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

NCT02189720 | Unknown

• 1 other identifier: EAP-001
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

Primary: The primary objective of this study under the original protocol was to provide neuromuscular specialists and neurologists access to amifampridine phosphate therapy for their patients with LEMS, CMS or downbeat nystagmus until the product became commercially available. Secondary: The secondary objective of this study under the original protocol was to provide additional long-term safety data on amifampridine phosphate in patients. Primary The primary objective of this study after its fifth amendment was to provide access to amifampridine phosphate therapy to pediatric patients with LEMS, and pediatric and adult patients with CMS until the product became commercially available for these indications or development of the product for the indication was terminated. Secondary: The secondary objective of this study after its fifth amendment was to assess the long-term safety of amifampridine phosphate in pediatric patients with LEMS, and pediatric and adult patients with CMS.

Conditions

Congenital Myasthenic Syndrome; Lambert-Eaton Myasthenic Syndrome; Nystagmus, Acquired

Keywords

Amifampridine Phosphate; Amifampridine; 3,4-Diaminopyridine Phosphate; 3,4-Diaminopyridine; 3,4-DAP; CMS; Congenital Myasthenic Syndrome; Neuromuscular; Expanded Access; Firdapse®; LEMS

Interventions

Amifampridine Phosphate DRUG

Dosage form: tablets containing the equivalent of 10 mg amifampridine per tablet. Amifampridine Phosphate given based on Investigator assessment of optimal neuromuscular benefit. In adult patients \> 16 years of age, Doses ranged from 30 mg to 80 mg, divided into doses taken 3 to 4 times per day and in pediatric patients age \< 16 years the maximum daily dose was 60 mg/day. No single dose to exceed 20 mg.

Also known as: 3,4-Diaminopyridine Phosphate, 3,4 DAP, Firdapse®

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female:
• 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.

You may not qualify if:

• 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.
• 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.
• 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.
• Male or female:
• Confirmed genetic diagnosis of CMS.
• Negative urine pregnancy test for females of childbearing potential at Screening.

Sponsors & Collaborators

• Catalyst Pharmaceuticals, Inc.: lead

Study Sites (1)

UCLA

Los Angeles, California, 90095, United States

Location

MeSH Terms

Conditions

Myasthenic Syndromes, Congenital; Lambert-Eaton Myasthenic Syndrome; Nystagmus, Pathologic

Interventions

Amifampridine

Condition Hierarchy (Ancestors)

Neuromuscular Junction Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Myasthenia Gravis; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Autoimmune Diseases; Immune System Diseases; Ocular Motility Disorders; Cranial Nerve Diseases; Eye Diseases

Intervention Hierarchy (Ancestors)

4-Aminopyridine; Aminopyridines; Amines; Organic Chemicals; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Perry Shieh, MD, PhD

  University of California, Los Angeles

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 5, 2014
• First Posted: July 15, 2014
• Last Updated: March 26, 2025

Record last verified: 2024-05

Locations

US (1)