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: Inclusion criteria: 1.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. 2.Male or female between the ages of 6 and 50 years. 3.Genetically confirmed diagnosis of SMA Type 3. 4.Able to walk independently for at least 30 meters. 5.Do not to take Nusinersen for the treatment of SMA (Nusinersen should be stopped at least 6 months before screening). Salbutamol is permitted only if the dose has been stable during the 6 months before screening. 6.Able to swallow oral medication. 7.Female patients of childbearing potential must have a negative pregnancy test (serum human chorionic gonadotropin [HCG] at Screening); and must practice an effective, reliable contraceptive regimen during the study and for up to 30 days following discontinuation of treatment. 8.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. 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: Exclusion criteria: 1.Epilepsy and currently on medication for epilepsy. 2.Concomitant use of medicinal products with a known potential to cause QTc prolongation. 3.Patients with long QT syndromes. 4.An electrocardiogram (ECG) within 6 months before starting treatment that shows clinically significant abnormalities, in the opinion of the Investigator. 5.Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study. 6.Treatment with an investigational drug (other than amifampridine), device, or biological agent within 6 months prior to Screening or while participating in this study. 7.Surgery for scoliosis or joint contractures within the previous 6 months. 8.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. 9.History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s). 10.Less than a 3-point improvement in HFSME from start of the Open label Run -in period to end of Run-in (Day 0).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To characterize the overall safety and tolerability of amifampridine phosphate compared with placebo in patients with SMA Type 3. To assess the clinical efficacy of amifampridine phosphate compared with placebo in ambulatory patients with SMA Type 3 using the Hammersmith Functional Motor Scale Expanded (HFMSE). ;Secondary Objective: To assess the clinical efficacy of amifampridine phosphate compared with placebo using the six-minute walk test, timed tests, and individual quality of life (INQoL) and pediatric quality of life inventory (PEDSQoL) assessments.;Primary end point(s): The primary efficacy endpoint of the study is the change in HFMSE score from Day 0 (baseline) for SMA subjects treated with amifampridine and placebo.;Timepoint(s) of evaluation of this end point: About 60 days from the start date of the treatment with IMP | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The change in the six-minute walk test from Day 0(baseline) ; The evaluation of the timed tests ; The change in the quality of life assessments from Day 0 (baseline) ; Safety of IMP 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 and concomitant medications will also be evaluated.;Timepoint(s) of evaluation of this end point: About 60 days from the start date of the treatment with IMP; About 60 days from the start date of the treatment with IMP; About 60 days from the start date of the treatment with IMP; The study period during which all non-serious AEs will be reported begins after the first administration of study drug through the termination visit or at the early termination visit. After informed consent but prior to initiation of study treatment, only SAEs associated with any protocol-imposed interventions will be reported. The reporting period for SAEs begins after informed consent is obtained and continues through 4 weeks after the last visit | — |
Countries
Italy
Contacts
Public ContactGary Ingenito
Catalyst Pharmaceuticals, Inc
Outcome results
None listed