SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4

• Status: Completed
• Trial end date: 2018-01-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: UMC Utrecht
• Treatments: Pyridostigmine Bromide

Criteria

Inclusion Criteria:

• A clinical diagnosis of SMA type 2, 3a, 3b or 4

• Genetically confirmed homozygous SMN1 deletion

• Ability to complete visits during trial period;

• Given oral and written informed consent when ≥18 years old;

• Given informed consent by the parents or legal representative(s) in case of patients aged ≥12 till <18 years old (in accordance with Dutch law)

• Ability of performing at least 2 subsequent rounds of the Nine Hole Peg test

• A maximum total Motor Function Measure (MFM) score of 80% (i.e.: a maximum score under 80% of the D1+D2+D3 subscores).

Exclusion Criteria:

• Known concomitant disorders of the NMJ (e.g. but not limited to: Lambert Eaton myasthenic syndrome, myasthenia gravis);

• Use of drugs that may alter NMJ function

• Classic SMA type 1;

• Apprehension against participation in EMG;

• Inability to meet study visits;

• Mechanical gastro-intestinal, urinary or biliary obstruction;

• 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;

• ECG abnormalities known as a contraindication for pyridostigmine use;

• Current pregnancy or breast-feeding

• Allergy to bromides

• Severe bronchial asthma (in case of uncertainty of diagnosis, we will contact treating pulmonologist or physician)

• Total MFM score at baseline (screening) > 80% (i.e.: a maximum total MFM score above 80% of the D1+D2+D3 subscores).