A Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS)
Condition: Lambert Eaton Myasthenic Syndrome
MeSH terms: Lambert-Eaton Myasthenic Syndrome; Syndrome
Intervention: Amifampridine Phosphate; Placebo
Intervention description: Amifampridine, 30-80 mg given 3-4 times per day with a maximum single dose of 20 mg (2 x 10 mg tablets), for 2 weeks.; Matching placebo tablets administered 3-4 times a day (to the individual patient's tablet count of active at baseline) over 2 weeks.
Intervention MeSH terms: Amifampridine
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 38.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      A Phase 3 study to evaluate the efficacy and safety of Amifampridine Phosphate in patients
      with Lambert-Eaton Myasthenic Syndrome (LEMS).
    

Eligibility criteria:

        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.