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Rekrutierende Studie = ; Fortlaufende Studie =
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Nationale klinische Studie(n)

BELGIEN
OOST-VLAANDEREN
GENT

SUNFISH: A two-part seamless, multi-center randomized, placebo-controlled, double blind study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patients - BE
Ghent University Hospital - UZ Gent
NMRC UZ Gent

DEUTSCHLAND
Baden-Württemberg
FREIBURG
Kann eine Behandlung mit humanan Wachstumshormonen die Muskelkraft bei Patienten mit spinaler Muskelatrophie Typ II und III erhöhen (Phase-II-Studie)?
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen

DEUTSCHLAND
Baden-Württemberg
FREIBURG

NURTURE: Eine offene Studie zur Untersuchung der Wirksamkeit, Sicherheit, Verträglichkeit und Pharmakokinetik von mehreren Dosen von intrathekalem ISIS 396443 bei Patienten mit genetisch gesicherter und präsymptomatischer spinaler Muskelatrophie (Phase II)
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen

DEUTSCHLAND
Bayern
MÜNCHEN

Eine offene Verlängerungsstudie für Patienten mit spinaler Muskelatrophie, die zuvor an den klinischen Studien mit ISIS 396443 teilgenommen haben (Phase III)
Friedrich-Baur-Institut
Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik

DEUTSCHLAND
Bayern
MÜNCHEN


OLEOS: Multizentrische, offene, einarmige Studie zur Untersuchung der Langzeit Sicherheit, Verträglichkeit und Wirksamkeit von 10mg/kg Olesoxime bei Patienten mit spinaler Muskelatrophie (Phase II)
Friedrich-Baur-Institut
Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik

NIEDERLANDE
Utrecht
UTRECHT
SPACE trial: A Phase II, Mono-center, Placebo-controlled, Double-blind, Crossover Trial to Investigate Effect and Efficacy of Pyridostigmine in Dutch Patients With Spinal Muscular Atrophy Types 2, 3 and 4
UMC Utrecht - Universitair Medisch Centrum Utrecht
Polikliniek Neuromusculaire Ziekten

SPANIEN
Andalucía
SEVILLA

SUNFISH: A two-part seamless, multi-center randomized, placebo-controlled, double blind study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patients (Phase II) - ES
Hospital Universitario Virgen del Rocío
Unidad de Neurología Pediátrica

SPANIEN
Castilla - La Mancha
ALCÁZAR DE SAN JUAN

A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101 - ES
Hospital General La Mancha Centro

SPANIEN
Cataluńa
BARCELONA

An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 (Phase III) - ES
Hospital Universitari Vall d'Hebron
Área de Genética Clínica y Molecular

SPANIEN
Cataluńa
BARCELONA

SUNFISH: A two-part seamless, multi-center randomized, placebo-controlled, double blind study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patients (Phase II) - ES
Hospital Universitari Vall d'Hebron
Área de Genética Clínica y Molecular

SPANIEN
Cataluńa
ESPLUGUES DE LLOBREGAT

An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 (Phase III) - ES
Hospital Sant Joan de Déu Barcelona
Unidad de Enfermedades Neuromusculares

SPANIEN
Cataluńa
ESPLUGUES DE LLOBREGAT

SUNFISH: A two-part seamless, multi-center randomized, placebo-controlled, double blind study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patients (Phase II) - ES
Hospital Sant Joan de Déu Barcelona
Servicio de Neurología

SPANIEN
Madrid
ADDRESS: NOT PROVIDED - ES
The TOPAZ study: Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy - ES
Institution: Information not provided - ES

SPANIEN
Madrid
MADRID

An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 (Phase III) - ES
Hospital Universitario La Paz
Servicio de Neurología infantil

SPANIEN
Madrid
MADRID

SUNFISH: A two-part seamless, multi-center randomized, placebo-controlled, double blind study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in type 2 and 3 spinal muscular atrophy patients (Phase II) - ES
Hospital Universitario La Paz
Servicio de Neurología infantil

BELGIEN
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
SHINE: An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 - BE
Erasme Hospital - ULB
Centre de Référence Neuromusculaire Erasme-HUDERF

BELGIEN
LIEGE
LIEGE


RAINBOWFISH: An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy - BE
Centre Hospitalier Régional de la Citadelle
Centre de Référence des Maladies Neuromusculaires (CRMN)

BELGIEN
OOST-VLAANDEREN
GENT

JEWELFISH: An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of RO7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy - BE
Ghent University Hospital - UZ Gent
NMRC UZ Gent

FRANKREICH
ILE-DE-FRANCE
PARIS


A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy - FR
CHU Paris Est - Hôpital d'Enfants Armand-Trousseau
Institut I-Motion - Centre de recherche pédiatrique en pathologies neuromusculaires

SPANIEN
Madrid
ADDRESS: NOT PROVIDED - ES

DEVOTE: Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy - ES
Institution: Information not provided - ES

VEREINIGTE STAATEN
Washington
ADDRESS: NOT PROVIDED - US


A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy - US
Institution: Information not provided - US

FRANKREICH
ILE-DE-FRANCE
GARCHES
Long-Term Effect of LIAM (Lung Insufflation Assist Maneuver) on Respiratory Performance in Non-invasive Ventilated Patients Suffering From Neuromuscular Disease
CHU Paris IdF Ouest - Hôpital Raymond Poincaré
Centre d'Investigation Clinique et Technologique 805 - CIC-IT de Garches

FRANKREICH
ILE-DE-FRANCE
GARCHES
Establishment of a Non-invasive and Indirect Measure of Volitional Pimax (PI MAX STIM)
CHU Paris IdF Ouest - Hôpital Raymond Poincaré
Service de Physiologie-Explorations Fonctionnelles
Multinationale klinische Studie(n)

ITALIEN
LAZIO
ROMA
Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients - coordination
IRCCS Ospedale Pediatrico Bambino Gesů - SEDE GIANICOLO
Ospedale Pediatrico Bambino Gesů

SCHWEIZ
Suisse Alémanique
BASEL