Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 (SMA3)
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ClinicalTrials.gov Identifier: NCT03819660 |
Recruitment Status :
Terminated
(Development of indication not being pursued)
First Posted : January 28, 2019
Last Update Posted : March 29, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Muscle Atrophy | Drug: Amifampridine Phosphate 10 MG Oral Tablet | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 12 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Intervention Model Description: | Open-label, long term safety treatment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3 |
Actual Study Start Date : | March 7, 2019 |
Actual Primary Completion Date : | September 13, 2021 |
Actual Study Completion Date : | September 13, 2021 |

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.
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Drug: Amifampridine Phosphate 10 MG Oral Tablet
Oral tablets |
- The number and severity of treatment related adverse events and serious adverse events as assessed by CTCAE v4.0 [ Time Frame: 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.

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Ages Eligible for Study: | 6 Years to 50 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.
- Uncontrolled asthma.
- Concomitant use with sultopride.
- Concomitant use with medicinal products with a narrow therapeutic window.
- Concomitant use with medicinal products with a known to cause QTc prolongation.
- Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator.
- Subjects with congenital QT syndromes.
- Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study.
- Intolerable amifampridine-related side effects
- Treatment with an investigational drug (other than amifampridine) or device while participating in this study.
- 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).

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03819660
Italy | |
Neurological Institute Carlo Besta | |
Milano, Lombardy, Italy, 20133 |
Principal Investigator: | Lorenzo Maggi, MD | Carlo Besta Institute, Milan, Italy |
Responsible Party: | Catalyst Pharmaceuticals, Inc. |
ClinicalTrials.gov Identifier: | NCT03819660 |
Other Study ID Numbers: |
SMA-002 |
First Posted: | January 28, 2019 Key Record Dates |
Last Update Posted: | March 29, 2022 |
Last Verified: | March 2022 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Type 3 |
Muscular Atrophy Muscular Atrophy, Spinal Atrophy Pathological Conditions, Anatomical Neuromuscular Manifestations Neurologic Manifestations Nervous System Diseases Spinal Cord Diseases Central Nervous System Diseases Motor Neuron Disease |
Neurodegenerative Diseases Neuromuscular Diseases Amifampridine Neuromuscular Agents Peripheral Nervous System Agents Physiological Effects of Drugs Potassium Channel Blockers Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action |