3. 脊髄性筋萎縮症 Spinal muscular atrophy Clinical trials / Disease details


臨床試験数 : 237 薬物数 : 123 - (DrugBank : 29) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 75

  
3 trials found
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1NCT02941328
(ClinicalTrials.gov)
December 201530/9/2016SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4A 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 4Spinal Muscular Atrophy;SMA;Kugelberg-Welander DiseaseDrug: Pyridostigmine;Drug: PlaceboUMC UtrechtNULLCompleted12 YearsN/AAll39Phase 2Netherlands
2EUCTR2011-004369-34-NL
(EUCTR)
22/04/201503/11/2014 SPACE trial SMA and Pyridostigmine in Adults and Children; Experimental trial to assess effect of pyridostigmine compared to placebo in patients with spinal muscular atrophy types 2, 3 and 4 SPACE trial SMA and Pyridostigmine in Adults and Children; Efficacy trial Phase II, mono-center, doubleblind, placebo-controlled, crossover trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2, 3 and 4 - SPACE trial Proximal spinal muscular atrophy (SMA) is characterized by weakness of predominantly axial and proximal muscle groups and is caused by homozygous deletion of the survival motor neuron 1 (SMN1)-gene. There are 4 SMA types (type 1-4), with a descending order of severity. Age at onset and achieved motor milestones are the characteristics to define severity. Treatment of SMA is exclusively supportive.
MedDRA version: 17.1;Level: LLT;Classification code 10068209;Term: Spinal muscular atrophy adult onset;System Organ Class: 100000004850
MedDRA version: 17.1;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: pyridostigmine bromide
Product Name: Mestinon
Product Code: RVG 03820
Universtiy Medical Center UtrechtNULLNot Recruiting Female: yes
Male: yes
45 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noNetherlands
3NCT02227823
(ClinicalTrials.gov)
July 201420/8/2014Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3Safety and Efficacy Study of Anti-cholinesterase Therapy on the Motor Functions in Patients With Spinal Muscular Atrophy Type 3.Spinal Muscular Atrophy Type 3Drug: Pyridostigmine BromideCentre Hospitalier Régional de la CitadelleNULLRecruiting6 YearsN/ABoth20Phase 2Belgium