Bitte wählen Sie:

Universitätsklinikum Essen
Direkteinstieg:
Forschung & Lehre

Klinische Studien

Duchenne Muskeldystrophie

  • MISSION SRP 4658-402  EudraCT 2018-001762-42
    A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping

  • MOMENTUM SRP 5051-201 EudraCT: 2019-000601-77
    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

  • ESSENCE SRP 4045-301 EUDRA CT NUMMER:2015-002069 5
    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 - Study of SRP-4045 and SRP-4053  in DMD Patients                                           

  • SRP 4045-302 EUDRACT Number: 2017-004625-32
    Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen

  • DSC/142357/51 EUDRACT: 2017-000397-10
    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.

  • PTC PASS
    Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care (Anwendungsbeobachtungsstudie PTC124-GD-025o-DMD)

Centronukleäre Myopathien

  • DYN101-C101 EudraCT: 2018-004089-33
    A Phase 1/2 trial on the 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.

Spinale Muskelatrophie

  • SHINE ISIS 396443-CS11 EudraCT No: 2015-001870-16
    An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443

Myasthenia gravis

  • ARGX-113-2006 EudraCT: 2020-005841-18
    Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis

Gliedergürteldystrophie

  • SRP-LGMD-501-NH
    Journey: A Global, Multicenter, Longitudinal Study of the Natural History of Subjects with Limb Girdle Muscular Dystrophy (LGMD) Type 2E (LGMD2E/R4), Type 2D (LGMD2D/R3), and Type 2C (LGMD2C/R5)

 In Vorbereitung

  • PFIZER C3391003 Eudra CT 2019-002921-31
    A phase 3, multicenter, randomized,   double-blind, placebo-controlled study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne Muscular Dystrophy

  • EMBARK SRP-9001-301 Eudra CT 2019-003374-91
    A Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy Who Are Aged 4-7 Years

  • DYNE251-DMD-201 EudraCT: 2021-005478-24
    A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

  • Biogen 232SM303 ASCEND EUDRA CT NUMBER: 2021-001294-23
    A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients with Spinal Muscular Atrophy Previously Treated with Risdiplam

  • Sapphire - SRK-015-003 EudraCT Number: 2021-005314-34
    Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

  • Antisense ATL1102 EudraCT No. 2021-005894-26
    A multicentre, randomised, double-blind, placebo-controlled study to assess The efficacy, safety, and pharmacokinetic profile of two dose levels of TL1102 administered by subcutaneous injection in non-ambulatory participants with Duchenne Muscular Dystrophy
    A study of ATL1102 in participants with Duchenne Muscular Dystrophy (DMD) Who cannot walk independently and require wheelchair assistance

  • MLB-01-005 LGMD  IND Number: 145713
    Protocol Title: A Phase 3 Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of BBP-418 (ribitol) in Patients with Limb Girdle Muscular Dystrophy 2I (LGMD2I)

  • BN 42644 Manatee  2021-003417-19
    A TWO-PART, SEAMLESS, MULTI-CENTER, RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE-BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7204239 IN COMBINATION WITH RISDIPLAM (RO7034067) IN  AMBULANT PATIENTS WITH SPINAL MUSCULAR ATROPHY

Univ.-Prof. Ulrike Schara-Schmidt

Univ.-Prof. Ulrike Schara-Schmidt

Ltd. Ärztin Kinderneurologie, Stellvertretende Direktorin Klinik für Kinderheilkunde I

0201-723-2508 E-Mail schreiben
Dr. med. Andrea Gangfuß

Dr. med. Andrea Gangfuß

Oberärztin, Fachärztin für Kinderheilkunde mit Schwerpunkt Neuropädiatrie

E-Mail schreiben
Dr. med. univ. Nele Baier
Ansprechpartnerin

Dr. med. univ. Nele Baier

Studienärztin

E-Mail schreiben
 Corinna Seifert
Ansprechpartner

Corinna Seifert

Studienkoordinatorin und Kinderkrankenschwester

E-Mail schreiben
 Karin Smuda
Ansprechpartner

Karin Smuda

Studienkoordinatorin und Kinderkrankenschwester

E-Mail schreiben
 Barbara Andres
Ansprechpartner

Barbara Andres

Physiotherapeutin, Klinische Studien

E-Mail schreiben