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

• ClinicalTrials.gov Identifier: NCT02189720
• Expanded Access Status: No longer available
• First Posted: July 15, 2014
• Last Update Posted: December 9, 2019
• Sponsor: Catalyst Pharmaceuticals, Inc.
• Information provided by (Responsible Party): Catalyst Pharmaceuticals, Inc.

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
Congenital Myasthenic Syndrome	Drug: Amifampridine Phosphate

Study Design

• Study Type: Expanded Access

See clinical trials of the intervention/treatment in this expanded access record.

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

Interventions

Intervention Details:

• Drug: Amifampridine Phosphate

  Dosage form: tablets containing the equivalent of 10 mg amifampridine per tablet.

  Amifampridine Phosphateis given based on Investigator assessment of optimal neuromuscular benefit. Doses range from 30 mg to 80 mg, divided into doses taken 3 to 4 times per day, with a maximum single dose of 20 mg.

  Other Names:

  • 3,4-Diaminopyridine Phosphate
  • 3,4 DAP

Eligibility Criteria

• Ages Eligible for Study: 2 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All

Criteria

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.

Contacts and Locations

Locations

United States, California

UCLA

Los Angeles, California, United States, 90095

Sponsors and Collaborators

Catalyst Pharmaceuticals, Inc.

Investigators

• Principal Investigator: Perry Shieh, MD, PhD; University of California, Los Angeles

More Information

• Responsible Party: Catalyst Pharmaceuticals, Inc.
• ClinicalTrials.gov Identifier: NCT02189720
• Other Study ID Numbers: EAP-001
• First Posted: July 15, 2014
• Last Update Posted: December 9, 2019
• Last Verified: December 2019

Keywords provided by Catalyst Pharmaceuticals, Inc.:

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

Additional relevant MeSH terms:

Lambert-Eaton Myasthenic Syndrome; Myasthenic Syndromes, Congenital; Syndrome; Disease; Pathologic Processes; Myasthenia Gravis; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Genetic Diseases, Inborn; Amifampridine; Neuromuscular Agents; Peripheral Nervous System Agents; Physiological Effects of Drugs; Potassium Channel Blockers; Membrane Transport Modulators; Molecular Mechanisms of Pharmacological Action