A Study of Oxytocin in Children and Adolescents With Autistic Disorder

NCT01308749 | Completed Phase 2 Results

• 1 other identifier: 11-0493
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

Brief Summary

The investigators propose to conduct this pilot study to evaluate oxytocin as a supplemental treatment for improving social difficulties in individuals with autism.

Conditions

Autism

Outcome Measures

Primary Outcomes (2)

• Number of Participants Who Could Tolerate Twice Daily Oxytocin

  This study will help to determine tolerability of intranasal oxytocin treatment in children with autism by measuring the ability of at least 80% of the sample to tolerate twice daily intranasal administration of oxytocin.

  Week 0 to week 8

• Number of Participants Who Could Tolerate Twice Daily Oxytocin

  This study will help to determine tolerability of intranasal oxytocin treatment in children with autism by measuring the ability of at least 80% of the sample to tolerate twice daily intranasal administration of oxytocin.

  Week 0 to week 16

Secondary Outcomes (9)

• Change in Mean Plasma Oxytocin Level During Period 1 - Double Blind Phase

  Week 0 to week 8

• Change in Mean Weight

  between weeks 0 and 8

• Change in Mean Total Social Social Responsiveness Scale (SRS) T-score

  0-8 weeks, blinded treatment, period 1

• Change in Mean Autism Diagnostic Observation Schedule (ADOS) Total Score

  Baseline to 16 Weeks

• Change in Mean Aberrant Behavior Checklist (ABC)-Social Withdrawal Subscale Score Over Both Periods

  Baseline to 16 Weeks

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Intervention: Drug: placebo

Drug: Placebo

Oxytocin

ACTIVE COMPARATOR

Intervention: Drug: Syntocinon® Nasal Spray

Drug: Oxytocin

Interventions

Oxytocin DRUG

Subjects will use the Syntocinon® Nasal Spray (oxytocin) twice daily for 8 weeks if they are randomized to that arm in the Randomized Phase. All subjects will use the Syntocinon® Nasal Spray twice daily for 8 weeks in the Open Label Phase. Subjects ages 3-10 years old will be titrated up to a maximum dose of 24 International units (IU). Subjects ages 11-17 years old will be titrated up to a maximum dose of 32IU.

Also known as: Syntocinon® Nasal Spray

Oxytocin

Placebo DRUG

Placebo Nasal Spray

Placebo

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Between 3 and 17 years old, inclusive.
• Have a clinical diagnosis of autistic disorder confirmed according to Diagnostic Statistical Manual of Mental Disorders-IV criteria by using the Autism Diagnostic Interview - Revised (ADI-R) and/or the Autism Diagnostic Observation Scale (ADOS, Lord et al., 1989).

You may not qualify if:

• Changes in allied health therapies, behavioral or educational interventions within the past 2 months of the baseline visit other than those associated with school holidays.
• Changes in psychotropic and alternative medication doses in the last 30 days of the baseline visit.
• Subjects with a medical condition that might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being. This includes, but is not limited to, Rett Syndrome, impairment of renal function, evidence or history of malignancy or any significant hematological, endocrine, cardiovascular (including any rhythm disorder and uncontrolled hypertension), respiratory, hepatic, or gastrointestinal disease.
• Marked sensory impairment such as deafness or blindness that would interfere with conduct of the study.
• Refusal to practice contraception if sexually active because the effects of exposure to high concentrations of oxytocin on sperm or newly conceived embryos are unknown. Sexually active men and women should not take part in this study if they and their partners are not both using an effective birth control method (for example, women use birth control pills, an intrauterine device (IUD) or a diaphragm and men use condoms).
• Inability of caretakers to speak English.
• Absence of a consistent caretaker to report on symptoms.
• Subjects who, in the Investigator's opinion, might not be suitable for the study.

Sponsors & Collaborators

• University of North Carolina, Chapel Hill: lead
• Autism Speaks: collaborator

Study Sites (1)

University of North Carolina Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

MeSH Terms

Conditions

Autistic Disorder

Interventions

Oxytocin

Condition Hierarchy (Ancestors)

Autism Spectrum Disorder; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Posterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Limitations and Caveats

This was a very small study with a very heterogeneous sample. Further the two groups included very different proportions of intellectually disabled non-verbal participants which may influence assessment of benefit.

Results Point of Contact

• Title: Linmarie Sikich, MD
• Organization: Duke University Center for Autism and Brain Development

linmarie.sikich@duke.edu

9196810026

Study Officials

• Linmarie Sikich, M.D.

  University of North Carolina, Chapel Hill

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: February 28, 2011
• First Posted: March 4, 2011
• Study Start: March 1, 2011
• Primary Completion: April 1, 2013
• Study Completion: April 1, 2013
• Last Updated: July 17, 2017
• Results First Posted: July 17, 2017

Record last verified: 2017-06

Locations

US (1)