Trial Outcomes & Findings for Riluzole to Treat Child and Adolescent Obsessive-Compulsive Disorder With or Without Autism Spectrum Disorders (NCT NCT00251303)

NCT ID: NCT00251303

Last Updated: 2014-07-15

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 78 participants
• Primary outcome timeframe: 12 weeks
• Results posted on: 2014-07-15

Participant Flow

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.

Participant milestones

Measure	Riluzole 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 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 Study STARTED	30	30
Overall Study COMPLETED	22	29
Overall Study NOT COMPLETED	8	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Riluzole n=30 Participants 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 n=30 Participants 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 n=60 Participants Total of all reporting groups
Age, Categorical <=18 years	30 Participants n=5 Participants	30 Participants n=7 Participants	60 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	14.78 years STANDARD_DEVIATION 2.10 • n=5 Participants	14.17 years STANDARD_DEVIATION 2.58 • n=7 Participants	14.47 years STANDARD_DEVIATION 2.35 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants	8 Participants n=7 Participants	16 Participants n=5 Participants
Sex: Female, Male Male	22 Participants n=5 Participants	22 Participants n=7 Participants	44 Participants n=5 Participants
Region of Enrollment United States	30 participants n=5 Participants	30 participants n=7 Participants	60 participants n=5 Participants

PRIMARY outcome

Timeframe: 12 weeks

Outcome measures

Measure	Riluzole n=22 Participants 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 n=29 Participants 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.
Much/Very Much Improved on Clinical Global Impressions - Improvement Score (CGI-I)	3 participants	4 participants

PRIMARY outcome

Timeframe: 12 weeks

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.

Outcome measures

Measure	Riluzole n=22 Participants 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 n=29 Participants 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.
Children's Yale-Brown Obsessive-Compulsive Scale Scores (CY-BOCS)	21.72 units on a scale Standard Deviation 6.43	23.30 units on a scale Standard Deviation 4.64

Adverse Events

Riluzole

Serious events: 1 serious events

Other events: 27 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 29 other events

Deaths: 0 deaths

Serious adverse events

Measure	Riluzole n=30 participants at risk 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 n=30 participants at risk 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.
Gastrointestinal disorders pancreatitis	3.3% 1/30 • Number of events 1 • 12 weeks double-blind period.	0.00% 0/30 • 12 weeks double-blind period.

Other adverse events

Measure	Riluzole n=30 participants at risk 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 n=30 participants at risk 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.
Cardiac disorders chest pain, hypotension, Tachycardia, EKG abnormality, and other cardiovascular	26.7% 8/30 • Number of events 11 • 12 weeks double-blind period.	16.7% 5/30 • Number of events 5 • 12 weeks double-blind period.
Ear and labyrinth disorders earache, poor hearing, Tinnitus, and ear other	43.3% 13/30 • Number of events 17 • 12 weeks double-blind period.	36.7% 11/30 • Number of events 13 • 12 weeks double-blind period.
Eye disorders eye irritation, blurred vision, eye other	20.0% 6/30 • Number of events 9 • 12 weeks double-blind period.	26.7% 8/30 • Number of events 10 • 12 weeks double-blind period.
Infections and infestations fever	20.0% 6/30 • Number of events 6 • 12 weeks double-blind period.	23.3% 7/30 • Number of events 7 • 12 weeks double-blind period.
Injury, poisoning and procedural complications Accidental Injury	13.3% 4/30 • Number of events 4 • 12 weeks double-blind period.	20.0% 6/30 • Number of events 6 • 12 weeks double-blind period.
Musculoskeletal and connective tissue disorders Muscle Bone joint pain, Dyskinesia, Muscle Rigidity, and Musclualrsketetal other	46.7% 14/30 • Number of events 14 • 12 weeks double-blind period.	40.0% 12/30 • Number of events 15 • 12 weeks double-blind period.
Gastrointestinal disorders Mouth Ulcer, Dry mouth, sore tongue, gum problems, Dental problems, and mouth other	36.7% 11/30 • Number of events 18 • 12 weeks double-blind period.	43.3% 13/30 • Number of events 19 • 12 weeks double-blind period.
Gastrointestinal disorders Stomach or abdominal discomfort, nausea, vomitting, diarrhea, constipation, stool discoloration	60.0% 18/30 • Number of events 37 • 12 weeks double-blind period.	70.0% 21/30 • Number of events 47 • 12 weeks double-blind period.
Gastrointestinal disorders appetite increase/decrease, taste abnormality, increased thirst, and gastrointestinal other	40.0% 12/30 • Number of events 23 • 12 weeks double-blind period.	63.3% 19/30 • Number of events 39 • 12 weeks double-blind period.
Nervous system disorders headache, dizziness, akathisia, tremor, tic movements, slurred speech, and confusion	76.7% 23/30 • Number of events 46 • 12 weeks double-blind period.	56.7% 17/30 • Number of events 32 • 12 weeks double-blind period.
Nervous system disorders concentration difficulty and memory problems	50.0% 15/30 • Number of events 21 • 12 weeks double-blind period.	43.3% 13/30 • Number of events 20 • 12 weeks double-blind period.
Renal and urinary disorders Painful urination, difficulty urinating, increased frequency, and enuresis	30.0% 9/30 • Number of events 11 • 12 weeks double-blind period.	43.3% 13/30 • Number of events 15 • 12 weeks double-blind period.
Skin and subcutaneous tissue disorders Dermatologic Skin Irritation, Sweating, hair problems, and skin other	66.7% 20/30 • Number of events 25 • 12 weeks double-blind period.	66.7% 20/30 • Number of events 28 • 12 weeks double-blind period.
Reproductive system and breast disorders breast pain swelling, discharge from nipples, menstrual irregularity, cramps, premenstral tension	87.5% 7/8 • Number of events 7 • 12 weeks double-blind period.	50.0% 4/8 • Number of events 8 • 12 weeks double-blind period.
Reproductive system and breast disorders genital discomfort, increased/decreased libido and genitourinary other	16.7% 5/30 • Number of events 7 • 12 weeks double-blind period.	6.7% 2/30 • Number of events 3 • 12 weeks double-blind period.
Respiratory, thoracic and mediastinal disorders nasal congestion, bloody nose, sore throat, difficulty swallowing, and flu upper respitory	66.7% 20/30 • Number of events 35 • 12 weeks double-blind period.	80.0% 24/30 • Number of events 44 • 12 weeks double-blind period.
Respiratory, thoracic and mediastinal disorders nose throat other, shortness of breath, wheezing, and coughing	50.0% 15/30 • Number of events 19 • 12 weeks double-blind period.	50.0% 15/30 • Number of events 26 • 12 weeks double-blind period.
Surgical and medical procedures medical or surgical procedure	3.3% 1/30 • Number of events 1 • 12 weeks double-blind period.	10.0% 3/30 • Number of events 3 • 12 weeks double-blind period.
Psychiatric disorders tiredness fatigue, increased/decreased motor activity, difficulty falling asleep	73.3% 22/30 • Number of events 36 • 12 weeks double-blind period.	73.3% 22/30 • Number of events 45 • 12 weeks double-blind period.
Psychiatric disorders early morning awakening, interrupted sleep, drowsiness sedation, hallucinations, depression, anxiety	80.0% 24/30 • Number of events 62 • 12 weeks double-blind period.	83.3% 25/30 • Number of events 66 • 12 weeks double-blind period.
Psychiatric disorders irritability, suicidal ideas, suicidal behavior, agression, and psychological behavior other	70.0% 21/30 • Number of events 35 • 12 weeks double-blind period.	70.0% 21/30 • Number of events 38 • 12 weeks double-blind period.
Musculoskeletal and connective tissue disorders swelling	6.7% 2/30 • Number of events 2 • 12 weeks double-blind period.	0.00% 0/30 • 12 weeks double-blind period.

Additional Information

Dr. Susan E. Swedo

National Institute of Mental Health, Pediatrics and Developmental Neuroscience Branch

Phone: 301-496-5323

Email: swedos@mail.nih.gov

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place