ASD, Autism Spectrum Disorders, PDD, Pervasive Developmental Disorders
Conditions
Keywords
Autism Spectrum Disorders, Pervasive Developmental Disorders, ASD, PDD, Oxytocin, Syntocinon, treatment
Brief summary
This study is an 8-week open-label trial testing oxytocin nasal spray (Syntocinon) as a treatment for social impairment in adolescents with autism spectrum disorders (ASD). We hypothesize that oxytocin nasal spray will be safe, tolerable, and effective in improving the core symptoms of autism spectrum disorders in adolescents ages 11-17.
Interventions
DRUGIntranasal Oxytocin
Sponsors
Massachusetts General Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
11 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
1. Male and female outpatients ages 11-17 years 2. DSM-IV-TR PDD diagnosis of autistic disorder, Asperger's disorder, or PDD-NOS as established by clinical diagnostic interview and with the aid of the MGH ASD Symptom Checklist (MGH-ASD-SCL). 3. At least moderate severity of ASD impairment as measured by a raw score of ≥80 on the SRS and a severity score of ≥4 on CGI-PDD. 4. Participants and their parent/guardian must be able to speak and understand English sufficiently to comprehend the nature of the study and to allow for the completion of all study procedures required per protocol. 5. Subjects and their parent/guardian must be considered reliable reporters. 6. Each subject and their parent/guardian must understand the nature of the study and provide written informed assent/consent. 7. Subjects must be able to participate in mandatory blood draws. 8. Subjects with mood, anxiety, or disruptive behavior disorders will be allowed to participate in the study provided they do not meet any exclusionary criteria.
Exclusion criteria
1. IQ \<85 2. Total lack of spoken language 3. DSM-IV-TR PDD diagnosis of Rett's disorder or childhood disintegrative disorder. 4. Clinically unstable psychiatric conditions or judged to be at serious suicidal risk as determined by evaluating investigator. 5. History of substance use (except nicotine or caffeine) within past 3 months 6. Serious, stable or unstable systemic illness including hepatic, renal, gastroenterological, respiratory, cardiovascular (including ischemic heart disease), endocrinologic, neurologic, immunologic, or hematologic disease. 7. Subjects with severe hepatic impairment (LFTs \> 3 times ULN) and those with severely impaired renal function (eGFR \< 30). 8. Pregnant or nursing females. 9. Known hypersensitivity to oxytocin. 10. Severe allergies or multiple adverse drug reactions. 11. A non-responder or history of intolerance to oxytocin, after treatment at adequate doses as determined by the clinician. 12. Subjects with significant nasal pathology (including atrophic rhinitis, recurrent nose bleeds, and history of hypophysectomy). 13. Investigator and his/her immediate family defined as the investigator's spouse, parent, child, grandparent, or grandchild. 14. Currently enrolled or recently participated (within the past 6 months) in a clinical trial of intranasal oxytocin.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in ASD Symptoms Social Responsiveness Scales 2 (SRS-2) Scales From Baseline to Week 8 | Week-8 | Change in ASD symptoms as measured by change from baseline on the Social Responsiveness Scale 2 (SRS-2) scale. The SRS-2 is a 65-item rating scale completed that is used to measure the severity of autism spectrum symptoms as they occur in natural settings. The SRS-2 School Age form is completed by a parent or guardian for patients ages 9-17 and the SRS-2 Adult Self-report is completed by patients ages 18-59. Total raw scores range from 0 to 195, with higher scores indicating increased symptom severity. |
| Number of Participants With ≥30% Reduction in SRS Raw Score and CGI Improvement Scores of ≤ 2 at Week 8 | Week-8 | The SRS-2 is a 65-item rating scale completed that is used to measure the severity of autism spectrum symptoms as they occur in natural settings. The SRS-2 School Age form is completed by a parent or guardian for patients ages 9-17 and the SRS-2 Adult Self-report is completed by patients ages 18-59. Total raw scores range from 0 to 195, with higher scores indicating increased symptom severity. The Clinical Global Impression Improvement scale (CGI-ASD-I) is a clinician rated measure of ASD improvement. Improvement scores range from 1 (very much improved) to 7 (very much worse). |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Participants Ages 11-17 Participants ages 11-17 enrolled to receive oxytocin treatment | 8 |
| Total | 8 |
Baseline characteristics
| Characteristic | Participants Ages 11-17 |
|---|---|
| Age, Categorical <=18 years | 8 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 6 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) White | 6 Participants |
| Sex: Female, Male Female | 5 Participants |
| Sex: Female, Male Male | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 5 / 8 |
| serious Total, serious adverse events | 0 / 8 |
Outcome results
Primary
Change in ASD Symptoms Social Responsiveness Scales 2 (SRS-2) Scales From Baseline to Week 8
Change in ASD symptoms as measured by change from baseline on the Social Responsiveness Scale 2 (SRS-2) scale. The SRS-2 is a 65-item rating scale completed that is used to measure the severity of autism spectrum symptoms as they occur in natural settings. The SRS-2 School Age form is completed by a parent or guardian for patients ages 9-17 and the SRS-2 Adult Self-report is completed by patients ages 18-59. Total raw scores range from 0 to 195, with higher scores indicating increased symptom severity.
Time frame: Week-8
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Participants Ages 11-17 | Change in ASD Symptoms Social Responsiveness Scales 2 (SRS-2) Scales From Baseline to Week 8 | -32.4 scores on a scale | Standard Deviation 17.25688 |
Primary
Number of Participants With ≥30% Reduction in SRS Raw Score and CGI Improvement Scores of ≤ 2 at Week 8
The SRS-2 is a 65-item rating scale completed that is used to measure the severity of autism spectrum symptoms as they occur in natural settings. The SRS-2 School Age form is completed by a parent or guardian for patients ages 9-17 and the SRS-2 Adult Self-report is completed by patients ages 18-59. Total raw scores range from 0 to 195, with higher scores indicating increased symptom severity. The Clinical Global Impression Improvement scale (CGI-ASD-I) is a clinician rated measure of ASD improvement. Improvement scores range from 1 (very much improved) to 7 (very much worse).
Time frame: Week-8
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Participants Ages 11-17 | Number of Participants With ≥30% Reduction in SRS Raw Score and CGI Improvement Scores of ≤ 2 at Week 8 | 2 Participants |