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A Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS)

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: NCT01377922
Recruitment Status : Completed
First Posted : June 22, 2011
Results First Posted : January 4, 2018
Last Update Posted : January 4, 2018
Sponsor:
Information provided by (Responsible Party):
Catalyst Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE June 17, 2011
First Posted Date  ICMJE June 22, 2011
Results First Submitted Date  ICMJE September 28, 2017
Results First Posted Date  ICMJE January 4, 2018
Last Update Posted Date January 4, 2018
Study Start Date  ICMJE June 2011
Actual Primary Completion Date July 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 3, 2018)
  • Change From Baseline Quantitative Myasthenia Gravis (QMG) at 14 Days [ Time Frame: Assessment at Baseline and Day 14 ]
    The QMG is a physician-rated test including 13 assessments, including facial strength, swallowing, grip strength, and duration of time that limbs can be maintained in outstretched positions. Each of the 13 items is scored from 0 (none) to 3 (severe). The total score can range from 0 to 39. Increased QMG total score correlates to worsening symptoms of LEMS.
  • Change in SGI Score [ Time Frame: Assessment at Baseline and Day 14 ]
    Subject Global Impression (SGI) is a measure of changes in subject's perception of change in overall wellbeing. The patient is asked to use the 7-point scale below to rate their impression of the effects of the study medication during the preceding 3 days on their physical well being.
    1. Terrible
    2. Mostly dissatisfied
    3. Mixed
    4. Partially satisfied
    5. Mostly satisfied
    6. Pleased
    7. Delighted
Original Primary Outcome Measures  ICMJE
 (submitted: June 20, 2011)
Change From Baseline Quantitative Myasthenia Gravis (QMG) at 14 Days [ Time Frame: Assessment at Baseline, Day 8, and Day 14 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 3, 2018)
  • Change From Baseline Timed 25 Foot Walking Test (T25FW) at 14 Days [ Time Frame: Assessment at Baseline and Day 14 ]
    The T25FW test, a component of the Multiple Sclerosis Functional Composite, was a quantitative mobility and leg function performance test based on a timed 25-foot walk (National Multiple Sclerosis Society). The patient was directed to walk a clearly marked 25-foot course as quickly and safely as possible. Following a rest of at least 5 minutes, the timed 25-foot walk was repeated. Patients could use assistive devices, such as canes, crutches, or walkers. All data were normalized to the number of feet per minute, so if the patient walked 25 feet in less than a minute, the result was a speed greater than 25 feet/minute. The measurement for the T25FW test was the average speed, expressed in feet/minute, of the 2 completed walks.
  • Change in CGI-I Score [ Time Frame: Baseline and Day 14 ]
    The Investigator completed the 7-point CGI I, based on changes in symptoms, behavior, and functional abilities, at the protocol-specified time points compared to the patient's condition at Day 0.
    1. = Very much improved
    2. = Much improved
    3. = Minimally improved
    4. = No change
    5. = Minimally worse
    6. = Much worse
    7. = Very much worse
Original Secondary Outcome Measures  ICMJE
 (submitted: June 20, 2011)
Change From Baseline Timed 25 Foot Walking Test (T25FW) at 14 Days [ Time Frame: Assessment at Baseline, Day 8, and Day 14 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS)
Official Title  ICMJE 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)
Brief Summary A Phase 3 study to evaluate the efficacy and safety of Amifampridine Phosphate in patients with Lambert-Eaton Myasthenic Syndrome (LEMS).
Detailed Description This multicenter, double-blind, placebo-controlled, randomized (1:1) discontinuation study is a 4-part study designed to evaluate the efficacy and safety of multiple dose administration of amifampridine phosphate in patients with LEMS. Data from parts 2 and 3 (the double-blind parts of the study) are presented in this record.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Lambert Eaton Myasthenic Syndrome
Intervention  ICMJE
  • Drug: Amifampridine Phosphate
    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.
    Other Names:
    • 3,4-diaminopyridine phosphate
    • 3,4-DAP phosphate
  • Drug: Placebo
    Matching placebo tablets administered 3-4 times a day (to the individual patient's tablet count of active at baseline) over 2 weeks.
Study Arms  ICMJE
  • Placebo Comparator: Placebo
    Matching placebo tablets administered 3-4 times a day (to the individual patient's tablet count of active at baseline) over 2 weeks.
    Intervention: Drug: Placebo
  • Experimental: Amifampridine Phosphate
    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.
    Intervention: Drug: Amifampridine Phosphate
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 26, 2016)
38
Original Estimated Enrollment  ICMJE
 (submitted: June 20, 2011)
30
Actual Study Completion Date  ICMJE July 2016
Actual Primary Completion Date July 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France,   Germany,   Hungary,   Poland,   Russian Federation,   Serbia,   Spain,   United States
Removed Location Countries Canada,   Czech Republic,   Italy
 
Administrative Information
NCT Number  ICMJE NCT01377922
Other Study ID Numbers  ICMJE LMS-002
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Catalyst Pharmaceuticals, Inc.
Study Sponsor  ICMJE Catalyst Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Charles W Gorodetzky, MD, PhD Chief Medical Officer
PRS Account Catalyst Pharmaceuticals, Inc.
Verification Date January 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP