- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00198120
Safety and Effectiveness of D-Cycloserine in Children With Autism
A Randomized Controlled Trial of D-Cycloserine in Autism
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
This project proposes to study the efficacy and safety of D-cycloserine in children with autism. The central hypothesis of this project is that D-cycloserine will be efficacious in reducing certain symptoms of autism including some aspects of social impairment.
Autism is a severe neuropsychiatric disorder with a prevalence of at least 0.1 %. Despite investigations into the pharmacologic treatment of autism, no drugs have been shown to consistently improve the core symptoms of the disorder, namely social and communication impairment. Pilot data has suggested that D-cycloserine, a drug that affects the N-methyl-D-aspartate (NMDA) subtype of glutamate receptor, has efficacy for the symptom of social withdrawal in autism. In this study, children with autism will be randomly assigned to treatment with either D-cycloserine or placebo for 8 weeks. Both the subjects and investigators will be blind to treatment assignment. Subjects will be rated on a variety of clinical measures to examine the effects of D-cycloserine on social withdrawal and other symptoms of autism. Safety data including side-effects, vital signs, blood tests, and electrocardiograms will be performed at the beginning and end of the study. This study will provide important information about the effects of D-cycloserine for treating core and associated symptoms of autism. It will also greatly expand the knowledge about glutamatergic agents in autism and provide crucial information regarding the pathophysiology and future design of drug studies in autism.
Study Type
Enrollment (Actual)
Phase
- Phase 3
Contacts and Locations
Study Locations
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Indiana
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Indianapolis, Indiana, United States, 46202
- Riley Hospital for Children, Christian Sarkine Autism Treatment Center
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Age 3 Years to 12 Years
- Diagnostic Statistical Manual Version -IV (DSM-IV) and Autism Diagnostic Interview - Revised (ADI-R)-confirmed Diagnosis of Autistic Disorder
- Aberrant Behavior Checklist (ABC) Lethargy Subscale Score of 13 or greater
Exclusion Criteria:
- Children with Severe to Profound Mental Retardation
- Weight at Screening Visit <11 kilograms
- Clinical Global Impressions-Severity Score of 7
- Presence of a Neurodevelopmental Disorder with Possible Associations to Autism: Subjects with Fragile X Syndrome, Tuberous Sclerosis, or other neurodevelopmental disorders known to be associated with autism or autistic features will be excluded.
- Presence of a Psychiatric Disorder that would Require a Specific Type of Treatment: Subjects with major depressive disorder, bipolar disorder, or a psychotic disorder will be excluded because treatment for these disorders often requires specific psychotropic agents. Subjects with an active substance use disorder will be excluded because of safety concerns and problems this would cause in assessing efficacy.
- Presence of a Medical Condition that would make Treatment with D-Cycloserine Less Safe: Subjects with significant cardiac, hepatic, or renal disease will be excluded due to concerns about pharmacokinetic alterations or adverse effects. Subjects with epilepsy or a history of seizures will be excluded due to rare reports of seizures with high doses of D-cycloserine. D-cycloserine is an U.S. FDA Pregnancy Category C drug. Because of the unknown effects of D-cycloserine on the developing human fetus, females of childbearing potential will be given a urine pregnancy test and required to use a suitable form of birth control during the study.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: 1
Participants will receive D-Cycloserine for 8 weeks.
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D-Cycloserine 0.6mg/kg/day in week 1 D-Cycloserine 1.1mg/kg/day in week 2 D-Cycloserine 1.7mg/kg/day in week 3-8 Flexible dosing based on response.
Capsule Strength: 10mg, 20mg
Other Names:
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Placebo Comparator: 2
Participants will receive placebo for 8 weeks.
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Placebo: same dosing schedule and capsule strength
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Clinical Global Impressions (CGI) Global Improvement
Time Frame: Change from Baseline at 8 Weeks
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All randomized subjects in the Double-Blind Phase will be assessed for change.
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Change from Baseline at 8 Weeks
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Change in Clinical Global Impressions (CGI) Global Improvement
Time Frame: Change from Open-Label Baseline at 8 Weeks
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All placebo non-responders will enter into an open-label phase after the Double-Blind Phase
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Change from Open-Label Baseline at 8 Weeks
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Change in Lethargy Subscale of the Aberrant Behavior Checklist (ABC)
Time Frame: Change from Baseline at 8 Weeks.
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All randomized subjects in the Double-Blind Phase will be assessed for change.
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Change from Baseline at 8 Weeks.
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Change in Lethargy Subscale of the Aberrant Behavior Checklist (ABC)
Time Frame: Change from Open-Label Baseline at 8 Weeks
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All placebo non-responders will enter into an open-label phase after the Double-Blind Phase
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Change from Open-Label Baseline at 8 Weeks
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Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Christopher J. McDougle, MD, Indiana University School of Medicine
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Mental Disorders
- Neurodevelopmental Disorders
- Child Development Disorders, Pervasive
- Autism Spectrum Disorder
- Autistic Disorder
- Molecular Mechanisms of Pharmacological Action
- Anti-Infective Agents
- Antimetabolites
- Anti-Bacterial Agents
- Antitubercular Agents
- Antibiotics, Antitubercular
- Anti-Infective Agents, Urinary
- Renal Agents
- Cycloserine
Other Study ID Numbers
- K23MH068627 (U.S. NIH Grant/Contract)
- 0305-30
- DDTR BK-TKND
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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