Development of indication not being pursued

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

This open-label outpatient extension study was designed to evaluate the long-term safety and tolerability of amifampridine in patients diagnosed with SMA Type 3. In addition, the evaluation of the effects of amifampridine on the QoL was performed. The study was planned to enroll those patients who had completed the SMA-001 study and after all final evaluations for that study had been completed, or those who demonstrated benefit after completing the dose titration period but failed to meet the randomization criteria on Day 0 of SMA-001.

The duration of participation for each patient was expected to be at least 12 months as patients could continue in the study until amifampridine was approved by Regulatory Agencies or the clinical development of amifampridine was terminated for this indication. In addition to amifampridine, patients continued to receive previous concomitant medications, as needed.

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).

27 months

The reporting period for SAEs began after obtaining informed consent and continued through 4 weeks after the last visit. A treatment emergent adverse event (TEAE) was defined as any AE that started at or after the first dose of study drug for the long-term study.

The results of efficacy are limited to the assessment of quality of life using the INQoL. The premature interruption of the study determined that few patients had available data of INQoL from Month 6 onwards.