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111 Resultado(s)

Ensaio (s) clínico (s) nacional (is)

A Phase 1/2 Study of ARQ 092 in Patients with Overgrowth Diseases and/or Vascular Anomalies with Genetic Alterations of the PI3K/AKT Pathway IRCCS Ospedale Pediatrico Bambino Gesù - SEDE GIANICOLO Ambulatorio Polispecialistico per le Malattie Rare

ABACHAI: Safety and effectiveness of Abatacept (s.c.) in patients with CTLA4 insufficiency or LRBA deficiency (study of the GAIN-Network) CCI am Universitätsklinikum Freiburg Centrum für Chronische Immundefizienz

ABACHAI: Safety and effectiveness of Abatacept (s.c.) in patients with CTLA4 insufficiency or LRBA deficiency (study of the GAIN-Network) Medizinische Klinik und Poliklinik IV - Standort Ziemssenstraße Medizinische Klinik und Poliklinik IV

FIRSTMAPPP study: First international randomised phase II study in malignant progressive Pheochromocytoma and Paraganglioma - DE Zentrum Innere Medizin (ZIM) Medizinische Klinik und Poliklinik I - Endokrinologie und Diabetologie

A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia -DE- Institution: Information not provided - DE

Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study -DE- Institution: Information not provided - DE

ABACHAI: Safety and effectiveness of Abatacept (s.c.) in patients with CTLA4 insufficiency or LRBA deficiency (study of the GAIN-Network) Universitätsklinikum Schleswig-Holstein - Campus Kiel Klinik für Innere Medizin I

An Open-Label Extension Study of the Safety of Relacorilant in the Treatment of the Signs and Symptoms of Cushing Syndrome (Phase II-III) - AT Institution: Information not provided - AT

OLE-IEDAT: Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - BE UZ Leuven - Campus Gasthuisberg Laboratory of Pediatric Immunology

Treat Wolfram: A pivotal, international, randomised, double-blind, efficacy and safety trial of sodium valporate in paediatric and adult patients with Wolfram Syndrome (Phase II) - ES Institution: Information not provided - ES

OLE-IEDAT: Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study (Phase III) - ES Hospital Universitario La Paz Servicio de Neurología

BROADEN Study: A Randomized, Double-Blind, Placebo-Controlled, With an Open Label Extension, Phase 2/3 Study of ISIS 304801 Administered Subcutaneously to Patients With Familial Partial Lipodystrophy - US University of Michigan UH B1 H410/0028 University of Michigan

Phase II Study on the Management of Pediatric Craniopharyngioma by a Combination of Partial Surgical Resection, and Protontherapy CLCC Institut Curie Département d'Oncologie pédiatrique, adolescents et jeunes adultes

FIRSTMAPPP: First International Randomized Study in Malignant Progressive Pheochromocytoma and Paraganglioma - FR CLCC Institut Gustave Roussy Cancers endocriniens - Surrénales et Tumeurs neuro-endocrines

Does Arginine Enhance Galactose Oxidative Capacity in Classic Galactosemia: A Pilot Study AZM - Academisch Ziekenhuis Maastricht Afdeling Kindergeneeskunde

A Two-Period, Open-label Trial Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism Royal Manchester Children's Hospital Department of Endocrinology

Pulsed glucocorticoid replacement therapy for patients with adrenocortical insufficiency secondary to Addisonâs disease and congenital adrenal hyperplasia - the pulses study University of Bristol Henry Wellcome Laboratories for Integrative Neuroscience and Endocrinology (LINE)

An Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon for the Treatment of Children With Congenital Hyperinsulinism-GB NHS Greater Glasgow and Clyde Corporate HQ Greater Glasgow and Clyde NHS Board

REAL4: A Trial Comparing the Effect and Safety of Once Weekly Dosing of Somapacitan With Daily Norditropin® in Children With Growth Hormone Deficiency (Phase 3) - AT Institution: Information not provided - AT

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES Hospital Universitario Miguel Servet Servicio de Pediatría

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES Hospital Universitario Central de Asturias Servicio de Pediatría

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES Hospital Germans Trias I Pujol Servicio de Pediatría

An exploratory, open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystrophie (cALD) to assess the effect of MIN-102 treatment on the progression of cerebral lesions (Phase II) - ES Hospital Sant Joan de Déu Barcelona

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES Hospital Sant Joan de Déu Barcelona Servicio de Endocrinología

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES Hospital Universitari de Girona Dr. Josep Trueta Servicio de Pediatría

Effect of plasma exchange by albumin replacement in Adrenomyeloneuropathy: unicentric, single arm, proof of concept trial (Phase II) - ES IDIBELL - Instituto de Investigación Biomédica de Bellvitge Laboratorio de Enfermedades Neurometabólicas

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES Corporación Sanitaria Parc Taulí. Hospital de Sabadell Unidad de Endocrinología Pediátrica

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES Hospital Clínico Universitario de Santiago Servicio de Pediatría

REAL4: A Trial Comparing the Effect and Safety of Once Weekly Dosing of Somapacitan With Daily Norditropin® in Children With Growth Hormone Deficiency - ES Institution: Information not provided - ES

Setmelanotide (RM-493): Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity - ES Hospital Infantil Universitario Niño Jesús Servicio de Endocrinología, Crecimiento y Metabolismo

A Phase 3 trial of Setmelanotide (RM-493), a Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and AlstrC6m syndrome (AS) Patients with Moderate to Severe Obesity - ES Hospital Infantil Universitario Niño Jesús Servicio de Endocrinología, Crecimiento y Metabolismo

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES Complejo Hospitalario de Navarra Servicio de Pediatría

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES Hospital Universitario Araba. Sede Txagorritxu Servicio de Endocrinología pediátrica

ARISE: A phase 3 multicenter, randomized, placebo-controlled study of SBC-102 in patients with Lysosomal Acid Lipase Deficiency - DE Alexion Pharmaceuticals, Inc.

A Multicenter, Open-Label Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency (Phase II) - DE Alexion Pharmaceuticals, Inc.

ARISE: A Multicenter Study of SBC-102 (Sebelipase Alfa) in Patients With Lysosomal Acid Lipase Deficiency - CA Synageva BioPharma Corp

Role of GLP-1 In Congenital Hyperinsulinism: Effect of Exendin-(9-39) on Glucose Requirements To Maintain Euglycemia (Phase 1/2) - US The Children's Hospital of Philadelphia Center of Congenital Hyperinsulinism

Phase 2 Study of Orlistat and SLx-4090 for the treatment of Type 1 Hyperlipoproteinemia - US University of Texas Southwestern Medical Center Department of Internal Medicine

A Phase 1 Multi-Center Study to Assess the Efficacy and Safety of Abiraterone Acetate as Adjunctive Therapy in Pre-Pubescent Children With Classic 21-Hydroxylase Deficiency - US University of Texas Southwestern Medical Center

ARISE: A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency (Phase III) - FR CHU de Nancy - Hôpitaux de Brabois Service de médecine infantile

ARISE: A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency (Phase III) - FR Hôpital Necker-Enfants Malades Service des Maladies Métaboliques pédiatriques

OXYJEUNE : Effects of Intranasal Administrations of Oxytocin on Behavioural Troubles, Hyperphagia and Social Skills in Children With Prader-Willi Syndrome Aged From 3 to 12 Years (Phase III) CHU de Toulouse - Hôpital des Enfants Centre de référence du syndrome de Prader-Willi

ARISE (LAL-CL02): A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency (Phase III) - GR Institution: Information not provided - GR

A Multicenter, Open-Label Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency (Phase II) - NL Amsterdam UMC, locatie AMC Polikliniek Kindermetabole Ziekten

Dutch Growth Hormone Cohort study in children with Prader-Willi syndrome: Long-term effects on growth, body composition, activity level and psychosocial development Stichting Kind en Groei

ARISE (LAL-CL02): A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency (Phase III) - PL Institution: Information not provided - PL

An Open-Label Phase 2 Study to Evaluate PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma-GB Addenbrooke's Hospital Addenbrookes Hospital

An open label, 52-week, safety and tolerability extension to a randomized, double-blind, placebo controlled study of LCQ908 in subjects with Familial Chylomicronemia Syndrome (phase III) - UK Institution: Information not provided - UK

A randomized, double-blind, placebo controlled study to assess efficacy, safety and tolerability of LCQ908 in subjects with Familial Chylomicronemia Syndrome (phase III) - UK Institution: Information not provided - UK

A Phase III study of efficacy, safety and tolerability of Chronocort® compared with standard glucocorticoid replacement therapy in the treatment of congenital adrenal hyperplasia.-GB Institution: Information not provided - UK

Effects of exenatide on appetite and ghrelin levels in patients with Prader-Willi Syndrome Fazakerley Hospital University Hospital Aintree - Diabetes and Endocrinology

Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity University Hospital Birmingham Marshfield Clinic

ARISE (LAL-CL02): A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency (Phase III) - UK University of Birmingham University of Birmingham HQ

ARISE (LAL-CL02): A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency (Phase III) - CZ Institution: Information not provided - CZ

ODYSSEY HoFH: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Homozygous Familial Hypercholesterolemia (Phase 3) - AT Institution: Information not provided - AT

A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders (Phase 4) - AT Institution: Information not provided - AT

A Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia (Phase 3) - AT Institution: Information not provided - AT

An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia (Phase 3) - AT Institution: Information not provided - AT

A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia (Phase 3) - AT Institution: Information not provided - AT

A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders (Phase IV) - ES Hospital Universitari Vall d'Hebron

A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders (Phase IV) - ES Hospital Clínico Universitario de Santiago

UNLOCKED: A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care - ES Institution: Information not provided - ES

Study of Growth Hormone Use in Patients With Pseudohypoparathyroidism Type 1a (Subtype of Albright Hereditary Osteodystrophy) (Phase 2/3) - US Kennedy Krieger Institute

VEDROP: Study of the Absorption of Vitamin E water-soluble form (pegylated) in the familial hypocholesterolemia with chylomicron retention - FR CHU de Lyon HCL - Direction de la recherche clinique Direction de la recherche clinique

The Triac Trial: Thyroid Hormone Analog Therapy of Patients With Severe Psychomotor Retardation Caused by Mutations in the MCT8 Thyroid Hormone Transporter Erasmus MC - Erasmus Medisch Centrum Polikliniek Endocrinologie

The Triac Trial II: Effects of the Thyroid Hormone Analog Triac on the Neurocognitive Phenotype in Patients With Severe Psychomotor Retardation Caused by Mutations in the MCT8 Thyroid Hormone Transporter (Phase II) - NL Erasmus MC - Erasmus Medisch Centrum Polikliniek Endocrinologie

MODE: Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) (Phase II) - UK Institution: Information not provided - UK

Ensaio (s) clínico (s) multinacional (ais)

An Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon for the Treatment of Children With Congenital Hyperinsulinism Zealand Pharma A/S

ADVANCE: A randomized, double-blind, placebo-controlled, multinational, multicenter study with open-label treatment extension to assess the effect of MIN-102 on the progression of adrenomyeloneuropathy in male patients with X-linked adrenoleukodystrophy Institution: Information not provided - ES

OLE-IEDAT: Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study Institution: Information not provided - IT

The KHENERGYZE Study: A Phase IIb Double-blind, Randomised, Placebo-controlled, Multi-centre, Confirmative Three-way Cross-over Study on Cognitive Function With Two Doses of KH176 in Subjects With a Genetically Confirmed Mitochondrial DNA tRNALeu(UUR) m.3243A>G Mutation Khondrion B.V.

TAIN: Treatment of Adrenal Insufficiency in neonates- Development of a Hydrocortisone Preparation for the treatment of Adrenal Insufficiency in neonatesand infants TAIN Project, University of Sheffield TAIN Project

A Trial Investigating Efficacy and Safety of Once-weekly NNC0195-0092 (Somapacitan) Treatment Compared to Daily Growth Hormone Treatment (Norditropin® FlexPro®) in Growth Hormone Treatment naïve Pre-pubertal Children With Growth Hormone Deficiency Novo Nordisk A/S

An exploratory, open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystrophie (cALD) to assess the effect of MIN-102 treatment on the progression of cerebral lesions. Minoryx Therapeutics S.L.

MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study Institution: Information not provided - US

A Phase 1/2 Study of ARQ 092 in Patients with Overgrowth Diseases and/or Vascular Anomalies with Genetic Alterations of the PI3K/AKT Pathway
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE GIANICOLO

Ambulatorio Polispecialistico per le Malattie Rare

ABACHAI: Safety and effectiveness of Abatacept (s.c.) in patients with CTLA4 insufficiency or LRBA deficiency (study of the GAIN-Network)
CCI am Universitätsklinikum Freiburg

Centrum für Chronische Immundefizienz

ABACHAI: Safety and effectiveness of Abatacept (s.c.) in patients with CTLA4 insufficiency or LRBA deficiency (study of the GAIN-Network)
Medizinische Klinik und Poliklinik IV - Standort Ziemssenstraße

Medizinische Klinik und Poliklinik IV

FIRSTMAPPP study: First international randomised phase II study in malignant progressive Pheochromocytoma and Paraganglioma - DE
Zentrum Innere Medizin (ZIM)

Medizinische Klinik und Poliklinik I - Endokrinologie und Diabetologie

A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia -DE-
Institution: Information not provided - DE

Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study -DE-
Institution: Information not provided - DE

ABACHAI: Safety and effectiveness of Abatacept (s.c.) in patients with CTLA4 insufficiency or LRBA deficiency (study of the GAIN-Network)
Universitätsklinikum Schleswig-Holstein - Campus Kiel

Klinik für Innere Medizin I

An Open-Label Extension Study of the Safety of Relacorilant in the Treatment of the Signs and Symptoms of Cushing Syndrome (Phase II-III) - AT
Institution: Information not provided - AT

OLE-IEDAT: Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - BE
UZ Leuven - Campus Gasthuisberg

Laboratory of Pediatric Immunology

Treat Wolfram: A pivotal, international, randomised, double-blind, efficacy and safety trial of sodium valporate in paediatric and adult patients with Wolfram Syndrome (Phase II) - ES
Institution: Information not provided - ES

OLE-IEDAT: Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study (Phase III) - ES
Hospital Universitario La Paz

Servicio de Neurología

BROADEN Study: A Randomized, Double-Blind, Placebo-Controlled, With an Open Label Extension, Phase 2/3 Study of ISIS 304801 Administered Subcutaneously to Patients With Familial Partial Lipodystrophy - US
University of Michigan UH B1 H410/0028

University of Michigan

Phase II Study on the Management of Pediatric Craniopharyngioma by a Combination of Partial Surgical Resection, and Protontherapy
CLCC Institut Curie

Département d'Oncologie pédiatrique, adolescents et jeunes adultes

FIRSTMAPPP: First International Randomized Study in Malignant Progressive Pheochromocytoma and Paraganglioma - FR
CLCC Institut Gustave Roussy

Cancers endocriniens - Surrénales et Tumeurs neuro-endocrines

Does Arginine Enhance Galactose Oxidative Capacity in Classic Galactosemia: A Pilot Study
AZM - Academisch Ziekenhuis Maastricht

Afdeling Kindergeneeskunde

A Two-Period, Open-label Trial Evaluating the Efficacy and Safety of Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism
Royal Manchester Children's Hospital

Department of Endocrinology

Pulsed glucocorticoid replacement therapy for patients with adrenocortical insufficiency secondary to Addisonâs disease and congenital adrenal hyperplasia - the pulses study
University of Bristol

Henry Wellcome Laboratories for Integrative Neuroscience and Endocrinology (LINE)

An Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon for the Treatment of Children With Congenital Hyperinsulinism-GB
NHS Greater Glasgow and Clyde Corporate HQ

Greater Glasgow and Clyde NHS Board

REAL4: A Trial Comparing the Effect and Safety of Once Weekly Dosing of Somapacitan With Daily Norditropin® in Children With Growth Hormone Deficiency (Phase 3) - AT
Institution: Information not provided - AT

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES
Hospital Universitario Miguel Servet

Servicio de Pediatría

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES
Hospital Universitario Central de Asturias

Servicio de Pediatría

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES
Hospital Germans Trias I Pujol

Servicio de Pediatría

An exploratory, open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystrophie (cALD) to assess the effect of MIN-102 treatment on the progression of cerebral lesions (Phase II) - ES
Hospital Sant Joan de Déu Barcelona

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES
Hospital Sant Joan de Déu Barcelona

Servicio de Endocrinología

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES
Hospital Universitari de Girona Dr. Josep Trueta

Servicio de Pediatría

Effect of plasma exchange by albumin replacement in Adrenomyeloneuropathy: unicentric, single arm, proof of concept trial (Phase II) - ES
IDIBELL - Instituto de Investigación Biomédica de Bellvitge

Laboratorio de Enfermedades Neurometabólicas

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES
Corporación Sanitaria Parc Taulí. Hospital de Sabadell

Unidad de Endocrinología Pediátrica

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES
Hospital Clínico Universitario de Santiago

Servicio de Pediatría

REAL4: A Trial Comparing the Effect and Safety of Once Weekly Dosing of Somapacitan With Daily Norditropin® in Children With Growth Hormone Deficiency - ES
Institution: Information not provided - ES

Setmelanotide (RM-493): Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity - ES
Hospital Infantil Universitario Niño Jesús

Servicio de Endocrinología, Crecimiento y Metabolismo

A Phase 3 trial of Setmelanotide (RM-493), a Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and AlstrC6m syndrome (AS) Patients with Moderate to Severe Obesity - ES
Hospital Infantil Universitario Niño Jesús

Servicio de Endocrinología, Crecimiento y Metabolismo

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES
Complejo Hospitalario de Navarra

Servicio de Pediatría

A phase 3, open-label, randomized, multicenter, 12 months, efficacy and safety study of weekly mod-4023 compared to daily genotropin® therapy in pre-pubertal children with growth hormone deficiency - ES
Hospital Universitario Araba. Sede Txagorritxu

Servicio de Endocrinología pediátrica

ARISE: A phase 3 multicenter, randomized, placebo-controlled study of SBC-102 in patients with Lysosomal Acid Lipase Deficiency - DE
Alexion Pharmaceuticals, Inc.

A Multicenter, Open-Label Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency (Phase II) - DE
Alexion Pharmaceuticals, Inc.

ARISE: A Multicenter Study of SBC-102 (Sebelipase Alfa) in Patients With Lysosomal Acid Lipase Deficiency - CA
Synageva BioPharma Corp

Role of GLP-1 In Congenital Hyperinsulinism: Effect of Exendin-(9-39) on Glucose Requirements To Maintain Euglycemia (Phase 1/2) - US
The Children's Hospital of Philadelphia

Center of Congenital Hyperinsulinism

Phase 2 Study of Orlistat and SLx-4090 for the treatment of Type 1 Hyperlipoproteinemia - US
University of Texas Southwestern Medical Center

Department of Internal Medicine

A Phase 1 Multi-Center Study to Assess the Efficacy and Safety of Abiraterone Acetate as Adjunctive Therapy in Pre-Pubescent Children With Classic 21-Hydroxylase Deficiency - US
University of Texas Southwestern Medical Center

ARISE: A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency (Phase III) - FR
CHU de Nancy - Hôpitaux de Brabois

Service de médecine infantile

ARISE: A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency (Phase III) - FR
Hôpital Necker-Enfants Malades

Service des Maladies Métaboliques pédiatriques

OXYJEUNE : Effects of Intranasal Administrations of Oxytocin on Behavioural Troubles, Hyperphagia and Social Skills in Children With Prader-Willi Syndrome Aged From 3 to 12 Years (Phase III)
CHU de Toulouse - Hôpital des Enfants

Centre de référence du syndrome de Prader-Willi

ARISE (LAL-CL02): A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency (Phase III) - GR
Institution: Information not provided - GR

A Multicenter, Open-Label Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency (Phase II) - NL
Amsterdam UMC, locatie AMC

Polikliniek Kindermetabole Ziekten

Dutch Growth Hormone Cohort study in children with Prader-Willi syndrome: Long-term effects on growth, body composition, activity level and psychosocial development
Stichting Kind en Groei

ARISE (LAL-CL02): A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency (Phase III) - PL
Institution: Information not provided - PL

An Open-Label Phase 2 Study to Evaluate PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma-GB
Addenbrooke's Hospital

Addenbrookes Hospital

An open label, 52-week, safety and tolerability extension to a randomized, double-blind, placebo controlled study of LCQ908 in subjects with Familial Chylomicronemia Syndrome (phase III) - UK
Institution: Information not provided - UK

A randomized, double-blind, placebo controlled study to assess efficacy, safety and tolerability of LCQ908 in subjects with Familial Chylomicronemia Syndrome (phase III) - UK
Institution: Information not provided - UK

A Phase III study of efficacy, safety and tolerability of Chronocort® compared with standard glucocorticoid replacement therapy in the treatment of congenital adrenal hyperplasia.-GB
Institution: Information not provided - UK

Effects of exenatide on appetite and ghrelin levels in patients with Prader-Willi Syndrome
Fazakerley Hospital

University Hospital Aintree - Diabetes and Endocrinology

Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity
University Hospital Birmingham

Marshfield Clinic

ARISE (LAL-CL02): A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency (Phase III) - UK
University of Birmingham

University of Birmingham HQ

ARISE (LAL-CL02): A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency (Phase III) - CZ
Institution: Information not provided - CZ

ODYSSEY HoFH: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of Alirocumab in Patients With Homozygous Familial Hypercholesterolemia (Phase 3) - AT
Institution: Information not provided - AT

A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders (Phase 4) - AT
Institution: Information not provided - AT

A Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia (Phase 3) - AT
Institution: Information not provided - AT

An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia (Phase 3) - AT
Institution: Information not provided - AT

A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia (Phase 3) - AT
Institution: Information not provided - AT

A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders (Phase IV) - ES
Hospital Universitari Vall d'Hebron

A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders (Phase IV) - ES
Hospital Clínico Universitario de Santiago

UNLOCKED: A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care - ES
Institution: Information not provided - ES

Study of Growth Hormone Use in Patients With Pseudohypoparathyroidism Type 1a (Subtype of Albright Hereditary Osteodystrophy) (Phase 2/3) - US
Kennedy Krieger Institute

VEDROP: Study of the Absorption of Vitamin E water-soluble form (pegylated) in the familial hypocholesterolemia with chylomicron retention - FR
CHU de Lyon HCL - Direction de la recherche clinique

Direction de la recherche clinique

The Triac Trial: Thyroid Hormone Analog Therapy of Patients With Severe Psychomotor Retardation Caused by Mutations in the MCT8 Thyroid Hormone Transporter
Erasmus MC - Erasmus Medisch Centrum

Polikliniek Endocrinologie

The Triac Trial II: Effects of the Thyroid Hormone Analog Triac on the Neurocognitive Phenotype in Patients With Severe Psychomotor Retardation Caused by Mutations in the MCT8 Thyroid Hormone Transporter (Phase II) - NL
Erasmus MC - Erasmus Medisch Centrum

Polikliniek Endocrinologie

MODE: Modifying Orphan Disease Evaluation (MODE) Study: A Multicenter, Open-Label Study of the Effects of CER-001 on Plaque Volume in Subjects with Homozygous Familial Hypercholesterolemia (HoFH) (Phase II) - UK
Institution: Information not provided - UK

An Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon for the Treatment of Children With Congenital Hyperinsulinism
Zealand Pharma A/S

ADVANCE: A randomized, double-blind, placebo-controlled, multinational, multicenter study with open-label treatment extension to assess the effect of MIN-102 on the progression of adrenomyeloneuropathy in male patients with X-linked adrenoleukodystrophy
Institution: Information not provided - ES

OLE-IEDAT: Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study
Institution: Information not provided - IT

The KHENERGYZE Study: A Phase IIb Double-blind, Randomised, Placebo-controlled, Multi-centre, Confirmative Three-way Cross-over Study on Cognitive Function With Two Doses of KH176 in Subjects With a Genetically Confirmed Mitochondrial DNA tRNALeu(UUR) m.3243A>G Mutation
Khondrion B.V.

TAIN: Treatment of Adrenal Insufficiency in neonates- Development of a Hydrocortisone Preparation for the treatment of Adrenal Insufficiency in neonatesand infants
TAIN Project, University of Sheffield

TAIN Project

A Trial Investigating Efficacy and Safety of Once-weekly NNC0195-0092 (Somapacitan) Treatment Compared to Daily Growth Hormone Treatment (Norditropin® FlexPro®) in Growth Hormone Treatment naïve Pre-pubertal Children With Growth Hormone Deficiency
Novo Nordisk A/S

An exploratory, open-label, multicenter study in male pediatric patients with cerebral X-linked Adrenoleukodystrophie (cALD) to assess the effect of MIN-102 treatment on the progression of cerebral lesions.
Minoryx Therapeutics S.L.

MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study
Institution: Information not provided - US

Safety and dose finding study of different MOD-4023 dose levelscompared to daily r-hGH therapy in pre-pubertal growth hormonedeficient children
OPKO Biologics Ltd.

ZEPHYR: A Phase 2b/3 Study to Evaluate the Safety, Tolerability, and Effects of Livoletide (AZP-531), an Unacylated Ghrelin Analog, on Food-related Behaviors in Patients With Prader-Willi Syndrome
Millendo Therapeutics Ltd

LAL-CL01: An Open Label Multicenter Study to Evaluate the Safety, Tolerability and Pharmacokinetics of SBC-102 in Adult Patients With Liver Dysfunction Due to Lysosomal Acid Lipase Deficiency (Phase I)
Institution: Information not provided - UK

ARISE (LAL-CL02): A Multicenter, Randomized, Placebo-Controlled Study of SBC-102 in Patients With Lysosomal Acid Lipase Deficiency (Phase III)- coordination
Institution: Information not provided - UK

A Randomised, Controlled, Open-Label Parallel Arm Study of Safety, PK and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate) Oral Liquid and Sodium Phenylbutyrate in Phenylbutyrate Treatment Naïve Patients With Urea Cycle Disorders
Horizon Pharma Inc.