Spinal Muscular Atrophy (SMA) Type 3 MedDRA version: 20.1 Level: PT Classification code 10041582 Term: Spinal muscular atrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
CATALYST PHARMACEUTICALS INC.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Patients 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 defined according to the Clinical Trial Facilitation Group - CTFG guidelines* 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. * According to CTFG guidelines a woman is considered of childbearing potential, i.e. fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 8 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - 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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the long-term safety and tolerability of amifampridine phosphate in patients with SMA Type 3;Secondary Objective: To assess the clinical efficacy of amifampridine phosphate over time in patients with SMA Type 3 based on changes in quality of life (QoL) ;Primary end point(s): The primary endpoint of the study is the safety and the tolerability of amifampridine. Safety will be assessed by the incidence of treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs). Vital signs, 12-lead ECGs, clinical laboratory tests, physical examination findings,and concomitant medications will also be evaluated;Timepoint(s) of evaluation of this end point: The study primary endpoint will be assessed at months 3, 6, 9, 12, 15, 21 and 27 from the start of treatment with amifampridine phosphate | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary endpoint is the efficacy of amifampridine phosphate based on changes of the Quality of Life (QoL) scores over time. The Individualized Quality of Life for Neuromuscular disease (INQoL) or the Pediatric Quality of Life (PEDSQLTM) will be assessed for adult or pediatric patients, respectively;Timepoint(s) of evaluation of this end point: The Quality of Life (QoL) will be assessed at months 3, 6, 9, 12, 15, 21 and 27 from the start of treatment with amifampridine phosphate | — |
Countries
Italy
Contacts
Public ContactGary Ingenito
Catalyst Pharmaceuticals Inc.
Outcome results
None listed