Trial of Dextromethorphan in Rett Syndrome

This study has been terminated.
(Study changed to a placebo controlled trial of dextromethorphan)
Sponsor:
Information provided by (Responsible Party):
SakkuBai Naidu, M.D., Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
ClinicalTrials.gov Identifier:
NCT00593957
First received: January 4, 2008
Last updated: March 26, 2014
Last verified: July 2013
Results First Received: March 8, 2013  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Rett Syndrome
Intervention: Drug: Dextromethorphan

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Recruitment was from 2004 to 2010. Study initiation was delayed due to the closure of Johns Hopkins Medicine Institutional Review Board(JHMIRB). Patients were recruited from our Kennedy Krieger Institute (KKI)medical clinics, physician referrals,and parent organizations. Parents were sent letters of invitation.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Pharmacokinetics in enrolled subjects required her to be a fast metabolizer of Dextromethorphan (DM).

Reporting Groups
  Description
DM1( 0.25 mg/kg /Day)

Dextromethorphan 0.25 mg/kg per day

Dextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.

Dextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.

DM2 (2.5 mg/kg/Day)

Dextromethorphan 2.5 mg/kg/day

Dextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.

Dextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.

DM3 (5mg/kg/Day)

Dextromethorphan 5mg/kg/day

Dextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.

Dextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.


Participant Flow:   Overall Study
    DM1( 0.25 mg/kg /Day)     DM2 (2.5 mg/kg/Day)     DM3 (5mg/kg/Day)  
STARTED     13     13     12  
COMPLETED     13     12     10  
NOT COMPLETED     0     1     2  
Physician Decision                 0                 1                 2  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
DM1( 0.25 mg/kg /Day)

Dextromethorphan 0.25 mg/kg per day

Dextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.

Dextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.

DM2 (2.5 mg/kg/Day)

Dextromethorphan 2.5 mg/kg/day

Dextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.

Dextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.

DM3 (5mg/kg/Day)

Dextromethorphan 5mg/kg/day

Dextromethorphan : Dextromethorphan polistirex. Doses are 0.25 mg/kg/day, 2.5mg/kg/day, and 5 mg/kg/day. The drug is given in two divided doses 12 hours apart for 6 months.

Dextromethorphan : Subjects will be randomized to receive one of three dosage groups either 0.25 mg/kg per day; or 2.5 mg/kg/day; or 5mg/kg/day of Dextromethorphan Polistirex (Delsym)oral syrup, which will be given exactly 12 hours apart in two divided doses during the 6 month trial.

Total Total of all reporting groups

Baseline Measures
    DM1( 0.25 mg/kg /Day)     DM2 (2.5 mg/kg/Day)     DM3 (5mg/kg/Day)     Total  
Number of Participants  
[units: participants]
  13     13     12     38  
Age  
[units: participants]
       
<=18 years     13     13     12     38  
Between 18 and 65 years     0     0     0     0  
>=65 years     0     0     0     0  
Age  
[units: years]
Mean ± Standard Deviation
  6.54  ± 2.70     6.31  ± 3.50     6.08  ± 3.06     6.32  ± 3.02  
Gender  
[units: participants]
       
Female     13     13     12     38  
Male     0     0     0     0  
Region of Enrollment  
[units: participants]
       
United States     13     13     12     38  



  Outcome Measures
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1.  Primary:   Difference in EEG Spike Counts at Six Months Compared to Baseline for Each Treatment Arm.   [ Time Frame: Initial and 6-month post-treatment ]

2.  Secondary:   Improvement in Receptive Language as Measured by the Mullen Scale.   [ Time Frame: Change in mean between Initial and 6-month follow-up ]

3.  Secondary:   Difference in SSI Mean Score at Six Months Compared to Baseline for Each Treatment Arm.   [ Time Frame: Initial and 6 month followup ]

4.  Secondary:   Mean SSI Score for Total Subjects at Baseline and 6 Months   [ Time Frame: 0-6 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
The trial was terminated due to the FDA requiring a placebo controlled trial instead of the ongoing open label trial. As recruitment was delayed,the total number of participants was also less than anticipated.


  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr Sakkubai Naidu Professor of Neurology and Pediatrics
Organization: Kennedy Krieger Institute
phone: 443-923-2778
e-mail: naidu@kennedykrieger.org


No publications provided


Responsible Party: SakkuBai Naidu, M.D., Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
ClinicalTrials.gov Identifier: NCT00593957     History of Changes
Other Study ID Numbers: FD2408
Study First Received: January 4, 2008
Results First Received: March 8, 2013
Last Updated: March 26, 2014
Health Authority: United States: Food and Drug Administration