncology Group Study

Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Acute Lymphoblastic Leukemia | Myelodysplastic Syndrome | Myeloid Leukemia Associated With Down Syndrome | Mixed Phenotype Acute Leukemia | Acute Myeloid Leukemia Post Cytotoxic Therapy | Myelodysplastic Syndrome Post Cytotoxic Therapy

United States, Canada, Australia, Puerto Rico, New Zealand
Northwestern University
Ann & Robert H Lurie Children's Hospital of Chicago

Completed

Absorption of Corticosteroids in Children With Juvenile Dermatomyositis

Connective Tissue Diseases | Vasculitis | Dermatomyositis | Vasculitis, Hypersensitivity

United States

Clinical Trials on Pompe's Disease Juvenile Onset

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Zimbabwe

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

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