Search clinical trials for: Congenital multiple pituitary hormone deficiency

Total 80840 results

UK Kidney Association

Recruiting

National Registry of Rare Kidney Diseases (RaDaR)

Vasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditions

United Kingdom
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
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
Odense University Hospital
Aarhus University Hospital; Rigshospitalet, Denmark; Aalborg University Hospital

Active, not recruiting

Incidence of cCPHD in Denmark - a National Observational Study (IcCPHD)

Combined Pituitary Hormone Deficiency Genetic Form | Combined Pituitary Hormone Deficiency

Denmark
Universitaire Ziekenhuizen KU Leuven
KU Leuven

Unknown

PREPL in Health and Disease (PHD)

Obesity | Healthy Volunteers | Prader-Willi Syndrome | Muscle Hypotonia | Hypotonia-Cystinuria Syndrome | Dwarfism, Growth Hormone Deficiency

Belgium
Ospedale San Raffaele

Recruiting

Tirzepatide Monotherapy in Patients With Wolfram Syndrome Type 1

Wolfram Syndrome

Italy
University of Minnesota

Recruiting

Natural History Study for DNA Repair Disorders

DNA Repair Disorder | Xeroderma Pigmentosum | Cockayne Syndrome | Trichothiodystrophy

United States
Mayo Clinic
Hopital du Sacre-Coeur de Montreal

Recruiting

PB to Treat Hereditary Nephrogenic Diabetes Insipidus, ADPKD Treated With Tolvaptan, and Severely Polyuric Patients With Previous Lithium Administration (SerendipityPB1)

Autosomal Dominant Polycystic Kidney Disease | Nephrogenic Diabetes Insipidus | Acquired Nephrogenic Diabetes Insipidus | Congenital Nephrogenic Diabetes Insipidus

United States
University Hospital, Strasbourg, France

Recruiting

Growth Curves in Cockayne Syndrome Type 1 and Type 2 (Curves-CS)

Cockayne Syndrome, Type I and II

France
Centre d'Etude des Cellules Souches
Genethon

Recruiting

Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome (AUDIOWOLF)

Wolfram Syndrome

France, Spain
Nemours Children's Clinic
Potentials Foundation; Walking with Giants Foundation

Recruiting

Primordial Dwarfism Registry

MOPDII | RNU4atac-opathy (e.g. MOPDI/III, Lowry-Wood Syndrome, Roifman Syndrome) | Meier-Gorlin Syndrome | Saul-Wilson Syndrome | Ligase 4 Syndrome | Microcephalic Primordial Dwarfism

United States
dr. Laura C. G. de Graaff-Herder

Recruiting

GROWing Up With Rare GENEtic Syndromes (GROW UR GENES)

Disorders of Sex Development | Congenital Adrenal Hyperplasia | Tuberous Sclerosis | Kallmann Syndrome | Prader-Willi Syndrome | Neurofibromatosis | Rett Syndrome | 22q11 Deletion Syndrome | Turner Syndrome | Noonan Syndrome | Allan-Herndon-Dudley Syndrome | Saethre-Chotzen Syndrome | Congenital Hypopituitarism | Cornelia... and other conditions

Netherlands
dr. Laura C. G. de Graaff-Herder

Not yet recruiting

Growing up With the Young Endocrine Support System (YESS!) (YESS)

Congenital Adrenal Hyperplasia | Hypogonadotropic Hypogonadism | Growth Hormone Deficiency | Turner Syndrome | Addison's Disease | Klinefelter Syndrome | Combined Pituitary Hormone Deficiency | Androgen Insensitivity Syndrome | Thyroid Dysgenesis

Spain, United Kingdom, Belgium, Netherlands
McGill University Health Centre/Research Institute...

Recruiting

Monogenic Diabetes Misdiagnosed as Type 1 (ADDAM)

Diabetes Mellitus, Type 1 | Wolfram Syndrome | Neonatal Diabetes | Monogenic Diabetes | Maturity-onset Diabetes in the Young (MODY) | Wolcott-Rallison Syndrome | Mitochondrial Diabetes

Canada
St. Jude Children's Research Hospital

Recruiting

Familial Investigations of Childhood Cancer Predisposition (SJFAMILY)

Pancreatic Cancer | Hodgkin Lymphoma | Lynch Syndrome | Tuberous Sclerosis | Fanconi Anemia | AML | Non Hodgkin Lymphoma | Familial Adenomatous Polyposis | Acute Leukemia | Nevoid Basal Cell Carcinoma Syndrome | Neurofibromatosis Type 1 | Neuroblastoma | Retinoblastoma | MDS | Rhabdomyosarcoma | Von Hippel-Lindau Disease | Adrenocortical Carcinoma 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
University Hospital, Strasbourg, France

Recruiting

Metabolic Study of Cockayne Syndrome (METABO-CS)

Cockayne Syndrome

France
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
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
Washington University School of Medicine

Recruiting

Immune Disorder HSCT Protocol

Severe Combined Immunodeficiency | Chronic Granulomatous Disease | DiGeorge Syndrome | Hemophagocytic Lymphohistiocytosis | Wiskott-Aldrich Syndrome | X-linked Lymphoproliferative Syndrome | X-linked Agammaglobulinemia | Chediak-Higashi Syndrome | IPEX | Autoimmune Lymphoproliferative Syndrome | Common Variable... and other conditions

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
Massachusetts General Hospital

Recruiting

Kisspeptin Administration in the Adult

PCOS | Kallmann Syndrome | Hypogonadotropic Hypogonadism | Polycystic Ovarian Syndrome | Hyperprolactinemia | GnRH Deficiency

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

Completed

Defining the Genetic Basis for the Development of Primary Pigmented Nodular Adrenocortical Disease (PPNAD) and the Carney Complex

Pituitary Adenoma | Cushing's Syndrome | Carney Complex | Primary Pigmented Nodular Adrenocortical Disease | Peutz-Jeghers Syndrome

United States
Amylyx Pharmaceuticals Inc.

Active, not recruiting

AMX0035 in Adult Patients With Wolfram Syndrome

Wolfram Syndrome

United States
Sanguine Biosciences

Completed

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

Depression | Melanoma | Epilepsy | Lymphoma | Multiple Sclerosis | Kidney Cancer | Cervical Cancer | Diabetes Mellitus | Leukemia | Breast Cancer | Chronic Kidney Diseases | Chronic Obstructive Pulmonary Disease | Parkinson's Disease | Systemic Lupus Erythematosus | Multiple Myeloma | Hepatitis B | Insomnia | Colorectal Cancer | HIV/AID... and other conditions

United States
Washington University School of Medicine

Enrolling by invitation

I-Tracking Neurodegeneration in Early Wolfram Syndrome (I-TRACK)

Wolfram Syndrome

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

Completed

A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome

Diabetes Mellitus | Ataxia | Wolfram Syndrome | Optic Nerve Atrophy

United States
National Cancer Institute (NCI)

Active, not recruiting

Examination of Clinical and Laboratory Abnormalities in Patients With Defective DNA Repair: Xeroderma Pigmentosum, Cockayne Syndrome, or Trichothiodystrophy

Skin Neoplasms | Xeroderma Pigmentosum | Cockayne Syndrome | Trichothiodystrophy Syndromes | Genodermatosis

United States
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
Rhythm Pharmaceuticals, Inc.

Completed

Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Patients With Specific Gene Defects in the MC4R Pathway

Bardet-Biedl Syndrome | POMC Deficiency

Netherlands, United States, Germany, Canada, Puerto Rico, United Kingdom
Haukeland University Hospital
Karolinska Institutet; University Hospitals Bristol and Weston NHS Foundation... and other collaborators

Completed

Dynamic Hormone Diagnostics in Endocrine Disease (ultradian)

Congenital Adrenal Hyperplasia | Acromegaly | Growth Hormone Deficiency | Cushing Syndrome | Primary Hyperaldosteronism | Adrenal Insufficiency

Norway, Sweden, Greece, United Kingdom
University of Birmingham

Active, not recruiting

Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome

Wolfram Syndrome

France, United Kingdom, Poland, Spain
Faculty Hospital Kralovske Vinohrady

Completed

The Epidemiology, Management and Comorbidities in Alopecia Areata in Czech Republic

Infections | Multiple Sclerosis | Autoimmune Diseases | Diabetes Mellitus, Type 1 | Rheumatoid Arthritis | Vaccine Reaction | Inflammatory Bowel Diseases | Psoriasis | Crohn Disease | Ulcerative Colitis | Down Syndrome | Atopic Dermatitis | Vitiligo | Alopecia Areata | COVID-19 Pandemic | Ankylosing Spondylitis | Lupus Erythematosus and other conditions

Czechia
Massachusetts General Hospital

Completed

Investigation of the Genetic Causes of Kallmann Syndrome and Reproductive Disorders

Kallmann Syndrome | Hypogonadotropic Hypogonadism | Puberty, Precocious | Puberty, Delayed | GnRH Deficiency

United States
Hadassah Medical Organization

Withdrawn

GLP Analogs for Diabetes in Wolfram Syndrome Patients

Wolfram Syndrome | Diabetes Mellitus Associated With Genetic Syndrome
Stanford University

Completed

Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study

Epidermolysis Bullosa Simplex | Epidermolysis Bullosa Simplex Kobner | Weber-Cockayne Syndrome

United States
University Hospital, Toulouse

Completed

Is There a Sensibility Increased in the Growth Hormone at Child With Prader-Willi Syndrome?

Prader-Willi Syndrome | Growth Hormone Deficiency

France
Mayo Clinic

Completed

Spontaneous Coronary Artery Dissection (SCAD) and Autoimmunity

Multiple Sclerosis | Rheumatoid Arthritis | Systemic Lupus Erythematosus | Uveitis | Vasculitis | Psoriasis | Graves Disease | Systemic Sclerosis | Celiac Disease | Crohn Disease | Type 1 Diabetes Mellitus | Myasthenia Gravis | Ulcerative Colitis | Dermatomyositis | Polymyositis | Vitiligo | Hepatitis, Autoimmune | Polymyalgia... and other conditions

United States
Novo Nordisk A/S

Completed

Investigation of Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Multiple Doses of Somapacitan in Subjects With Various Degrees of Impaired Renal Function Compared to Subjects With Normal Renal Function

Growth Hormone Disorder | Growth Hormone Deficiency in Children | Adult Growth Hormone Deficiency

Germany
Roswell Park Cancer Institute

Completed

NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders

Lymphoma | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Fanconi Anemia | Multiple Myeloma and Plasma Cell Neoplasm | Myelodysplastic/Myeloproliferative Diseases | Aplastic Anemia

United States
Novo Nordisk A/S

Completed

Investigation of Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Multiple Doses of Somapacitan in Subjects With Mild and Moderate Degrees of Hepatic Impairment Compared to Subjects With Normal Hepatic Function.

Growth Hormone Disorder | Growth Hormone Deficiency in Children | Adult Growth Hormone Deficiency

Slovakia
University Hospital, Angers
University of Angers, Angers

Unknown

Neuropsychological Assessment of Children and Adolescents With Turner Syndrome (ENEAST)

Isolated Growth Hormone Deficiency | Turner Syndrome | Pediatrics | Social Cognition | Cognitive Functions
Washington University School of Medicine
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Completed

Tracking Neurodegeneration in Early Wolfram Syndrome (TRACK)

Diabetes Mellitus | Type 1 Diabetes | Diabetes Insipidus | Wolfram Syndrome

United States
Merck KGaA, Darmstadt, Germany

Completed

Predictive Markers in Growth Hormone Deficiency (GHD) and Turner Syndrome (TS) Children Treated With SAIZEN®

Growth Hormone Deficiency | Turner Syndrome

Argentina, Australia, Austria, Canada, France, Germany, Italy, Norway, Russian Federation, Singapore, Spain, Sweden, United Kingdom
Masonic Cancer Center, University of Minnesota
National Cancer Institute (NCI)

Terminated

Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer

Lymphoma | Myelodysplastic Syndromes | Leukemia | Chronic Myeloproliferative Disorders | Fanconi Anemia | Multiple Myeloma and Plasma Cell Neoplasm | Myelodysplastic/Myeloproliferative Diseases | Graft Versus Host Disease | Diamond-blackfan Anemia

United States
Premier Specialists, Australia

Unknown

Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study

Epidermolysis Bullosa Simplex | Epidermolysis Bullosa Simplex Kobner | Weber-Cockayne Syndrome

Australia
Novo Nordisk A/S

Completed

Observational Prospective Study on Patients Treated With Norditropin®

Chronic Kidney Disease | Turner Syndrome | Noonan Syndrome | Chronic Renal Insufficiency | Growth Hormone Disorder | Growth Hormone Deficiency in Children | Adult Growth Hormone Deficiency | Genetic Disorder | Foetal Growth Problem | Small for Gestational Age

Israel, Saudi Arabia, Spain, Argentina, Russian Federation, Germany, Netherlands, Italy, Belgium, United Kingdom, Slovenia, Ireland, Sweden, Denmark, Hungary, Finland, Norway, France, Czechia, Lithuania, Luxembourg, Serbia, Switzerland
Novo Nordisk A/S

Completed

Evaluating Acceptance of New Liquid Somatropin Formulation in Children With Growth Hormone Deficiency

Turner Syndrome | Growth Hormone Disorder | Growth Hormone Deficiency in Children | Genetic Disorder

Israel
Hadassah Medical Organization

Unknown

Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone and Incretin Based Therapy

Diabetes Mellitus | Iron Metabolism Disorders | Optic Atrophy | Sensorineural Hearing Loss | Platelet Dysfunction | Gastroduodenal Ulcer

Israel
Novo Nordisk A/S

Completed

Non-interventional Study of Patients Using Norditropin® for Growth Hormone Deficiency or Turner Syndrome (NordiPAD)

Turner Syndrome | Growth Hormone Disorder | Growth Hormone Deficiency in Children | Genetic Disorder

Japan

Search clinical trials for: Congenital multiple pituitary hormone deficiency

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