Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 18 September 2023
• Main ID: NCT02189720
• Date of registration: 05/07/2014
• Prospective Registration: Yes
• Primary sponsor: Catalyst Pharmaceuticals, Inc.
• Public title: Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome EAP-001
• Scientific title: An Open-Label, Expanded Access Protocol for Firdapse® (Amifampridine Phosphate; 3,4-Diaminopyridine Phosphate) Treatment in Pediatric Patients With Lambert-Eaton Myasthenic Syndrome (LEMS), and in Pediatric and Adult Patients With Congenital Myasthenic Syndromes (CMSs)
• Date of first enrolment: August 5, 2014
• Recruitment status: No longer available
• URL: https://clinicaltrials.gov/ct2/show/NCT02189720
• Study type: Expanded Access

Countries of recruitment

United States

Contacts

• Name: Perry Shieh, MD, PhD
• Affiliation: University of California, Los Angeles

Key inclusion & exclusion criteria

Inclusion Criteria - Amendment 5 v6 - most recent version

- Male or female:

- 2 years of age

- Confirmed physician diagnosis of LEMS in pediatric patients or CMS in either pediatric
or adult patients.

- Completion of anti-cancer treatment at least 3 months (90 days) before treatment.

- 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.

- Any subject currently participating in study CMS 001 is immediately eligible for
enrollment into study EAP-001, as long as inclusion/exclusion criteria are still met.

- 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- Amendment 5 v6

- History of epilepsy and on medication/treatment for the same.

- CMS subtypes including slow-channel syndrome, LRP4 deficiency, plectin deficiency and
acetylcholinesterase deficiency.

- Any subject with a LEMS diagnosis and =18 years of age.

- Known active brain metastasis. Patients with treated brain metastasis (radiotherapy
and/or surgery) who have completed treatment for their brain metastasis >90 days
before Screening, are neurologically stable (neurological symptoms grade <1), are on a
stable dose of corticosteroids and have no evidence of new disease on magnetic
resonance imaging (MRI) are eligible, provided they meet the other inclusion/exclusion
criteria.

- 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 for pediatric subjects <18 years of age.

- Breastfeeding or pregnant or planning to become pregnant (self or partner). Male
patients with breastfeeding partners are not excluded from the study.

- Patients with end stage kidney disease on dialysis.

- Any condition that, in the view of the Principal Investigator, places the patient at
risk, or patients with poor treatment compliance.

Inclusion Criteria: Protocol Amendment 1 ( first version IRB approved)

- 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: Protocol Amendment 1 ( first version IRB approved)

- 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.



Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Lambert-Eaton Myasthenic Syndrome

Nystagmus, Acquired

Congenital Myasthenic Syndrome

Intervention(s)

Drug: Amifampridine Phosphate

Secondary ID(s)

EAP-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors