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Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

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: NCT02562066
Recruitment Status : Completed
First Posted : September 29, 2015
Results First Posted : April 2, 2021
Last Update Posted : April 2, 2021
Sponsor:
Information provided by (Responsible Party):
Catalyst Pharmaceuticals, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Myasthenic Syndromes, Congenital
Interventions Drug: amifampridine phosphate
Drug: Placebo
Enrollment 20
Recruitment Details The study was conducted from 08 February 2016 - 24 January 2019 and was conducted at six sites in the United States and two sites in Canada, although one site (Montreal Neurological Institute and Hospital) did not enroll any patients.
Pre-assignment Details Screening ensured patients met I/E criteria. Patients naïve to amifampridine began open-label titration until a stable dose and frequency was achieved for 7 days. Demonstrated efficacy (assessed by improvement on the MFM) was required to participate in the randomized portion of the study. Patients already on a stable dose with hostory of meaningful improvement were eligible immediately and transitioned to an equivalent dose of amifampridine phosphate for 7 days prior to randomization.
Arm/Group Title Amifampridine Phosphate - Placebo Placebo - Amifampridine Phosphate Amifampridine Phosphate Only
Hide Arm/Group Description

Each patient will participate in an open-label unblinded drug escalation/treatment run-in phase for up to 4 weeks until stable dose and frequency of amifampridine phosphate is achieved for 7 days. After this phase, half of the subjects will be randomized to receive amifampridine in Period I and then crossed over to receive placebo in Period II. Each randomized treatment period is 7 days in duration and is separated by a re-stabilization period of approximately two weeks where the subject will be returned to the stable dose administered at the end of the open-label run-in period. Dosing will be between 20 - 80 mg per day and frequency will be between 2-4 times per day.

Amifampridine Phosphate: Amifampridine phosphate tablets 10 mg will be provided in round, white-scored tablets, and containing amifampridine phosphate formulated to be the equivalent of 10 mg amifampridine base per tablet.

Placebo: A placebo equivalent will be provided as tablets indistinguishable from the amifampridine phosphate tablets. The placebo will be administered consistent with the dose regimen of amifampridine phosphate.

Each patient will participate in an open-label unblinded drug escalation/treatment run-in phase for up to 4 weeks until stable dose and frequency of amifampridine phosphate is achieved for 7 days. After this phase, half of the subjects will be randomized to receive placebo in Period I and then crossed over to receive amifampridine in Period II. Each randomized treatment period is 7 days in duration and separated by a re-stabilization period of approximately two weeks where the subject will be returned to the stable dose administered at the end of the open-label run-in period. Dosing will be between 20 - 80 mg per day and frequency will be between 2-4 times per day.

amifampridine phosphate: Amifampridine phosphate tablets 10 mg will be provided in round, white-scored tablets, and containing amifampridine phosphate formulated to be the equivalent of 10 mg amifampridine base per tablet.

Placebo: A placebo equivalent will be provided as tablets indistinguishable from the amifampridine phosphate tablets. The placebo will be administered consistent with the dose regimen of amifampridine phosphate.

Patients receiving amifampridine during the open-label run-in period but were not randomized for the crossover portion of the study.
Period Title: Overall Study
Started 8 8 4
Completed 7 7 0
Not Completed 1 1 4
Arm/Group Title Amifampridine Phosphate - Placebo Placebo - Amifampridine Phosphate Total
Hide Arm/Group Description

Each patient will participate in an open-label unblinded drug escalation/treatment run-in phase for up to 4 weeks until stable dose and frequency of amifampridine phosphate is achieved for 7 days. After this phase, half of the subjects will be randomized to receive amifampridine in Period I and then crossed over to receive placebo in Period II. Each randomized treatment period is 7 days in duration and is separated by a re-stabilization period of approximately two weeks where the subject will be returned to the stable dose administered at the end of the open-label run-in period. Dosing will be between 20 - 80 mg per day and frequency will be between 2-4 times per day.

Amifampridine Phosphate: Amifampridine phosphate tablets 10 mg will be provided in round, white-scored tablets, and containing amifampridine phosphate formulated to be the equivalent of 10 mg amifampridine base per tablet.

Placebo: A placebo equivalent will be provided as tablets indistinguishable from the amifampridine phosphate tablets. The placebo will be administered consistent with the dose regimen of amifampridine phosphate.

Each patient will participate in an open-label unblinded drug escalation/treatment run-in phase for up to 4 weeks until stable dose and frequency of amifampridine phosphate is achieved for 7 days. After this phase, half of the subjects will be randomized to receive placebo in Period I and then crossed over to receive amifampridine in Period II. Each randomized treatment period is 7 days in duration and separated by a re-stabilization period of approximately two weeks where the subject will be returned to the stable dose administered at the end of the open-label run-in period. Dosing will be between 20 - 80 mg per day and frequency will be between 2-4 times per day.

amifampridine phosphate: Amifampridine phosphate tablets 10 mg will be provided in round, white-scored tablets, and containing amifampridine phosphate formulated to be the equivalent of 10 mg amifampridine base per tablet.

Placebo: A placebo equivalent will be provided as tablets indistinguishable from the amifampridine phosphate tablets. The placebo will be administered consistent with the dose regimen of amifampridine phosphate.

Total of all reporting groups
Overall Number of Baseline Participants 8 8 16
Hide Baseline Analysis Population Description
Safety population: All randomized patients who have received at least one dose of study medication. Note that subjects in the Amifampridine Phosphate Only arm are excluded from this analysis, as they were not randomized for inclusion in the crossover portion of the study.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 8 participants 16 participants
<=18 years
7
  87.5%
5
  62.5%
12
  75.0%
Between 18 and 65 years
1
  12.5%
2
  25.0%
3
  18.8%
>=65 years
0
   0.0%
1
  12.5%
1
   6.3%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants 8 participants 16 participants
12.59  (15.70) 27.35  (25.00) 19.97  (21.56)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 8 participants 16 participants
Female
5
  62.5%
3
  37.5%
8
  50.0%
Male
3
  37.5%
5
  62.5%
8
  50.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 8 participants 16 participants
Hispanic or Latino
0
   0.0%
1
  12.5%
1
   6.3%
Not Hispanic or Latino
8
 100.0%
7
  87.5%
15
  93.8%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants 8 participants 16 participants
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%
1
  12.5%
1
   6.3%
White
8
 100.0%
5
  62.5%
13
  81.3%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
2
  25.0%
2
  12.5%
1.Primary Outcome
Title Subject Global Impression (SGI) Score Summary: Mann-Whitney Main Effects Test Results; SGI Score Mixed Model Analysis
Hide Description Subject Global Impression (SGI) rates the subject's impression of the effects of the study medication during the preceding week with max score =7 (most satisfied) and min score =1 (least satisfied). It will be completed at D0 (baseline for Study Period 1), D8, D21 (baseline for Study Period 2), and D29. Change from baseline (CFB) will be assessed for Study Period 1 (difference in SGI score from D0 to D8), Study Period 2 (difference in SGI score from D21 to D29) and the change in CFB from Study Period 1 to Study Period 2 (difference between CFB during Study Period 1 and CFB during Study Period 2). A Mann-Whitney-Wilcoxon test for equality of the CFB results in the two treatments in Period 1 and for the equality of the CFB results in the two treatment sequences will be conducted. A mixed effects liner model will be fit with the SGI raw scores as a response and study arm, treatment, period, age group and mutation type as fixed effect terms and patient as a random effect.
Time Frame Study Period 1: Baseline (Day 0), Day 8; Study Period 2: Baseline (Day 21), Day 29
Hide Outcome Measure Data
Hide Analysis Population Description
The Full Analysis Set consists of all randomized patients who receive at least one dose of study medication and have at least one post-treatment efficacy assessment.
Arm/Group Title Amifampridine Phosphate - Placebo Placebo - Amifampridine Phosphate
Hide Arm/Group Description:

Each patient will participate in an open-label unblinded drug escalation/treatment run-in phase for up to 4 weeks until stable dose and frequency of amifampridine phosphate is achieved for 7 days. After this phase, half of the subjects will be randomized to receive amifampridine in Period I and then crossed over to receive placebo in Period II. Each randomized treatment period is 7 days in duration and is separated by a re-stabilization period of approximately two weeks where the subject will be returned to the stable dose administered at the end of the open-label run-in period. Dosing will be between 20 - 80 mg per day and frequency will be between 2-4 times per day.

Amifampridine Phosphate: Amifampridine phosphate tablets 10 mg will be provided in round, white-scored tablets, and containing amifampridine phosphate formulated to be the equivalent of 10 mg amifampridine base per tablet.

Placebo: A placebo equivalent will be provided as tablets indistinguishable from the amifampridine phosphate tablets. The placebo will be administered consistent with the dose regimen of amifampridine phosphate.

Each patient will participate in an open-label unblinded drug escalation/treatment run-in phase for up to 4 weeks until stable dose and frequency of amifampridine phosphate is achieved for 7 days. After this phase, half of the subjects will be randomized to receive placebo in Period I and then crossed over to receive amifampridine in Period II. Each randomized treatment period is 7 days in duration and separated by a re-stabilization period of approximately two weeks where the subject will be returned to the stable dose administered at the end of the open-label run-in period. Dosing will be between 20 - 80 mg per day and frequency will be between 2-4 times per day.

amifampridine phosphate: Amifampridine phosphate tablets 10 mg will be provided in round, white-scored tablets, and containing amifampridine phosphate formulated to be the equivalent of 10 mg amifampridine base per tablet.

Placebo: A placebo equivalent will be provided as tablets indistinguishable from the amifampridine phosphate tablets. The placebo will be administered consistent with the dose regimen of amifampridine phosphate.

Overall Number of Participants Analyzed 8 8
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
Study Period 1: CFB in SGI Score at Day 8 -0.13  (1.81) -1.75  (2.12)
Study Period 2 : CFB in SGI Score at Day 29 -0.75  (1.75) -1.14  (1.68)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Amifampridine Phosphate - Placebo, Placebo - Amifampridine Phosphate
Comments A Mann-Whitney-Wilcoxon test for equality of the change from baseline (CFB) in the two treatments in Period 1 will be presented.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.085
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.63
Estimation Comments Results show the difference in mean CFB at the end of Study Period 1.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Amifampridine Phosphate - Placebo
Comments A mixed effects linear model will be fit to the data with SGI raw scores as the response and fixed effect terms for treatment, period, age group, mutation type and sequence*period, and a random effect for patient. The LSMeans for the estimated difference between treatments in Period 1 along with a 95% confidence interval for this difference.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value [Not Specified]
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.56
Confidence Interval (2-Sided) 95%
-0.97 to 2.09
Estimation Comments Results show the LSMeans for the estimated difference between treatments in Period 1 along with a 95% confidence interval for this difference.
2.Secondary Outcome
Title Motor Function Measure 32 (MFM-32) Score Summary: Mann-Whitney Main Effect Test Results, MFM-32 Score Mixed Model Analysis
Hide Description Motor Function Measure 32 (MFM-32) evaluates the severity and progression of motor function in patients 7 years of age or older with max total score = 96 (best score) and min total score =0 (worst score). It will be completed at D0 (baseline for Study Period 1), D8, D21 (baseline for Study Period 2) and D29. CFB will be assessed for Study Period 1 (difference in MFM-32 from D0 to D8), Study Period 2 (difference in MFM-32 from D21 to D28) and the change in CFB from Study Period 1 to Study Period 2 (difference between CFB during Study Period 1 and CFB during Study Period 2). A Mann-Whitney-Wilcoxon test for equality of CFB results in the two treatments in Period 1 and for the equality of the CFB results in the two treatment sequences will be conducted. A mixed effects linear model will be fit to the overall MFM-32 score as the response variable and fixed effect terms for treatment, period, age group, genetic mutation type, and sequence*period and a random effect for patient.
Time Frame Study Period 1: Baseline (Day 0), Day 8; Study Period 2: Baseline (Day 21), Day 29
Hide Outcome Measure Data
Hide Analysis Population Description
The Full Analysis Set consists of all randomized patients who receive at least one dose of study medication and have at least one post-treatment efficacy assessment.
Arm/Group Title Amifampridine Phosphate - Placebo Placebo - Amifampridine Phosphate
Hide Arm/Group Description:

Each patient will participate in an open-label unblinded drug escalation/treatment run-in phase for up to 4 weeks until stable dose and frequency of amifampridine phosphate is achieved for 7 days. After this phase, half of the subjects will be randomized to receive amifampridine in Period I and then crossed over to receive placebo in Period II. Each randomized treatment period is 7 days in duration and is separated by a re-stabilization period of approximately two weeks where the subject will be returned to the stable dose administered at the end of the open-label run-in period. Dosing will be between 20 - 80 mg per day and frequency will be between 2-4 times per day.

Amifampridine Phosphate: Amifampridine phosphate tablets 10 mg will be provided in round, white-scored tablets, and containing amifampridine phosphate formulated to be the equivalent of 10 mg amifampridine base per tablet.

Placebo: A placebo equivalent will be provided as tablets indistinguishable from the amifampridine phosphate tablets. The placebo will be administered consistent with the dose regimen of amifampridine phosphate.

Each patient will participate in an open-label unblinded drug escalation/treatment run-in phase for up to 4 weeks until stable dose and frequency of amifampridine phosphate is achieved for 7 days. After this phase, half of the subjects will be randomized to receive placebo in Period I and then crossed over to receive amifampridine in Period II. Each randomized treatment period is 7 days in duration and separated by a re-stabilization period of approximately two weeks where the subject will be returned to the stable dose administered at the end of the open-label run-in period. Dosing will be between 20 - 80 mg per day and frequency will be between 2-4 times per day.

amifampridine phosphate: Amifampridine phosphate tablets 10 mg will be provided in round, white-scored tablets, and containing amifampridine phosphate formulated to be the equivalent of 10 mg amifampridine base per tablet.

Placebo: A placebo equivalent will be provided as tablets indistinguishable from the amifampridine phosphate tablets. The placebo will be administered consistent with the dose regimen of amifampridine phosphate.

Overall Number of Participants Analyzed 8 8
Mean (Standard Deviation)
Unit of Measure: Score on a Scale
Study Period 1: CFB in Overall MFM-32 Score at Day 8 Number Analyzed 4 participants 6 participants
3.00  (3.92) -0.17  (4.58)
Study Period 2: CFB in Overall MFM-32 Score at Day 29 Number Analyzed 4 participants 5 participants
0.75  (2.22) -0.60  (0.89)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Amifampridine Phosphate - Placebo, Placebo - Amifampridine Phosphate
Comments A Mann-Whitney-Wilcoxon test for equality of the change from baseline (CFB) in the two treatments in Period 1 will be presented.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.376
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 3.17
Estimation Comments Results show the difference in mean CFB at the end of Study Period 1.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Amifampridine Phosphate - Placebo, Placebo - Amifampridine Phosphate
Comments A mixed effects linear model will be fit to the data with Overall MFM-32 scores as the response and fixed effect terms for treatment, period, age group, mutation type and sequence*period, and a random effect for patient. The LSMeans for the estimated difference between treatments in Period 1 along with a 95% confidence interval for this difference.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value [Not Specified]
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.14
Confidence Interval (2-Sided) 95%
-2.31 to 4.58
Estimation Comments Results show the LSMeans for the estimated difference between treatments in Period 1 along with a 95% confidence interval for this difference.
3.Secondary Outcome
Title Motor Function Measure 20 (MFM-20) Score Mixed Model Analysis, MFM-20 Score Mixed Model Analysis
Hide Description Motor Function Measure 20 (MFM-20) evaluates the severity and progression of motor function in patients 2 to 6 years of age with max total score = 60 (best score) and min total score =0 (worst score). It will be completed at D0 (baseline for Study Period 1), D8, D21 (baseline for Study Period 2) and D29. CFB will be assessed for Study Period 1 (difference in MFM-20 from D0 to D8), Study Period 2 (difference in MFM-20 from D21 to D28) and the change in CFB from Study Period 1 to Study Period 2 (difference between CFB during Study Period 1 and CFB during Study Period 2). A Mann-Whitney-Wilcoxon test for equality of CFB results in the two treatments in Period 1 and for the equality of the CFB results in the two treatment sequences will be conducted. A mixed effects linear model will be fit to the overall MFM-32 score as the response variable and fixed effect terms for treatment, period, age group, genetic mutation type, and sequence*period and a random effect for patient.
Time Frame Study Period 1: Baseline (Day 0), Day 8; Study Period 2: Baseline (Day 21), Day 29
Hide Outcome Measure Data
Hide Analysis Population Description
The Full Analysis Set consists of all randomized patients who receive at least one dose of study medication and have at least one post-treatment efficacy assessment.
Arm/Group Title Amifampridine Phosphate - Placebo Placebo - Amifampridine Phosphate
Hide Arm/Group Description:

Each patient will participate in an open-label unblinded drug escalation/treatment run-in phase for up to 4 weeks until stable dose and frequency of amifampridine phosphate is achieved for 7 days. After this phase, half of the subjects will be randomized to receive amifampridine in Period I and then crossed over to receive placebo in Period II. Each randomized treatment period is 7 days in duration and is separated by a re-stabilization period of approximately two weeks where the subject will be returned to the stable dose administered at the end of the open-label run-in period. Dosing will be between 20 - 80 mg per day and frequency will be between 2-4 times per day.

Amifampridine Phosphate: Amifampridine phosphate tablets 10 mg will be provided in round, white-scored tablets, and containing amifampridine phosphate formulated to be the equivalent of 10 mg amifampridine base per tablet.

Placebo: A placebo equivalent will be provided as tablets indistinguishable from the amifampridine phosphate tablets. The placebo will be administered consistent with the dose regimen of amifampridine phosphate.

Each patient will participate in an open-label unblinded drug escalation/treatment run-in phase for up to 4 weeks until stable dose and frequency of amifampridine phosphate is achieved for 7 days. After this phase, half of the subjects will be randomized to receive placebo in Period I and then crossed over to receive amifampridine in Period II. Each randomized treatment period is 7 days in duration and separated by a re-stabilization period of approximately two weeks where the subject will be returned to the stable dose administered at the end of the open-label run-in period. Dosing will be between 20 - 80 mg per day and frequency will be between 2-4 times per day.

amifampridine phosphate: Amifampridine phosphate tablets 10 mg will be provided in round, white-scored tablets, and containing amifampridine phosphate formulated to be the equivalent of 10 mg amifampridine base per tablet.

Placebo: A placebo equivalent will be provided as tablets indistinguishable from the amifampridine phosphate tablets. The placebo will be administered consistent with the dose regimen of amifampridine phosphate.

Overall Number of Participants Analyzed 8 8
Mean (Standard Deviation)
Unit of Measure: Score on a Scale
Study Period 1: CFB in Overall MFM-20 Score at Day 8 Number Analyzed 4 participants 2 participants
-1.50  (4.20) -0.50  (6.36)
Study Period 2: CFB in Overall MFM-20 Score at Day 29 Number Analyzed 4 participants 2 participants
-0.75  (0.50) 1.50  (2.12)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Amifampridine Phosphate - Placebo, Placebo - Amifampridine Phosphate
Comments A Mann-Whitney-Wilcoxon test for equality of the change from baseline (CFB) in the two treatments in Period 1 will be presented.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.800
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1.00
Estimation Comments Results show the difference in mean CFB at the end of Study Period 1.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Amifampridine Phosphate - Placebo, Placebo - Amifampridine Phosphate
Comments A mixed effects linear model will be fit to the data with Overall MFM-20 scores as the response and fixed effect terms for treatment, period, age group, mutation type and sequence*period, and a random effect for patient. The LSMeans for the estimated difference between treatments in Period 1 along with a 95% confidence interval for this difference.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value [Not Specified]
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.63
Confidence Interval (2-Sided) 95%
-5.25 to 6.50
Estimation Comments Results show the LSMeans for the estimated difference between treatments in Period 1 along with a 95% confidence interval for this difference.
Time Frame Serious adverse events (SAEs) were recorded for each patient from the time informed consent was obtained and continues through four weeks after the last dose or at the early discontinuation visit, for a total of approximately 14 weeks. Non-serious AEs were recorded for each patient after informed consent is obtained and the first administration of study drug (on Day 1 of run-in) through the last visit or at the early discontinuation visit, for a total of approximately 10 weeks .
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Amifampridine Phosphate Placebo
Hide Arm/Group Description Treatment administered to the patient at the time of onset of the AE. Includes patients who received amifampridine phosphate during the run-in period who were not randomized for inclusion in the crossover portion of the study. Treatment administered to the patient at the time of onset of the AE.
All-Cause Mortality
Amifampridine Phosphate Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/20 (0.00%)   0/20 (0.00%) 
Hide Serious Adverse Events
Amifampridine Phosphate Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   1/20 (5.00%)   1/20 (5.00%) 
General disorders     
Pyrexia  1  0/20 (0.00%)  1/20 (5.00%) 
Infections and infestations     
Parainfluenzae virus infection  1  0/20 (0.00%)  1/20 (5.00%) 
Rhinovirus infection  1  1/20 (5.00%)  0/20 (0.00%) 
Vascular disorders     
Hypotension  1  0/20 (0.00%)  1/20 (5.00%) 
1
Term from vocabulary, MedDRA 19.1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Amifampridine Phosphate Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   11/20 (55.00%)   6/20 (30.00%) 
Ear and labyrinth disorders     
Ear haemorrhage  1  1/20 (5.00%)  0/20 (0.00%) 
Ear pain  1  0/20 (0.00%)  1/20 (5.00%) 
Gastrointestinal disorders     
Diarrhoea  1  1/20 (5.00%)  0/20 (0.00%) 
Food poisoning  1  1/20 (5.00%)  0/20 (0.00%) 
Nausea  1  1/20 (5.00%)  1/20 (5.00%) 
Paraesthesia oral  1  1/20 (5.00%)  0/20 (0.00%) 
Retching  1  1/20 (5.00%)  0/20 (0.00%) 
Vomiting  1  3/20 (15.00%)  1/20 (5.00%) 
General disorders     
Pyrexia  1  1/20 (5.00%)  0/20 (0.00%) 
Immune system disorders     
Allergy to animal  1  0/20 (0.00%)  1/20 (5.00%) 
Infections and infestations     
Conjunctivitis  1  0/20 (0.00%)  1/20 (5.00%) 
Nasopharyngitis  1  3/20 (15.00%)  0/20 (0.00%) 
Upper respiratory tract infection  1  2/20 (10.00%)  0/20 (0.00%) 
Musculoskeletal and connective tissue disorders     
Pain in extremity  1  1/20 (5.00%)  0/20 (0.00%) 
Nervous system disorders     
Headache  1  3/20 (15.00%)  0/20 (0.00%) 
Hypoaesthesia  1  0/20 (0.00%)  1/20 (5.00%) 
Migraine  1  0/20 (0.00%)  1/20 (5.00%) 
Paraesthesia  1  2/20 (10.00%)  0/20 (0.00%) 
Psychiatric disorders     
Agitation  1  1/20 (5.00%)  0/20 (0.00%) 
Anger  1  1/20 (5.00%)  0/20 (0.00%) 
Irritability  1  1/20 (5.00%)  0/20 (0.00%) 
Reproductive system and breast disorders     
Menstruation irregular  1  1/20 (5.00%)  0/20 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Asthma  1  1/20 (5.00%)  0/20 (0.00%) 
Skin and subcutaneous tissue disorders     
Acne  1  0/20 (0.00%)  1/20 (5.00%) 
Vascular disorders     
Haematoma  1  1/20 (5.00%)  0/20 (0.00%) 
1
Term from vocabulary, MedDRA 19.1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Results Point of Contact
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Name/Title: Gary Ingenito
Organization: Catalyst Pharmaceuticals, Inc.
Phone: 305-420-3200
EMail: gingenito@catalystpharma.com
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Responsible Party: Catalyst Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02562066    
Other Study ID Numbers: CMS-001
First Submitted: September 24, 2015
First Posted: September 29, 2015
Results First Submitted: February 8, 2021
Results First Posted: April 2, 2021
Last Update Posted: April 2, 2021