Ignite Creation Date:
2024-05-06 @ 9:15 AM
Last Modification Date:
2024-10-26 @ 12:12 PM
Study NCT ID:
NCT02941328
Status:
COMPLETED
Last Update Posted:
2018-02-05
First Post:
2016-09-30
Brief Title:
SPACE Trial Pyridostigmine vs Placebo in SMA Types 2 3 and 4
Sponsor:
UMC Utrecht
Organization:
UMC Utrecht
Study Overview
Official Title:
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
Status:
COMPLETED
Status Verified Date:
2018-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
SPACE
Brief Summary:
A trial investigating the effects of pyridostigmine mestinon versus a placebo in a double-blind cross over trial in patients with hereditary proximal spinal muscular atrophy SMA types 2 3 and 4
Detailed Description:
In this cross-over trial we will investigate the effects of pyridostigmine mestinon versus a placebo on fatigability in patients with hereditary proximal spinal muscular atrophy SMA types 2 3 and 4 aged 12 and older The study participants will be using a placebo during 8 weeks and mestinon during 8 weeks Both investigators and patients are blinded to the treatment allocation in both periods Patients will be randomly assigned to use placebo or mestinon first At the end of the study patients will have used both mestinon and a placebo during 8 weeks
In both phases periods of the cross over study study medication dosage will slowly be increased to a maximum dosage of 6 mgkgday spread over 4 doses daily in order to either prevent side effects or to manage them as good as possible This scheme allows us to assist our participants in case side effects do occur early Although we strive to have all patients on the same dosage per kg of body weight serious side effects will obviously lead to lowering the dosage
Before the start of medication use either placebo or pyridostigmine baseline measurements will be taken a physical examination and data concerning both primary and secondary outcomes MFM MRC scores fatigability tests etc will be collected After use a drug mestinonplacebo for 8 weeks these tests will be repeated to evaluate possible medication effect After a wash-out period of approximately 1-2 weeks the same scheme is used for the second study period ie the cross-over design Unblinding follows after the entire study is completed ie last included patients finishes participation
In both phases periods of the cross over study study medication dosage will slowly be increased to a maximum dosage of 6 mgkgday spread over 4 doses daily in order to either prevent side effects or to manage them as good as possible This scheme allows us to assist our participants in case side effects do occur early Although we strive to have all patients on the same dosage per kg of body weight serious side effects will obviously lead to lowering the dosage
Before the start of medication use either placebo or pyridostigmine baseline measurements will be taken a physical examination and data concerning both primary and secondary outcomes MFM MRC scores fatigability tests etc will be collected After use a drug mestinonplacebo for 8 weeks these tests will be repeated to evaluate possible medication effect After a wash-out period of approximately 1-2 weeks the same scheme is used for the second study period ie the cross-over design Unblinding follows after the entire study is completed ie last included patients finishes participation
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None