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Riluzole to Treat Child and Adolescent Obsessive-Compulsive Disorder With or Without Autism Spectrum Disorders

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ClinicalTrials.gov Identifier: NCT00251303
Recruitment Status : Completed
First Posted : November 9, 2005
Results First Posted : July 15, 2014
Last Update Posted : July 15, 2014
Sponsor:
Information provided by (Responsible Party):
Susan Swedo, M.D., National Institutes of Health Clinical Center (CC)

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Obsessive-Compulsive Disorder
Autism Spectrum Disorder
Autism
Asperger Disorder
Developmental Disorder
Interventions Drug: Riluzole
Drug: Placebo
Enrollment 78

Recruitment Details  
Pre-assignment Details Subjects had to be symptomatic for at least 6 weeks, with a score of at least 20 CY-BOCS; had to have had at least one standard-of-care treatment for childhood OCD for adequate periods of time; had to have been stable for at least 6 weeks on any medicine; had to meet entrance criteria at a screening and 2 weeks later.
Arm/Group Title Riluzole Placebo
Hide Arm/Group Description Riluzole was titrated upward to at least 100 mg of active drug. If adverse effects are noted, dose titration was slowed, stopped, or reversed, or the drug was discontinued. Maximum permitted dose of riluzole was 120 mg/day. Placebo capsules were prepared by NIH Clinical Center Pharmacy to appear identical to active drug capsules. Dose was titrated upward as if active drug. Follow-up and laboratory studies were identical for active drug and placebo arms.
Period Title: Overall Study
Started 30 [1] 30
Completed 22 29
Not Completed 8 1
[1]
78 subjects signed any consent. 60 started drug (30 riluzole & 30 placebo).
Arm/Group Title Riluzole Placebo Total
Hide Arm/Group Description

In a 12-weeks long double-blind phase, the clinicians and subjects and their guardians were blind to active drug or placebo. There were approx. twice monthly in-person or telephone contacts.

Riluzole was titrated upward to at least 100 mg of active drug. If adverse effects are noted, dose titration was slowed, stopped, or reversed, or the drug was discontinued. Maximum permitted dose of riluzole was 120 mg/day. At the end of the 12 weeks study period, subjects and their families could elect to take open-label riluzole.Since neither subjects nor investigators knew whether subjects had been receiving active drug,the open-label administration was also titrated. Laboratory assays were obtained at 2, 4, 8, and 12 weeks after starting the open-label riluzole, as they had been during double-blind. Subsequently, testing was less frequent.

Placebo capsules were prepared by NIH Clinical Center Pharmacy to appear identical to active drug capsules. Dose was titrated upward as if active drug. Follow-up and laboratory studies were identical for active drug and placebo arms. At the end of the double-blind phase, subjects could elect to take open-label drug, which was titrated upward to the maximum dose, 100 mg daily.

Study blind was not broken for subjects or investigators until the final subject had completed the double-blind phase of the study.

Total of all reporting groups
Overall Number of Baseline Participants 30 30 60
Hide Baseline Analysis Population Description
These data apply only to double-blind phase.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 30 participants 30 participants 60 participants
<=18 years
30
 100.0%
30
 100.0%
60
 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 30 participants 30 participants 60 participants
14.78  (2.10) 14.17  (2.58) 14.47  (2.35)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 30 participants 30 participants 60 participants
Female
8
  26.7%
8
  26.7%
16
  26.7%
Male
22
  73.3%
22
  73.3%
44
  73.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 30 participants 30 participants 60 participants
30 30 60
1.Primary Outcome
Title Much/Very Much Improved on Clinical Global Impressions - Improvement Score (CGI-I)
Hide Description [Not Specified]
Time Frame 12 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Riluzole Placebo
Hide Arm/Group Description:

In a 12-weeks long double-blind phase, the clinicians and subjects and their guardians were blind to active drug or placebo. There were approx. twice monthly in-person or telephone contacts.

Riluzole was titrated upward to at least 100 mg of active drug. If adverse effects are noted, dose titration was slowed, stopped, or reversed, or the drug was discontinued. Maximum permitted dose of riluzole was 120 mg/day. At the end of the 12 weeks study period, subjects and their families could elect to take open-label riluzole.Since neither subjects nor investigators knew whether subjects had been receiving active drug,the open-label administration was also titrated. Laboratory assays were obtained at 2, 4, 8, and 12 weeks after starting the open-label riluzole, as they had been during double-blind. Subsequently, testing was less frequent.

Placebo capsules were prepared by NIH Clinical Center Pharmacy to appear identical to active drug capsules. Dose was titrated upward as if active drug. Follow-up and laboratory studies were identical for active drug and placebo arms. At the end of the double-blind phase, subjects could elect to take open-label drug, which was titrated upward to the maximum dose, 100 mg daily.

Study blind was not broken for subjects or investigators until the final subject had completed the double-blind phase of the study.

Overall Number of Participants Analyzed 22 29
Measure Type: Number
Unit of Measure: participants
3 4
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Riluzole, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.959
Comments [Not Specified]
Method Chi-squared
Comments [Not Specified]
2.Primary Outcome
Title Children's Yale-Brown Obsessive-Compulsive Scale Scores (CY-BOCS)
Hide Description CY-BOCS is a 0-40 point scale of obsessive-compulsive symptom severity, higher number indicates more severe obsessive-compulsive symptoms. Comparison of 12 weeks scores for placebo and riluzole groups.
Time Frame 12 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Riluzole Placebo
Hide Arm/Group Description:
Riluzole was titrated upward to at least 100 mg of active drug. If adverse effects are noted, dose titration was slowed, stopped, or reversed, or the drug was discontinued. Maximum permitted dose of riluzole was 120 mg/day.
Placebo capsules were prepared by NIH Clinical Center Pharmacy to appear identical to active drug capsules. Dose was titrated upward as if active drug. Follow-up and laboratory studies were identical for active drug and placebo arms.
Overall Number of Participants Analyzed 22 29
Mean (Standard Deviation)
Unit of Measure: units on a scale
21.72  (6.43) 23.30  (4.64)
Time Frame 12 weeks double-blind period.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Riluzole Placebo
Hide Arm/Group Description

In a 12-weeks long double-blind phase, the clinicians and subjects and their guardians were blind to active drug or placebo. There were approx. twice monthly in-person or telephone contacts.

Riluzole was titrated upward to at least 100 mg of active drug. If adverse effects are noted, dose titration was slowed, stopped, or reversed, or the drug was discontinued. Maximum permitted dose of riluzole was 120 mg/day. At the end of the 12 weeks study period, subjects and their families could elect to take open-label riluzole.Since neither subjects nor investigators knew whether subjects had been receiving active drug,the open-label administration was also titrated. Laboratory assays were obtained at 2, 4, 8, and 12 weeks after starting the open-label riluzole, as they had been during double-blind. Subsequently, testing was less frequent.

Placebo capsules were prepared by NIH Clinical Center Pharmacy to appear identical to active drug capsules. Dose was titrated upward as if active drug. Follow-up and laboratory studies were identical for active drug and placebo arms. At the end of the double-blind phase, subjects could elect to take open-label drug, which was titrated upward to the maximum dose, 100 mg daily.

Study blind was not broken for subjects or investigators until the final subject had completed the double-blind phase of the study.

All-Cause Mortality
Riluzole Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Riluzole Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/30 (3.33%)      0/30 (0.00%)    
Gastrointestinal disorders     
pancreatitis   1/30 (3.33%)  1 0/30 (0.00%)  0
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Riluzole Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   27/30 (90.00%)      29/30 (96.67%)    
Cardiac disorders     
chest pain, hypotension, Tachycardia, EKG abnormality, and other cardiovascular   8/30 (26.67%)  11 5/30 (16.67%)  5
Ear and labyrinth disorders     
earache, poor hearing, Tinnitus, and ear other   13/30 (43.33%)  17 11/30 (36.67%)  13
Eye disorders     
eye irritation, blurred vision, eye other   6/30 (20.00%)  9 8/30 (26.67%)  10
Gastrointestinal disorders     
Mouth Ulcer, Dry mouth, sore tongue, gum problems, Dental problems, and mouth other   11/30 (36.67%)  18 13/30 (43.33%)  19
Stomach or abdominal discomfort, nausea, vomitting, diarrhea, constipation, stool discoloration   18/30 (60.00%)  37 21/30 (70.00%)  47
appetite increase/decrease, taste abnormality, increased thirst, and gastrointestinal other   12/30 (40.00%)  23 19/30 (63.33%)  39
Infections and infestations     
fever   6/30 (20.00%)  6 7/30 (23.33%)  7
Injury, poisoning and procedural complications     
Accidental Injury   4/30 (13.33%)  4 6/30 (20.00%)  6
Musculoskeletal and connective tissue disorders     
Muscle Bone joint pain, Dyskinesia, Muscle Rigidity, and Musclualrsketetal other   14/30 (46.67%)  14 12/30 (40.00%)  15
swelling   2/30 (6.67%)  2 0/30 (0.00%)  0
Nervous system disorders     
headache, dizziness, akathisia, tremor, tic movements, slurred speech, and confusion   23/30 (76.67%)  46 17/30 (56.67%)  32
concentration difficulty and memory problems   15/30 (50.00%)  21 13/30 (43.33%)  20
Psychiatric disorders     
tiredness fatigue, increased/decreased motor activity, difficulty falling asleep   22/30 (73.33%)  36 22/30 (73.33%)  45
early morning awakening, interrupted sleep, drowsiness sedation, hallucinations, depression, anxiety   24/30 (80.00%)  62 25/30 (83.33%)  66
irritability, suicidal ideas, suicidal behavior, agression, and psychological behavior other   21/30 (70.00%)  35 21/30 (70.00%)  38
Renal and urinary disorders     
Painful urination, difficulty urinating, increased frequency, and enuresis   9/30 (30.00%)  11 13/30 (43.33%)  15
Reproductive system and breast disorders     
breast pain swelling, discharge from nipples, menstrual irregularity, cramps, premenstral tension   7/8 (87.50%)  7 4/8 (50.00%)  8
genital discomfort, increased/decreased libido and genitourinary other   5/30 (16.67%)  7 2/30 (6.67%)  3
Respiratory, thoracic and mediastinal disorders     
nasal congestion, bloody nose, sore throat, difficulty swallowing, and flu upper respitory   20/30 (66.67%)  35 24/30 (80.00%)  44
nose throat other, shortness of breath, wheezing, and coughing   15/30 (50.00%)  19 15/30 (50.00%)  26
Skin and subcutaneous tissue disorders     
Dermatologic Skin Irritation, Sweating, hair problems, and skin other   20/30 (66.67%)  25 20/30 (66.67%)  28
Surgical and medical procedures     
medical or surgical procedure   1/30 (3.33%)  1 3/30 (10.00%)  3
Indicates events were collected by systematic assessment
Only treatment-refractory subjects were eligible for study participation. This may have decreased treatment response. Further, 98% were taking psychotropic drugs at baseline, which may have reduced between-group differences.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Dr. Susan E. Swedo
Organization: National Institute of Mental Health, Pediatrics and Developmental Neuroscience Branch
Phone: 301-496-5323
Other Publications:
Responsible Party: Susan Swedo, M.D., National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT00251303     History of Changes
Other Study ID Numbers: 050225
05-M-0225 ( Other Identifier: NIMH Office of Protocol Services )
First Submitted: November 9, 2005
First Posted: November 9, 2005
Results First Submitted: May 16, 2013
Results First Posted: July 15, 2014
Last Update Posted: July 15, 2014