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Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenic Syndrome (CMS), or Downbeat Nystagmus Patients (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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02189720
Expanded Access Status : Available
First Posted : July 15, 2014
Last Update Posted : October 16, 2018
Information provided by (Responsible Party):
Catalyst Pharmaceuticals, Inc.

Brief Summary:

The primary objective of the study is:

• To provide patients with LEMS/CMS/downbeat nystagmus access to amifampridine phosphate therapy until the product becomes commercially available.

The secondary objective of the study is:

• To assess the long-term safety of amifampridine phosphate in patients with LEMS/CMS/downbeat nystagmus

Condition or disease Intervention/treatment
Lambert-Eaton Myasthenic Syndrome Congenital Myasthenic Syndrome Downbeat Nystagmus 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 Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenic Syndrome (CMS) and Downbeat Nystagmus

Intervention Details:
  • Drug: Amifampridine Phosphate

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

    Amifampridine Phosphate is taken with food and each patient is given an individualized dose 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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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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:

    • 2 years of age at 5 pediatric CMS study sites
    • 10 years of age at other study sites.
  • Confirmed physician diagnosis of LEMS, CMS or downbeat nystagmus.
  • 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 studies LMS-002, LMS-002EXT, or 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:

  • History of epilepsy.
  • CMS subtypes including slow-channel syndrome, LRP4 deficiency, and acetylcholinesterase deficiency.
  • 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.
  • History of additional risk factors for torsade de pointes (e.g. history of surviving a near drowning due to loss of consciousness, family history of congenital QT syndrome, long QT syndrome, family history of unexplained early sudden death, or heart failure).
  • Breastfeeding or pregnant or planning to become pregnant (self or partner). Male patients with breastfeeding partners are not excluded from the study.
  • History of severe renal impairment or evidence of severe renal impairment at time of Screening on laboratory tests, specifically a creatinine clearance <30 mL/min (within 30 days) as calculated using the Cockcroft Gault formula.
  • History at time of Screening of laboratory tests (within 30 days) indicating hepatic impairment:

    o In patients without liver metastases from cancer, alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or total bilirubin >1.5 × upper limit of normal (ULN).

  • 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 identifier (NCT number): NCT02189720

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Contact: Jonathan Rubine, MD 305-420-3200 ext 133

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United States, California
Los Angeles, California, United States, 90095
Contact: Clara Sam    310-825-3264   
Contact: Gary Ingenito, MD, PhD    305-420-3200 ext 123   
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. Identifier: NCT02189720     History of Changes
Other Study ID Numbers: EAP-001
First Posted: July 15, 2014    Key Record Dates
Last Update Posted: October 16, 2018
Last Verified: October 2018

Keywords provided by Catalyst Pharmaceuticals, Inc.:
Amifampridine Phosphate
3,4-Diaminopyridine Phosphate
Lambert-Eaton Myasthenic Syndrome
Congenital Myasthenic Syndrome
Neuromuscular disorders
eye movement
Expanded Access
Downbeat Nystagmus

Additional relevant MeSH terms:
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Lambert-Eaton Myasthenic Syndrome
Nystagmus, Pathologic
Myasthenic Syndromes, Congenital
Pathologic Processes
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
Ocular Motility Disorders
Cranial Nerve Diseases
Eye 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