Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-26 @ 11:11 AM
Ignite Modification Date:
2025-12-26 @ 11:11 AM
NCT ID:
NCT02941328
Eligibility 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).
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
12 Years
Study:
NCT02941328
Study Brief:
Protocol Section:
NCT02941328