Ignite Creation Date:
2025-12-25 @ 2:06 AM
Ignite Modification Date:
2026-01-07 @ 1:24 AM
Study NCT ID:
NCT03819660
Status:
TERMINATED
Last Update Posted:
2023-11-30
First Post:
2019-01-23
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
Sponsor:
Catalyst Pharmaceuticals, Inc.
Organization:
Study Overview
Official Title:
Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3
Status:
TERMINATED
Status Verified Date:
2023-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Development of indication not being pursued
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
SMA3
Brief Summary:
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.
Detailed Description:
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.
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.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: