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Placebo Controlled Trial of Dextromethorphan in Rett Syndrome (PCTDMRTT)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01520363
Recruitment Status : Completed
First Posted : January 27, 2012
Results First Posted : October 23, 2018
Last Update Posted : December 4, 2018
Sponsor:
Collaborator:
The Johns Hopkins Institute for Clinical and Translational Research (ICTR)
Information provided by (Responsible Party):
SakkuBai Naidu, M.D., Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Rett Syndrome
Interventions Drug: dextromethorphan
Drug: placebo
Enrollment 57
Recruitment Details  
Pre-assignment Details A total of 57 participants were consented. Of these 5 were slow metabolizers who did not meet post consent eligibility criteria.
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group
Hide Arm/Group Description

MECP2 mutation positive subjects randomized to receive DM

dextromethorphan: The DM group will take 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study. The pharmacists will dispense the DM to the study participants.

MECP2 positive subjects randomized to the placebo compound

placebo: The placebo will be dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months. The Research pharmacist will dispense the placebo to the participants.

Period Title: Overall Study
Started 26 26
Completed 24 26
Not Completed 2 0
Reason Not Completed
Withdrawal by Subject             2             0
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Total
Hide Arm/Group Description

MECP2 mutation positive subjects randomized to receive DM

dextromethorphan: The DM group will take 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study. The pharmacists will dispense the DM to the study participants.

MECP2 positive subjects randomized to the placebo compound

placebo: The placebo will be dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months. The Research pharmacist will dispense the placebo to the participants.

Total of all reporting groups
Overall Number of Baseline Participants 26 26 52
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 26 participants 26 participants 52 participants
<=18 years
26
 100.0%
26
 100.0%
52
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 26 participants 26 participants 52 participants
4.75  (2.52) 4.91  (1.91) 4.83  (2.22)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 26 participants 26 participants 52 participants
Female
26
 100.0%
26
 100.0%
52
 100.0%
Male
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 26 participants 26 participants 52 participants
United States
25
  96.2%
25
  96.2%
50
  96.2%
Canada
0
   0.0%
1
   3.8%
1
   1.9%
Mexico
1
   3.8%
0
   0.0%
1
   1.9%
1.Primary Outcome
Title Change in Mullen; Visual Reception Sub-scale Scores, Pre- and Post-Intervention
Hide Description The Mullen Scales of Early Learning (MULLEN) Visual reception subscale raw scores range from Minimum=0 to Maximum=50. A higher score is a better outcome. Age equivalents from 1 month to 70 months can be computed for each subscale separately.
Time Frame Initial evaluation and at the end of the 3 month trial
Hide Outcome Measure Data
Hide Analysis Population Description
Mutation positive participants who are fast metabolizers at baseline. 2 were noncompliant and removed from the study drug group. At 3 months 22 were analyzed in the study drug group and 25 in the placebo group due to incomplete data.
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug Group at 3 Months Placebo at 3 Months
Hide Arm/Group Description:

MECP2 mutation positive subjects randomized to receive DM at baseline

dextromethorphan: The DM group will take 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study. The pharmacists will dispense the DM to the study participants.

MECP2 positive subjects randomized to the placebo at baseline

placebo: The placebo will be dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months. The Research pharmacist will dispense the placebo to the participants.

MECP2 mutation positive subjects randomized to DM at 3 months
MECP2 mutation positive subjects randomized to placebo at 3 months
Overall Number of Participants Analyzed 24 26 22 25
Mean (Standard Deviation)
Unit of Measure: score on a scale
5.45  (3.419) 6.61  (6.308) 6.64  (7.135) 6.57  (5.008)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Study Drug Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .211
Comments [Not Specified]
Method t-test, 2 sided
Comments df=21
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1.182
Confidence Interval (2-Sided) 95%
-3.086 to .722
Parameter Dispersion
Type: Standard Deviation
Value: 4.294
Estimation Comments [Not Specified]
Other Statistical Analysis t= -1.291; p=.211
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Group, Placebo at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .949
Comments [Not Specified]
Method t-test, 2 sided
Comments df=22
Method of Estimation Estimation Parameter Median Difference (Final Values)
Estimated Value .043
Confidence Interval (2-Sided) 95%
-1.345 to 1.432
Parameter Dispersion
Type: Standard Error of the Mean
Value: .670
Estimation Comments [Not Specified]
2.Primary Outcome
Title Change in Mullen; Fine Motor Sub-scale Scores, Pre- and Post-Intervention
Hide Description The Mullen Scales of Early Learning (MULLEN) Fine motor scale raw scores range from Minimum=0 to Maximum=49. A higher score is a better outcome. Age equivalents from 1 month to 70 months can be computed for each subscale separately.
Time Frame Baseline and 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
Mutation positive participants who are fast metabolizers at baseline. 2 were noncompliant and removed from the study drug group. At 3 months 22 were analyzed in the study drug group and 25 in the placebo group due to incomplete data.
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug Group at 3 Months Placebo at 3 Months
Hide Arm/Group Description:

MECP2 mutation positive subjects randomized to receive DM at baseline

dextromethorphan: The DM group will take 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study. The pharmacists will dispense the DM to the study participants.

MECP2 positive subjects randomized to the placebo at baseline

placebo: The placebo will be dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months. The Research pharmacist will dispense the placebo to the participants.

MECP2 mutation positive subjects randomized to DM at 3 months
MECP2 mutation positive subjects randomized to placebo at 3 months
Overall Number of Participants Analyzed 24 26 22 25
Mean (Standard Deviation)
Unit of Measure: score on a scale
5.38  (5.5) 7.00  (6.474) 5.05  (6.103) 7.04  (6.564)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Study Drug Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .541
Comments df=20
Method t-test, 2 sided
Comments df=20
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.333
Confidence Interval (2-Sided) 95%
-0.785 to 1.451
Parameter Dispersion
Type: Standard Deviation
Value: 2.456
Estimation Comments [Not Specified]
Other Statistical Analysis t=0.622; p=.541
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Group, Placebo at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .946
Comments df=23
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -.042
Confidence Interval (2-Sided) 95%
-1.290 to 1.206
Parameter Dispersion
Type: Standard Deviation
Value: 2.956
Estimation Comments [Not Specified]
Other Statistical Analysis t=-.069; p=0.946
3.Primary Outcome
Title Change in Mullen; Receptive Language Subscale Scores, Pre- and Post-Intervention
Hide Description The Mullen Scales of Early Learning (MULLEN) Receptive Language scale raw scores range from Minimum=0 to Maximum=50. A higher score is a better outcome. Age equivalents from 1 month to 70 months can be computed for each subscale separately.
Time Frame Baseline and 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
Mutation positive participants who are fast metabolizers at baseline. 2 were noncompliant and removed from the study drug group. At 3 months 22 were analyzed in the study drug group and 25 in the placebo group due to incomplete data.
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug Group at 3 Months Placebo at 3 Months
Hide Arm/Group Description:

MECP2 mutation positive subjects randomized to receive DM at baseline

dextromethorphan: The DM group will take 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study. The pharmacists will dispense the DM to the study participants.

MECP2 positive subjects randomized to the placebo at baseline

placebo: The placebo will be dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months. The Research pharmacist will dispense the placebo to the participants.

MECP2 mutation positive subjects randomized to DM at 3 months
MECP2 mutation positive subjects randomized to placebo at 3 months
Overall Number of Participants Analyzed 24 26 22 25
Mean (Standard Deviation)
Unit of Measure: score on a scale
6.45  (5.307) 6.95  (5.269) 6.68  (5.472) 6.55  (6.085)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Study Drug Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .736
Comments [Not Specified]
Method t-test, 2 sided
Comments df=21
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.227
Confidence Interval (2-Sided) 95%
-1.609 to 1.154
Parameter Dispersion
Type: Standard Deviation
Value: 3.116
Estimation Comments [Not Specified]
Other Statistical Analysis t=-0.342; p=0.736
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Group, Placebo at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .589
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.409
Confidence Interval (2-Sided) 95%
-1.143 to 1.961
Parameter Dispersion
Type: Standard Deviation
Value: 3.500
Estimation Comments [Not Specified]
Other Statistical Analysis t=0.548; p=0.589
4.Primary Outcome
Title Change in Mullen, Expressive Language Sub-scale Scores, Pre- and Post-Intervention
Hide Description The Mullen Scales of Early Learning (MULLEN) Expressive Language scale raw scores range from Minimum=0 to Maximum=50. A higher score is a better outcome. Age equivalents from 1 month to 70 months can be computed for each subscale separately.
Time Frame Baseline and 3 months
Hide Outcome Measure Data
Hide Analysis Population Description
Mutation positive participants who are fast metabolizers at baseline. 2 were noncompliant and removed from the study drug group. At 3 months 22 were analyzed in the study drug group and 25 in the placebo group due to incomplete data.
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug Group at 3 Months Placebo at 3 Months
Hide Arm/Group Description:

MECP2 mutation positive subjects randomized to receive DM at baseline

dextromethorphan: The DM group will take 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study. The pharmacists will dispense the DM to the study participants.

MECP2 positive subjects randomized to the placebo at baseline

placebo: The placebo will be dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months. The Research pharmacist will dispense the placebo to the participants.

MECP2 mutation positive subjects randomized to DM at 3 months
MECP2 mutation positive subjects randomized to placebo at 3 months
Overall Number of Participants Analyzed 24 26 22 25
Mean (Standard Deviation)
Unit of Measure: score on a scale
5.33  (3.665) 6.73  (3.8) 6.76  (5.504) 7.29  (7.123)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Study Drug Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value .05
Comments [Not Specified]
Method t-test, 2 sided
Comments df=20
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1.429
Confidence Interval (2-Sided) 95%
-2.886 to 0.029
Parameter Dispersion
Type: Standard Deviation
Value: 3.203
Estimation Comments [Not Specified]
Other Statistical Analysis t=-2.044; p=0.05
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Group, Placebo at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.958
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.042
Confidence Interval (2-Sided) 95%
-1.589 to 1.672
Parameter Dispersion
Type: Standard Deviation
Value: 3.862
Estimation Comments [Not Specified]
Other Statistical Analysis t=0.053; p=0.958
5.Secondary Outcome
Title Change in VABS: Motor Skills Domain Scores, Pre- and Post-Intervention
Hide Description Vineland Adaptive Behavior Scales-II (VABS): Motor Skills Domain Scores individual items are scored on a Likert scale from 2=Usually, 1=Sometimes or Partially, 0= Seldom or Never. Motor Skills Domain raw scores range from: Minimum=0 to Maximum=100. A higher score is a better outcome.
Time Frame Baseline evaluation and at the end of the 3 month study
Hide Outcome Measure Data
Hide Analysis Population Description
MECP2 Mutation positive participants who are fast metabolizers were randomized to placebo or DM at baseline. Two participants in the baseline DM group were noncompliant and removed from the study at baseline. At 3 months 22 were analyzed in the DM study drug group and 25 in the placebo group due to incomplete data.
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug-dextromethorphan (DM) at 3 Months Placebo Group at 3 Months
Hide Arm/Group Description:
MECP2 mutation positive subjects randomized to receive DM (dextromethorphan) at baseline: The DM group will take 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study.
MECP2 positive subjects randomized to the placebo compound at baseline. The placebo will be dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months.
MECP2 mutation positive subjects randomized to receive DM (dextromethorphan) who completed study assessments at the 3 month time point.
MECP2 positive subjects randomized to the placebo compound who completed the assessments at the 3 month time point.
Overall Number of Participants Analyzed 24 26 22 25
Mean (Standard Deviation)
Unit of Measure: score on a scale
9.86  (3.821) 11.36  (4.838) 9.64  (3.959) 11.12  (4.755)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Study Drug-dextromethorphan (DM) at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.488
Comments [Not Specified]
Method t-test, 2 sided
Comments df=21
Method of Estimation Estimation Parameter Median Difference (Net)
Estimated Value 0.227
Confidence Interval (2-Sided) 95%
-0.442 to 0.897
Parameter Dispersion
Type: Standard Deviation
Value: 1.510
Estimation Comments [Not Specified]
Other Statistical Analysis t=0.706; p=0.488
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Group, Placebo Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.442
Comments [Not Specified]
Method t-test, 2 sided
Comments df=24
Method of Estimation Estimation Parameter Median Difference (Net)
Estimated Value 0.24
Confidence Interval (2-Sided) 95%
-0.394 to 0.874
Parameter Dispersion
Type: Standard Deviation
Value: 1.535
Estimation Comments [Not Specified]
Other Statistical Analysis t=0.782; p=0.442
6.Secondary Outcome
Title Change in VABS:Daily Living Skills Domain Scores, Pre- and Post-Intervention
Hide Description Vineland Adaptive Behavior Scales-II (VABS): Daily Living Skills Domain individual items are scored on a Likert scale from 2=Usually, 1=Sometimes or Partially, 0= Seldom or Never. The Daily Living Skills Domain measures personal behavior as well as domestic and community interaction skills. Daily Living Skills Domain raw scores range from Minimum=0 to Maximum=218.
Time Frame Baseline and at the end of the 3 month trial
Hide Outcome Measure Data
Hide Analysis Population Description
52 MECP2 Mutation positive participants who are fast metabolizers were enrolled in the study. Two participants in the baseline DM group were noncompliant and removed from the study at baseline. At 3 months 22 were analyzed in the DM study drug group and 25 in the placebo group due to incomplete data.
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug-dextromethorphan (DM) at 3 Months Placebo Group at 3 Months
Hide Arm/Group Description:
MECP2 mutation positive subjects randomized to receive DM (dextromethorphan) at baseline: The DM group took 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study.
MECP2 positive subjects randomized to the placebo compound at baseline: The placebo was dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months. The Research pharmacist dispensed the placebo to the participants.
MECP2 mutation positive subjects randomized to receive DM (dextromethorphan) who completed the study at 3 months
MECP2 positive subjects randomized to the placebo compound were still active in the study at 3 months.
Overall Number of Participants Analyzed 24 26 22 25
Mean (Standard Deviation)
Unit of Measure: score on a scale
21.64  (6.939) 21.00  (5.307) 20.95  (6.388) 19.84  (4.589)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Study Drug-dextromethorphan (DM) at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.182
Comments [Not Specified]
Method t-test, 2 sided
Comments df=21
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.682
Confidence Interval (2-Sided) 95%
-0.346 to 1.709
Parameter Dispersion
Type: Standard Deviation
Value: 2.317
Estimation Comments [Not Specified]
Other Statistical Analysis t=1.380; p=0.182
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Group, Placebo Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.005
Comments [Not Specified]
Method t-test, 2 sided
Comments df=24
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 1.160
Confidence Interval (2-Sided) 95%
0.391 to 1.929
Parameter Dispersion
Type: Standard Deviation
Value: 1.864
Estimation Comments [Not Specified]
Other Statistical Analysis t=3.112; p=0.005
7.Secondary Outcome
Title Change in VABS: Socialization Domain Scores, Pre- and Post-Intervention
Hide Description Vineland Adaptive Behavior Scales-II (VABS): Socialization Domain. Critical behaviors are scored on a Likert scale from 2=Usually, 1=Sometimes or Partially, 0= Seldom or Never. Socialization Domain raw scores range from: Minimum=0 to Maximum=152. A higher score is a better outcome.
Time Frame Baseline and at the end of the 3 month trial
Hide Outcome Measure Data
Hide Analysis Population Description
MECP2 Mutation positive participants who are fast metabolizers were randomized to placebo or DM at baseline. Two participants in the baseline DM group were noncompliant and removed from the study at baseline. At 3 months 22 were analyzed in the DM study drug group and 25 in the placebo group due to incomplete data.
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug-dextromethorphan (DM) at 3 Months Placebo Group at 3 Months
Hide Arm/Group Description:
MECP2 mutation positive subjects randomized to receive DM (dextromethorphan) at baseline: The DM group will take 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study. The pharmacists dispensed the DM to the study participants.
MECP2 positive subjects randomized to the placebo compound at baseline: The placebo was dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months. The Research pharmacist dispensed the placebo to the participants.
MECP2 mutation positive subjects randomized to receive DM (dextromethorphan) at 3 month time period.
MECP2 mutation positive subjects randomized to receive DM placebo at 3 month time period.
Overall Number of Participants Analyzed 24 26 22 25
Mean (Standard Deviation)
Unit of Measure: score on a scale
24.14  (5.557) 22.96  (4.954) 24.05  (6.514) 22.48  (5.524)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Study Drug-dextromethorphan (DM) at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.90
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value .091
Confidence Interval (2-Sided) 95%
-1.463 to 1.644
Parameter Dispersion
Type: Standard Deviation
Value: 3.504
Estimation Comments [Not Specified]
Other Statistical Analysis t=0.122; p=0.90
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Group, Placebo Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.356
Comments [Not Specified]
Method t-test, 2 sided
Comments df=24
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.480
Confidence Interval (2-Sided) 95%
-0.573 to 1.533
Parameter Dispersion
Type: Standard Deviation
Value: 2.551
Estimation Comments [Not Specified]
Other Statistical Analysis t=0.941; p=0.356
8.Secondary Outcome
Title Change in VABS:Communication Domain Scores, Pre- and Post-Intervention
Hide Description Vineland Adaptive Behavior Scales (VABS)-II Communication Domain Scores. The Communication Domain evaluates the receptive, expressive, and written communication skills of the child. Critical behaviors in each Subdomain item are rated as 2=Usually, 1=Sometimes or Partially, 0= Seldom or Never. Communication Domain raw scores range from: Minimum=0 to Maximum=198. A higher score is a better outcome.
Time Frame Baseline and at the end of the 3 month trial
Hide Outcome Measure Data
Hide Analysis Population Description
52 MECP2 Mutation positive participants who are fast metabolizers were enrolled at baseline. Two participants in the baseline DM group were noncompliant and removed from the study. At 3 months 22 were analyzed in the DM study drug group and 25 in the placebo group due to incomplete data.
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug-dextromethorphan (DM) at 3 Months Placebo Group at 3 Months
Hide Arm/Group Description:
MECP2 mutation positive subjects randomized to receive DM (dextromethorphan) at baseline: The DM group will take 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study. The pharmacists will dispense the DM to the study participants.

MECP2 positive subjects randomized to the placebo compound

placebo: The placebo will be dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months. The Research pharmacist will dispense the placebo to the participants.

MECP2 mutation positive subjects randomized to receive DM (dextromethorphan) and active in study at 3 month time point.
MECP2 mutation positive subjects randomized to receive Placebo and active in study at 3 month time point.
Overall Number of Participants Analyzed 24 26 22 25
Mean (Standard Deviation)
Unit of Measure: score on a scale
18.05  (4.146) 18.08  (4.261) 17.91  (5.032) 17.96  (4.835)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Study Drug-dextromethorphan (DM) at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.836
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.136
Confidence Interval (2-Sided) 95%
-1.220 to 1.493
Parameter Dispersion
Type: Standard Deviation
Value: 3.060
Estimation Comments [Not Specified]
Other Statistical Analysis t=0.209; p=0.836
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Group, Placebo Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.826
Comments [Not Specified]
Method t-test, 2 sided
Comments df=24
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.120
Confidence Interval (2-Sided) 95%
-0.993 to 1.233
Parameter Dispersion
Type: Standard Deviation
Value: 2.698
Estimation Comments [Not Specified]
Other Statistical Analysis t=0.222; p=0.826
9.Secondary Outcome
Title Change in Ghuman-Folstein Screen for Social Interaction (SSI) Score, Pre- and Post-Intervention.
Hide Description

The Ghuman-Folstein Screen for Social Interaction (SSI) assesses the change in behavior and temperament dysregulation as a total score.

The score ranges from 0-162, with 0 being most Impaired /has the strongest autism features and 162 having no impairment/no autism features.

Time Frame Initial evaluation and at the end of the 3 month study. The test lasts 45 minutes
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug Group at 3 Months Placebo at 3 Months
Hide Arm/Group Description:

MECP2 mutation positive subjects randomized to receive DM at baseline

dextromethorphan: The DM group will take 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study. The pharmacists will dispense the DM to the study participants.

MECP2 positive subjects randomized to the placebo at baseline

placebo: The placebo will be dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months. The Research pharmacist will dispense the placebo to the participants.

MECP2 mutation positive subjects randomized to DM at 3 months
MECP2 mutation positive subjects randomized to placebo at 3 months
Overall Number of Participants Analyzed 22 24 22 24
Mean (Standard Deviation)
Unit of Measure: units on a scale
73.1  (22.86) 71.3  (17.26) 73.1  (25.97) 68.0  (16.21)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Placebo Group, Study Drug Group at 3 Months, Placebo at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.42
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -3.38
Confidence Interval (2-Sided) 95%
-11.80 to 5.05
Parameter Dispersion
Type: Standard Error of the Mean
Value: 4.18
Estimation Comments [Not Specified]
10.Secondary Outcome
Title Change in Rett Syndrome Behavior Questionnaire Score, Pre- and Post-Intervention
Hide Description The Rett Syndrome Behavior Questionnaire (RSBQ) total score was assessed. The total score ranges from 0 to 90, with 0 exhibiting no Rett syndrome related symptoms and 90 showing the greatest amount of symptoms (worse outcome).
Time Frame Initial evaluation and at the end of the 3 month study
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug-dextromethorphan (DM) at 3 Months Placebo Group at 3 Months
Hide Arm/Group Description:
MECP2 mutation positive subjects randomized to receive DM (dextromethorphan) at baseline: The DM group will take 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study.
MECP2 positive subjects randomized to the placebo compound at baseline. The placebo will be dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months.
MECP2 mutation positive subjects randomized to receive DM (dextromethorphan) who completed study assessments at the 3 month time point.
MECP2 positive subjects randomized to the placebo compound who completed the assessments at the 3 month time point.
Overall Number of Participants Analyzed 22 24 22 24
Mean (Standard Deviation)
Unit of Measure: units on a scale
33.8  (13.21) 33.3  (12.26) 33.8  (13.21) 33.3  (12.26)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Placebo Group, Study Drug-dextromethorphan (DM) at 3 Months, Placebo Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.73
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -1.03
Confidence Interval (2-Sided) 95%
-7.02 to 4.96
Parameter Dispersion
Type: Standard Error of the Mean
Value: 2.97
Estimation Comments [Not Specified]
11.Other Pre-specified Outcome
Title Change in PedsQL School Functioning Subscale Score, Pre- and Post-Intervention
Hide Description Pediatric Quality of Life Inventory (PedsQL version 4). School Functioning subscale. 5-point Likert scale from 0 (Never) to 4 (Almost always); Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. Higher scores indicate better Health Related Quality of Life (QOL).
Time Frame Baseline evaluation and at the end of the 3 month study
Hide Outcome Measure Data
Hide Analysis Population Description
MECP2 Mutation positive participants who are fast metabolizers were randomized to placebo or DM at baseline. Two participants in the baseline DM group were noncompliant and removed from the study at baseline. At 3 months, 22 were analyzed in the DM study drug group and 25 in the placebo group due to incomplete data.
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug Group at 3 Months Placebo at 3 Months
Hide Arm/Group Description:
MECP2 mutation positive subjects randomized to receive dextromethorphan(DM) at baseline
MECP2 positive subjects randomized to the placebo at baseline.
MECP2 mutation positive subjects randomized to DM at 3 months
MECP2 mutation positive subjects randomized to placebo at 3 months
Overall Number of Participants Analyzed 24 26 22 25
Mean (Standard Deviation)
Unit of Measure: score on a scale
64.231  (23.3682) 49.583  (20.3793) 55.769  (25.2782) 44.592  (14.2084)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Study Drug Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.371
Comments [Not Specified]
Method t-test, 2 sided
Comments df=12
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 8.4615
Confidence Interval (2-Sided) 95%
-11.3603 to 28.2834
Parameter Dispersion
Type: Standard Deviation
Value: 32.8016
Estimation Comments [Not Specified]
Other Statistical Analysis t=0.903; p=0.371
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Group, Placebo at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.358
Comments [Not Specified]
Method t-test, 2 sided
Comments df=11
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 4.9917
Confidence Interval (2-Sided) 95%
-6.4553 to 16.4387
Parameter Dispersion
Type: Standard Deviation
Value: 18.0163
Estimation Comments t=0.960; p=0.358
12.Other Pre-specified Outcome
Title Change in PedsQL Total Score, Pre- and Post-Intervention
Hide Description Pediatric Quality of Life Inventory (PedsQL version 4) total score. Each item is rated on a 5-point Likert scale from 0 (Never) to 4 (Almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. The Total Score is the sum of all the items over the number of items answered on all the Scales. Higher scores indicate better HRQOL.
Time Frame Baseline evaluation and at the end of the 3 month study
Hide Outcome Measure Data
Hide Analysis Population Description
MECP2 Mutation positive participants who are fast metabolizers were randomized to placebo or DM at baseline. Two participants in the baseline DM group were noncompliant and removed from the study at baseline. At 3 months, 22 were analyzed in the DM study drug group and 25 in the placebo group due to incomplete data.
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug Group at 3 Months Placebo at 3 Months
Hide Arm/Group Description:
MECP2 mutation positive subjects randomized to receive dextromethorphan(DM) at baseline
MECP2 positive subjects randomized to the placebo at baseline.
MECP2 mutation positive subjects randomized to DM at 3 months
MECP2 mutation positive subjects randomized to placebo at 3 months
Overall Number of Participants Analyzed 24 26 22 25
Mean (Standard Deviation)
Unit of Measure: score on a scale
54.018  (18.5915) 54.157  (14.9519) 50.9235  (19.11019) 51.7286  (18.83983)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Study Drug Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.603
Comments [Not Specified]
Method t-test, 2 sided
Comments df=16
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 3.09412
Confidence Interval (2-Sided) 95%
-9.27756 to 15.46580
Parameter Dispersion
Type: Standard Deviation
Value: 24.06228
Estimation Comments t=0.530; p=0.603
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Group, Placebo at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.432
Comments [Not Specified]
Method t-test, 2 sided
Comments df=13
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 2.42857
Confidence Interval (2-Sided) 95%
-4.04665 to 8.90380
Parameter Dispersion
Type: Standard Deviation
Value: 11.21479
Estimation Comments t=0.810; p=0.432
13.Other Pre-specified Outcome
Title Change in PedsQL Social Functioning Subscale Score, Pre- and Post-Intervention
Hide Description Pediatric Quality of Life Inventory (PedsQL version 4). Social Functioning subscale. 5-point Likert scale from 0 (Never) to 4 (Almost always); Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. Higher scores indicate better Health Related Quality of Life (QOL).
Time Frame Baseline and at the end of the 3 month trial
Hide Outcome Measure Data
Hide Analysis Population Description
MECP2 Mutation positive participants who are fast metabolizers were randomized to placebo or DM at baseline. Two participants in the baseline DM group were noncompliant and removed from the study at baseline. At 3 months, 22 were analyzed in the DM study drug group and 25 in the placebo group due to incomplete data.
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug Group at 3 Months Placebo at 3 Months
Hide Arm/Group Description:
MECP2 mutation positive subjects randomized to receive dextromethorphan(DM) at baseline
MECP2 positive subjects randomized to the placebo at baseline.
MECP2 mutation positive subjects randomized to DM at 3 months
MECP2 mutation positive subjects randomized to placebo at 3 months
Overall Number of Participants Analyzed 24 26 22 25
Mean (Standard Deviation)
Unit of Measure: score on a scale
58.53  (21.849) 54.64  (18.341) 46.76  (16.002) 47.14  (20.448)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Study Drug Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.083
Comments [Not Specified]
Method t-test, 2 sided
Comments df=16
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 11.765
Confidence Interval (2-Sided) 95%
-1.745 to 25.275
Parameter Dispersion
Type: Standard Deviation
Value: 26.276
Estimation Comments [Not Specified]
Other Statistical Analysis t=1.846; p=0.083
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Group, Placebo at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.073
Comments [Not Specified]
Method t-test, 2 sided
Comments df=13
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 7.500
Confidence Interval (2-Sided) 95%
-0.802 to 15.802
Parameter Dispersion
Type: Standard Deviation
Value: 14.378
Estimation Comments [Not Specified]
Other Statistical Analysis t=1.952; p=0.073
14.Other Pre-specified Outcome
Title Change in PedsQL Emotional Functioning Subscale Score, Pre- and Post-Intervention
Hide Description Pediatric Quality of Life Inventory (PedsQL version 4). Emotional Functioning subscale. 5-point Likert scale from 0 (Never) to 4 (Almost always); Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. Higher scores indicate better Health Related Quality of Life (QOL).
Time Frame Baseline evaluation and at the end of the 3 month study
Hide Outcome Measure Data
Hide Analysis Population Description
MECP2 Mutation positive participants who are fast metabolizers were randomized to placebo or DM at baseline. Two participants in the baseline DM group were noncompliant and removed from the study at baseline. At 3 months, 22 were analyzed in the DM study drug group and 25 in the placebo group due to incomplete data.
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug-dextromethorphan (DM) at 3 Months Placebo Group at 3 Months
Hide Arm/Group Description:
MECP2 mutation positive subjects randomized to receive DM (dextromethorphan) at baseline: The DM group will take 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study.
MECP2 positive subjects randomized to the placebo compound at baseline. The placebo will be dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months.
MECP2 mutation positive subjects randomized to receive DM (dextromethorphan) who completed study assessments at the 3 month time point.
MECP2 positive subjects randomized to the placebo compound who completed the assessments at the 3 month time point.
Overall Number of Participants Analyzed 24 26 22 25
Mean (Standard Deviation)
Unit of Measure: score on a scale
72.37  (17.979) 68.93  (13.182) 68.621  (24.3816) 68.214  (113.8129)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Study Drug-dextromethorphan (DM) at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.549
Comments [Not Specified]
Method t-test, 2 sided
Comments df=18
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 3.7474
Confidence Interval (2-Sided) 95%
-9.1269 to 16.6217
Parameter Dispersion
Type: Standard Deviation
Value: 26.7110
Estimation Comments t=0.612; p=0.549
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Group, Placebo Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.874
Comments [Not Specified]
Method t-test, 2 sided
Comments df=13
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.7143
Confidence Interval (2-Sided) 95%
-8.8174 to 10.2460
Parameter Dispersion
Type: Standard Deviation
Value: 16.5084
Estimation Comments t=0.162; p=0.874
15.Other Pre-specified Outcome
Title Change in PedsQL Physical Functioning Subscale Score, Pre- and Post-Intervention
Hide Description Pediatric Quality of Life Inventory (PedsQL version 4). Physical Functioning subscale. 5-point Likert scale from 0 (Never) to 4 (Almost always); Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. Higher scores indicate better Health Related Quality of Life (QOL).
Time Frame Initial evaluation and at the end of the 3 month study
Hide Outcome Measure Data
Hide Analysis Population Description
MECP2 Mutation positive participants who are fast metabolizers were randomized to placebo or DM at baseline. Two participants in the baseline DM group were noncompliant and removed from the study at baseline. At 3 months, 22 were analyzed in the DM study drug group and 25 in the placebo group due to incomplete data.
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug Group at 3 Months Placebo at 3 Months
Hide Arm/Group Description:
MECP2 mutation positive subjects randomized to receive dextromethorphan(DM) at baseline
MECP2 positive subjects randomized to the placebo at baseline.
MECP2 mutation positive subjects randomized to DM at 3 months
MECP2 mutation positive subjects randomized to placebo at 3 months
Overall Number of Participants Analyzed 24 26 22 25
Mean (Standard Deviation)
Unit of Measure: score on a scale
42.717  (30.3026) 48.973  (29.5643) 38.900  (29.7848) 48.133  (34.9777)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Study Drug Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.693
Comments [Not Specified]
Method t-test, 2 sided
Comments df=17
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 3.8167
Confidence Interval (2-Sided) 95%
-16.2046 to 23.8379
Parameter Dispersion
Type: Standard Deviation
Value: 40.2609
Estimation Comments t=0.402; p=0.693
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo Group, Placebo at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.909
Comments [Not Specified]
Method t-test, 2 sided
Comments df=14
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.8400
Confidence Interval (2-Sided) 95%
-14.6102 to 16.2902
Parameter Dispersion
Type: Standard Deviation
Value: 27.8994
Estimation Comments t=0.117; p=0.909
16.Other Pre-specified Outcome
Title Change in Seizure Frequency, Pre- and Post-Intervention, 0-4 Year Age Group
Hide Description Change in Frequency of seizure count baseline to follow-up for children aged 0-4 years
Time Frame Baseline evaluation and at the end of the 3 month study
Hide Outcome Measure Data
Hide Analysis Population Description
Children enrolled at baseline aged 0 to 4 years
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug-dextromethorphan (DM) at 3 Months Placebo Group at 3 Months
Hide Arm/Group Description:

MECP2 mutation positive subjects randomized to receive DM

dextromethorphan: The DM group will take 5mg/kg/day orally in 2 divided doses 12 hours apart for the 3 month period of the study. The pharmacists will dispense the DM to the study participants.

MECP2 positive subjects randomized to the placebo compound

placebo: The placebo will be dispensed to equal the volume of DM of 5mg/kg/day. It is taken orally in 2 divided doses 12 hours apart during the study period of 3 months. The Research pharmacist will dispense the placebo to the participants.

MECP2 mutation positive subjects randomized to DM at 3 months.
MECP2 mutation positive subjects randomized to placebo at 3 months,
Overall Number of Participants Analyzed 11 10 11 10
Mean (Standard Deviation)
Unit of Measure: seizure count
3  (1.41) 3.2  (1.32) 3.7  (.90) 3.1  (1.45)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Placebo Group, Study Drug-dextromethorphan (DM) at 3 Months, Placebo Group at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.21
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value -0.83
Confidence Interval (2-Sided) 95%
-2.17 to 0.52
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.64
Estimation Comments [Not Specified]
17.Other Pre-specified Outcome
Title Change in Seizure Frequency, Pre-and Post-Intervention, 5-10 Year Age Group
Hide Description Change in Frequency of seizures baseline to follow-up for children aged 5-10 years
Time Frame Baseline evaluation and at the end of the 3 month study
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug Group at 3 Months Placebo at 3 Months
Hide Arm/Group Description:
MECP2 mutation positive subjects randomized to receive dextromethorphan(DM) at baseline
MECP2 positive subjects randomized to the placebo at baseline.
MECP2 mutation positive subjects randomized to DM at 3 months
MECP2 mutation positive subjects randomized to placebo at 3 months
Overall Number of Participants Analyzed 13 16 11 16
Mean (Standard Deviation)
Unit of Measure: seizure count
3.6  (1.19) 2.8  (1.47) 3.36  (1.12) 3.37  (1.20)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Study Drug-dextromethorphan (DM), Placebo Group, Study Drug Group at 3 Months, Placebo at 3 Months
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.28
Comments [Not Specified]
Method t-test, 2 sided
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Net)
Estimated Value 0.74
Confidence Interval (2-Sided) 95%
-0.66 to 2.15
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.68
Estimation Comments [Not Specified]
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Study Drug-dextromethorphan (DM) Placebo Group Study Drug Group at 3 Months Placebo at 3 Months
Hide Arm/Group Description MECP2 mutation positive subjects randomized to receive dextromethorphan(DM) at baseline MECP2 positive subjects randomized to the placebo at baseline. MECP2 mutation positive subjects randomized to DM at 3 months MECP2 mutation positive subjects randomized to placebo at 3 months
All-Cause Mortality
Study Drug-dextromethorphan (DM) Placebo Group Study Drug Group at 3 Months Placebo at 3 Months
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/26 (0.00%)      0/26 (0.00%)      0/24 (0.00%)      0/26 (0.00%)    
Hide Serious Adverse Events
Study Drug-dextromethorphan (DM) Placebo Group Study Drug Group at 3 Months Placebo at 3 Months
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/26 (0.00%)      0/26 (0.00%)      0/24 (0.00%)      0/26 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Study Drug-dextromethorphan (DM) Placebo Group Study Drug Group at 3 Months Placebo at 3 Months
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/26 (0.00%)      0/26 (0.00%)      18/24 (75.00%)      22/26 (84.62%)    
Blood and lymphatic system disorders         
increased platelets   0/26 (0.00%)  0 0/26 (0.00%)  0 3/24 (12.50%)  3 2/26 (7.69%)  2
Hepatobiliary disorders         
Increased alkaline phosphatase   0/26 (0.00%)  0 0/26 (0.00%)  0 3/24 (12.50%)  3 1/26 (3.85%)  1
Nervous system disorders         
seizures   0/26 (0.00%)  0 0/26 (0.00%)  0 4/24 (16.67%)  4 8/26 (30.77%)  8
Respiratory, thoracic and mediastinal disorders         
Upper Respiratory Infection   0/26 (0.00%)  0 0/26 (0.00%)  0 8/24 (33.33%)  8 11/26 (42.31%)  11
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Sakkubai Naidu
Organization: Hugo W. Moser Research Institute at Kennedy Krieger , Inc.
Phone: 443 923 2778
EMail: naidu@kennedykrieger.org
Layout table for additonal information
Responsible Party: SakkuBai Naidu, M.D., Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
ClinicalTrials.gov Identifier: NCT01520363    
Other Study ID Numbers: FD-004247-01
First Submitted: January 25, 2012
First Posted: January 27, 2012
Results First Submitted: July 31, 2018
Results First Posted: October 23, 2018
Last Update Posted: December 4, 2018