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Trial record 21 of 73 for:    "Asperger syndrome"

A Study of Oral N-Acetylcysteine in Children With Autism Spectrum Disorders

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00453180
Recruitment Status : Completed
First Posted : March 28, 2007
Results First Posted : June 14, 2017
Last Update Posted : June 14, 2017
Sponsor:
Collaborator:
National Alliance for Autism Research
Information provided by (Responsible Party):
Indiana University ( Indiana University School of Medicine )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Conditions Autistic Disorder
Asperger Syndrome
Child Development Disorders, Pervasive
Interventions Drug: N-acetylcysteine
Drug: Placebo
Enrollment 31
Recruitment Details  
Pre-assignment Details  
Arm/Group Title N-acetylcysteine Placebo
Hide Arm/Group Description

Target dose for n-acetylcysteine is 60 mg/kg/day. Capsules available in 300 mg and 600 mg strengths.

N-acetylcysteine: Capsules available in 300 mg or 600mg strength. Target dose of n-acetylcysteine will be 60mg/kg/day TID. Dosage will be increased to this target dose from week 1 to week 3 barring side effects. Dose reduction will be allowed at any time for adverse side effects. Maximum dose of n-acetylcysteine will be 4200mg/day.

Subjects randomized to placebo arm will receive capsules identical in size and appearance to those subjects receiving study drug. Placebo capsules contain inactive ingredients.

Placebo: Subjects randomized to placebo arm will receive placebo pill for duration of study.

Period Title: Overall Study
Started 16 15
Completed 13 12
Not Completed 3 3
Reason Not Completed
Lost to Follow-up             2             1
Adverse Event             1             2
Arm/Group Title N-acetylcysteine Placebo Total
Hide Arm/Group Description

Target dose for n-acetylcysteine is 60 mg/kg/day. Capsules available in 300 mg and 600 mg strengths.

N-acetylcysteine: Capsules available in 300 mg or 600mg strength. Target dose of n-acetylcysteine will be 60mg/kg/day TID. Dosage will be increased to this target dose from week 1 to week 3 barring side effects. Dose reduction will be allowed at any time for adverse side effects. Maximum dose of n-acetylcysteine will be 4200mg/day.

Subjects randomized to placebo arm will receive capsules identical in size and appearance to those subjects receiving study drug. Placebo capsules contain inactive ingredients.

Placebo: Subjects randomized to placebo arm will receive placebo pill for duration of study.

Total of all reporting groups
Overall Number of Baseline Participants 16 15 31
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 15 participants 31 participants
<=18 years
16
 100.0%
15
 100.0%
31
 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 16 participants 15 participants 31 participants
7.6  (2.5) 8.2  (2.9) 7.8  (2.7)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 15 participants 31 participants
Female
4
  25.0%
3
  20.0%
7
  22.6%
Male
12
  75.0%
12
  80.0%
24
  77.4%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 16 participants 15 participants 31 participants
16 15 31
Clinical Global Impression-Severity   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 15 participants 31 participants
1. Normal, not al all ill
0
   0.0%
0
   0.0%
0
   0.0%
2. Borderline ill
0
   0.0%
0
   0.0%
0
   0.0%
3..Mildly ill
0
   0.0%
0
   0.0%
0
   0.0%
4. Moderatly ill
6
  37.5%
9
  60.0%
15
  48.4%
5. Markedly ill
9
  56.3%
6
  40.0%
15
  48.4%
6. Severly ill
1
   6.3%
0
   0.0%
1
   3.2%
7. Extremly ill
0
   0.0%
0
   0.0%
0
   0.0%
[1]
Measure Description: The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill.
1.Primary Outcome
Title Clinical Global Impression - Severity
Hide Description The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill.
Time Frame Week 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title N-acetylcysteine Placebo
Hide Arm/Group Description:

Target dose for n-acetylcysteine is 60 mg/kg/day. Capsules available in 300 mg and 600 mg strengths.

N-acetylcysteine: Capsules available in 300 mg or 600mg strength. Target dose of n-acetylcysteine will be 60mg/kg/day TID. Dosage will be increased to this target dose from week 1 to week 3 barring side effects. Dose reduction will be allowed at any time for adverse side effects. Maximum dose of n-acetylcysteine will be 4200mg/day.

Subjects randomized to placebo arm will receive capsules identical in size and appearance to those subjects receiving study drug. Placebo capsules contain inactive ingredients.

Placebo: Subjects randomized to placebo arm will receive placebo pill for duration of study.

Overall Number of Participants Analyzed 13 12
Measure Type: Count of Participants
Unit of Measure: Participants
1.Normal, not at all ill 0 0
2.Borderline ill 0 0
3.Mildly ill 3 0
4.Moderately ill 5 12
5.Markedly ill 5 0
6.Severely ill 0 0
7.Extremely ill 0 0
2.Primary Outcome
Title Clinical Global Impression - Improvement
Hide Description

Clinical Global Impression - Improvement (CGI-I) is designed to take into account all factors to arrive at an assessment of response to treatment.

The CGI-I scale ranges from 1 to 7 (1=very much improved; 2=much improved, 3=minimally Improved, 4=no change, 5=minimally worse, 6= much worse and 7=very much worse). Participants with a CGI-I score of 1 or 2 were classified as improved. Participants with a CGI score of 3, 4 or 5 were classified as no response. No participants scored 6 or 7.

Time Frame Week 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title N-acetylcysteine Placebo
Hide Arm/Group Description:

Target dose for n-acetylcysteine is 60 mg/kg/day. Capsules available in 300 mg and 600 mg strengths.

N-acetylcysteine: Capsules available in 300 mg or 600mg strength. Target dose of n-acetylcysteine will be 60mg/kg/day TID. Dosage will be increased to this target dose from week 1 to week 3 barring side effects. Dose reduction will be allowed at any time for adverse side effects. Maximum dose of n-acetylcysteine will be 4200mg/day.

Subjects randomized to placebo arm will receive capsules identical in size and appearance to those subjects receiving study drug. Placebo capsules contain inactive ingredients.

Placebo: Subjects randomized to placebo arm will receive placebo pill for duration of study.

Overall Number of Participants Analyzed 13 12
Measure Type: Count of Participants
Unit of Measure: Participants
Improved
6
  46.2%
5
  41.7%
No Response
7
  53.8%
7
  58.3%
3.Secondary Outcome
Title Aberrant Behavior Checklist
Hide Description The Aberrant Behavior Checklist (ABC) is a 58-item measure of maladaptive behaviors and is used as a measure of drug effects. Each of the 58 items are rated from 0 (not at all) to 3 (severe).The ABC has 5 subscales: Irritability (15 items) ranging from 0 (not at all) to 45 (severe), Lethargy (16 items) ranging from 0 (not at all) to 48 (severe), Stereotypy (7 items) ranging from 0 (not at all) to 21 (severe), Hyperactivity (16 items) ranging from 0 (not at all) to 48 (severe), and Inappropriate Speech (4 items) ranging from 0 (not at all) to 12 (severe). Higher scores indicate a higher level of maladaptive behavior.
Time Frame Week 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title N-acetylcysteine Placebo
Hide Arm/Group Description:

Target dose for n-acetylcysteine is 60 mg/kg/day. Capsules available in 300 mg and 600 mg strengths.

N-acetylcysteine: Capsules available in 300 mg or 600mg strength. Target dose of n-acetylcysteine will be 60mg/kg/day TID. Dosage will be increased to this target dose from week 1 to week 3 barring side effects. Dose reduction will be allowed at any time for adverse side effects. Maximum dose of n-acetylcysteine will be 4200mg/day.

Subjects randomized to placebo arm will receive capsules identical in size and appearance to those subjects receiving study drug. Placebo capsules contain inactive ingredients.

Placebo: Subjects randomized to placebo arm will receive placebo pill for duration of study.

Overall Number of Participants Analyzed 13 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
Irritablity 14.9  (14.0) 12.0  (7.3)
Lethargy 10.0  (7.1) 7.9  (4.7)
Stereotypy 3.9  (5.1) 5.8  (4.0)
Hyperactivity 17.4  (16.4) 15.1  (10.8)
Inappropriate Speech 4.1  (3.9) 5.14  (3.5)
4.Secondary Outcome
Title Social Responsiveness Scale
Hide Description The Social Responsiveness Scale (SRS) is a 65-item scale that assesses social impairment in the aspects of social awareness, social cognition, social communication, social motivation and autistic mannerisms. Each item is scored from 0 (not true) to 3 (almost always true). The total SRS raw score may range from 0-195, where higher scores indicate greater severity.
Time Frame Week 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title N-acetylcysteine Placebo
Hide Arm/Group Description:

Target dose for n-acetylcysteine is 60 mg/kg/day. Capsules available in 300 mg and 600 mg strengths.

N-acetylcysteine: Capsules available in 300 mg or 600mg strength. Target dose of n-acetylcysteine will be 60mg/kg/day TID. Dosage will be increased to this target dose from week 1 to week 3 barring side effects. Dose reduction will be allowed at any time for adverse side effects. Maximum dose of n-acetylcysteine will be 4200mg/day.

Subjects randomized to placebo arm will receive capsules identical in size and appearance to those subjects receiving study drug. Placebo capsules contain inactive ingredients.

Placebo: Subjects randomized to placebo arm will receive placebo pill for duration of study.

Overall Number of Participants Analyzed 13 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
85.8  (34.9) 89.1  (26.3)
5.Secondary Outcome
Title Pervasive Developmental Disorder Behavior Index
Hide Description The PDD Behavior Inventory (PDDBI) is a rating scale filled out by caregivers or teachers that was designed to assess children having a Pervasive Developmental Disorder (PDD; autism, Asperger disorder, PDD-NOS, or childhood disintegrative disorder). Both adaptive and maladaptive behaviors are assessed in the scale, making it useful for treatment studies in which decreases in maladaptive behaviors and improvements in adaptive social and language skills relevant to PDD are expected.
Time Frame Week 12
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Hide Analysis Population Description
Less than 25% of participants in each group received a score on the PDDBI due to a significant floor effect. As a result, the data for these participants are not considered reliable given the significant floor effect.
Arm/Group Title N-acetylcysteine Placebo
Hide Arm/Group Description:

Target dose for n-acetylcysteine is 60 mg/kg/day. Capsules available in 300 mg and 600 mg strengths.

N-acetylcysteine: Capsules available in 300 mg or 600mg strength. Target dose of n-acetylcysteine will be 60mg/kg/day TID. Dosage will be increased to this target dose from week 1 to week 3 barring side effects. Dose reduction will be allowed at any time for adverse side effects. Maximum dose of n-acetylcysteine will be 4200mg/day.

Subjects randomized to placebo arm will receive capsules identical in size and appearance to those subjects receiving study drug. Placebo capsules contain inactive ingredients.

Placebo: Subjects randomized to placebo arm will receive placebo pill for duration of study.

Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
6.Secondary Outcome
Title Vineland Adaptive Behavior Scales-II (VABS-II)
Hide Description The VABS-II is a semi-structured interview designed to assess adaptive functioning in the domains of communication, daily living skills and socialization. Items in each domain are rated as either 0 (does not), 1(sometimes) or 2(independently) performs a given behavior or skill. The communication domain has 99 items with scores ranging from 0-198. The daily living skills domain has 109 items with scores ranging from 0-218. The socialization domain has 99 items with scores ranging from 0-198. The domains scores are combined to form the adaptive composite score (ranging from 20-160). The raw scores from the communication, daily living skills and socialization domains along with the composite score were selected for use in this study. Higher scores indicate a higher level of adaptive functioning.
Time Frame Week 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title N-acetylcysteine Placebo
Hide Arm/Group Description:

Target dose for n-acetylcysteine is 60 mg/kg/day. Capsules available in 300 mg and 600 mg strengths.

N-acetylcysteine: Capsules available in 300 mg or 600mg strength. Target dose of n-acetylcysteine will be 60mg/kg/day TID. Dosage will be increased to this target dose from week 1 to week 3 barring side effects. Dose reduction will be allowed at any time for adverse side effects. Maximum dose of n-acetylcysteine will be 4200mg/day.

Subjects randomized to placebo arm will receive capsules identical in size and appearance to those subjects receiving study drug. Placebo capsules contain inactive ingredients.

Placebo: Subjects randomized to placebo arm will receive placebo pill for duration of study.

Overall Number of Participants Analyzed 13 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
Communication 88.5  (32.5) 95.0  (30.3)
Daily Living Skills 99.0  (37.1) 98.6  (33.4)
Socialization 71.7  (18.7) 75.9  (21.0)
Adaptive Behavior Composite 62.9  (17.1) 60.5  (18.8)
Time Frame Twelve Weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title N-acetylcysteine Placebo
Hide Arm/Group Description

Target dose for n-acetylcysteine is 60 mg/kg/day. Capsules available in 300 mg and 600 mg strengths.

N-acetylcysteine: Capsules available in 300 mg or 600mg strength. Target dose of n-acetylcysteine will be 60mg/kg/day TID. Dosage will be increased to this target dose from week 1 to week 3 barring side effects. Dose reduction will be allowed at any time for adverse side effects. Maximum dose of n-acetylcysteine will be 4200mg/day.

Subjects randomized to placebo arm will receive capsules identical in size and appearance to those subjects receiving study drug. Placebo capsules contain in active ingredients.

Placebo: Subjects randomized to placebo arm will receive placebo pill for duration of study.

All-Cause Mortality
N-acetylcysteine Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
N-acetylcysteine Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/16 (0.00%)   0/15 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
N-acetylcysteine Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   16/16 (100.00%)   15/15 (100.00%) 
Ear and labyrinth disorders     
Ear Infection *  2/16 (12.50%)  1/15 (6.67%) 
Endocrine disorders     
Hypoglycemia *  1/16 (6.25%)  0/15 (0.00%) 
Swollen neck lymph node of unknown origin *  1/16 (6.25%)  0/15 (0.00%) 
Gastrointestinal disorders     
Constipation *  0/16 (0.00%)  1/15 (6.67%) 
Diarrhea *  0/16 (0.00%)  1/15 (6.67%) 
Encopresis *  0/16 (0.00%)  1/15 (6.67%) 
Enuresis *  1/16 (6.25%)  1/15 (6.67%) 
Intermittent Dyspepsia *  1/16 (6.25%)  0/15 (0.00%) 
Nausea *  2/16 (12.50%)  3/15 (20.00%) 
General disorders     
Appetite Increase *  1/16 (6.25%)  0/15 (0.00%) 
Dry Mouth *  0/16 (0.00%)  1/15 (6.67%) 
Fever *  2/16 (12.50%)  0/15 (0.00%) 
Weight Gain *  0/16 (0.00%)  1/15 (6.67%) 
Hepatobiliary disorders     
Elevated liver enzymes *  1/16 (6.25%)  0/15 (0.00%) 
Infections and infestations     
Influenza *  1/16 (6.25%)  0/15 (0.00%) 
Musculoskeletal and connective tissue disorders     
Accidental arm injury *  0/16 (0.00%)  1/15 (6.67%) 
Nervous system disorders     
Hand Movements *  0/16 (0.00%)  2/15 (13.33%) 
Headache *  3/16 (18.75%)  3/15 (20.00%) 
Stuttering *  1/16 (6.25%)  0/15 (0.00%) 
Psychiatric disorders     
Aggression   3/16 (18.75%)  2/15 (13.33%) 
Behavior Worse *  1/16 (6.25%)  2/15 (13.33%) 
Difficulty Falling Asleep *  4/16 (25.00%)  1/15 (6.67%) 
Skin Picking/SIB *  0/16 (0.00%)  1/15 (6.67%) 
Threats to hurt self *  0/16 (0.00%)  1/15 (6.67%) 
Renal and urinary disorders     
Urinary Tract Infection *  0/16 (0.00%)  1/15 (6.67%) 
Respiratory, thoracic and mediastinal disorders     
Cold *  5/16 (31.25%)  5/15 (33.33%) 
Cough *  2/16 (12.50%)  0/15 (0.00%) 
Nasal Congestion *  1/16 (6.25%)  0/15 (0.00%) 
Skin and subcutaneous tissue disorders     
Hives *  0/16 (0.00%)  1/15 (6.67%) 
Itchy Skin *  0/16 (0.00%)  1/15 (6.67%) 
Poison Ivy *  0/16 (0.00%)  1/15 (6.67%) 
Localized Rash *  1/16 (6.25%)  0/15 (0.00%) 
Scabies *  0/16 (0.00%)  1/15 (6.67%) 
Surgical and medical procedures     
Nasal Cauterization *  1/16 (6.25%)  0/15 (0.00%) 
Vascular disorders     
Increased Swelling of Feet *  1/16 (6.25%)  0/15 (0.00%) 
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Craig Erickson
Organization: Cincinnati Childrens Hospital
Phone: 513-636-6258
Responsible Party: Indiana University ( Indiana University School of Medicine )
ClinicalTrials.gov Identifier: NCT00453180     History of Changes
Other Study ID Numbers: 0611-10
First Submitted: March 27, 2007
First Posted: March 28, 2007
Results First Submitted: March 29, 2017
Results First Posted: June 14, 2017
Last Update Posted: June 14, 2017