Trial Outcomes & Findings for ERG/5-HTP in Fragile X Syndrome (FXS) (NCT NCT05030129)
NCT ID: NCT05030129
Last Updated: 2024-09-19
Results Overview
Adverse Events (AEs), including clinically meaningful laboratory abnormalities and significant behavioral changes will be recorded from the time of the subject's first dose of study drug to the end of the study (Week 17) or longer if needed. Adverse events will be assessed at each visit as well as during each phone call and will be tabulated for each treatment period.
COMPLETED
PHASE2
15 participants
Baseline through Week 17
2024-09-19
Participant Flow
Participant milestones
| Measure |
Adult Males With Fragile X Syndrome
Fifteen participants with Fragile X syndrome ages 18-45 years old will be enrolled in this single-blind study. Eligible participants will be started on EM for 4 weeks in Treatment Period 1, then will take EM and 5-HTP for 4 weeks in Treatment Period 2, then, 5-HTP for 4 weeks in Treatment Period 3, then placebo for 4 weeks in Treatment Period 4.
|
|---|---|
|
Screening Period
STARTED
|
15
|
|
Screening Period
COMPLETED
|
15
|
|
Screening Period
NOT COMPLETED
|
0
|
|
Treatment Period 1: EM & Placebo
STARTED
|
15
|
|
Treatment Period 1: EM & Placebo
COMPLETED
|
15
|
|
Treatment Period 1: EM & Placebo
NOT COMPLETED
|
0
|
|
Treatment Period 2: EM & 5-HTP
STARTED
|
15
|
|
Treatment Period 2: EM & 5-HTP
COMPLETED
|
15
|
|
Treatment Period 2: EM & 5-HTP
NOT COMPLETED
|
0
|
|
Treatment Period 3: 5-HTP & Placebo
STARTED
|
15
|
|
Treatment Period 3: 5-HTP & Placebo
COMPLETED
|
15
|
|
Treatment Period 3: 5-HTP & Placebo
NOT COMPLETED
|
0
|
|
Treatment Period 4: Placebo
STARTED
|
15
|
|
Treatment Period 4: Placebo
COMPLETED
|
15
|
|
Treatment Period 4: Placebo
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
ERG/5-HTP in Fragile X Syndrome (FXS)
Baseline characteristics by cohort
| Measure |
Adult Males With Fragile X Syndrome
n=15 Participants
Fifteen participants with Fragile X syndrome ages 18-45 years old will be enrolled in this single-blind study. Eligible participants will be started on EM for 4 weeks in Treatment Period 1, then will take EM and 5-HTP for 4 weeks in Treatment Period 2, then, 5-HTP for 4 weeks in Treatment Period 3, then placebo for 4 weeks in Treatment Period 4.
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|---|---|
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Vineland-3 Adaptive Behavior Scale (VABS-3) - Subdomain Growth Scale Values
Written Communication Subdomain
|
91 score on a scale
n=5 Participants
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Vineland-3 Adaptive Behavior Scale (VABS-3) - Subdomain Growth Scale Values
Daily Living Skills - Community Subdomain
|
67.35714286 score on a scale
n=5 Participants
|
|
Vineland-3 Adaptive Behavior Scale (VABS-3) - Subdomain Growth Scale Values
Daily Living Skills - Domestic Subdomain
|
60.92857143 score on a scale
n=5 Participants
|
|
Vineland-3 Adaptive Behavior Scale (VABS-3) - Subdomain Growth Scale Values
Social - Interpersonal Relationships Subdomain
|
101.7857143 score on a scale
n=5 Participants
|
|
Vineland-3 Adaptive Behavior Scale (VABS-3) - Subdomain Growth Scale Values
Social - Play and Leisure Subdomain
|
105.9230769 score on a scale
n=5 Participants
|
|
Vineland-3 Adaptive Behavior Scale (VABS-3) - Subdomain Growth Scale Values
Social - Coping Skills Subdomain
|
69.5 score on a scale
n=5 Participants
|
|
NIH Toolbox Cognitive Battery for Intellectual Disabilities (NIH-TCB) Total Cognition Score
|
57.3 score on a scale
n=5 Participants
|
|
Visual Analog Scale (VAS) Assessment Domain Scores
Target Language Issues
|
5.241333333 score on a scale
n=5 Participants
|
|
Visual Analog Scale (VAS) Assessment Domain Scores
Daily Function Issues
|
6.11 score on a scale
n=5 Participants
|
|
Visual Analog Scale (VAS) Assessment Domain Scores
Anxiety or Irritability Issues
|
5.426666667 score on a scale
n=5 Participants
|
|
Age, Continuous
|
31 years
STANDARD_DEVIATION 7.70 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
14 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
15 Participants
n=5 Participants
|
|
KiTAP Executive Battery - Alertness Subscore (Reaction Time)
|
482.7857143 milliseconds
n=5 Participants
|
|
KiTAP Executive Battery Distractibility Subtest - Total Number of Correct Responses
|
11.78 Correct Responses
n=5 Participants
|
|
KiTAP Executive Battery Distractibility Subtest - Total Number of Errors
|
6.214285714 Errors
n=5 Participants
|
|
KiTAP Executive Battery Flexibility Subtest - Total Number of Correct Responses
|
14.64 Correct Responses
n=5 Participants
|
|
KiTAP Executive Battery Flexibility Subtest - Total Number of Errors
|
12.29 Errors
n=5 Participants
|
|
KiTAP Test of Attentional Performance Go-NoGo Subtest - Total Number of Correct Responses
|
14.86 Correct Responses
n=5 Participants
|
|
KiTAP Test of Attentional Performance Go-NoGo Subtest - Total Number of Errors
|
9.57 Errors
n=5 Participants
|
|
Clinical Global Impression - Severity (CGI-S) Scale Overall Score
Extremely Severe
|
0 Participants
n=5 Participants
|
|
Clinical Global Impression - Severity (CGI-S) Scale Overall Score
Severe
|
0 Participants
n=5 Participants
|
|
Clinical Global Impression - Severity (CGI-S) Scale Overall Score
Marked
|
5 Participants
n=5 Participants
|
|
Clinical Global Impression - Severity (CGI-S) Scale Overall Score
Moderate
|
8 Participants
n=5 Participants
|
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Clinical Global Impression - Severity (CGI-S) Scale Overall Score
Mild
|
2 Participants
n=5 Participants
|
|
Clinical Global Impression - Severity (CGI-S) Scale Overall Score
Very Mild
|
0 Participants
n=5 Participants
|
|
Clinical Global Impression - Severity (CGI-S) Scale Overall Score
Not Ill
|
0 Participants
n=5 Participants
|
|
Aberrant Behavior Checklist (ABC) Subscores
Irritability Subscale
|
6.666666667 scores on a scale
n=5 Participants
|
|
Aberrant Behavior Checklist (ABC) Subscores
Lethargy Subscale
|
4.8 scores on a scale
n=5 Participants
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|
Aberrant Behavior Checklist (ABC) Subscores
Stereotypes Subscale
|
2.666666667 scores on a scale
n=5 Participants
|
|
Aberrant Behavior Checklist (ABC) Subscores
Hyperactivity Subscale
|
4.733333333 scores on a scale
n=5 Participants
|
|
Aberrant Behavior Checklist (ABC) Subscores
Inappropriate Speech Subscale
|
4.4 scores on a scale
n=5 Participants
|
|
Aberrant Behavior Checklist (ABC) Subscores
Social Avoidance Subscale
|
4.133333333 scores on a scale
n=5 Participants
|
|
Anxiety, Depression, and Mood Scale (ADAMS) Subscores
Manic/Hyperactive Behavior Subscore
|
5.333333333 scores on a scale
n=5 Participants
|
|
Anxiety, Depression, and Mood Scale (ADAMS) Subscores
Depressed Mood Subscore
|
3.2 scores on a scale
n=5 Participants
|
|
Anxiety, Depression, and Mood Scale (ADAMS) Subscores
Social Avoidance Subscore
|
8 scores on a scale
n=5 Participants
|
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Anxiety, Depression, and Mood Scale (ADAMS) Subscores
General Anxiety Subscore
|
6.666666667 scores on a scale
n=5 Participants
|
|
Anxiety, Depression, and Mood Scale (ADAMS) Subscores
Obsessive/Compulsive Behavior Subscore
|
1.866666667 scores on a scale
n=5 Participants
|
|
Vineland-3 Adaptive Behavior Scale (VABS-3) - Subdomain Growth Scale Values
Receptive Communication Subdomain
|
126.5 score on a scale
n=5 Participants
|
|
Event-Related Potentials (ERP) - Gamma Band Absolute Power at Resting State (Frontal)
Gamma 1 (30-55 Hz) Absolute Power
|
0.047656355 V^2/Hz
STANDARD_DEVIATION 0.018258564 • n=5 Participants
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Event-Related Potentials (ERP) - Gamma Band Absolute Power at Resting State (Frontal)
Gamma 2 (65-80 Hz) Absolute Power
|
0.026220458 V^2/Hz
STANDARD_DEVIATION 0.011025469 • n=5 Participants
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Event-Related Potential (ERP) Components in Response to Standard Tones
P1 Component
|
0.01171 μV*seconds
STANDARD_DEVIATION 0.010617538 • n=5 Participants
|
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Event-Related Potential (ERP) Components in Response to Standard Tones
P2 Component
|
0.0345626 μV*seconds
STANDARD_DEVIATION 0.032675507 • n=5 Participants
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|
Event-Related Potential (ERP) Components in Response to Standard Tones
N1 Component
|
0.0164247 μV*seconds
STANDARD_DEVIATION 0.022616337 • n=5 Participants
|
|
Event-Related Potential (ERP) Components in Response to Standard Tones
N2 Component
|
0.0360871 μV*seconds
STANDARD_DEVIATION 0.041661709 • n=5 Participants
|
|
Event-Related Potentials - Frontal Alpha Asymmetry at Resting State
|
-0.049005225 unitless
STANDARD_DEVIATION 0.326769814 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline through Week 17Population: The Safety population will include all participants who received at least one dose of study treatment.
Adverse Events (AEs), including clinically meaningful laboratory abnormalities and significant behavioral changes will be recorded from the time of the subject's first dose of study drug to the end of the study (Week 17) or longer if needed. Adverse events will be assessed at each visit as well as during each phone call and will be tabulated for each treatment period.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
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|---|---|---|---|---|
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Safety and Tolerability - Number of Treatment-Emergent Adverse Events
|
2 Number of Adverse Events
|
1 Number of Adverse Events
|
1 Number of Adverse Events
|
3 Number of Adverse Events
|
PRIMARY outcome
Timeframe: Baseline through Week 17Population: The Safety population will include all participants who received at least one dose of study treatment.
Adverse Events (AEs), including clinically meaningful laboratory abnormalities and significant behavioral changes will be tabulated for each treatment period. The Investigator is responsible for assessing the severity (intensity) of each adverse event as mild, moderate, or severe according to the following definitions: Mild - An event that is easily tolerated and generally not interfering with normal daily activities. Moderate - An event that is sufficiently discomforting to interfere with normal daily activities. Severe - An event that is incapacitating with inability to work or perform normal daily activities.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
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|---|---|---|---|---|
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Safety and Tolerability - Severity of Treatment Emergent Adverse Events
Mild
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2 Number of Adverse Events
|
1 Number of Adverse Events
|
1 Number of Adverse Events
|
2 Number of Adverse Events
|
|
Safety and Tolerability - Severity of Treatment Emergent Adverse Events
Severe
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
|
Safety and Tolerability - Severity of Treatment Emergent Adverse Events
Moderate
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
0 Number of Adverse Events
|
1 Number of Adverse Events
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: Intent to treat (ITT) efficacy population, which will include all participants who received at least one dose of treatment and returned for at least one follow-up visit.
The KiTAP is a computerized executive function battery that consists of eight nonverbal subtests measuring different basal as well as higher-order components of attention and executive functioning. The alertness subtest requires subjects to tap a button every time a stimulus appears on the screen. KiTAP was completed at Baseline as well as Week 4, Week 8, Week 12, and Week 16 (the end of each treatment period). Mean reaction time was measured over 90 seconds and change from baseline was calculated. A lower reaction time (net decrease in reaction time) indicates better performance.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
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|---|---|---|---|---|
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Change From Baseline in KiTAP Executive Battery - Alertness Subscore (Reaction Time)
|
-78.2678 milliseconds
Standard Error 107.4594
|
-0.52675 milliseconds
Standard Error 107.4594
|
14.3989 milliseconds
Standard Error 109.7533
|
31.48097 milliseconds
Standard Error 109.7533
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: Intent to treat (ITT) efficacy population, which includes all participants who received at least one dose of treatment and returned for at least one follow-up visit.
The KiTAP is a computerized executive function battery that consists of eight nonverbal subtests measuring different basal as well as higher-order components of attention and executive functioning. The distractibility subtest measures how easily the subject is distracted by extraneous stimuli. This subtest requires subjects to tap a button when a target stimulus appears on the screen while ignoring distracters that appear shortly before the stimulus. KiTAP was completed at Baseline as well as Week 4, Week 8, Week 12, and Week 16 (the end of each treatment period). Mean number of correct responses and errors were recorded, and change from baseline was calculated. For the distractability subtest, a higher mean number of correct responses and lower mean number of errors over the 3 minutes indicates better performance.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Change From Baseline in Total Number of Correct Responses on KiTAP Executive Battery Distractibility Subtest
|
2.142857 Correct Responses
Standard Error 2.9003
|
-3.1705358 Correct Responses
Standard Error 2.9201
|
0.8607271 Correct Responses
Standard Error 2.9201
|
-4.0571867 Correct Responses
Standard Error 2.9201
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: Intent to treat (ITT) efficacy population, which includes all participants who received at least one dose of treatment and returned for at least one follow-up visit.
The KiTAP is a computerized executive function battery that consists of eight nonverbal subtests measuring different basal as well as higher-order components of attention and executive functioning. The distractibility subtest measures how easily the subject is distracted by extraneous stimuli. This subtest requires subjects to tap a button when a target stimulus appears on the screen while ignoring distracters that appear shortly before the stimulus. KiTAP was completed at Baseline as well as Week 4, Week 8, Week 12, and Week 16 (the end of each treatment period). Mean number of correct responses and errors were recorded, and change from baseline was calculated. For the distractability subtest, a higher mean number of correct responses and lower mean number of errors over the 3 minutes indicates better performance.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Change From Baseline in Total Number of Errors on KiTAP Executive Battery Distractibility Subtest
|
2.64285 Errors
Standard Error 2.7233
|
0.53599 Errors
Standard Error 2.8155
|
0.79141 Errors
Standard Error 2.8155
|
-1.9874 Errors
Standard Error 2.7474
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: Intent to treat (ITT) efficacy population, which will include all participants who received at least one dose of treatment and returned for at least one follow-up visit.
The KiTAP is a computerized executive function battery that consists of eight nonverbal subtests measuring different basal as well as higher-order components of attention and executive functioning. The flexibility subtest measures the subject's ability to be flexible about shifting from one response type to another. This subtest is 2 minutes long and requires subjects to alternate between identifying blue and green dragons which seem on random sides of the screen by tapping one of two buttons. KiTAP was completed at Baseline as well as Week 4, Week 8, Week 12, and Week 16 (the end of each treatment period). Mean number of correct responses and errors were recorded and change from baseline was calculated. For the flexibility subtest, a higher mean number of correct responses and lower mean number of errors indicates better performance.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Change From Baseline in Total Number of Correct Responses on KiTAP Executive Battery Flexibility Subtest
|
-1.928571 Correct Responses
Standard Error 1.1029
|
-1.452112 Correct Responses
Standard Error 1.1124
|
-3.340254 Correct Responses
Standard Error 1.1124
|
-1.237821 Correct Responses
Standard Error 1.1124
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: Intent to treat (ITT) efficacy population, which will include all participants who received at least one dose of treatment and returned for at least one follow-up visit.
The KiTAP is a computerized executive function battery that consists of eight nonverbal subtests measuring different basal as well as higher-order components of attention and executive functioning. The flexibility subtest measures the subject's ability to be flexible about shifting from one response type to another. This subtest is 2 minutes long and requires subjects to alternate between identifying blue and green dragons which seem on random sides of the screen by tapping one of two buttons. KiTAP was completed at Baseline as well as Week 4, Week 8, Week 12, and Week 16 (the end of each treatment period). Mean number of correct responses and errors were recorded and change from baseline was calculated. For the flexibility subtest, a higher mean number of correct responses and lower mean number of errors indicates better performance.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Change From Baseline in Total Number of Errors on KiTAP Executive Battery Flexibility Subtest
|
1.5 Errors
Standard Error 0.7120
|
1.93979 Errors
Standard Error 0.7178
|
2.05569 Errors
Standard Error 0.7178
|
1.56290 Errors
Standard Error 0.7178
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: The primary efficacy population will be the intent to treat (ITT) efficacy population, which will include all participants who received at least one dose of treatment and returned for at least one follow-up visit.
The KiTAP is a computerized executive function battery that consists of eight nonverbal subtests measuring different basal as well as higher-order components of attention and executive functioning. The Go-NoGo subtest measures a subject's impulsivity by requiring subjects to tap a button when the target stimulus is presented, while refraining from hitting the button for the non-target stimulus. KiTAP was completed at Baseline as well as Week 4, Week 8, Week 12, and Week 16 (the end of each treatment period). This subtest takes place over 2 minutes 30 seconds. Mean number of correct responses and errors were recorded, and change from baseline was calculated. For the Go-NoGo subtest, a higher mean number of correct responses and a lower mean number of errors indicates better performance.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Change From Baseline in Total Number of Correct Responses on KiTAP Test of Attentional Performance Go-NoGo Subtest
|
0.3966214 Correct Responses
Standard Error 1.5644
|
1.8198629 Correct Responses
Standard Error 1.5962
|
0.4632881 Correct Responses
Standard Error 1.5644
|
-1.1383753 Correct Responses
Standard Error 1.5962
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: The primary efficacy population will be the intent to treat (ITT) efficacy population, which will include all participants who received at least one dose of treatment and returned for at least one follow-up visit.
The KiTAP is a computerized executive function battery that consists of eight nonverbal subtests measuring different basal as well as higher-order components of attention and executive functioning. The Go-NoGo subtest measures a subject's impulsivity by requiring subjects to tap a button when the target stimulus is presented, while refraining from hitting the button for the non-target stimulus. KiTAP was completed at Baseline as well as Week 4, Week 8, Week 12, and Week 16 (the end of each treatment period). This subtest takes place over 2 minutes 30 seconds. Mean number of correct responses and errors were recorded, and change from baseline was calculated. For the Go-NoGo subtest, a higher mean number of correct responses and a lower mean number of errors indicates better performance.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Change From Baseline in Total Number of Errors on KiTAP Test of Attentional Performance Go-NoGo Subtest
|
-0.1177 Errors
Standard Error 1.5019
|
0.55828 Errors
Standard Error 1.5335
|
0.28221 Errors
Standard Error 1.5019
|
0.73542 Errors
Standard Error 1.5335
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: Intent to treat (ITT) efficacy population, which will include all participants who received at least one dose of treatment and returned for at least one follow-up visit.
The Clinical Global Impression-Severity (CGI-S) is a global measure to provide a clinical judgment of a participant's overall condition based on a trained clinician's assessment of cognition, behavior and activities of daily living. The clinician compares the subject with individuals of the same age and sex. The assessment of severity is with a 7-point scale: 1, not ill; 2, very mild; 3, mild; 4, moderate; 5, marked; 6, severe; 7, extremely severe. A net decrease in severity score over time indicates a positive outcome.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Change From Baseline in Clinical Global Impression Severity Scale Overall Score
|
-0.06667 scores on a scale
Standard Error 0.0805
|
-0.06667 scores on a scale
Standard Error 0.0805
|
-0.06667 scores on a scale
Standard Error 0.0805
|
-0.06667 scores on a scale
Standard Error 0.0805
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: Intent to treat (ITT) efficacy population, which will include all participants who received at least one dose of treatment and returned for at least one follow-up visit.
This assessment is a parent/caregiver-rated scale with six subscales to assess irritability, stereotypes, lethargy, hyperactivity, inappropriate speech, and social avoidance, using ABC-FX factoring system. The ABC was was completed at Baseline as well as Week 4, Week 8, Week 12, and Week 16 (the end of each treatment period). Mean score for each subscale was measured and change from baseline was calculated. Lower mean scores on each subscale indicate fewer aberrant behaviors (better functioning).The range of possible scores on each subscale are irritability (0-54), lethargy (0-39), stereotypes (0-18), hyperactivity (0-30), inappropriate speech (0-12), and social avoidance (0-12).
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscores
Irritability Subscale
|
-2.59088 score on a scale
Standard Error 0.9919
|
-2.36774 score on a scale
Standard Error 0.9919
|
-2.53333 score on a scale
Standard Error 0.9706
|
-2.13333 score on a scale
Standard Error 0.9706
|
|
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscores
Lethargy Subscale
|
-0.86667 score on a scale
Standard Error 0.8081
|
-0.34695 score on a scale
Standard Error 0.8258
|
-0.79147 score on a scale
Standard Error 0.8258
|
-1.8 score on a scale
Standard Error 0.8081
|
|
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscores
Hyperactivity Subscale
|
-0.6 score on a scale
Standard Error 0.5241
|
-1.31738 score on a scale
Standard Error 0.5355
|
-1.46667 score on a scale
Standard Error 0.5241
|
-1.4 score on a scale
Standard Error 0.5241
|
|
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscores
Stereotypes Subscale
|
-0.73333 score on a scale
Standard Error 0.5678
|
-0.82132 score on a scale
Standard Error 0.5801
|
-1.06667 score on a scale
Standard Error 0.5678
|
-0.8 score on a scale
Standard Error 0.5678
|
|
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscores
Inappropriate Speech Subscale
|
-0.53333 score on a scale
Standard Error 0.3355
|
-0.9934 score on a scale
Standard Error 0.3429
|
-1.46667 score on a scale
Standard Error 0.3355
|
-1.46667 score on a scale
Standard Error 0.3355
|
|
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscores
Social Avoidance Subscale
|
-0.73333 score on a scale
Standard Error 0.3362
|
-0.64283 score on a scale
Standard Error 0.3520
|
-0.50848 score on a scale
Standard Error 0.3436
|
-0.4 score on a scale
Standard Error 0.3362
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: Intent to treat (ITT) efficacy population, which will include all participants who received at least one dose of treatment and returned for at least one follow-up visit.
The ADAMS is a parent/caregiver rated scale with five subscores to assess manic/hyperactive behavior, depressed mood, social avoidance, general anxiety, and obsessive/compulsive behavior. The ADAMs was completed at Baseline as well as Week 4, Week 8, Week 12, and Week 16 (the end of each treatment period). Mean score for each subscale was recorded, and change from baseline was calculated for each subscore. The range of possible scores for each subscale is 0-21. Lower mean scores on each subscale indicate better functioning.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Change From Baseline in Anxiety, Depression, and Mood Scale (ADAMS) Subscores
Social Avoidance Total Score
|
-1.733333 score on a scale
Standard Error 0.6911
|
-1.333333 score on a scale
Standard Error 0.6911
|
-2.666667 score on a scale
Standard Error 0.6911
|
-1.866667 score on a scale
Standard Error 0.6911
|
|
Change From Baseline in Anxiety, Depression, and Mood Scale (ADAMS) Subscores
Manic/Hyperactive Behavior Total Score
|
-1.4 score on a scale
Standard Error 0.4076
|
-1.7333 score on a scale
Standard Error 0.4076
|
-2.26667 score on a scale
Standard Error 0.4076
|
-1.8 score on a scale
Standard Error 0.4076
|
|
Change From Baseline in Anxiety, Depression, and Mood Scale (ADAMS) Subscores
Depressed Mood Total Score
|
-1.13333 score on a scale
Standard Error 0.6433
|
-0.06667 score on a scale
Standard Error 0.6433
|
-1.13333333 score on a scale
Standard Error 0.6433
|
-1.6 score on a scale
Standard Error 0.6433
|
|
Change From Baseline in Anxiety, Depression, and Mood Scale (ADAMS) Subscores
General Anxiety Total Score
|
-1.533333 score on a scale
Standard Error 0.6770
|
-1.8 score on a scale
Standard Error 0.6770
|
-2.946642 score on a scale
Standard Error 0.6770
|
-2.266667 score on a scale
Standard Error 0.6770
|
|
Change From Baseline in Anxiety, Depression, and Mood Scale (ADAMS) Subscores
Obsessive/Compulsive Behavior Total Score
|
-0.13333333 score on a scale
Standard Error 0.3792
|
0.06666667 score on a scale
Standard Error 0.3792
|
-0.2 score on a scale
Standard Error 0.3792
|
-0.4 score on a scale
Standard Error 0.3792
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: Intent to treat (ITT) efficacy population, which will include all participants who received at least one dose of treatment and returned for at least one follow-up visit.
VABS-3 is a clinician-administered standardized interview. For this study, we collected data in domains of communication (receptive \& written language), daily living skills (domestic \& community skills), \& socialization (interpersonal relationships, play \& leisure time, \& coping abilities). This assessment was administered at Baseline as well as at Week 4, Week 8, Week 12, and Week 16 (end of each treatment period). Mean growth scale value (GSV) for each subdomain was recorded at each time point and change from baseline was calculated. The possible GSVs for each subdomain are receptive (10-162), written (10-163), domestic (10-110), community (10-136), interpersonal (10-152), play and leisure (10-164), \& coping skills (10-120). Higher GSVs for each subdomain indicate better functioning.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Change From Baseline on Vineland-3 Adaptive Behavior Scale - Subdomain Growth Scale Values
Daily Living Skills - Domestic Subdomain
|
-1.940166 score on a scale
Standard Error 2.4600
|
1.421966 score on a scale
Standard Error 2.3207
|
5.013375 score on a scale
Standard Error 2.0873
|
5.501518 score on a scale
Standard Error 2.1852
|
|
Change From Baseline on Vineland-3 Adaptive Behavior Scale - Subdomain Growth Scale Values
Social - Play and Leisure Subdomain
|
-10.989137 score on a scale
Standard Error 6.4126
|
-3.47187 score on a scale
Standard Error 6.0588
|
-1.203629 score on a scale
Standard Error 5.5469
|
5.592143 score on a scale
Standard Error 5.8006
|
|
Change From Baseline on Vineland-3 Adaptive Behavior Scale - Subdomain Growth Scale Values
Social - Coping Skills Subdomain
|
1.460214 score on a scale
Standard Error 2.8857
|
2.756048 score on a scale
Standard Error 2.7214
|
2.077872 score on a scale
Standard Error 2.4548
|
7.090882 score on a scale
Standard Error 2.5673
|
|
Change From Baseline on Vineland-3 Adaptive Behavior Scale - Subdomain Growth Scale Values
Daily Living Skills - Community Subdomain
|
2.2145 score on a scale
Standard Error 1.9946
|
3.437951 score on a scale
Standard Error 1.8816
|
2.733986 score on a scale
Standard Error 1.6921
|
4.313574 score on a scale
Standard Error 1.7715
|
|
Change From Baseline on Vineland-3 Adaptive Behavior Scale - Subdomain Growth Scale Values
Social - Interpersonal Relationships Subdomain
|
6.01876 score on a scale
Standard Error 2.3460
|
6.042022 score on a scale
Standard Error 2.2132
|
6.664025 score on a scale
Standard Error 1.9898
|
5.801988 score on a scale
Standard Error 2.0834
|
|
Change From Baseline on Vineland-3 Adaptive Behavior Scale - Subdomain Growth Scale Values
Receptive Communication Subdomain
|
0.3603766 score on a scale
Standard Error 3.0827
|
4.4457568 score on a scale
Standard Error 2.9079
|
3.9678919 score on a scale
Standard Error 2.6167
|
5.2990546 score on a scale
Standard Error 2.7389
|
|
Change From Baseline on Vineland-3 Adaptive Behavior Scale - Subdomain Growth Scale Values
Written Communication Subdomain
|
3.926537 score on a scale
Standard Error 2.6030
|
3.915339 score on a scale
Standard Error 2.4556
|
5.287075 score on a scale
Standard Error 2.2088
|
5.051526 score on a scale
Standard Error 2.3123
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: Intent to treat (ITT) efficacy population, which includes all participants who received at least one dose of treatment and returned for at least one follow-up visit.
The NIH-TCB is a battery of extensively validated computer-administered cognitive tests. The NIH-TCB includes 7 evaluations measuring cognitive flexibility, inhibition \& visual attention, episodic memory, immediate recall \& sequencing of different visually \& orally presented stimuli, processing speed, recognition of letters and words, \& receptive vocabulary. This assessment was administered at Baseline as well as at Week 4, Week 8, Week 12, and Week 16 (end of each treatment period). This battery produces fluid and crystallized cognition composite scores. The Total Cognition Composite Score is found by converting raw fluid and crystallized scores to standard scores, averaging these two scores, then deriving standard scores based on this new distribution. Scores range from 0-140. The normative mean of the uncorrected standard score is 100 and the standard deviation is 15. A higher mean total cognition composite score indicates better performance.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Change From Baseline in NIH Toolbox Cognitive Battery for Intellectual Disabilities (NIH-TCB) Total Cognition Score
|
2.10074 scores on a scale
Standard Error 4.5470
|
-9.156292 scores on a scale
Standard Error 6.9968
|
-4.734578 scores on a scale
Standard Error 5.0309
|
-2.531407 scores on a scale
Standard Error 4.1965
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: Intent to treat (ITT) efficacy population, which will include all participants who received at least one dose of treatment and returned for at least one follow-up visit.
IIn an attempt to measure the level of behavioral difficulty experienced by the parent/caregiver with respect to the child with FXS, the VAS allows parents to mark on a visual line measuring 10 cm with one side marked "worst behavior" and the other side marked "best behavior." The caregiver rated the participant's behavior with respect to three domains: daily functioning, anxiety/irritability and language. Distances closer to 10 cm are considered worse behavior and distances closer to 0 cm are considered better behavior. Average distance of the mark was recorded for each domain at Baseline then again at Week 4, Week 8, Week 12, and Week 16 (end of each treatment period). Change from baseline was calculated for each domain. Lower scores indicate a better outcome (closer distance to best behavior side).
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Change From Baseline in Visual Analog Scale (VAS) Assessment Domain Scores
Target Language Issues
|
0.9786667 score on a scale
Standard Error 0.5869
|
0.9786667 score on a scale
Standard Error 0.5869
|
0.712 score on a scale
Standard Error 0.5869
|
0.5253333 score on a scale
Standard Error 0.5869
|
|
Change From Baseline in Visual Analog Scale (VAS) Assessment Domain Scores
Daily Function Issues
|
0.6833333 score on a scale
Standard Error 0.5763
|
0.8433333 score on a scale
Standard Error 0.5763
|
0.6566667 score on a scale
Standard Error 0.5763
|
0.93 score on a scale
Standard Error 0.5763
|
|
Change From Baseline in Visual Analog Scale (VAS) Assessment Domain Scores
Anxiety or Irritability Issues
|
0.2533333 score on a scale
Standard Error 0.6165
|
1.1233333 score on a scale
Standard Error 0.6165
|
0.6433333 score on a scale
Standard Error 0.6165
|
-0.2933333 score on a scale
Standard Error 0.6165
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: Intent to treat (ITT) efficacy population, which will include all participants who received at least one dose of treatment and returned for at least one follow-up visit.
Event-Related Potentials (ERPs) enable extraction of neural responses associated with specific sensory, cognitive, or motor events from an overall EEG. Gamma 1 and gamma 2 waves were measured for various parts of the brain at a resting state. ERP was performed at Baseline as well as at Week 4, Week 8, Week 12, Week 16 (at the end of each treatment period). Mean power of gamma 1 and gamma 2 bands, measured by frontal EEG leads, was recorded for each time point and change from baseline was calculated. FXS patients have typically been found to exhibit greater gamma frequency band power than typically developing controls, which may be related to social and sensory processing difficulties. Therefore, lower gamma band power would indicate better functioning.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Event-Related Potentials (ERP) - Gamma Band Absolute Power at Resting State (Frontal)
Gamma 1 (30-55 Hz) Absolute Power
|
0.054717778 V^2/Hz
Standard Error 0.009638014
|
0.051050595 V^2/Hz
Standard Error 0.006690128
|
0.061321956 V^2/Hz
Standard Error 0.007923461
|
0.050649034 V^2/Hz
Standard Error 0.010533546
|
|
Event-Related Potentials (ERP) - Gamma Band Absolute Power at Resting State (Frontal)
Gamma 2 (65-80 Hz) Absolute Power
|
0.027005807 V^2/Hz
Standard Error 0.005267368
|
0.025525849 V^2/Hz
Standard Error 0.004251649
|
0.031589661 V^2/Hz
Standard Error 0.004834075
|
0.025235128 V^2/Hz
Standard Error 0.004757463
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: Intent to treat (ITT) efficacy population, which will include all participants who received at least one dose of treatment and returned for at least one follow-up visit.
Event-Related Potentials (ERPs) enable extraction of neural responses associated with specific sensory, cognitive, or motor events from an overall EEG. Auditory stimuli are presented and EEG events are assessed in relation to timing of the stimuli. ERP was performed at Baseline as well as at week Week 4, Week 8, Week 12, Week 16 (end of each treatment period). The area under the curve was measured for different components of the ERP including P1 and P2 (positive components) and N1 and N2 (negative components). The average area under the curve was recorded for each component at each time point and change from baseline was calculated. Individuals with FXS are thought to have excessive ERP response, so a decrease in area under each curve compared to baseline would indicate a favorable outcome.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Event-Related Potentials (ERP) Components in Response to Standard Tones
P1 Component
|
0.009766667 μV*seconds
Standard Error 0.001838821
|
0.010784615 μV*seconds
Standard Error 0.002861501
|
0.012972727 μV*seconds
Standard Error 0.003341777
|
0.00621 μV*seconds
Standard Error 0.001656264
|
|
Event-Related Potentials (ERP) Components in Response to Standard Tones
P2 Component
|
0.041122167 μV*seconds
Standard Error 0.007281467
|
0.031379615 μV*seconds
Standard Error 0.009411693
|
0.046629091 μV*seconds
Standard Error 0.009927824
|
0.0558891 μV*seconds
Standard Error 0.014525993
|
|
Event-Related Potentials (ERP) Components in Response to Standard Tones
N1 Component
|
0.025633667 μV*seconds
Standard Error 0.006968832
|
0.029363077 μV*seconds
Standard Error 0.006649054
|
0.035257182 μV*seconds
Standard Error 0.007841523
|
0.0354182 μV*seconds
Standard Error 0.008270948
|
|
Event-Related Potentials (ERP) Components in Response to Standard Tones
N2 Component
|
0.033444167 μV*seconds
Standard Error 0.009811161
|
0.038742385 μV*seconds
Standard Error 0.01309467
|
0.028213 μV*seconds
Standard Error 0.012192131
|
0.0185623 μV*seconds
Standard Error 0.009189006
|
SECONDARY outcome
Timeframe: Baseline, Week 4 (EM Treatment), Week 8 (EM+5-HTP Treatment), Week 12 (5-HTP Treatment), Week 16 (Placebo Treatment)Population: Intent to treat (ITT) efficacy population, which will include all participants who received at least one dose of treatment and returned for at least one follow-up visit.
Event-Related Potentials (ERPs) enable extraction of neural responses associated with specific sensory, cognitive, or motor events from an overall EEG. EEG data was collected when the subject was in a resting state. Frontal alpha asymmetry measures differences in alpha activity level of the right and left hemisphere of the frontal lobe of the brain. ERP was performed at Baseline as well as at Week 4, Week 8, Week 12, Week 16 (end of each treatment period). Mean alpha asymmetry at resting state was recorded and change from baseline was calculated for each time point. Higher alpha asymmetry scores indicate greater alpha power in the right hemisphere, which corresponds to increased neural activity of the left hemisphere. High activity of the left frontal lobe is hypothesized to correspond with approach behaviors, and high activity of the right frontal lobe is thought to correspond to withdrawal behaviors. In this study, higher alpha asymmetry scores indicate a better outcome.
Outcome measures
| Measure |
Ergoloid Mesylates (EM)
n=15 Participants
During treatment period 1, subjects will receive EM 1 mg (Medisca) and matching placebo for 5-HTP 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 2, subjects will receive EM 1 mg (Medisca) and 5-HTP 100 mg (5-HTP, Basic Vitamins). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
5-hydroxytryptophan (5-HTP)
n=15 Participants
During treatment period 3, subjects will receive 5-HTP 100 mg (5-HTP, Basic Vitamins) and matching placebo for EM 1 mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
Placebo
n=15 Participants
During treatment period 4, subjects will receive matching placebo for EM 1mg (ascorbic acid powder) and matching placebo for 5-Hydroxytryptophan 100mg (ascorbic acid powder). One of each capsule (both size 00) will be taken three times per day for four weeks.
|
|---|---|---|---|---|
|
Event-Related Potentials - Frontal Alpha Asymmetry at Resting State
|
0.044133817 unitless
Standard Error 0.085312863
|
-0.048217532 unitless
Standard Error 0.094117681
|
-0.133404775 unitless
Standard Error 0.085879258
|
0.191055011 unitless
Standard Error 0.106432066
|
Adverse Events
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
Placebo
Ergoloid Mesylates (EM) and Placebo
5-hydroxytryptophan (5-HTP) and Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Ergoloid Mesylates (EM) and 5-hydroxytryptophan (5-HTP)
n=15 participants at risk
Ergoloid mesylates (EM) 1 mg three times daily and 5-hydroxytryptophan (5-HTP) 100 mg three times daily for 4 weeks
5-Hydroxytryptophan / Vitamin B6: 5-hydroxytryptophan, also known as 5-HTP has a nutraceutical status and has never been approved as a drug for any indication, but as a dietary supplement has been used extensively for several disorders for many years such as in the therapy of depression, fibromyalgia, obesity, insomnia and chronic headache
Ergoloid Mesylate: Ergoloid Mesylates, trade name Hydergine, is a mixture of the methanesulfonate salts of three dihydrogenated ergot alkaloids, dihydroergocristine, dihydroergocornine, and alpha- and beta-dihydroergocryptine. Ergot alkaloids are dopamine agonists which activate dopamine receptors (in the basal ganglia and other parts of the brain involved in motor function) and a prolactin inhibitor. Ergot is a strong vasoconstrictor and thus helps to reduce bleeding by narrowing of the blood vessels.
|
Placebo
n=15 participants at risk
2 placebo capsules three times daily for 4 weeks
Placebo: Placebo capsules
|
Ergoloid Mesylates (EM) and Placebo
n=15 participants at risk
Ergoloid mesylates (EM) 1 mg three times daily and 1 placebo capsule three times daily for 4 weeks.
Ergoloid Mesylate: Ergoloid Mesylates, trade name Hydergine, is a mixture of the methanesulfonate salts of three dihydrogenated ergot alkaloids, dihydroergocristine, dihydroergocornine, and alpha- and beta-dihydroergocryptine. Ergot alkaloids are dopamine agonists which activate dopamine receptors (in the basal ganglia and other parts of the brain involved in motor function) and a prolactin inhibitor. Ergot is a strong vasoconstrictor and thus helps to reduce bleeding by narrowing of the blood vessels.
Placebo: Placebo capsules
|
5-hydroxytryptophan (5-HTP) and Placebo
n=15 participants at risk
5-hydroxytryptophan (5-HTP) 100 mg three times daily and 1 placebo capsule three times daily for 4 weeks.
5-Hydroxytryptophan / Vitamin B6: 5-hydroxytryptophan, also known as 5-HTP has a nutraceutical status and has never been approved as a drug for any indication, but as a dietary supplement has been used extensively for several disorders for many years such as in the therapy of depression, fibromyalgia, obesity, insomnia and chronic headache
Placebo: Placebo capsules
|
|---|---|---|---|---|
|
Psychiatric disorders
Increased Irritability
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
13.3%
2/15 • Number of events 2 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
|
Gastrointestinal disorders
Diarrhea
|
6.7%
1/15 • Number of events 1 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
|
Nervous system disorders
Headache
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
6.7%
1/15 • Number of events 1 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
6.7%
1/15 • Number of events 1 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
|
Infections and infestations
COVID-19
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
6.7%
1/15 • Number of events 1 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
|
Gastrointestinal disorders
Gastroenteritis
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
6.7%
1/15 • Number of events 1 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
|
Infections and infestations
Upper Respiratory Infection
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
6.7%
1/15 • Number of events 1 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
0.00%
0/15 • Adverse events, which include abnormal and clinically significant clinical laboratory test variables, will be monitored and documented from the time Informed Consent until study participation is complete, 1 week after the final study visit (~21 weeks total).
Participants who experience AEs will be monitored with relevant clinical assessments and laboratory tests, as determined by the Investigator. Per good medical practice, all AEs must be followed to satisfactory resolution or stabilization of the event(s), or, if a chronic condition, until fully characterized. Participants who have non-serious AEs that are ongoing at study completion or study withdrawal must be followed until resolution of the AEs or for 30 days after the last dose of study drug.
|
Additional Information
Elizabeth Berry-Kravis, MD PhD
Rush University Medical Center
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place