Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 (SMA3)
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|ClinicalTrials.gov Identifier: NCT03819660|
Recruitment Status : Enrolling by invitation
First Posted : January 28, 2019
Last Update Posted : March 29, 2019
|Condition or disease||Intervention/treatment||Phase|
|Muscle Atrophy||Drug: Amifampridine Phosphate 10 MG Oral Tablet||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||12 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||Open-label, long term safety treatment|
|Masking:||None (Open Label)|
|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|
|Estimated Primary Completion Date :||December 31, 2020|
|Estimated Study Completion Date :||April 1, 2021|
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
- 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.
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
|Neurological Institute Carlo Besta|
|Milano, Lombardy, Italy, 20133|
|Principal Investigator:||Lorenzo Maggi, MD||Carlo Besta Institute, Milan, Italy|