Don't get left behind! The modernized ClinicalTrials.gov is coming. Check it out now.
Say goodbye to ClinicalTrials.gov!
The new site is coming soon - go to the modernized ClinicalTrials.gov
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01970345
Recruitment Status : Recruiting
First Posted : October 28, 2013
Last Update Posted : July 5, 2022
Sponsor:
Collaborator:
Autism Science Foundation
Information provided by (Responsible Party):
Alexander Kolevzon, Icahn School of Medicine at Mount Sinai

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
The proposed project will pilot the use of IGF-1 as a novel treatment for core symptoms of autism. We will use a double-blind, placebo-controlled crossover trial design in five children with autism to evaluate the impact of IGF-1 treatment on autism-specific impairments in socialization, language, and repetitive behaviors. We expect to provide evidence for the safety and feasibility of IGF-1 in ameliorating social withdrawal in children with Autistic Disorder. Further, we expect to demonstrate that IGF-1 is associated with improvement on secondary outcomes of social impairment, language delay, and repetitive behavior, as well as on functional outcomes of global severity.

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder Drug: IGF-1 Drug: Placebo/saline Phase 2

Detailed Description:
IGF-1 is an FDA approved, commercially available compound that crosses the blood-brain barrier and has beneficial effects on synaptic development by promoting neuronal cell survival, synaptic maturation, and synaptic plasticity. IGF-1 is effective in reversing mouse and neuronal models of Rett syndrome and Phelan McDermid syndrome, both single gene causes of ASD and may therefore be effective in treating autism spectrum disorders (ASD) more broadly.
Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder
Study Start Date : May 2014
Estimated Primary Completion Date : July 2024
Estimated Study Completion Date : July 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
Experimental: IGF-1

Randomized, placebo-controlled, crossover format with 12 weeks in each treatment arm (IGF-1 and placebo), separated by a four-week wash-out phase.

Dose titration will be initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and preprandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well tolerated dose is established.

 Drug: IGF-1
IGF-1 is an FDA-approved (IND exemption #113450), commercially available compound that crosses the blood-brain barrier and has beneficial effects on synaptic development by promoting neuronal cell survival, synaptic maturation, and synaptic plasticity.
Other Names:
  • Increlex
  • mecasermin
Placebo Comparator: Placebo
Placebo
 Drug: Placebo/saline
Placebo
Other Name: Saline


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Aberrant Behavior Checklist - Social Withdrawal Subscale [ Time Frame: at 12 weeks ]
    Aberrant Behavior Checklist - Social Withdrawal Subscale


Secondary Outcome Measures :
  1. Social [ Time Frame: at 12 weeks ]
    Social: Pervasive Developmental Disorder-Behavior Inventory (PDD-BI; Cohen 2003).

  2. Language [ Time Frame: at 12 weeks ]
    Language: Vineland Adaptive Behavior Communication Domain (Sparrow, 1984).

  3. Repetitive Behavior [ Time Frame: at 12 weeks ]
    Repetitive Behavior: Repetitive Behavior Scale (RBS; Bodfish et al., 1999); Children's Yale-Brown Obsessive Compulsive Disorder Scale for Pervasive Developmental Disorders (CY-BOCS-PDD; Scahill et al., 2006

  4. Overall Functional Outcomes [ Time Frame: at 12 weeks ]
    Overall Functional Outcomes: CGI-Improvement and Severity Scales (Guy, 1976); Vineland Adaptive Behavior Composite

  5. Caregiver Strain [ Time Frame: at 12 weeks ]
    Caregiver Strain Index (CSI; Brannan et al., 1997).


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   5 Years to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Meet DSM-5 criteria for Autism Spectrum Disorder confirmed by the Autism Diagnostic Interview-Revised (ADI-R) and the Autism Diagnostic Observation Schedule- Generic (ADOS-G)
  • Children between the ages of 5-12 years of age
  • Language delay (lack of fluent phrase speech) reflected by use of ADOS Module 1 or 2
  • Must be on stable medication regimens for at least three months prior to enrollment, assuming the concomitant medication is safe for use with IGF-1

Exclusion Criteria:

  • Closed epiphyses
  • Active or suspected neoplasia
  • Intracranial hypertension
  • Hepatic insufficiency
  • Renal insufficiency
  • Cardiomegaly/valvulopathy
  • History of allergy to IGF-1
  • Patients with comorbid conditions deemed too medically compromised to tolerate the risk of experimental treatment with IGF-1
Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01970345


Contacts
Layout table for location contacts
Contact: Lexie Massa 212-241-3692 alexandra.massa@mssm.edu

Locations
Layout table for location information
United States, New York
Icahn School of Medicine at Mount Sinai Recruiting
New York, New York, United States, 10029
Principal Investigator: Alexander Kolevzon, MD         
Sponsors and Collaborators
Icahn School of Medicine at Mount Sinai
Autism Science Foundation
Investigators
Layout table for investigator information
Principal Investigator: Alexander Kolevzon, MD Icahn School of Medicine at Mount Sinai
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
Responsible Party: Alexander Kolevzon, Clinical Director, Seaver Autism Center for Research and Treatment, Icahn School of Medicine at Mount Sinai
ClinicalTrials.gov Identifier: NCT01970345    
Other Study ID Numbers: GCO 13-1206
First Posted: October 28, 2013    Key Record Dates
Last Update Posted: July 5, 2022
Last Verified: June 2022
Keywords provided by Alexander Kolevzon, Icahn School of Medicine at Mount Sinai:
Autism Spectrum Disorder
IGF-1
Additional relevant MeSH terms:
Layout table for MeSH terms
Disease
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Pathologic Processes
 Neurodevelopmental Disorders
Mental Disorders
Mecasermin
Growth Substances
Physiological Effects of Drugs