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

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ClinicalTrials.gov Identifier: NCT01970345
Recruitment Status : Recruiting
First Posted : October 28, 2013
Last Update Posted : January 17, 2018
Sponsor:
Collaborator:
Autism Science Foundation
Information provided by (Responsible Party):
Alexander Kolevzon, Icahn School of Medicine at Mount Sinai

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 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 : January 2020
Estimated Study Completion Date : January 2020


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



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).



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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

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
Contact: Jordana Norry 212-241-3072 jordana.norry@mssm.edu

Locations
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
Principal Investigator: Alexander Kolevzon, MD Icahn School of Medicine at Mount Sinai

Responsible Party: Alexander Kolevzon, Clinical Director, Seaver Autism Center for Research and Treatment, Icahn School of Medicine at Mount Sinai
ClinicalTrials.gov Identifier: NCT01970345     History of Changes
Other Study ID Numbers: GCO 13-1206
First Posted: October 28, 2013    Key Record Dates
Last Update Posted: January 17, 2018
Last Verified: January 2018

Keywords provided by Alexander Kolevzon, Icahn School of Medicine at Mount Sinai:
Autism Spectrum Disorder
IGF-1

Additional relevant MeSH terms:
Disease
Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Pathologic Processes
Neurodevelopmental Disorders
Mental Disorders
Mitogens
Mecasermin
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action
Growth Substances
Physiological Effects of Drugs