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Ensaio (s) clínico (s) nacional (is)
ESPANHA
Madrid
MADRID
The FENDEEP Study: Fenfluramine for the treatment of different types of developmental and epileptic encephalopathies: a pilot trial exploring epileptic and non-epileptic outcomes - ES
Hospital Ruber Internacional
Servicio de Neurología
AUSTRIA
WIEN
ADDRESS: 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
FRANCA
ILE-DE-FRANCE
PARIS
ACTHYF: Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children - FR
Institut Jérôme Lejeune
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
REAL 8: A Study Comparing the Effect and Safety of Once Weekly Dosing of Somapacitan With Daily Norditropin® as Well as Evaluating Long-term Safety of Somapacitan in a Basket Study Design in Children With Short Stature Either Born Small for Gestational Age or With Turner Syndrome, Noonan Syndrome, or Idiopathic Short Stature - AT
Institution: Information not provided - AT
ESPANHA
Madrid
ADDRESS: NOT PROVIDED - ES
An open-label, multicenter study to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of RO7248824 in participants with Angelman syndrome -ES
Institution: Information not provided - ES
ITALIA
SICILIA
MESSINA
Randomized, Placebo-controlled, Cross-over, Double-blind Study of a Metabolic Support Therapy With Q10 Ubiquinol and a Multivitamin B and E Complex in Two Cohorts of Patients With Idiopathic and Syndromic Autism (Phelan-McDermid Syndrome)
Università degli Studi di Messina
ALEMANHA
Berlin
BERLIN
HYDRO-PROTECT: HYDROchlorothiazide to PROTECT Polycystic Kidney Disease Patients and Improve Their Quality of Life - DE
Charité - Universitätsmedizin Berlin
BELGICA
VLAAMS BRABANT
LEUVEN
HYDRO-PROTECT: HYDROchlorothiazide to PROTECT Polycystic Kidney Disease Patients and Improve Their Quality of Life - BE
UZ Leuven - Campus Gasthuisberg
Dienst nefrologie/ nephrology department
BELGICA
VLAAMS BRABANT
LEUVEN
International, Multi-center, Open Label, 9-month Follow-up Extension Study Assessing the Long-term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A - BE
UZ Leuven - Campus Gasthuisberg
Dienst neurologie/ Department of Neurology
ESPANHA
Comunidad Valenciana
VALENCIA
PLEO-CMT-FU: International, multi-center, double blind 9-month FOLLOW-UP extension study assessing the long term safety and tolerability of PXT3003 in patients with Charcot-Marie-Tooth Disease type 1A -ES
Hospital Universitario y Politécnico La Fe
Servicio de Neurología
ESPANHA
Madrid
ADDRESS: NOT PROVIDED - ES
PREMIER: A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A) -ES
Institution: Information not provided - ES
ESPANHA
Madrid
ADDRESS: NOT PROVIDED - ES
HYDRO-PROTECT: HYDROchlorothiazide to PROTECT Polycystic Kidney Disease Patients and Improve Their Quality of Life -ES
Institution: Information not provided - ES
ESPANHA
Madrid
ADDRESS: NOT PROVIDED - ES
A phase II, randomized, double-blind, placebo-controlled, parallel group study to evaluate the safety, efficacy, and pharmacodynamics of 52 weeks of treatment with basmisanil in children with dup15q syndrome -ES
Institution: Information not provided - ES
ESTADOS UNIDOS
Washington
ADDRESS: NOT PROVIDED - US
International, Multi-center, Open Label, 9-month Follow-up Extension Study Assessing the Long-term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A -GB
Institution: Information not provided - US
FRANCA
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
HYDRO-PROTECT: HYDROchlorothiazide to PROTECT Polycystic Kidney Disease Patients and Improve Their Quality of Life -FR
Institution: Information not provided - FR
FRANCA
ILE-DE-FRANCE
CRÉTEIL
SERENDEM : MD1003 in Patients Suffering From Demyelinating Neuropathies, an Open Label Pilot Study (Phase I-II)
Hôpitaux Universitaires Henri Mondor
Service de neurologie
FRANCA
PROVENCE-ALPES-COTE D'AZUR
MARSEILLE
International, multi-center, randomized, double-blind, placebo-controlled phase III study assessing in parallel groups the efficacy and safety of 2 doses of PXT3003 in patients with Charcot-Marie-Tooth disease type 1A treated 15 months (PLEO-CMT) - FR
CHU de Marseille - Hôpital de la Timone
Service de Neurologie - Maladies neuromusculaires et SLA
REINO UNIDO
Greater London
ADDRESS: NOT PROVIDED - GB
HYDRO-PROTECT: HYDROchlorothiazide to PROTECT Polycystic Kidney Disease Patients and Improve Their Quality of Life -GB
Institution: Information not provided - GB
Ensaio (s) clínico (s) multinacional (ais)
ESTADOS UNIDOS
Washington
ADDRESS: NOT PROVIDED - US
Phase III Clinical Trial for the Treatment of Myeloid Leukemia in Children with Down Syndrome 2018 - ML-DS 2018
Institution: Information not provided - US
ESTADOS UNIDOS
Washington
ADDRESS: NOT PROVIDED - US
An Open-Label, Multicenter Study To Investigate The Safety, Tolerability, Pharmacokinetics And Pharmacodynamics Of RO7248824 In Participants With Angelman Syndrome
Institution: Information not provided - US
CANADA
Québec
ADDRESS: NOT PROVIDED - CA
TAM4MTM: A Phase 1/2 Randomized, Placebo-Controlled, Double-Blinded, Single Crossover Study to Determine the Safety and Efficacy of Tamoxifen Therapy for Myotubular Myopathy (XLMTM)
Institution: Information not provided - CA
ESTADOS UNIDOS
Washington
ADDRESS: NOT PROVIDED - US
International, Multi-center, Open Label, 9-month Follow-up Extension Study Assessing the Long-term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A
Institution: Information not provided - US
ESTADOS UNIDOS
Washington
ADDRESS: NOT PROVIDED - US
A Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant Adeno-associated Virus Vector Expressing RPGR (rAAV2tYF-GRK1-RPGR), Compared to an Untreated Control Group in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene
Institution: Information not provided - US
PAISES BAIXOS
Groningen
GRONINGEN