Trial · NCT01520363

Placebo Controlled Trial of Dextromethorphan in Rett Syndrome

NCT01520363, Phase 2, Completed, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; results first posted 2018-10-23

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ConductScience Library. Placebo Controlled Trial of Dextromethorphan in Rett Syndrome. Trials, record NCT01520363. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01520363

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Placebo Controlled Trial of Dextromethorphan in Rett Syndrome
NCT number: NCT01520363
Status: Completed
Phases: Phase 2
Enrollment: 57 participants, actual
Lead sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Conditions: Rett Syndrome
Interventions: dextromethorphan, placebo
First posted: 2012-01-27
Last updated: 2018-12-04
Results first posted: 2018-10-23

Source ClinicalTrials.gov

Registered outcomes17

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change in Mullen; Visual Reception Sub-scale Scores, Pre- and Post-Intervention	Initial evaluation and at the end of the 3 month trial	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.
Primary	Change in Mullen; Fine Motor Sub-scale Scores, Pre- and Post-Intervention	Baseline and 3 months	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.
Primary	Change in Mullen; Receptive Language Subscale Scores, Pre- and Post-Intervention	Baseline and 3 months	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.
Primary	Change in Mullen, Expressive Language Sub-scale Scores, Pre- and Post-Intervention	Baseline and 3 months	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.
Secondary	Change in VABS: Motor Skills Domain Scores, Pre- and Post-Intervention	Baseline evaluation and at the end of the 3 month study	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.
Secondary	Change in VABS:Daily Living Skills Domain Scores, Pre- and Post-Intervention	Baseline and at the end of the 3 month trial	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.
Secondary	Change in VABS: Socialization Domain Scores, Pre- and Post-Intervention	Baseline and at the end of the 3 month trial	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.
Secondary	Change in VABS:Communication Domain Scores, Pre- and Post-Intervention	Baseline and at the end of the 3 month trial	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.
Secondary	Change in Ghuman-Folstein Screen for Social Interaction (SSI) Score, Pre- and Post-Intervention.	Initial evaluation and at the end of the 3 month study. The test lasts 45 minutes	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.
Secondary	Change in Rett Syndrome Behavior Questionnaire Score, Pre- and Post-Intervention	Initial evaluation and at the end of the 3 month study	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).

1 to 10 of 17Source ClinicalTrials.gov

See all 17 registered outcomes

Outcomes17

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Change in Mullen; Visual Reception Sub-scale Scores, Pre- and Post-Intervention	Initial evaluation and at the end of the 3 month trial	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.	score on a scale	MEAN
PRIMARY	Change in Mullen; Fine Motor Sub-scale Scores, Pre- and Post-Intervention	Baseline and 3 months	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.	score on a scale	MEAN
PRIMARY	Change in Mullen; Receptive Language Subscale Scores, Pre- and Post-Intervention	Baseline and 3 months	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.	score on a scale	MEAN
PRIMARY	Change in Mullen, Expressive Language Sub-scale Scores, Pre- and Post-Intervention	Baseline and 3 months	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.	score on a scale	MEAN
SECONDARY	Change in VABS: Motor Skills Domain Scores, Pre- and Post-Intervention	Baseline evaluation and at the end of the 3 month study	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.	score on a scale	MEAN
SECONDARY	Change in VABS:Daily Living Skills Domain Scores, Pre- and Post-Intervention	Baseline and at the end of the 3 month trial	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.	score on a scale	MEAN
SECONDARY	Change in VABS: Socialization Domain Scores, Pre- and Post-Intervention	Baseline and at the end of the 3 month trial	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.	score on a scale	MEAN
SECONDARY	Change in VABS:Communication Domain Scores, Pre- and Post-Intervention	Baseline and at the end of the 3 month trial	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.	score on a scale	MEAN
SECONDARY	Change in Ghuman-Folstein Screen for Social Interaction (SSI) Score, Pre- and Post-Intervention.	Initial evaluation and at the end of the 3 month study. The test lasts 45 minutes		units on a scale	MEAN
SECONDARY	Change in Rett Syndrome Behavior Questionnaire Score, Pre- and Post-Intervention	Initial evaluation and at the end of the 3 month study		units on a scale	MEAN

1 to 10 of 17Source ClinicalTrials.gov

See all 17 outcomes

Results by group68

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Change in Mullen; Visual Reception Sub-scale Scores, Pre- and Post-Intervention	Study Drug-dextromethorphan (DM)	5.45	Standard Deviation 3.419	score on a scale
Change in Mullen; Visual Reception Sub-scale Scores, Pre- and Post-Intervention	Placebo Group	6.61	Standard Deviation 6.308	score on a scale
Change in Mullen; Visual Reception Sub-scale Scores, Pre- and Post-Intervention	Study Drug Group at 3 Months	6.64	Standard Deviation 7.135	score on a scale
Change in Mullen; Visual Reception Sub-scale Scores, Pre- and Post-Intervention	Placebo at 3 Months	6.57	Standard Deviation 5.008	score on a scale
Change in Mullen; Fine Motor Sub-scale Scores, Pre- and Post-Intervention	Study Drug-dextromethorphan (DM)	5.38	Standard Deviation 5.5	score on a scale
Change in Mullen; Fine Motor Sub-scale Scores, Pre- and Post-Intervention	Placebo Group	7.00	Standard Deviation 6.474	score on a scale
Change in Mullen; Fine Motor Sub-scale Scores, Pre- and Post-Intervention	Study Drug Group at 3 Months	5.05	Standard Deviation 6.103	score on a scale
Change in Mullen; Fine Motor Sub-scale Scores, Pre- and Post-Intervention	Placebo at 3 Months	7.04	Standard Deviation 6.564	score on a scale
Change in Mullen; Receptive Language Subscale Scores, Pre- and Post-Intervention	Study Drug-dextromethorphan (DM)	6.45	Standard Deviation 5.307	score on a scale
Change in Mullen; Receptive Language Subscale Scores, Pre- and Post-Intervention	Placebo Group	6.95	Standard Deviation 5.269	score on a scale

1 to 10 of 68Source ClinicalTrials.gov

See all 68 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Study Drug-dextromethorphan (DM)	26
Overall Study	STARTED	Placebo Group	26
Overall Study	COMPLETED	Study Drug-dextromethorphan (DM)	24
Overall Study	COMPLETED	Placebo Group	26
Overall Study	NOT COMPLETED	Study Drug-dextromethorphan (DM)	2
Overall Study	NOT COMPLETED	Placebo Group	0

Source ClinicalTrials.gov

Adverse events16

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Upper Respiratory Infection	Respiratory, thoracic and mediastinal disorders	Study Drug-dextromethorphan (DM)	0	26	0
other	Upper Respiratory Infection	Respiratory, thoracic and mediastinal disorders	Placebo Group	0	26	0
other	Upper Respiratory Infection	Respiratory, thoracic and mediastinal disorders	Study Drug Group at 3 Months	8	24	8
other	Upper Respiratory Infection	Respiratory, thoracic and mediastinal disorders	Placebo at 3 Months	11	26	11
other	seizures	Nervous system disorders	Study Drug-dextromethorphan (DM)	0	26	0
other	seizures	Nervous system disorders	Placebo Group	0	26	0
other	seizures	Nervous system disorders	Study Drug Group at 3 Months	4	24	4
other	seizures	Nervous system disorders	Placebo at 3 Months	8	26	8
other	Increased alkaline phosphatase	Hepatobiliary disorders	Study Drug-dextromethorphan (DM)	0	26	0
other	Increased alkaline phosphatase	Hepatobiliary disorders	Placebo Group	0	26	0

1 to 10 of 16Source ClinicalTrials.gov

See all 16 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Study Drug-dextromethorphan (DM)	0	26
Total, all-cause mortality	Placebo Group	0	26
Total, all-cause mortality	Study Drug Group at 3 Months	0	24
Total, all-cause mortality	Placebo at 3 Months	0	26
Total, other adverse events	Study Drug-dextromethorphan (DM)	0	26
Total, other adverse events	Placebo Group	0	26
Total, other adverse events	Study Drug Group at 3 Months	18	24
Total, other adverse events	Placebo at 3 Months	22	26
Total, serious adverse events	Study Drug-dextromethorphan (DM)	0	26
Total, serious adverse events	Placebo Group	0	26

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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