Trial Outcomes & Findings for A Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS) (NCT NCT01377922)

Last Updated: 2018-01-04

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

38 participants

Primary outcome timeframe

Assessment at Baseline and Day 14

Results posted on

2018-01-04

Participant Flow

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.

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.

Participant milestones

Participant milestones
Measure	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).
Overall Study STARTED	22	16
Overall Study COMPLETED	21	16
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	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).
Overall Study rescue tx after part 2, entered part 4	1	0

Baseline Characteristics

Data only for those subjects (7 in the placebo group, 3 in the amifampridine group) who were taking amifampridine prior to enrollment.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Part 2 and Part 3 Placebo n=22 Participants 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 n=16 Participants 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 n=38 Participants Total of all reporting groups
Age, Continuous	51.5 years STANDARD_DEVIATION 17.57 • n=22 Participants	51.6 years STANDARD_DEVIATION 12.05 • n=16 Participants	51.5 years STANDARD_DEVIATION 15.30 • n=38 Participants
Sex: Female, Male Female	14 Participants n=22 Participants	9 Participants n=16 Participants	23 Participants n=38 Participants
Sex: Female, Male Male	8 Participants n=22 Participants	7 Participants n=16 Participants	15 Participants n=38 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=22 Participants	3 Participants n=16 Participants	3 Participants n=38 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	22 Participants n=22 Participants	12 Participants n=16 Participants	34 Participants n=38 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=22 Participants	1 Participants n=16 Participants	1 Participants n=38 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=22 Participants	0 Participants n=16 Participants	0 Participants n=38 Participants
Race (NIH/OMB) Asian	0 Participants n=22 Participants	0 Participants n=16 Participants	0 Participants n=38 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=22 Participants	0 Participants n=16 Participants	0 Participants n=38 Participants
Race (NIH/OMB) Black or African American	0 Participants n=22 Participants	0 Participants n=16 Participants	0 Participants n=38 Participants
Race (NIH/OMB) White	21 Participants n=22 Participants	13 Participants n=16 Participants	34 Participants n=38 Participants
Race (NIH/OMB) More than one race	0 Participants n=22 Participants	2 Participants n=16 Participants	2 Participants n=38 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=22 Participants	1 Participants n=16 Participants	2 Participants n=38 Participants
Was the patient taking amifampridine (base or phosphate) immediately prior to enrollment? Yes	7 Participants n=22 Participants	3 Participants n=16 Participants	10 Participants n=38 Participants
Was the patient taking amifampridine (base or phosphate) immediately prior to enrollment? No	15 Participants n=22 Participants	13 Participants n=16 Participants	28 Participants n=38 Participants
If yes, number of continuous days of amifampridine exposure immediately prior to enrollment	1287.1 days STANDARD_DEVIATION 1525.73 • n=7 Participants • Data only for those subjects (7 in the placebo group, 3 in the amifampridine group) who were taking amifampridine prior to enrollment.	2143.3 days STANDARD_DEVIATION 3080.16 • n=3 Participants • Data only for those subjects (7 in the placebo group, 3 in the amifampridine group) who were taking amifampridine prior to enrollment.	1544.0 days STANDARD_DEVIATION 1957.36 • n=10 Participants • Data only for those subjects (7 in the placebo group, 3 in the amifampridine group) who were taking amifampridine prior to enrollment.

PRIMARY outcome

Timeframe: Assessment at Baseline and Day 14

Outcome measures

Outcome measures
Measure	Placebo n=21 Participants Matching placebo tablets administered 3-4 times a day over 2 weeks. Placebo: Matching number of tablets to the individual patient's tablet count of active at baseline.	Amifampridine Phosphate n=16 Participants 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).
Change From Baseline Quantitative Myasthenia Gravis (QMG) at 14 Days Baseline	5.6 QMG Score Standard Deviation 3.99	6.4 QMG Score Standard Deviation 3.22
Change From Baseline Quantitative Myasthenia Gravis (QMG) at 14 Days Day 14	7.9 QMG Score Standard Deviation 2.85	6.7 QMG Score Standard Deviation 4.09
Change From Baseline Quantitative Myasthenia Gravis (QMG) at 14 Days Change from Baseline	2.2 QMG Score Standard Deviation 2.93	0.3 QMG Score Standard Deviation 2.60

PRIMARY outcome

Timeframe: Assessment at Baseline and Day 14

Population: FAS

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

Outcome measures

Outcome measures
Measure	Placebo n=21 Participants Matching placebo tablets administered 3-4 times a day over 2 weeks. Placebo: Matching number of tablets to the individual patient's tablet count of active at baseline.	Amifampridine Phosphate n=16 Participants 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).
Change in SGI Score Baseline	5.9 SGI score Standard Deviation 1.22	5.6 SGI score Standard Deviation 1.26
Change in SGI Score Day 14	3.2 SGI score Standard Deviation 1.7	4.9 SGI score Standard Deviation 1.57
Change in SGI Score Change from Baseline	-2.7 SGI score Standard Deviation 2.29	-0.7 SGI score Standard Deviation 1.82

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Placebo n=21 Participants Matching placebo tablets administered 3-4 times a day over 2 weeks. Placebo: Matching number of tablets to the individual patient's tablet count of active at baseline.	Amifampridine Phosphate n=16 Participants 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).
Change From Baseline Timed 25 Foot Walking Test (T25FW) at 14 Days Baseline	255 feet/minute Standard Deviation 111	254 feet/minute Standard Deviation 126
Change From Baseline Timed 25 Foot Walking Test (T25FW) at 14 Days Day 14	244 feet/minute Standard Deviation 116	253 feet/minute Standard Deviation 126
Change From Baseline Timed 25 Foot Walking Test (T25FW) at 14 Days Change from Baseline	-10.4 feet/minute Standard Deviation 53.1	-1.46 feet/minute Standard Deviation 52.5

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	Placebo n=21 Participants Matching placebo tablets administered 3-4 times a day over 2 weeks. Placebo: Matching number of tablets to the individual patient's tablet count of active at baseline.	Amifampridine Phosphate n=16 Participants 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).
Change in CGI-I Score Baseline	2.5 CGI-I score Standard Deviation 0.98	2.6 CGI-I score Standard Deviation 0.63
Change in CGI-I Score Day 14	4.8 CGI-I score Standard Deviation 1.45	3.6 CGI-I score Standard Deviation 1.50

Adverse Events

Part 2 and Part 3 Placebo

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Part 2 and Part 3 Amifampridine Phosphate

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Part 2 and Part 3 Placebo n=21 participants at risk；n=22 participants at risk 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 n=16 participants at risk 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).
Infections and infestations Nasopharyngitis	0.00% 0/21 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.	6.2% 1/16 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.
Infections and infestations Otitis externa	0.00% 0/21 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.	6.2% 1/16 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.
Infections and infestations Periodontitis	4.8% 1/21 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.	0.00% 0/16 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.
Infections and infestations Pulpitis dental	4.8% 1/21 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.	0.00% 0/16 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.
Infections and infestations Urinary tract infection	0.00% 0/21 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.	6.2% 1/16 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.
General disorders Asthenia	9.5% 2/21 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.	0.00% 0/16 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.
General disorders Fatigue	4.8% 1/21 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.	6.2% 1/16 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.
Musculoskeletal and connective tissue disorders Muscular weakness	4.8% 1/21 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.	0.00% 0/16 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.
Musculoskeletal and connective tissue disorders Sensation of heaviness	4.8% 1/21 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.	0.00% 0/16 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.
Investigations Blood creatine phosphokinase increased	4.8% 1/21 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.	0.00% 0/16 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.
Psychiatric disorders Depression	4.8% 1/21 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.	0.00% 0/16 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.
Respiratory, thoracic and mediastinal disorders Dyspnoea	4.8% 1/21 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.	0.00% 0/16 • The reporting period for non-serious AEs was the first administration of study drug through the termination visit or at the early termination visit. The reporting period for SAEs began after a signed ICF was obtained and continued through 4 weeks after the last dose or at the early termination visit. For this study, the following additional events were considered SAEs: * A generalized tonic-clonic convulsion/seizure; * A normal liver function test for a patient at Screening, but the patient developed the following constellation of findings: * ALT or AST \>3 times the ULN; * total bilirubin \>2 times the ULN; and * no other explanation, such as concurrent liver disease or treatment with a potentially hepatotoxic concurrent medication, was found.

Additional Information

Gary Ingenito

Catalyst Pharmaceuticals, Inc.

Phone: 305-420-3200

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60