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Sperimentazioni cliniche nazionali

A Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE) -AU Institution: Information not provided - AU

A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency -AU Institution: Information not provided - AU

COMPASS: A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II -AU Institution: Information not provided - AU

MitoFibrate CT1: Clinical study for the assessment of safety and efficacy of Bezafibrate as a drug therapy for patients with mitochondrial myopathies (phase II) - AT Universitätsklinikum der Paracelsus Medizinischen Privatuniversität Universitätsklinik für Kinder- und Jugendheilkunde

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 Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD) - AT Institution: Information not provided - AT

Phase III Clinical Trial for CPX-351 in Myeloid Leukemia in Children with Down Syndrome 2018 - AT Institution: Information not provided - AT

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study To Examine The Efficacy And Safety Of ZX008 In Subjects With CDKL5 Deficiency Disorder Followed By An Open-Label Extension - AT Institution: Information not provided - AT

SIDEROS: A Phase III Double-blind, Randomized, Placebo-Controlled Study Assessing the Efficacy, Safety and Tolerability of Idebenone in Patients With Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - AT Gottfried von Preyer'sches Kinderspital Abteilung für Kinder- und Jugendheilkunde

SIDEROS-E: A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study - AT Gottfried von Preyer'sches Kinderspital Abteilung für Kinder- und Jugendheilkunde

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD) - AT Gottfried von Preyer'sches Kinderspital Abteilung für Kinder- und Jugendheilkunde

Fenfluramine as anti-epilepticum in Dravet syndrome - BE Institution: Information not provided - BE

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study To Examine The Efficacy And Safety Of ZX008 In Subjects With CDKL5 Deficiency Disorder Followed By An Open-Label Extension - BE Institution: Information not provided - BE

CUV152: A Proof of Concept, Phase IIa, Open Label Study to Evaluate the Safety and Efficacy of Subcutaneous Implants of Afamelanotide in Patients with Xeroderma Pigmentosum C and V (XPC and XPV) - BE Institution: Information not provided - BE

TAMDMD (Tamoxifen in Duchenne muscular dystrophy): Multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial - BE Cliniques Universitaires de Bruxelles - Hôpital Erasme Centre de Référence Neuromusculaire Erasme-HUDERF

EXIST-LT: Long-term follow-up study to monitor the growth and development of pediatric patients previously treated with everolimus in study CRAD001M2301- BE Universitair Ziekenhuis Brussel Pediatric neurology / Neurologie Kinderen

STELLAR: A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene -BE Ghent University Hospital - UZ Gent Department of Ophthalmology/Dienst Oogheelkunde - UZ Gent

An open-label safety, tolerability and pharmacokinetics study of Eteplirsen in young patients with Duchenne dystrophy amenable to exon 51 skipping - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

Long-term, open-label extension study for patients with Duchenne muscular dystrophy enrolled in clinical trials evaluating Casimersen or Golodirsen - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

MOMENTUM: A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD) - BE Ghent University Hospital - UZ Gent NMRC UZ Gent

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

Add-on Therapy With Low Dose Fenfluramine in Lennox Gastaut Epilepsy UZ Leuven - Campus Gasthuisberg Kinderen neurologie / Paediatric Neurology

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - BE UZ Leuven - Campus Gasthuisberg Kinderen neurologie / Paediatric Neurology

A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophy - BE UZ Leuven - Campus Gasthuisberg NMRC (afdeling kinderen) - UZ Leuven

A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy - BE UZ Leuven - Campus Gasthuisberg NMRC (afdeling kinderen) - UZ Leuven

Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies -BE UZ Leuven - Campus Gasthuisberg NMRC (afdeling kinderen) - UZ Leuven

A Long-Term Study to Assess the Ongoing Safety and Efficacy of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency - BE UZ Leuven - Campus Gasthuisberg Centrum metabole ziekten - UZ Leuven

Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients (Phase I-II) London Health Sciences Centre Children's Hospital

PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies (Phase II-III) Institution: Information not provided - CA

MOVE Trial: A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) -GB Institution: Information not provided - CA

A Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE) -CA Institution: Information not provided - CA

A Single-center, Un-controlled, Open-labeled Trial of the Long-term Safety of Lamazym Aftercare Treatment of Subjects With Alpha-Mannosidosis Whom Previously Participated in Lamazym Trials Institution: Information not provided - DK

EXIST-2: A Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) - ES (Phase III) Institution: Information not provided - FR

A multi-center, un-controlled, open-labeled trial of the long-term safety of Lamazym aftercare treatment of subjects with alpha-Mannosidosis who previously participated in Lamazym-trials (Phase III) - FR Institution: Information not provided - FR

A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy-FR Institution: Information not provided - FR

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) -FR Institution: Information not provided - FR

An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT) -FR Institution: Information not provided - FR

A Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE) -FR Institution: Information not provided - FR

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study To Examine The Efficacy And Safety Of ZX008 In Subjects With CDKL5 Deficiency Disorder Followed By An Open-Label Extension -FR Institution: Information not provided - FR

A Randomized, Double-blind, Placebo-controlled, Multi-center, 2-part, Phase 2 Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome -FR Institution: Information not provided - FR

A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency -FR Institution: Information not provided - FR

COMPASS: A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II -FR Institution: Information not provided - FR

ESSENCE: A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - FR Institution: Information not provided - FR

STELLAR: A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene -FR Centre hospitalier national d'ophtalmologie des Quinze-Vingts Centre de Référence REFERET

FancoMob : Pilot Study Assessing the Feasibility of CD34+ Cells Mobilization and Collection After Treatment With G-CSF and Plerixafor in Patients With Fanconi Anemia for Subsequent Treatment by Gene Therapy (Phase I-II) Hôpital Necker-Enfants Malades Service d'Hématologie adulte

A Phase 2, Open-Label Extension, Efficacy and Safety Study of a RARy Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects With Fibrodysplasia Ossificans Progressiva (FOP) - FR Hôpital Necker-Enfants Malades Service de Génétique Moléculaire

A Phase 2, Open-Label, Efficacy and Safety Study of an RARy Specific Agonist (Palovarotene) to Prevent Heterotopic Ossification in Subjects With Fibrodysplasia Ossificans Progressiva (FOP) Hôpital Necker-Enfants Malades Service de Génétique Moléculaire

A Phase 2a Study of TPN-101 in Patients With Aicardi-Goutières Syndrome (AGS) -FR IMAGINE - Institut des Maladies Génétiques Laboratoire de neurogénétique et neuroinflammation

ACTHYF: Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children - FR Institut Jérôme Lejeune

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

Safety and Efficacy of a Unilateral Subretinal Administration of HORA PDE6B in Patients with Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression (Phase I-II) CHU de Nantes - Hôtel Dieu Centre d'investigation clinique (CIC)

IRIS 2: Compensation for Blindness With the Intelligent Retinal Implant System (IRIS V2) in Patients With Retinal Dystrophy - FR CHU de Nantes - Hôtel Dieu Service d'ophtalmologie

Effectiveness and safety of mTOR-inhibitor in patients with tuberous sclerosis complex-KD Universitätsklinikum Heidelberg

A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency -DE Universitätsklinikum Heidelberg

PDE6A gene therapy for retinitis pigmentosa (RD-Cure Gene Therapy Trial) Department für Augenheilkunde Tübingen Universitäts-Augenklinik

STOPFOP: Saracatinib Trial TO Prevent FOP. A 6-month double blind randomized controlled trial of AZD0530 versus placebo, followed by a 12 month open label extension phase -DE Klinikum Garmisch-Partenkirchen GmbH Abteilung für Kinder- & Jugendmedizin

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

Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies (Phase III) -DE- Institution: Information not provided - DE

MOVE TRIAL: A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) -DE- Institution: Information not provided - DE

CUV156: A Proof of Concept, Phase IIa, Open Label Study to Evaluate the Safety and Efficacy of Subcutaneous Implants of Afamelanotide in Patients with Xeroderma Pigmentosum (XP) Institution: Information not provided - DE

A Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE) -DE Institution: Information not provided - DE

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study To Examine The Efficacy And Safety Of ZX008 In Subjects With CDKL5 Deficiency Disorder Followed By An Open-Label Extension - DE Institution: Information not provided - DE

COMPASS: A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II - DE Institution: Information not provided - DE

TAMDMD (Tamoxifen in Duchenne muscular dystrophy): Multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial -DE- DRK Kliniken Berlin Westend Klinik für Kinder- und Jugendmedizin

COMPASS: CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren - DE UKE - Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study -DE UKE - Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin

EPIDYS study: Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy -DE UKE - Universitätsklinikum Hamburg-Eppendorf Klinik und Poliklinik für Kinder- und Jugendmedizin

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 -DE- Universitätsklinikum Frankfurt Schwerpunkt Allergologie, Pneumologie und Mukoviszidose

An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT) -DE Universitätsmedizin Göttingen Klinik für Kinder- und Jugendmedizin

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - DE Universitätsklinikum Essen Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

TAMDMD (Tamoxifen in Duchenne muscular dystrophy): Multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial -DE- Universitätsklinikum Essen Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

EPIDYS study: Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy -DE Universitätsklinikum Essen Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy - DE Universitätsklinikum Essen

FFA-MAE: A proof of concept and assessment of maximal effect study with low dose Fenfluramine as add-on therapy in Myoclonic Astatic Epilepsy (Doose-Syndrome) Universitätsklinikum Schleswig-Holstein - Campus Kiel Klinik für Kinder- und Jugendmedizin II : Neuropädiatrie und Sozialpädiatrie

A Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE) -AU
Institution: Information not provided - AU

A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency -AU
Institution: Information not provided - AU

COMPASS: A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II -AU
Institution: Information not provided - AU

MitoFibrate CT1: Clinical study for the assessment of safety and efficacy of Bezafibrate as a drug therapy for patients with mitochondrial myopathies (phase II) - AT
Universitätsklinikum der Paracelsus Medizinischen Privatuniversität

Universitätsklinik für Kinder- und Jugendheilkunde

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 Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD) - AT
Institution: Information not provided - AT

Phase III Clinical Trial for CPX-351 in Myeloid Leukemia in Children with Down Syndrome 2018 - AT
Institution: Information not provided - AT

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study To Examine The Efficacy And Safety Of ZX008 In Subjects With CDKL5 Deficiency Disorder Followed By An Open-Label Extension - AT
Institution: Information not provided - AT

SIDEROS-E: A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study - AT
Gottfried von Preyer'sches Kinderspital

Abteilung für Kinder- und Jugendheilkunde

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD) - AT
Gottfried von Preyer'sches Kinderspital

Abteilung für Kinder- und Jugendheilkunde

Fenfluramine as anti-epilepticum in Dravet syndrome - BE
Institution: Information not provided - BE

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study To Examine The Efficacy And Safety Of ZX008 In Subjects With CDKL5 Deficiency Disorder Followed By An Open-Label Extension - BE
Institution: Information not provided - BE

CUV152: A Proof of Concept, Phase IIa, Open Label Study to Evaluate the Safety and Efficacy of Subcutaneous Implants of Afamelanotide in Patients with Xeroderma Pigmentosum C and V (XPC and XPV) - BE
Institution: Information not provided - BE

TAMDMD (Tamoxifen in Duchenne muscular dystrophy): Multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial - BE
Cliniques Universitaires de Bruxelles - Hôpital Erasme

Centre de Référence Neuromusculaire Erasme-HUDERF

EXIST-LT: Long-term follow-up study to monitor the growth and development of pediatric patients previously treated with everolimus in study CRAD001M2301- BE
Universitair Ziekenhuis Brussel

Pediatric neurology / Neurologie Kinderen

STELLAR: A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene -BE
Ghent University Hospital - UZ Gent

Department of Ophthalmology/Dienst Oogheelkunde - UZ Gent

An open-label safety, tolerability and pharmacokinetics study of Eteplirsen in young patients with Duchenne dystrophy amenable to exon 51 skipping - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

Long-term, open-label extension study for patients with Duchenne muscular dystrophy enrolled in clinical trials evaluating Casimersen or Golodirsen - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

MOMENTUM: A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD) - BE
Ghent University Hospital - UZ Gent

NMRC UZ Gent

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

Add-on Therapy With Low Dose Fenfluramine in Lennox Gastaut Epilepsy
UZ Leuven - Campus Gasthuisberg

Kinderen neurologie / Paediatric Neurology

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study - BE
UZ Leuven - Campus Gasthuisberg

Kinderen neurologie / Paediatric Neurology

A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophy - BE
UZ Leuven - Campus Gasthuisberg

NMRC (afdeling kinderen) - UZ Leuven

A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy - BE
UZ Leuven - Campus Gasthuisberg

NMRC (afdeling kinderen) - UZ Leuven

Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies -BE
UZ Leuven - Campus Gasthuisberg

NMRC (afdeling kinderen) - UZ Leuven

A Long-Term Study to Assess the Ongoing Safety and Efficacy of Recombinant Human Acid Sphingomyelinase in Patients With Acid Sphingomyelinase Deficiency - BE
UZ Leuven - Campus Gasthuisberg

Centrum metabole ziekten - UZ Leuven

Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients (Phase I-II)
London Health Sciences Centre

Children's Hospital

PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies (Phase II-III)
Institution: Information not provided - CA

MOVE Trial: A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) -GB
Institution: Information not provided - CA

A Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE) -CA
Institution: Information not provided - CA

A Single-center, Un-controlled, Open-labeled Trial of the Long-term Safety of Lamazym Aftercare Treatment of Subjects With Alpha-Mannosidosis Whom Previously Participated in Lamazym Trials
Institution: Information not provided - DK

EXIST-2: A Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) - ES (Phase III)
Institution: Information not provided - FR

A multi-center, un-controlled, open-labeled trial of the long-term safety of Lamazym aftercare treatment of subjects with alpha-Mannosidosis who previously participated in Lamazym-trials (Phase III) - FR
Institution: Information not provided - FR

A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy-FR
Institution: Information not provided - FR

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) -FR
Institution: Information not provided - FR

An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT) -FR
Institution: Information not provided - FR

A Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE) -FR
Institution: Information not provided - FR

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study To Examine The Efficacy And Safety Of ZX008 In Subjects With CDKL5 Deficiency Disorder Followed By An Open-Label Extension -FR
Institution: Information not provided - FR

A Randomized, Double-blind, Placebo-controlled, Multi-center, 2-part, Phase 2 Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome -FR
Institution: Information not provided - FR

A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency -FR
Institution: Information not provided - FR

COMPASS: A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II -FR
Institution: Information not provided - FR

ESSENCE: A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - FR
Institution: Information not provided - FR

STELLAR: A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene -FR
Centre hospitalier national d'ophtalmologie des Quinze-Vingts

Centre de Référence REFERET

FancoMob : Pilot Study Assessing the Feasibility of CD34+ Cells Mobilization and Collection After Treatment With G-CSF and Plerixafor in Patients With Fanconi Anemia for Subsequent Treatment by Gene Therapy (Phase I-II)
Hôpital Necker-Enfants Malades

Service d'Hématologie adulte

A Phase 2, Open-Label Extension, Efficacy and Safety Study of a RARy Specific Agonist (Palovarotene) in the Treatment of Preosseous Flare-ups in Subjects With Fibrodysplasia Ossificans Progressiva (FOP) - FR
Hôpital Necker-Enfants Malades

Service de Génétique Moléculaire

A Phase 2, Open-Label, Efficacy and Safety Study of an RARy Specific Agonist (Palovarotene) to Prevent Heterotopic Ossification in Subjects With Fibrodysplasia Ossificans Progressiva (FOP)
Hôpital Necker-Enfants Malades

Service de Génétique Moléculaire

A Phase 2a Study of TPN-101 in Patients With Aicardi-Goutières Syndrome (AGS) -FR
IMAGINE - Institut des Maladies Génétiques

Laboratoire de neurogénétique et neuroinflammation

ACTHYF: Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children - FR
Institut Jérôme Lejeune

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

Safety and Efficacy of a Unilateral Subretinal Administration of HORA PDE6B in Patients with Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß Expression (Phase I-II)
CHU de Nantes - Hôtel Dieu

Centre d'investigation clinique (CIC)

IRIS 2: Compensation for Blindness With the Intelligent Retinal Implant System (IRIS V2) in Patients With Retinal Dystrophy - FR
CHU de Nantes - Hôtel Dieu

Service d'ophtalmologie

Effectiveness and safety of mTOR-inhibitor in patients with tuberous sclerosis complex-KD
Universitätsklinikum Heidelberg

A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency -DE
Universitätsklinikum Heidelberg

PDE6A gene therapy for retinitis pigmentosa (RD-Cure Gene Therapy Trial)
Department für Augenheilkunde Tübingen

Universitäts-Augenklinik

STOPFOP: Saracatinib Trial TO Prevent FOP. A 6-month double blind randomized controlled trial of AZD0530 versus placebo, followed by a 12 month open label extension phase -DE
Klinikum Garmisch-Partenkirchen GmbH

Abteilung für Kinder- & Jugendmedizin

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

Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies (Phase III) -DE-
Institution: Information not provided - DE

MOVE TRIAL: A Phase 3, Efficacy and Safety Study of Oral Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP) -DE-
Institution: Information not provided - DE

CUV156: A Proof of Concept, Phase IIa, Open Label Study to Evaluate the Safety and Efficacy of Subcutaneous Implants of Afamelanotide in Patients with Xeroderma Pigmentosum (XP)
Institution: Information not provided - DE

A Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE) -DE
Institution: Information not provided - DE

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study To Examine The Efficacy And Safety Of ZX008 In Subjects With CDKL5 Deficiency Disorder Followed By An Open-Label Extension - DE
Institution: Information not provided - DE

COMPASS: A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II - DE
Institution: Information not provided - DE

TAMDMD (Tamoxifen in Duchenne muscular dystrophy): Multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial -DE-
DRK Kliniken Berlin Westend

Klinik für Kinder- und Jugendmedizin

COMPASS: CNS Unmet Medical Need in Mucopolysaccharidosis: A Phase 2 Safety and Pharmacokinetics Study of Ataluren - DE
UKE - Universitätsklinikum Hamburg-Eppendorf

Klinik und Poliklinik für Kinder- und Jugendmedizin

SIDEROS-E: A phase III open-label extension study to assess the long-term safety and efficacy of idebenone in patients with Duchenne muscular dystrophy (DMD) who completed the SIDEROS study -DE
UKE - Universitätsklinikum Hamburg-Eppendorf

Klinik und Poliklinik für Kinder- und Jugendmedizin

EPIDYS study: Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy -DE
UKE - Universitätsklinikum Hamburg-Eppendorf

Klinik und Poliklinik für Kinder- und Jugendmedizin

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 -DE-
Universitätsklinikum Frankfurt

Schwerpunkt Allergologie, Pneumologie und Mukoviszidose

An Open-label, Multicenter Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT) -DE
Universitätsmedizin Göttingen

Klinik für Kinder- und Jugendmedizin

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - DE
Universitätsklinikum Essen

Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

TAMDMD (Tamoxifen in Duchenne muscular dystrophy): Multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial -DE-
Universitätsklinikum Essen

Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

EPIDYS study: Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy -DE
Universitätsklinikum Essen

Klinik für Kinderheilkunde I - Bereich Neuropädiatrie und SPZ

A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy - DE
Universitätsklinikum Essen

FFA-MAE: A proof of concept and assessment of maximal effect study with low dose Fenfluramine as add-on therapy in Myoclonic Astatic Epilepsy (Doose-Syndrome)
Universitätsklinikum Schleswig-Holstein - Campus Kiel

Klinik für Kinder- und Jugendmedizin II : Neuropädiatrie und Sozialpädiatrie

FFA-MAE: A proof of concept and assessment of maximal effect study with low dose Fenfluramine as add-on therapy in Myoclonic Astatic Epilepsy (Doose-Syndrome)
Universitätsklinikum Schleswig-Holstein - Campus Kiel

Multicenter randomized double-blind comparison test followed by open-label continuous administration test of NPC-12T for Fibrodysplasia Ossificans Progressiva
Institution: Information not provided - JP

Placebo controlled double blind randomized Phase II Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders?
Institution: Information not provided - JP

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy - IE
Institution: Information not provided - IE

A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids (SIDEROS)-IE
Department of Neurology - Children's Health Ireland

Department of Neurology