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Universitätsklinikum Essen
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Forschung & Lehre

Klinische Studien

Muskeldystrophie Duchenne

  • PTC-PASS (PTC124-GD-25o-DMD): Langfristige Beobachtungsstudie zur Sicherheit und Wirksamkeit von Translarna bei der üblichen Versorgung
  • FOR-DMD - Anwendungsbeobachtungsstudie: Vergleich von drei Cortison-Schemata
  • SIDEROS  (SNT-III-012): 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
  • SAREPTA (4045-301): 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
  • DSC/14/2357/48 (Givinostat): Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.
  • WN40227 ( Antimyostatin): A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys with Duchenne Muscular Dystrophy
  • in Vorbereitung: TAM-DMD / Tamoxifen in Duchenn muscular dystrophy: A multicenter, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial

Spinale Muskelatrophie

  • Biogen ISIS 396443-CS11: An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443
  • BN29854/Olesoxime: Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA – beendet
  • in Vorbereitung: AveXis-101: Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy, SMA Typ
  • in Vorbereitung: Jewelfish: A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants with Spinal Muscular Atrophy
  • in Vorbereitung: TOPAZ /SRK-015-002: Phase 2 active treatment study to evaluate the efficacy and safety of SRK-015 in patients with later-onset spinal muscular atrophy

GLUT-1 Transporter Defekt

  • UX007G-CL301/ Ultagenyx: A Phase 3, randomized, double-blind, placebo-controlled, crossover study to assess the efficacy and safety of UX007 in the treatment of movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome – beendet

 Myotubuläre Myopathie

  •  NatHis-MTM: Prospective, Longitudinal Study of the Natural History and functional status of patients with MyoTubular Myopathy
  • in Vorbereitung: Dynacure: A phase 1/2 trial on safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of DYN101 in patients ≥ 16 years of age with centronuclear myopathies caused by mutations in DNM2 or MTM1.

Juvenile Myasthenia gravis

  • in Vorbereitung: Alexion: An open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of Eculizumab in pediatric patients with refractory generalized myasthenia Gravis
Leitung

Univ.-Prof. Ulrike Schara

Ltd. Ärztin Kinderneurologie, Stellvertretende Direktorin Klinik für Kinderheilkunde I

Ansprechpartner

Dr. med. Maria Henrich

Fachärztin für Kinderheilkunde

Ansprechpartner

Corinna Seifert

Studienkoordinatorin und Kinderkrankenschwester

Ansprechpartner

Karin Smuda

Studienkoordinatorin und Kinderkrankenschwester

Ansprechpartner

Barbara Andres

Physiotherapeutin, Klinische Studien