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A Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS)
Not Recruiting
Trial ID: NCT01377922
Purpose
A Phase 3 study to evaluate the efficacy and safety of Amifampridine Phosphate in patients
with Lambert-Eaton Myasthenic Syndrome (LEMS).
Official Title
A Phase 3, Double-blind, Placebo-controlled, Randomized Discontinuation Study Followed by Open-label Extension Evaluating Efficacy and Safety of Amifampridine Phosphate in Patients With Lambert-Eaton Myasthenic Syndrome (LEMS)
Stanford Investigator(s)
Sarada Sakamuri, MD
Clinical Associate Professor, Neurology & Neurological Sciences Clinical Associate Professor (By courtesy), Neurosurgery
Eligibility
Inclusion Criteria: Individuals eligible to participate in this study must meet all of the
following inclusion criteria:
- ≥18 years of age
- Confirmed diagnosis of LEMS
- Normal respiratory function
- Normal swallowing function
- If receiving peripherally acting cholinesterase inhibitors a stable dose is required
for at least 7 days prior to Screening.
- If receiving oral immunosuppressants a stable dose is required for at least 90 days
prior to Screening.
- Negative pregnancy test for females of childbearing potential
- If sexually active, willing to use 2 acceptable methods of contraception
- Willing to perform all study procedures as physically possible.
- Willing and able to provide written informed consent after the nature of the study has
been explained and prior to the start of any research-related procedures.
Exclusion Criteria: Individuals who meet any of the following exclusion criteria are not
eligible to participate in the study:
- History of epilepsy or seizure.
- Known active brain metastasis.
- Use of Fampridine (4-aminopyridine), and any form of 3,4-diaminopyridine other than
the IP provided, such as amifampridine base or Firdapse, during the study.
- Use of medications known to lower the epileptic threshold within 7 days or 5
half-lives.
- Use of medications which inhibit neuromuscular junction function within 7 days or 5
half-lives.
- Use of IVIG, plasmapheresis (plasma exchange), or immunoadsorption within 90 days
- Use of guanidine hydrochloride within 7 days
- Use of rituximab within 12 months
- History of drug allergy to any pyridine-containing substances or any amifampridine
phosphate excipient(s).
- Use of any other investigational productwithin 30 days
- Treatment with a concomitant medication that prolongs the QT/QTc interval within 7
days or 5 half-lives.
- Treatment with sultopride
(4-amino-N-[(1-ethylpyrrolidin-2-yl)methyl]-5-ethylsulfonyl-2-methoxybenzamide) within
7 days.
- An abnormal electrocardiogram (ECG).
- Documented history of arrhythmias.
- History of additional risk factors for torsade de pointes.
- Breastfeeding or pregnant or planning to become pregnant (self or partner) at any time
during the study.
- Likely or expected to require treatment for cancer within 3 months (90 days) after
entering.
- History of severe renal impairment or evidence of severe renal impairment
- Any condition that places the patient at high risk of poor treatment compliance or of
not completing the study.
- History of uncontrolled asthma.
Intervention(s):
drug: Amifampridine Phosphate
drug: Placebo
Not Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305