Congenital Myasthenic Syndromes (CMS) MedDRA version: 21.1 Level: PT Classification code 10028424 Term: Myasthenic syndrome System Organ Class: 10029205 - Nervous system disorders MedDRA version: 20.0 Level: LLT Classification code 10028425 Term: Myasthenic syndromes in diseases classified elsewhere System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Trade Name: Firdapse
Product Name: Amifampridina fosfato
Product Code: [Amifampridina]
Pharmaceutical Form: Tablet
INN or Proposed INN: AMIFAMPRIDINA
CAS Number: 446254-47-3
Current Sponsor code: DAPP
Sponsors
CATALYST PHARMACEUTICALS INC.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Participated in the CMS-001 study or failed to meet screening or randomization criteria of Protocol CMS-001 but considered to have potential clinical benefit from treatment with amifampridine phosphate. 2. Willing and able to provide written informed consent by patient or guardian after the nature of the study has been explained and before the start of any research-related procedures. 3. Female patients of childbearing potential defined according to the Clinical Trial Facilitation Group - CTFG guidelines must have a negative urine pregnancy test and must practice an effective, reliable contraceptive regimen according to the CTFG guidelines criteria during the study and for up to 30 days following discontinuation of treatment. 4. 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 13 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: Individuals who meet any of the exclusion criteria in the protocol CMS-001 or those listed below are not eligible to participate in the study: 1. Epilepsy and currently on medication. 2. Clinically significant abnormalities in 12 lead ECG, in the opinion of the Investigator. 3. Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study. 4. Intolerable amifampridine-related side effects 5. Treatment with an investigational drug (other than amifampridine) or device while participating in this study. 6. 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To characterize the long-term safety and tolerability of amifampridine phosphate in patients with Congenital Myasthenic Syndromes (CMS).;Secondary Objective: To assess the clinical efficacy of amifampridine phosphate over time in patients with Congenital Myasthenic Syndromes (CMS) based on change in Subject Global Impression (SGI) Scores.;Primary end point(s): The primary endpoint of the study is the safety and 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 safety and tolerability of amifampridine will be evaluated at months 3, 6, 9, 12, 15 and 21 after inclusion in the study. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary endpoint of the study is changes in the Subject Global Impression (SGI) scores over time.;Timepoint(s) of evaluation of this end point: The secondary endpoint of the study will be assessed from baseline at months 3, 6, 9, 12, 15 and 21. | — |
Countries
Italy
Contacts
Public ContactGary Ingenito
Catalyst Pharmaceuticals, Inc
Outcome results
None listed