Clinical Trials on Hemophilia B, Congenital

Total 15421 results

Bellicum Pharmaceuticals

Terminated

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

Hemoglobinopathies | Hemophagocytic Lymphohistiocytosis | Metabolic Disorders | Primary Immune Deficiency Disorders | Inherited Bone Marrow Failure Syndrome

United States
University of Nebraska

Completed

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers (SLS)

Sjogren-Larsson Syndrome (SLS)

United States
University of Alabama at Birmingham
Lakeshore Foundation; YMCA of Greater Birmingham

Active, not recruiting

Movement-2-Music: Lakeshore Examination of Activity, Disability, and Exercise Response Study (M2M LEADERS)

Stroke | Multiple Sclerosis | Cerebral Palsy | Parkinson Disease | Traumatic Brain Injury | Spinal Cord Injuries | Spina Bifida

United States
Aldeyra Therapeutics, Inc.

Completed

A Study of Topical NS2 Cream to Treat Ichthyosis in Sjögren-Larsson Syndrome (SLS)

Sjögren-Larsson Syndrome

United States
Gillette Children's Specialty Healthcare

Completed

Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

Cerebral Palsy | Muscular Dystrophy | Osteopenia | Rett Syndrome | Spinal Muscular Atrophy | Spina Bifida

United States
University of Split, School of Medicine
General Hospital Zadar

Unknown

QoL and Stress in Parents of Children With Developmental Disabilities and Chronic Disease

Epilepsy | Cerebral Palsy | Diabetes Mellitus, Type 1 | Stress | Asthma | Chronic Disease | Autism Spectrum Disorder | Down Syndrome | Developmental Disability | Pervasive Developmental Disorder

Croatia
Novartis Pharmaceuticals

Completed

Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) (EXIST-2)

Lymphangioleiomyomatosis (LAM) | Tuberous Sclerosis Complex (TSC)

United States, United Kingdom, Germany, Italy, Russian Federation, Netherlands, Japan, Canada, Poland, France, Spain
Masonic Cancer Center, University of Minnesota

Recruiting

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditions

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI)

Completed

Biological Therapy in Treating Patients With Advanced Myelodysplastic Syndrome, Acute or Chronic Myeloid Leukemia, or Acute Lymphoblastic Leukemia Who Are Undergoing Stem Cell Transplantation

Polycythemia Vera | Essential Thrombocythemia | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) | Adult... and other conditions

United States
Motric Bio

Recruiting

Single and Multiple Ascending Dose Study of MTR-601 in Healthy Individuals

Stroke | Multiple Sclerosis | Cerebral Palsy | Spinal Cord Injuries | Dystonia | Muscle Spasticity | Hereditary Spastic Paraplegia | Hypertonia, Muscle

United States
Jazz Pharmaceuticals

Recruiting

Assessment of Adjunctive Cannabidiol Oral Solution (GWP42003-P) in Children With Tuberous Sclerosis Complex (TSC), Dravet Syndrome (DS), or Lennox-Gastaut Syndrome (LGS) Who Experience Inadequately-controlled Seizures

Seizure in Participants With Tuberous Sclerosis Complex | Seizure in Participants With Dravet Syndrome | Seizure in Participants With Lennox-Gastaut Syndrome

United States
Centre Hospitalier Universitaire Vaudois
Swiss National Science Foundation

Recruiting

Genetics of Reproductive Disorders (Including Kallmann Syndrome) and Cleft Lip and/or Palate

Kallmann Syndrome | Hypogonadotropic Hypogonadism | Hypothalamic Amenorrhea | Polycystic Ovarian Syndrome | Precocious Puberty | Cleft Lip and Palate | Cleft Palate | Cleft Lip

Switzerland
National Institutes of Health Clinical Center (CC)

Enrolling by invitation

Use of Tracking Devices to Locate Abnormalities During Invasive Procedures

Adenocarcinoma | Renal Cell Carcinoma | Prostate Cancer | Prostate Neoplasms | Von Hippel Lindau Disease

United States
Assistance Publique - Hôpitaux de Paris

Completed

The Primordial Dwarfisms: Diagnosis, Identification of the Molecular Basis of Seckel Syndrome and Microcephalic Osteodysplastic Primordial Dwarfism Type II (NANPIM)

Seckel Syndrome | Microcephalic Osteodysplastic Primordial Dwarfism Type II

France
Boston Children's Hospital

Completed

Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome

Smith-Lemli-Opitz Syndrome

United States
Forbes Porter, M.D.
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Completed

Simvastatin Therapy in Smith-Lemli-Opitz Syndrome

Smith-Lemli-Opitz Syndrome

United States
Medical University of Graz

Completed

Use of an SPF30 Sunscreen and an After-sun-lotion in Skin Cancer Risk Patients

Skin Cancer | Basal Cell Nevus Syndrome | Xeroderma Pigmentosum

Austria
National Cancer Institute (NCI)

Completed

Vorinostat With or Without Isotretinoin in Treating Young Patients With Recurrent or Refractory Solid Tumors, Lymphoma, or Leukemia

Unspecified Childhood Solid Tumor, Protocol Specific | Juvenile Myelomonocytic Leukemia | Recurrent Childhood Medulloblastoma | Childhood Atypical Teratoid/Rhabdoid Tumor | Childhood Acute Promyelocytic Leukemia (M3) | Childhood Chronic Myelogenous Leukemia | Recurrent Childhood Acute Lymphoblastic... and other conditions

United States
Rigshospitalet, Denmark
University College, London

Enrolling by invitation

EuroSIDA - Clinical and Virological Outcome of European Patients Infected With HIV (EuroSIDA)

End Stage Liver Disease | Cardiovascular Diseases | Fractures, Bone | Diabetes Mellitus | Renal Insufficiency | Hepatitis B | Hepatitis C | Kidney Failure, Chronic | Proteinuria | HIV | Coinfection | Acidosis, Lactic | AIDS

Spain, Switzerland, Germany, United Kingdom, Austria, Sweden, Finland, Israel, Denmark, France, Italy, Iceland, Belgium, Slovakia, Norway, Lithuania, Portugal, Poland, Croatia, Czechia, Greece, Hungary, Luxembourg, Argentina, Belarus, Bosnia... and more
RTI International
University of North Carolina, Chapel Hill

Enrolling by invitation

Parent and Infant Inter(X)Action Intervention (PIXI)

Tuberous Sclerosis | Down Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Rett Syndrome | Turner Syndrome | Williams Syndrome | Angelman Syndrome | Chromosome 22q11.2 Deletion Syndrome | Klinefelter Syndrome | Phelan-McDermid Syndrome | Dup15Q Syndrome | Smith Magenis Syndrome

United States
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia

Chronic Myelomonocytic Leukemia | Fanconi Anemia | Childhood Acute Erythroleukemia (M6) | Childhood Acute Megakaryocytic Leukemia (M7) | Childhood Acute Monoblastic Leukemia (M5a) | Childhood Acute Monocytic Leukemia (M5b) | Childhood Acute Myeloblastic Leukemia With Maturation (M2) | Childhood Acute... and other conditions

United States
TScan Therapeutics, Inc.

Recruiting

A Basket Study of Customized Autologous TCR-T Cell Therapies in Patients With Locally Advanced (Unresectable) or Metastatic Solid Tumors

Melanoma | Cervical Cancer | Head and Neck Cancer | HPV-Related Malignancy | Ovarian Cancer | HPV-Related Carcinoma | HPV-Related Cervical Carcinoma | HPV Positive Oropharyngeal Squamous Cell Carcinoma | Non-small Cell Carcinoma | HPV - Anogenital Human Papilloma Virus Infection | HPV-Related Adenocarcinoma | HPV-Related Adenosquamous Carcinoma and other conditions

United States
Institut National de la Santé Et de la Recherche...

Recruiting

IDMet (RaDiCo Cohort) (RaDiCo-IDMet) (IDMet)

Prader-Willi Syndrome | Angelman Syndrome | Beckwith-Wiedemann Syndrome | Pseudohypoparathyroidism | Silver Russell Syndrome | Transient Neonatal Diabetes Mellitus | Temple Syndrome | Kagami-Ogata Syndrome | Familial Precocious Puberty

France
Fundacion para la Investigacion Biomedica del Hospital...
University of Alcala; Hospital Universitario Ramon y Cajal; Instituto de Salud... and other collaborators

Recruiting

Prevalence of Hyperandrogenism in Type 1 Diabetes

Polycystic Ovary Syndrome | Hyperandrogenism | Type 1 Diabetes | Hirsutism | Oligomenorrhea | Ovulation Disorder

Spain
Joshua Mann, MPH
National Organization for Rare Disorders

Recruiting

MyVHL: Patient Natural History Study (MyVHL)

Von Hippel-Lindau Disease | Hereditary Leiomyomatosis and Renal Cell Cancer | Birt-Hogg-Dube Syndrome | SDHB Gene Mutation

United States
Washington University School of Medicine
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators

Recruiting

Wolfram Syndrome and WFS1-related Disorders International Registry and Clinical Study

Diabetes Mellitus | Deafness | Ataxia | Diabetes Insipidus | Wolfram Syndrome | Optic Nerve Atrophy

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
University of Valencia
Hospital Universitario La Fe; Hospital General Universitario de Valencia; Hospital...

Completed

Intervention in Chronic Pediatric Patients and Their Families. (FACTORADAPT)

Asthma | Type 1 Diabetes | Allergic Rhinitis | Cystic Fibrosis | Pulmonary Disease | Atopic Dermatitis | Food Allergy | Allergic Asthma | Rhinoconjunctivitis | Primary Ciliary Dyskinesia | Short Stature

Spain
Cure HHT
University of Colorado, Denver; Massachusetts General Hospital; Mayo Clinic; Children... and other collaborators

Recruiting

Comprehensive HHT Outcomes Registry of the United States (CHORUS) (CHORUS)

Arteriovenous Malformations | Epistaxis | Hereditary Hemorrhagic Telangiectasia | GastroIntestinal Bleeding | Telangiectasia | Vascular Malformation | Cerebral Arteriovenous Malformations

United States
Travere Therapeutics, Inc.

Recruiting

Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases (EPPIK)

Focal Segmental Glomerulosclerosis | Immunoglobulin A Nephropathy | Minimal Change Disease | Alport Syndrome | IgA Vasculitis

United States, Italy, Netherlands, Poland, Spain, United Kingdom, Germany, Sweden
Nemours Children's Clinic

Recruiting

Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases

Charcot-Marie-Tooth Disease | Motor Neuron Disease | Amyotrophic Lateral Sclerosis | Muscular Dystrophy | Spinal Muscular Atrophy | Neuromuscular Disease | Spinal Muscular Atrophy With Respiratory Distress 1 | Peroneal Muscular Atrophy

United States
Children's Oncology Group
National Cancer Institute (NCI)

Active, not recruiting

Combination Chemotherapy and Surgery in Treating Young Patients With Wilms Tumor

Stage I Kidney Wilms Tumor | Stage II Kidney Wilms Tumor | Stage III Kidney Wilms Tumor | Stage IV Kidney Wilms Tumor | Adult Kidney Wilms Tumor | Beckwith-Wiedemann Syndrome | Childhood Kidney Wilms Tumor | Diffuse Hyperplastic Perilobar Nephroblastomatosis | Rhabdoid Tumor of the Kidney | Stage V Kidney...

United States, Canada, Australia, New Zealand, Puerto Rico, Israel
Masonic Cancer Center, University of Minnesota

Terminated

Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases

Sickle Cell Disease | Thalassemia | Cerebral Adrenoleukodystrophy | Inherited Metabolic Disorders | High Risk Hematologic Disorders

United States
Rush University Medical Center
Emory University

Enrolling by invitation

Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities

Tuberous Sclerosis | Fragile X Syndrome | Rett Syndrome | Hamartoma Syndrome, Multiple | Angelman Syndrome | Creatine Deficiency, X-linked | Telomeric 22Q13 Monosomy Syndrome | Chromosome 15Q, Partial Deletion

United States
University of Virginia

Completed

Effects of Prebiotics on Gut Microbiome in Patients Undergoing HSCT (HCTDiet)

Acute Myeloid Leukemia | Myelodysplastic Syndromes | Multiple Myeloma | Hodgkin Disease | Hodgkin Lymphoma | Myelofibrosis | Acute Lymphoblastic Leukemia | Mantle Cell Lymphoma | Sickle Cell Disease | Non Hodgkin Lymphoma | Acute Lymphocytic Leukemia | Myeloproliferative Neoplasm

United States
Phoenix Children's Hospital

Completed

A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT

Myelodysplastic Syndromes | Vitamin D Deficiency | Pediatric Cancer | Aplastic Anemia | Stem Cell Transplant Complications | Sickle Cell Anemia in Children | Thalassemia in Children | Blood Disorder | Pediatric Acute Myeloid Leukemia | Pediatric Acute Lymphoid Leukemia

United States
Institute for Neurodegenerative Disorders

Completed

A PET Brain Imaging Study of mGluR5 in Subjects With Neuropsychiatric Conditions (FPEB)

Parkinson Disease | Alzheimer Disease | Mild Cognitive Impairment | Fragile X Syndrome | Huntington Disease | Autistic Spectrum Disorders

United States
Stanford University

Completed

Screening for Cervical Intraepithelial Neoplasia Using Self-collected Menstrual Blood

HPV - Anogenital Human Papilloma Virus Infection

United States
M.D. Anderson Cancer Center

Active, not recruiting

Combination Chemotherapy and Ponatinib Hydrochloride in Treating Patients With Acute Lymphoblastic Leukemia

Acute Lymphoblastic Leukemia | Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive | Philadelphia Chromosome Positive | B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1 | Untreated Adult Acute Lymphoblastic Leukemia | Accelerated Phase Chronic Myelogenous Leukemia,... and other conditions

United States
University of Arizona

Active, not recruiting

Haploidentical BMT With Post-Transplant Cyclophosphamide and Bendamustine

Lymphoma, Follicular | Myelodysplastic Syndromes | Burkitt Lymphoma | Acute Lymphoblastic Leukemia | Marginal Zone Lymphoma | Acute Myelogenous Leukemia | Mantle-Cell Lymphoma | Chronic Myelogenous Leukemia | Lymphoma, Hodgkin | Gray Zone Lymphoma | Lymphoma,Non-Hodgkin | Large Cell Lymphoma | High Risk Undifferentiated...

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI)

Completed

Fludarabine and Total-Body Irradiation Followed By Donor Stem Cell Transplant and Cyclosporine and Mycophenolate Mofetil in Treating HIV-Positive Patients With or Without Cancer

HIV Infection | Unspecified Childhood Solid Tumor, Protocol Specific | Primary Myelofibrosis | Polycythemia Vera | Essential Thrombocythemia | Stage I Multiple Myeloma | Stage II Multiple Myeloma | Stage III Multiple Myeloma | Chronic Myelomonocytic Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal... and other conditions

United States
National Cancer Institute (NCI)
Childrens Oncology Group (COG); LLS PedAL LLC

Recruiting

Highest Dose of Uproleselan in Combination With Fludarabine and Cytarabine for Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Mixed Phenotype Acute Leukemia Relapsed or Refractory and That Expresses E-selectin Ligand on the Cell Membrane

Down Syndrome | Recurrent Acute Myeloid Leukemia | Recurrent Myelodysplastic Syndrome | Refractory Acute Myeloid Leukemia | Refractory Myelodysplastic Syndrome | Recurrent Mixed Phenotype Acute Leukemia | Refractory Mixed Phenotype Acute Leukemia | Acute Myeloid Leukemia Post Cytotoxic Therapy | Myelodysplastic...

United States
Eunice Kennedy Shriver National Institute of Child...

Completed

Study of Smith-Lemli-Opitz Syndrome

Smith-Lemli-Opitz Syndrome

United States
University of Nebraska
University of Colorado, Denver; University of Pittsburgh; Children's Hospital...

Completed

Smith-Lemli-Opitz Syndrome and Cholic Acid

Smith-Lemli-Opitz Syndrome

United States
University of California, San Francisco

Completed

Trial of Bilateral Sagittal Split Osteotomy Induced Paresthesia Using Ultrasonic vs. Reciprocating Saw Instrumentation

Paresthesia | Nerve Injury | Fracture | Surgical Complication | Mandibular Hypoplasia | Mandibular Nerve Injuries | Mandibular Retrognathism | Mandibular Prognathism | Nerve Entrapments | Mandibular Hyperplasia

United States
Lebanese University

Completed

Comparative Study on the Efficacy of Ovarian Stimulation Protocols on the Success Rate of ICSI in Female Infertility (CS-EOSP-ICSI)

Polycystic Ovary Syndrome | Endometriosis | Ectopic Pregnancy | Premature Ovarian Failure | Female Infertility Endocrine | Female Infertility | Fibroids | Salpingitis | Hydrosalpinx | Female Infertility Due to Ovulatory Disorder | Female Infertility of Tubal Origin | Female Infertility Due to Tubal Block | Female... and other conditions

Lebanon
Hadassah Medical Organization

Withdrawn

GLP Analogs for Diabetes in Wolfram Syndrome Patients

Wolfram Syndrome | Diabetes Mellitus Associated With Genetic Syndrome
M.D. Anderson Cancer Center
National Cancer Institute (NCI)

Terminated

68Ga-DOTA-TOC PET/CT in Imaging Participants With Neuroendocrine Tumors

Meningioma | Neuroendocrine Neoplasm | Multiple Endocrine Neoplasia Type 1 | Von Hippel-Lindau Syndrome | Metastatic Well Differentiated Neuroendocrine Neoplasm | Somatostatin Positive Neoplastic Cells Present

United States
Mercy Research

Completed

Stereotactic Body Radiotherapy for Spine Tumors

Arteriovenous Malformations | Neurofibroma | Chordoma | Meningioma | Schwannoma | Spinal Metastases | Paragangliomas | Vertebral Metastases | Benign Spinal Tumors

United States
Eunice Kennedy Shriver National Institute of Child...

Completed

Characterization of WAGR Syndrome and Other Chromosome 11 Gene Deletions

Urogenital Abnormalities | Mental Retardation | Wilm's Tumor | WAGR Syndrome | Aniridia

United States

Clinical Trials on Hemophilia B, Congenital

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