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

Primary Purpose

Autism Spectrum Disorder

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
IGF-1
Placebo/saline
Sponsored by
Icahn School of Medicine at Mount Sinai
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Autism Spectrum Disorder focused on measuring Autism Spectrum Disorder, IGF-1

Eligibility Criteria

5 Years - 12 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Icahn School of Medicine at Mount SinaiRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

IGF-1

Placebo

Arm Description

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.

Placebo

Outcomes

Primary Outcome Measures

Aberrant Behavior Checklist - Social Withdrawal Subscale
Aberrant Behavior Checklist - Social Withdrawal Subscale

Secondary Outcome Measures

Social
Social: Pervasive Developmental Disorder-Behavior Inventory (PDD-BI; Cohen 2003).
Language
Language: Vineland Adaptive Behavior Communication Domain (Sparrow, 1984).
Repetitive Behavior
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
Overall Functional Outcomes
Overall Functional Outcomes: CGI-Improvement and Severity Scales (Guy, 1976); Vineland Adaptive Behavior Composite
Caregiver Strain
Caregiver Strain Index (CSI; Brannan et al., 1997).

Full Information

First Posted
October 22, 2013
Last Updated
June 30, 2022
Sponsor
Icahn School of Medicine at Mount Sinai
Collaborators
Autism Science Foundation
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1. Study Identification

Unique Protocol Identification Number
NCT01970345
Brief Title
A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder
Official Title
A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder
Study Type
Interventional

2. Study Status

Record Verification Date
June 2022
Overall Recruitment Status
Recruiting
Study Start Date
May 2014 (undefined)
Primary Completion Date
July 2024 (Anticipated)
Study Completion Date
July 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Icahn School of Medicine at Mount Sinai
Collaborators
Autism Science Foundation

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autism Spectrum Disorder
Keywords
Autism Spectrum Disorder, IGF-1

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
IGF-1
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo
Intervention Type
Drug
Intervention Name(s)
IGF-1
Other Intervention Name(s)
Increlex, mecasermin
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Placebo/saline
Other Intervention Name(s)
Saline
Intervention Description
Placebo
Primary Outcome Measure Information:
Title
Aberrant Behavior Checklist - Social Withdrawal Subscale
Description
Aberrant Behavior Checklist - Social Withdrawal Subscale
Time Frame
at 12 weeks
Secondary Outcome Measure Information:
Title
Social
Description
Social: Pervasive Developmental Disorder-Behavior Inventory (PDD-BI; Cohen 2003).
Time Frame
at 12 weeks
Title
Language
Description
Language: Vineland Adaptive Behavior Communication Domain (Sparrow, 1984).
Time Frame
at 12 weeks
Title
Repetitive Behavior
Description
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
Time Frame
at 12 weeks
Title
Overall Functional Outcomes
Description
Overall Functional Outcomes: CGI-Improvement and Severity Scales (Guy, 1976); Vineland Adaptive Behavior Composite
Time Frame
at 12 weeks
Title
Caregiver Strain
Description
Caregiver Strain Index (CSI; Brannan et al., 1997).
Time Frame
at 12 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Lexie Massa
Phone
212-241-3692
Email
alexandra.massa@mssm.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Alexander Kolevzon, MD
Organizational Affiliation
Icahn School of Medicine at Mount Sinai
Official's Role
Principal Investigator
Facility Information:
Facility Name
Icahn School of Medicine at Mount Sinai
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Alexander Kolevzon, MD

12. IPD Sharing Statement

Learn more about this trial

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

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