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

Phase 2

Completed

Conditions: 3 and 4
Spinal muscular atrophy type 2
10029299
10029317

Registration Number: NL-OMON41713

Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 38

Inclusion Criteria

Patients with SMA will be included following the predefined criteria: 1) a clinical diagnosis of SMA type 2, 3a, 3b or 4 and a genetically confirmed homozygous SMN1 deletion 2) ability to complete visits during trial period; 3) given oral and written informed consent when >=18 years old; 4) given informed consent by the parents or legal representative in case of patients aged >=12 till 18 years old; 5) ability of performing at least 2 subsequent rounds of the Nine Hole Peg test; 6) A maximum Motor Function Measure score of 80%

Exclusion Criteria

Exclusion criteria for patients are 1) known concomitant disorders of the NMJ (Lambert Eaton myasthenic syndrome, myasthenia gravis); 2) use of drugs that may alter NMJ function; 3) SMA type 1; 4) apprehension against participation in EMG; 5) inability to meet study visits; 6) mechanical gastro-intestinal, urinary or biliary obstruction; 6) clinical significant alterations of laboratory tests (electrolytes, liver function, kidney function, thyroid function or blood dysplasia) drawn within 14 days prior to start of study entry; 7) electrocardiofysiology abnormalities known as a contraindication for pyridostigmine use; 8) pregnancy 9) allergy to bromides 10) severe bronchial astma (incase of uncertainty of diagnosis we will contact the treating pulmonologist or physician)

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Main parameter is the change in the Nine- hole peg test and Motor Function<br /><br>Measure before and after treatment. </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary outcome measures are scores on ESWT, ESBBT, MRC sumscore, scales on<br /><br>daily activities (SMA-FRS, SF=36, PedsQL, PedsQL fatique, VAS, FSS and<br /><br>fatiqueabililty guestionaire), CMAP and presence of decrement upon repetitive<br /><br>nerve stimulation, and adverse events.</p><br>