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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02189720
Expanded Access Status : No longer available
First Posted : July 15, 2014
Last Update Posted : December 9, 2019
Information provided by (Responsible Party):
Catalyst Pharmaceuticals, Inc.

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

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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)

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

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02189720

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United States, California
Los Angeles, California, United States, 90095
Sponsors and Collaborators
Catalyst Pharmaceuticals, Inc.
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Principal Investigator: Perry Shieh, MD, PhD University of California, Los Angeles
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Responsible Party: Catalyst Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02189720    
Other Study ID Numbers: EAP-001
First Posted: July 15, 2014    Key Record Dates
Last Update Posted: December 9, 2019
Last Verified: December 2019
Keywords provided by Catalyst Pharmaceuticals, Inc.:
Amifampridine Phosphate
3,4-Diaminopyridine Phosphate
Congenital Myasthenic Syndrome
Expanded Access
Additional relevant MeSH terms:
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Lambert-Eaton Myasthenic Syndrome
Myasthenic Syndromes, Congenital
Pathologic Processes
Myasthenia Gravis
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
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
Neuromuscular Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action