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Autistic DisorderPhase 3NCT00198120

Safety and Effectiveness of D-Cycloserine in Children With Autism

A Phase 3 study testing D-cycloserine in people with Autistic Disorder, run by Indiana University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This study will determine the effectiveness of D-cycloserine in reducing symptoms of autism in autistic children. Read more about this study

Who is it for?
Ages 3 to 12 years
Conditions the study is for
Autistic Disorder
What is being tested?
D-cycloserine
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
80 participants
Where
1 siteUnited States
Study dates
Feb 2004to Aug 2010
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 3 to 12 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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

Who cannot join

  • 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.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Riley Hospital for Children, Christian Sarkine Autism Treatment Center

    Indianapolis, Indiana, 46202

Other trials in United States

About this study

Official title: A Randomized Controlled Trial of D-Cycloserine in Autism

This study will determine the effectiveness of D-cycloserine in reducing symptoms of autism in autistic children.

Read the 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.

Being tested
D-cycloserine, Placebo
Keywords
Autistic Disorder, cycloserine, pharmacology, glutamatergic agents, communication, social interaction, Double-Blind Method
Medical subject headings
Autistic Disorder, Communication

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • D-cycloserineDrug

    Also called Seromycin, Cycloserine

    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

  • PlaceboDrug

    Placebo: same dosing schedule and capsule strength

Study groups in NCT00198120
Study groupRoleWhat they receive
1ExperimentalParticipants will receive D-Cycloserine for 8 weeks.
Receives: Drug: D-cycloserine
2Placebo comparatorParticipants will receive placebo for 8 weeks.
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change in Clinical Global Impressions (CGI) Global Improvement

    All randomized subjects in the Double-Blind Phase will be assessed for change.

    Measured: Change from Baseline at 8 Weeks

  2. Change in Clinical Global Impressions (CGI) Global Improvement

    All placebo non-responders will enter into an open-label phase after the Double-Blind Phase

    Measured: Change from Open-Label Baseline at 8 Weeks

  3. Change in Lethargy Subscale of the Aberrant Behavior Checklist (ABC)

    All randomized subjects in the Double-Blind Phase will be assessed for change.

    Measured: Change from Baseline at 8 Weeks.

  4. Change in Lethargy Subscale of the Aberrant Behavior Checklist (ABC)

    All placebo non-responders will enter into an open-label phase after the Double-Blind Phase

    Measured: Change from Open-Label Baseline at 8 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Sep 2005 (estimated)
  2. Study startFeb 2004
  3. Primary completionSep 2007
  4. Study completionAug 2010

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
80 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00198120
Sponsor's protocol ID
K23MH068627
Other IDs
K23MH068627, 0305-30, DDTR BK-TKND
Status last verified
Apr 2016
First submitted
12 Sep 2005
Last updated
12 Apr 2016
Results posted
No
Data monitoring committee
Yes

Publications

  • Iffland M, Livingstone N, Jorgensen M, Hazell P, Gillies D. Pharmacological intervention for irritability, aggression, and self-injury in autism spectrum disorder (ASD). Cochrane Database Syst Rev. 2023 Oct 9;10(10):CD011769. doi: 10.1002/14651858.CD011769.pub2.

    PubMed 37811711

  • Aye SZ, Ni H, Sein HH, Mon ST, Zheng Q, Wong YKY. The effectiveness and adverse effects of D-cycloserine compared with placebo on social and communication skills in individuals with autism spectrum disorder. Cochrane Database Syst Rev. 2021 Feb 14;2(2):CD013457. doi: 10.1002/14651858.CD013457.pub2.

    PubMed 33583058

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing D-cycloserine in people with Autistic Disorder, run by Indiana University at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 12 Apr 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change in Clinical Global Impressions (CGI) Global Improvement (time frame: Change from Baseline at 8 Weeks). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00198120, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Apr 2016.

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