Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
Condition: Spinal Muscular Atrophy Type 3
MeSH terms: Muscular Atrophy; Muscular Atrophy, Spinal; Atrophy
Intervention: Amifampridine Phosphate 10 MG Oral Tablet
Intervention description: Oral tablets
Intervention MeSH terms: Amifampridine
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Intervention model description: Open-label, extension study , long term safety and tolerability
Masking: None (Open Label)
Enrollment: 13.0
Sex: All
Minimum age: 6 Years
Maximum age: 50 Years
Healthy volunteers: No
Keywords: Type 3

Summary:

      A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy
      (SMA) Type 3.
    

Eligibility criteria:

        Inclusion Criteria:

        Individuals eligible to participate in this study must meet all the following inclusion
        criteria:

          1. Participated in the SMA-001 study

          2. Willing and able to provide written informed consent after the nature of the study has
             been explained and before the start of any research-related procedures.

          3. Female patients of childbearing potential must have a negative pregnancy test (urine
             human chorionic gonadotropin [HCG] at the end of SMA-001 study); and must practice an
             effective, reliable contraceptive regimen during the study and for up to 30 days
             following discontinuation of treatment.

          4. Ability to participate in the study based on overall health of the patient and disease
             prognosis, as applicable, in the opinion of the Investigator; and able to comply with
             all requirements of the protocol, including completion of study questionnaires.

        Exclusion Criteria:

        Individuals who met any of the exclusion criteria in the original protocol or those listed
        below are not eligible to participate in the study:

          1. Epilepsy and currently on medication.

          2. Uncontrolled asthma.

          3. Concomitant use with sultopride.

          4. Concomitant use with medicinal products with a narrow therapeutic window.

          5. Concomitant use with medicinal products with a known to cause QTc prolongation.

          6. Clinically significant abnormalities in 12 lead ECG, in the opinion of the
             Investigator.

          7. Subjects with congenital QT syndromes.

          8. Breastfeeding or pregnant at Screening or planning to become pregnant at any time
             during the study.

          9. Intolerable amifampridine-related side effects

         10. Treatment with an investigational drug (other than amifampridine) or device while
             participating in this study.

         11. Any medical condition that, in the opinion of the Investigator, might interfere with
             the patient's participation in the study, poses an added risk for the patient, or
             confound the assessment of the patient.

         12. History of drug allergy to any pyridine-containing substances or any amifampridine
             excipient(s).