Search clinical trials for: Krabbe Disease

Total 379056 results

Regeneron Pharmaceuticals

Not yet recruiting

Short-Term Linvoseltamab Treatment, on Top of Chronic Dupilumab Treatment, for Adults With Severe Immunoglobulin E (IgE)-Mediated Food Allergy

Food Allergy
Forge Biologics, Inc

Not yet recruiting

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients

Krabbe Disease
Marianne Barbu-Roth
Assistance Publique - Hôpitaux de Paris; San Francisco State University; Ecole... and other collaborators

Recruiting

Early Intervention Based on Neonatal Crawling in Very Premature Infants at Risk For Neurodevelopmental Disorder (Premalocom2)

Prematurity | Brain Damage | Infant Development | Extreme Prematurity | Motor and Developmental Delay

France
Forge Biologics, Inc

Recruiting

Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT (REKLAIM)

Krabbe Disease

United States
Fondazione I.R.C.C.S. Istituto Neurologico Carlo...

Recruiting

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

Neurologic Disorder | Rare Diseases | Metabolic Disease | Inherited Disease | Undiagnosed Disease | Neuro-Degenerative Disease

Italy
Children's Hospital of Philadelphia
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Recruiting

Locomotor Learning in Infants at High Risk for Cerebral Palsy

Cerebral Palsy

United States
Aria CV, Inc

Recruiting

Treatment of Pulmonary Arterial Hypertension Using the Aria CV Pulmonary Hypertension System (ASPIRE PH)

Pulmonary Arterial Hypertension | Pulmonary Hypertension | Right Heart Dysfunction

United States
Paul Szabolcs

Recruiting

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC (PRO-RIC)

Congenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias

United States
Ancora Heart, Inc.

Recruiting

Clinical Evaluation of the AccuCinch® Ventricular Restoration System in Patients Who Present With Symptomatic Heart Failure With Reduced Ejection Fraction (HFrEF): The CORCINCH-HF Study

Dilated Cardiomyopathy | Heart Failure With Reduced Ejection Fraction (HFrEF)

United States, France, Netherlands, Czechia, Belarus, Belgium, Serbia
Asklepios Biopharmaceutical, Inc.

Recruiting

NAN-101 in Patients With Class III Heart Failure (NAN-CS101)

Cardiovascular Diseases | Heart Failure | Heart Failure, Diastolic | Congestive Heart Failure | Heart Failure, Systolic | Heart Disease, Ischemic | Heart Failure，Congestive | Heart Arrhythmia | Heart; Complications

United States
University of Pittsburgh

Recruiting

Longitudinal Study of Neurodegenerative Disorders

Lysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditions

United States
Children's Hospital of Philadelphia
Eli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaborators

Recruiting

The Myelin Disorders Biorepository Project (MDBP)

Mucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... and other conditions

United States
State University of New York at Buffalo
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Recruiting

Krabbe Disease Global Patient Registry

Krabbe Disease

United States
Joanne Kurtzberg, MD
The Marcus Foundation

Recruiting

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells (DUOC-01)

Mucopolysaccharidosis II | Brain Diseases, Metabolic, Inborn | Adrenoleukodystrophy | Batten Disease | Leukodystrophy, Metachromatic | Leukodystrophy, Globoid Cell | Alpha-Mannosidosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | Neimann Pick Disease | Sanfilippo Mucopolysaccharidoses

United States
Paul Szabolcs

Recruiting

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT (HSCT+RIC)

Congenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias | Systemic Juvenile Idiopathic Arthritis (sJIA)

United States
Forge Biologics, Inc

Active, not recruiting

Gene Transfer Clinical Trial for Krabbe Disease (RESKUE)

Krabbe Disease

United States
Philips Electronics Nederland B.V. acting through...
UMC Utrecht

Completed

Clinical Relevance of Scratching and Sleep in Atopic Dermatitis

Atopic Dermatitis

Netherlands
RTI International
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

Early Check: Expanded Screening in Newborns

Primary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditions

United States
University Hospital, Basel, Switzerland

Active, not recruiting

The Effect of Cooling With a Bed Topper and Linen on Itch in Clinical Routine

Itch

Switzerland
Mohamed Tahar Maamouri University Hospital

Completed

Anatomical Research of the Clavicular Pedicled Flap for Mandibular Reconstruction

Maxillofacial Injuries | Mandibular Reconstruction

Tunisia
Children's Hospital of Philadelphia
Illumina, Inc.

Active, not recruiting

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Mucopolysaccharidoses | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2 Gangliosidosis | Zellweger... and other conditions

United States
Passage Bio, Inc.

Suspended

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease (GALax-C)

Leukodystrophy, Globoid Cell

United States, Brazil, Netherlands, United Kingdom, Canada, Israel
Arbeitsgemeinschaft medikamentoese Tumortherapie
Amgen; Assign Data Management and Biostatistics GmbH

Active, not recruiting

Denosumab for High Risk SMM and SLiM CRAB Positive, Early Myeloma Patients (DEFENCE)

Multiple Myeloma

Austria, Germany, Israel
HealthPartners Institute

Completed

Supraclavicular Nerve During Clavicle ORIF

Clavicle Fracture
University of Pittsburgh
National Institute of Neurological Disorders and Stroke (NINDS)

Active, not recruiting

Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease

Krabbe Disease

United States
Talaris Therapeutics Inc.
Duke University

Terminated

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Niemann-Pick Disease | Alpha-mannosidosis | Tay Sachs Disease | Sandhoff Disease | Metachromatic Leukodystrophy (MLD) | Hurler-Scheie Syndrome | Hurler Syndrome (MPS I) | Hunter Syndrome (MPS II) | Sanfilippo Syndrome (MPS III) | Krabbe Disease (Globoid Leukodystrophy) | Adrenoleukodystrophy (ALD and AMN) | Pelizaeus...

United States
Philips Clinical & Medical Affairs Global

Completed

Scratching Validation Study

Atopic Dermatitis

United States
Hong Kong Sanatorium & Hospital
Chinese University of Hong Kong

Terminated

Pilot Study on Traditional Chinese Medicine and Food Allergy (TCMFA)

Allergy to Fish | Allergy to Shrimp | Allergy to Crab

Hong Kong
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Krabbe Disease (BioKrabbe) (BIOKRABBE)

Krabbe Disease

Egypt, Germany, India, Sri Lanka
Joanne Kurtzberg, MD
Duke University; Miltenyi Biotec, Inc.

Available

Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

Hematologic Malignancies | Immune Deficiencies | Inborn Errors of Metabolism Disorders

United States
New York Medical College

Completed

Human Placental-Derived Stem Cell Transplantation (HPDSC)

Mucopolysaccharidosis I | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Fucosidosis | Gaucher... and other conditions

United States
Marianne Barbu-Roth
Ecole Pratique des Hautes Etudes

Active, not recruiting

Early Intervention Based on Neonatal Crawling in Very Premature Infants Without Major Brain Damage (Premalocom1)

Prematurity

France
Magenta Therapeutics, Inc.

Completed

MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

Inherited Metabolic Disorders (IMD)

United States
Shire
PharmaNet; Nextrials, Inc.

Withdrawn

An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)

Leukodystrophy, Globoid Cell
University of Chicago
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Completed

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Krabbe Disease | Mucopolysaccharidosis Type II (MPS II) | Mucopolysaccharidosis Type I (MPS I) | Mucopolysaccharidosis Type III (MPS III) | Mucopolysaccharidosis Type VI (MPS VI)

United States
Masonic Cancer Center, University of Minnesota

Completed

HSCT for High Risk Inherited Inborn Errors

Wolman Disease | Adrenoleukodystrophy | Metachromatic Leukodystrophy | Sanfilippo Syndrome | GM1 Gangliosidosis | Globoid Cell Leukodystrophy | Tay Sachs Disease | Sandhoffs Disease | I-Cell Disease

United States
Novartis Pharmaceuticals

Withdrawn

Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)

Inherited Metabolic Disorders IMD
Wake Forest University
Medicis Pharmaceutical Corporation

Completed

Efficacy of Fluocinonide Cream 0.1% (Vanos(R)) in Reducing Itch in Subjects With Atopic Dermatitis

Atopic Dermatitis

United States
Simon Krabbe
Zealand University Hospital; University of Aarhus; University of Southern Denmark

Completed

Predicting Hospitalized Infection in Patients With Chronic Inflammatory Arthritis Treated With Biological Drugs

Rheumatoid Arthritis | Psoriatic Arthritis | Spondyloarthritis
Zymenex A/S

Completed

The Natural History of Infantile Globoid Cell Leukodystrophy

Infantile Globoid Cell Leukodystrophy

United States
Masonic Cancer Center, University of Minnesota

Completed

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Mucopolysaccharidosis | Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Krabbe Disease | Adrenoleukodystrophy (ALD) | Hunter Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Metachromatic Leukodystrophy (MLD) | Maroteaux-Lamy Syndrome

United States
Masonic Cancer Center, University of Minnesota

Terminated

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Sphingolipidoses | Niemann-Pick Disease, Type C | Krabbe Disease | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Hurler's Syndrome | Maroteaux-Lamy Syndrome | Wolman's Disease | Niemann-Pick Disease Type B

United States
Joanne Kurtzberg, MD
Aldagen

Completed

Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells

Anemia, Aplastic | MDS | Inborn Errors of Metabolism | Congenital Marrow Failure | Congenital Immunodeficiency Syndrome

United States
Joanne Kurtzberg, MD
Aldagen

Withdrawn

Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases (IUHST-001)

Lysosomal Storage Diseases | Inborn Errors of Metabolism

United States
Centre Hospitalier Universitaire Vaudois

Completed

Interscalene Brachial Plexus Block to Treat Pain After Clavicular Surgery

Acute Pain

Switzerland
University of Michigan Rogel Cancer Center
Novartis

Completed

Study of PTK787 in the Treatment of Patients With Non-Metastatic Androgen Independent Prostate Cancer

Prostate Cancer
Duke University

Completed

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

Hemoglobinopathies | Thalassemia | Lysosomal Storage Disease | Immunodeficiencies | Inborn Errors of Metabolism | Sickle Cell | Non Malignant Disorders | Congenital Marrow Failures

United States
Aldagen

Terminated

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases (UCBT-002)

Mucopolysaccharidosis | Inborn Errors of Metabolism | Lysosomal Storage Disorders | Inherited Metabolic Diseases | Peroxisomal Storage Diseases

United States
Mansoura University

Unknown

Endometrial Scratching During Laproscopic Ovarian Drilling in Subfertile PCOS Women (ESLOD)

Infertility | Polycystic Ovarian Syndrome

Egypt
Medical Research Foundation, The Netherlands

Completed

Quality Assessment Creatinines in Plasma and Urine

Chronic Kidney Disease

Netherlands

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