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

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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.

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition: Lambert Eaton Myasthenic Syndrome
Interventions: Drug: Amifampridine Phosphate
Drug: Placebo

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations

The study was conducted at 13 clinical sites in 8 countries, including France, Germany, Hungary, Poland, Russia, Serbia, Spain, and the United States.

The study was conducted from 03Jun2011 to 08Jul2016.


Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Open-label Run-in (Part 1): titration to the optimal amifampridine dose (well tolerated and resulted in a ≥3 point improvement in QMG score from Screening for patients without previous amifampridine use) for each individual patient. Patients who did not receive amifampridine prior to Run-in and who did not reach the optimal dose were discontinued.

Reporting Groups
  Description
Part 2 and Part 3 Placebo

Matching placebo tablets, administered 3-4 times a day for 2 weeks.

Placebo tablets indistinguishable from amifampridine phosphate tablets. The placebo was administered consistent with the dose and dose regimen of amifampridine phosphate.

Part 2 and Part 3 Amifampridine Phosphate

Matching amifampridine phosphate tablets, 10 mg, administered 3-4 times a day for 2 weeks.

Amifampridine Phosphate: 30-80 mg given 3-4 times per day with a maximum single dose of 20 mg (2 x 10 mg tablets).


Participant Flow:   Overall Study
    Part 2 and Part 3 Placebo   Part 2 and Part 3 Amifampridine Phosphate
STARTED   22   16 
COMPLETED   21   16 
NOT COMPLETED   1   0 
rescue tx after part 2, entered part 4                1                0 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Part 2 and Part 3 Placebo

Matching placebo tablets, administered 3-4 times a day for 2 weeks.

Placebo tablets indistinguishable from amifampridine phosphate tablets. The placebo was administered consistent with the dose and dose regimen of amifampridine phosphate.

Part 2 and Part 3 Amifampridine Phosphate

Matching amifampridine phosphate tablets, 10 mg, administered 3-4 times a day for 2 weeks.

Amifampridine Phosphate: 30-80 mg given 3-4 times per day with a maximum single dose of 20 mg (2 x 10 mg tablets).

Total Total of all reporting groups

Baseline Measures
   Part 2 and Part 3 Placebo   Part 2 and Part 3 Amifampridine Phosphate   Total 
Overall Participants Analyzed 
[Units: Participants]
 22   16   38 
Age 
[Units: Years]
Mean (Standard Deviation)
     
Participants Analyzed   22   16   38 
   51.5  (17.57)   51.6  (12.05)   51.5  (15.30) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Participants Analyzed   22   16   38 
Female      14  63.6%      9  56.3%      23  60.5% 
Male      8  36.4%      7  43.8%      15  39.5% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
     
Participants Analyzed   22   16   38 
Hispanic or Latino   0   3   3 
Not Hispanic or Latino   22   12   34 
Unknown or Not Reported   0   1   1 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
     
Participants Analyzed   22   16   38 
American Indian or Alaska Native      0   0.0%      0   0.0%      0   0.0% 
Asian      0   0.0%      0   0.0%      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      0   0.0%      0   0.0%      0   0.0% 
White      21  95.5%      13  81.3%      34  89.5% 
More than one race      0   0.0%      2  12.5%      2   5.3% 
Unknown or Not Reported      1   4.5%      1   6.3%      2   5.3% 
Was the patient taking amifampridine (base or phosphate) immediately prior to enrollment? 
[Units: Participants]
Count of Participants
     
Participants Analyzed   22   16   38 
Yes      7  31.8%      3  18.8%      10  26.3% 
No      15  68.2%      13  81.3%      28  73.7% 
If yes, number of continuous days of amifampridine exposure immediately prior to enrollment [1] 
[Units: Days]
Mean (Standard Deviation)
     
Participants Analyzed   7   3   10 
   1287.1  (1525.73)   2143.3  (3080.16)   1544.0  (1957.36) 
[1] Data only for those subjects (7 in the placebo group, 3 in the amifampridine group) who were taking amifampridine prior to enrollment.


  Outcome Measures

1.  Primary:   Change From Baseline Quantitative Myasthenia Gravis (QMG) at 14 Days   [ Time Frame: Assessment at Baseline and Day 14 ]

2.  Primary:   Change in SGI Score   [ Time Frame: Assessment at Baseline and Day 14 ]

3.  Secondary:   Change From Baseline Timed 25 Foot Walking Test (T25FW) at 14 Days   [ Time Frame: Assessment at Baseline and Day 14 ]

4.  Secondary:   Change in CGI-I Score   [ Time Frame: Baseline and Day 14 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Gary Ingenito
Organization: Catalyst Pharmaceuticals, Inc.
phone: 305-420-3200
e-mail: gingenito@catalystpharma.com



Responsible Party: Catalyst Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT01377922     History of Changes
Other Study ID Numbers: LMS-002
First Submitted: June 17, 2011
First Posted: June 22, 2011
Results First Submitted: September 28, 2017
Results First Posted: January 4, 2018
Last Update Posted: January 4, 2018