SMA3: Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
Study Details
Study Description
Brief Summary
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Detailed Description
This open-label, long term safety study is designed to evaluate the safety of amifampridine phosphate in ambulatory patients diagnosed SMA Type 3 over an extended period. The study is planned to include approximately 12 male and female SMA Type 3 patients. The planned duration of participation for each patient may be up to 1 year. Patients must have demonstrated benefit during the initial SMA-001 trial.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: amifampridine phosphate Oral tablets, 15 to 80 mg per day in divided doses 3 to 4 times a day for up to 18 months. |
Drug: Amifampridine Phosphate 10 MG Oral Tablet
Oral tablets
|
Outcome Measures
Primary Outcome Measures
- The number and severity of treatment related adverse events and serious adverse events as assessed by CTCAE v4.0 [18 months]
Safety analyses will be conducted on the safety population (i.e. all patients who receive at least 1 dose of amifampridine. The safety analysis will be descriptive and will be presented on observed data only. All AEs will be coded using the Medical Dictionary for Regulatory Activities (MedDRA). The incidence of TEAEs will be summarized by system organ class, preferred term, relationship to treatment, and severity.
Eligibility Criteria
Criteria
Inclusion Criteria:
Individuals eligible to participate in this study must meet all the following inclusion criteria:
-
Participated in the SMA-001 study
-
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.
-
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.
-
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:
CRITERIA FOR INCLUSION AND EXCLUSION:
Individuals eligible to participate in this study must meet all the following inclusion criteria:
-
Participated in the SMA-001 study
-
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.
-
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.
-
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:
-
Epilepsy and currently on medication.
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Uncontrolled asthma.
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Concomitant use with sultopride.
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Concomitant use with medicinal products with a narrow therapeutic window.
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Concomitant use with medicinal products with a known to cause QTc prolongation.
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Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator.
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Subjects with congenital QT syndromes.
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Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.
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Intolerable amifampridine-related side effects
-
Treatment with an investigational drug (other than amifampridine) or device while participating in this study.
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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.
-
History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s).
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Neurological Institute Carlo Besta | Milano | Lombardy | Italy | 20133 |
Sponsors and Collaborators
- Catalyst Pharmaceuticals, Inc.
Investigators
- Principal Investigator: Lorenzo Maggi, MD, Carlo Besta Institute, Milan, Italy
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- SMA-002