Clinical Trials on Emphysema Secondary to Congenital AATD

Total 255 results

Hospital Universitario Dr. Jose E. Gonzalez

Completed

Sterile Single-use vs Reused Polyvinylchloride Catheter For Intermittent Catheterization In Neurogenic Bladder (Catheters)

Neurogenic Bladder Due to Spina Bifida (Disorder)
Second Affiliated Hospital of Nanchang University

Enrolling by invitation

Superselective Adrenal Arterial Embolization for Bilateral Idiopathic Hyperaldosteronism: A Prospective Cohort Study (SAAE-BIH)

Primary Aldosteronism Due to Adrenal Hyperplasia (Bilateral)

China
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
Third Military Medical University

Not yet recruiting

Spatial Proteomics Profiles of Aldosterone-producing Adenoma and Unilateral Hyperplasia

Primary Aldosteronism Due to Aldosterone Producing Adenoma | Primary Aldosteronism Due to Adrenal Hyperplasia (Bilateral)

China
Regeneron Pharmaceuticals

Available

Expanded Access to REGN4461 for Patients With Lipodystrophy or Monogenic Obesity With Complications of Leptin Deficiency or Deficient Leptin Signaling

Partial Lipodystrophy | Generalized Lipodystrophy | Monogenic Obesity Due to LEP or LEPR Loss of Function
Noel Jabbour
National Institute on Deafness and Other Communication Disorders (NIDCD)

Terminated

Exercises for Improving Soft Palate and Eustachian Tube Function in Children With Ear Tubes With or Without Cleft Palate

Cleft Palate | Tube Disorders Eustachian | Velopharyngeal Incompetence Due to Cleft Palate | Velopharyngeal Insufficiency

United States
HemaQuest Pharmaceuticals Inc.

Terminated

Effects of HQK-1001 in Patients With Sickle Cell Disease

Sickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin S

United States, Lebanon, Egypt, Canada, Jamaica
HemaQuest Pharmaceuticals Inc.

Completed

A Study of HQK-1001 in Patients With Sickle Cell Disease

Sickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin S

United States, Lebanon, Canada, Egypt, Jamaica
Children's Cancer and Leukaemia Group

Unknown

Treatment Outcome and Quality of Life in Patients With Pediatric Extra-Cranial Germ Cell Tumors Previously Treated on Clinical Trial CCLG-GC-1979-01 or CCLG-GC-1989-01

Infertility | Ovarian Cancer | Sexual Dysfunction | Neurotoxicity | Long-term Effects Secondary to Cancer Therapy in Children | Gastrointestinal Complications | Urinary Complications | Pulmonary Complications | Extragonadal Germ Cell Tumor | Childhood Germ Cell Tumor

Ireland, United Kingdom
Noel Jabbour
National Institute on Deafness and Other Communication Disorders (NIDCD)

Not yet recruiting

Expiratory Muscle Strength Training for Hypernasal Speech in Children

Velopharyngeal Incompetence Due to Cleft Palate | Velopharyngeal Insufficiency | Palatopharyngeal Incompetence | Inadequate Velopharyngeal Closure | Hypernasality

United States
Oslo University Hospital
Centre for Appearance Research, University of the West of England, UK

Enrolling by invitation

Online Psychosocial Support for Young People With a Visible Difference: A Randomised Control Study (UNGFaceIT)

Burns | Cleft Lip and Palate | Craniofacial Abnormalities | Skin Condition | Other Conditions Leading to a Visible Difference

Norway
Axcella Health, Inc

Completed

To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease

Sickle Cell Disease | Anemia, Sickle Cell | Sickle Cell Anemia | To Assess the Safety and Tolerabiltiy of an Amino Acid Composition in Subjects With Sickle Cell Disease | Sickle Cell Disorders

United States
National Human Genome Research Institute (NHGRI)

Completed

Evaluation of Patients With Unresolved Chromosome Abnormalities

Microcephaly | Failure to Thrive | Abnormalities | Mental Retardation

United States
Imperial College London
Royal Brompton & Harefield NHS Foundation Trust; St George's, University of... and other collaborators

Recruiting

Low QRS Voltages in Young Healthy Individuals and Athletes

Dilated Cardiomyopathy | Sudden Cardiac Death | Sudden Cardiac Death Due to Cardiac Arrhythmia | Sudden Cardiac Arrest | Arrhythmogenic Right Ventricular Cardiomyopathy | Arrhythmogenic Left Ventricular Cardiomyopathy

United Kingdom
Changi General Hospital
Singapore General Hospital

Recruiting

Outcome of Patients With Primary Aldosteronism (PA_Outcome)

Primary Aldosteronism | Hypokalemia | Chronic Renal Disease | Cardiovascular Morbidity | Mineralocorticoid Excess | Adrenalectomy; Status | Primary Aldosteronism Due to Aldosterone Producing Adenoma | Primary Aldosteronism Due to Adrenal Hyperplasia (Bilateral) | Mineralocorticoid Antagonists [Aldosterone...

Singapore
Vanderbilt University Medical Center

Active, not recruiting

A Trial for Prevention of Recurrent Ischemic Priapism in Men With Sickle Cell Disease: A Pilot Study (PIN)

Priapism Due to Sickle Cell Disease

Nigeria
Rennes University Hospital

Completed

CACOLAC : Citrulline Administration in the Hospital Patient in Intensive Care for COVID-19 Acute Respiratory Distress Syndrome (CACOLAC)

ARDS Secondary to COVID-19 Pneumonia

France
Gruppo Italiano Malattie EMatologiche dell'Adulto

Recruiting

Investigation on the Incidence and Course of COVID-19 in Patients With Prior, Ongoing or de Novo ITP. Evaluation of the Impact of COVID-19 Pandemic on the Management of ITP

Covid19 | Immune Thrombocytopenia | ITP | ITP Secondary to Infection

Italy
Children's Cancer and Leukaemia Group

Unknown

Quality of Life in Patients Who Have Undergone Previous Treatment for Primitive Neuroectodermal Tumors

Brain and Central Nervous System Tumors | Cognitive/Functional Effects | Long-term Effects Secondary to Cancer Therapy in Children | Psychosocial Effects of Cancer and Its Treatment

United Kingdom, Ireland
Gesellschaft fur Padiatrische Onkologie und Hamatologie...

Completed

Observation of Young Patients Who Are Undergoing Surgery for Craniopharyngioma

Brain and Central Nervous System Tumors | Perioperative/Postoperative Complications | Long-term Effects Secondary to Cancer Therapy in Children | Psychosocial Effects of Cancer and Its Treatment | Weight Changes

Switzerland, Germany, Austria, Sweden
Heart of England NHS Trust
University of Warwick; University of Birmingham

Completed

Chest Wall Motion Analysis in Disease (CWM)

Lung Neoplasms | Pulmonary Disease, Chronic Obstructive | Surgery | Cystic Fibrosis | Mesothelioma | Empyema, Pleural | Pectus Excavatum | Pectus Carinatum

United Kingdom
Brigham and Women's Hospital
University of Michigan

Completed

Calcium Channel Blockade in Primary Aldosteronism (CCB-PA)

Primary Aldosteronism | Primary Aldosteronism Due to Adrenal Hyperplasia (Bilateral)

United States
OHSU Knight Cancer Institute
National Cancer Institute (NCI)

Terminated

Acetylcysteine, Mannitol, Combination Chemotherapy, and Sodium Thiosulfate in Treating Children With Malignant Brain Tumors

Brain and Central Nervous System Tumors | Long-term Effects Secondary to Cancer Therapy in Children | Drug/Agent Toxicity by Tissue/Organ | Bone Marrow Suppression

United States
Pontificia Universidad Catolica de Chile
Ferring Pharmaceuticals

Completed

Severe Aortic Stenosis and Acquired Von Willebrand´s Disease: The Impact of Desmopressin in Valve-Replacement Surgery

Severe Aortic Stenosis | Acquired Von Willebrand Disease Secondary to Severe Aortic Stenosis | Heye´s Syndrome

Chile
Alaa Mamdouh

Completed

Efficacy of an Arabic Articulatory Error Remediation Software Program in Patients With Velopharyngeal Valve Dysfunction:

Articulation Disorders | Velopharyngeal Insufficiency | Hypernasality Syndrome Due to Velopharyngeal Weakness

Egypt
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Brain Function in Young Patients Receiving Methotrexate for Acute Lymphoblastic Leukemia

Untreated Childhood Acute Lymphoblastic Leukemia | Childhood T Acute Lymphoblastic Leukemia | Neurotoxicity Syndrome | Childhood B Acute Lymphoblastic Leukemia | Cognitive Side Effects of Cancer Therapy | Psychological Impact of Cancer | Long-Term Effects Secondary to Cancer Therapy in Children

United States, Canada, Australia, New Zealand
Wuhan University
Peking University; Pfizer

Unknown

Efficacy and Safety of Ziprasidone to Treat Depressive Symptoms in Patients With Schizophrenia

The Primary Evaluation is the Change From Baseline at End of Study for CDSS Total Scores. | The Secondary Efficacy Evaluations Include: MADRS, PANSS Total Scores and Subscales and Responder Rate; CGI-S and CGI-I Scales. | Safety Evaluations Include: Laboratory Test Abnormalities; BARS... and other conditions
Oslo University Hospital

Enrolling by invitation

Physical Activity, Motor Competence, Pulmonary Function, and Quality of Life in Children With Severe Spinal Disease

Scoliosis | Scoliosis; Juvenile | Scoliosis; Idiopathic, Infantile | Scoliosis Neuromuscular | Scoliosis in Neurofibromatosis | Scoliosis; Congenital, Postural | Scoliosis in Skeletal Dysplasia | Scoliosis Associated With Other Conditions | Scoliosis; Congenital, Due to Bony Malformation

Norway
Children's Cancer and Leukaemia Group

Active, not recruiting

Observing Young Patients With Ependymoma Undergoing Standard Combination Chemotherapy

Brain and Central Nervous System Tumors | Ototoxicity | Cognitive/Functional Effects | Long-term Effects Secondary to Cancer Therapy in Children

United Kingdom
National Cancer Institute (NCI)

Recruiting

Imatinib to Increase RUNX1 Activity in Participants With Germline RUNX1 Deficiency

Inherited Bone Marrow Failure Syndrome | Familial Platelet Disorder With Predisposition to Myeloid Malignancies

United States
Novo Nordisk A/S

Completed

To Quantify the Range of Main Psychosocial Factors Affecting Patients and Caregivers in Their Daily Lives (HERO)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia B | Relatives to/Carers of Patients

Brazil
Novo Nordisk A/S

Completed

Survey Evaluating the Psychosocial Effects of Living With Haemophilia (HERO)

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia B | Relatives to/Carers of Patients

Spain, Canada, United Kingdom
Wake Forest University Health Sciences
National Cancer Institute (NCI)

Withdrawn

KeraStat Skin Therapy in Treating Radiation Dermatitis in Patients With Newly Diagnosed Stage 0-IIIA Breast Cancer

Stage IIIA Breast Cancer | Stage IA Breast Cancer | Stage IB Breast Cancer | Stage IIA Breast Cancer | Stage IIB Breast Cancer | Ductal Breast Carcinoma in Situ | Skin Reactions Secondary to Radiation Therapy

United States
Pharnext SA

Completed

Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A

Charcot-Marie-Tooth Disease | Hereditary Neuropathy With Liability to Pressure Palsies | Genetic Disorders

France
National Heart, Lung, and Blood Institute (NHLBI)
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Completed

National Registry of Genetically Triggered Thoracic Aortic Aneurysms and Cardiovascular Conditions (GenTAC)

Marfan Syndrome | Turner Syndrome | Ehlers-Danlos Syndrome | Loeys-Dietz Syndrome | Shprintzen-Goldberg Syndrome | FBN1, TGFBR1, TGFBR2, ACTA2 or MYH11 Genetic Mutation | Bicuspid Aortic Valve Without Known Family History | Bicuspid Aortic Valve With Family History | Bicuspid Aortic Valve With Coarctation and other conditions

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

Completed

Combination Chemotherapy in Treating Young Patients With Down Syndrome and Acute Myeloid Leukemia or Myelodysplastic Syndromes

Secondary Acute Myeloid Leukemia | 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 Myeloblastic... and other conditions

United States, Canada, Australia, Puerto Rico
Roswell Park Cancer Institute

Active, not recruiting

Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer

Acute Myeloid Leukemia | Polycythemia Vera | Myelofibrosis | Chronic Myelomonocytic Leukemia | Waldenstrom Macroglobulinemia | Acute Lymphoblastic Leukemia | Chronic Lymphocytic Leukemia | Secondary Acute Myeloid Leukemia | Sickle Cell Disease | Myelodysplastic Syndrome | Plasma Cell Myeloma | Chronic Granulomatous... and other conditions

United States
ProgenaBiome

Recruiting

A Non-Interventional Pilot Study to Explore the Role of Gut Flora in Autism

Autism Spectrum Disorder | Autistic Disorder | Autism | Autism, Akinetic | Autism; Atypical | Autism; Psychopathy | Autism Fragile Syndrome X | Autism With High Cognitive Abilities | Autism-Related Speech Delay | Autism, Susceptibility to, 6 | Autism Spectrum Disorder High-Functioning | Autistic Thinking | Autism... and other conditions

United States
Cure CMD

Recruiting

Congenital Muscle Disease Study of Patient and Family Reported Medical Information (CMDPROS)

Emery-Dreifuss Muscular Dystrophy | Congenital Myasthenic Syndrome | Limb-Girdle Muscular Dystrophy | Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency | Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy) and other conditions

United States
Royal Marsden NHS Foundation Trust

Unknown

Hyperbaric Oxygen Therapy in Treating Long-Term Gastrointestinal Adverse Effects Caused by Radiation Therapy in Patients With Pelvic Cancer

Sarcoma | Cervical Cancer | Testicular Germ Cell Tumor | Colorectal Cancer | Ovarian Cancer | Prostate Cancer | Bladder Cancer | Endometrial Cancer | Radiation Toxicity | Vaginal Cancer | Gastrointestinal Complications | Long-term Effects Secondary to Cancer Therapy in Adults

United Kingdom
Kartal Kosuyolu Yuksek Ihtisas Education and Research...
Trakya University

Completed

The Use of Midazolam and Remifentanil During Dialysis Access Procedures

Chronic Kidney Diseases | Patient Satisfaction | Arteriovenous Fistula | Adverse Reaction to Drug | Analgesic Adverse Reaction | Sedative Adverse Reaction | Midazolam Adverse Reaction
Soutien aux Actions contre les Maladies du Globule...
Novartis

Completed

"Association of Proteinuria and Progression of Kidney Dysfunction in Sickle Cell Disease"Disease (CSEG101A0FR01)

Change in Albumin to Creatinine Ratio and Glomerular Filtration Rate | Progression of Kidney Failure and or All-cause Mortality

France
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
RFM Renal Treatment Services

Unknown

Serum Sclerostin Levels, Cardiovascular Parameters and Carpal Tunnel Syndrome in Maintenance Hemodialysis Patients

Chronic Kidney Disease | Left Ventricular Hypertrophy | Renal Osteodystrophy

Turkey
Christopher Dvorak

No longer available

Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant

Acute Myeloid Leukemia | Leukocyte Disorders | Acute Lymphoblastic Leukemia | Chronic Myeloid Leukemia | Myelodysplastic Syndrome | Cytopenias | Immune Deficiency | Lymphomas | Bone Marrow Failure | Osteopetrosis | Hemoglobinopathy | Anemia Due to Intrinsic Red Cell Abnormality

United States
Sanford Health
National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators

Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)

Mitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditions

United States, Australia
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
National Human Genome Research Institute (NHGRI)

Recruiting

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

Metabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditions

United States
Centre Hospitalier Universitaire de Liege
Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaborators

Recruiting

Baby Detect : Genomic Newborn Screening

Congenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditions

Belgium
Roswell Park Cancer Institute

Completed

Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Primary Myelofibrosis | Polycythemia Vera | Chronic Myelomonocytic Leukemia | Recurrent Adult Acute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue | Nodal Marginal Zone B-cell Lymphoma | Recurrent Adult Burkitt Lymphoma and other conditions

United States

Clinical Trials on Emphysema Secondary to Congenital AATD

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