EAP-001: Expanded Access Study Amifampridine Phosphate in Congenital Myasthenic Syndrome (CMS)

Sponsor

Catalyst Pharmaceuticals, Inc. (Industry)

Overall Status

No longer available

CT.gov ID

NCT02189720

Collaborator

(none)

Location

Study Details

Study Description

Brief Summary

The primary objective of the study is:

• To provide patients with CMSaccess to amifampridine phosphate therapy until the product becomes commercially available or development is discontinued.

The secondary objective of the study is:

• To assess the long-term safety of amifampridine phosphate in patients with CMS

Condition or Disease	Intervention/Treatment	Phase
Congenital Myasthenic Syndrome	Drug: Amifampridine Phosphate

Study Design

Study Type:

Expanded Access

Official Title:

An Open-Label, Expanded Access Protocol for Amifampridine Phosphate Treatment in Patients With Congenital Myasthenic Syndrome (CMS)

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years and Older

Sexes Eligible for Study:

All

Inclusion Criteria:

Male or female:
Confirmed genetic diagnosis of CMS.
Negative urine pregnancy test for females of childbearing potential at Screening.
If sexually active and of childbearing potential, willing to use 2 acceptable methods of contraception from screening visit until 3 months after the last dose of investigational product. No adequate clinical data on exposed pregnancies are available for amifampridine. No nonclinical safety data are available regarding the effects of amifampridine on reproductive function. Amifampridine phosphate should not be used during pregnancy. It is unknown whether amifampridine is excreted in human breast milk. The excretion of amifampridine in milk has not been studied in animals. Amifampridine phosphate should not be used during breastfeeding.
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.

Exclusion Criteria:

History of epilepsy and on medication/treatment for the same.
CMS subtypes including slow-channel syndrome, LRP4 deficiency, and acetylcholinesterase deficiency.
Current use of dalfampridine (Ampyra®; 4-aminopyridine), and any form of 3,4 DAP other than the investigational product provided, such as amifampridine base and does not agree to discontinue use for the duration of the study.
Use of guanidine hydrochloride within 7 days of starting amifampridine phosphate treatment.
History of drug allergy to any pyridine-containing substances or any amifampridine phosphate excipients (i.e. microcrystalline cellulose, colloidal silicon dioxide or calcium stearate).
Use of any other investigational product (other than 3,4 DAP or amifampridine phosphate) or investigational medical device within 30 days before starting treatment or requirement for any investigational agent before completion of all scheduled study assessments.
An electrocardiogram (ECG) within 6 months before starting treatment that shows clinically significant abnormality(ies), in the opinion of the patient's personal physician.
Breastfeeding or pregnant or planning to become pregnant (self or partner). Male patients with breastfeeding partners are not excluded from the study.
Any condition that, in the view of the Principal Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.