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

NCT01970345 | Terminated Phase 2 Results DMC

• 1 other identifier: GCO 13-1206
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

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.

Conditions

Autism Spectrum Disorder

Keywords

Autism Spectrum Disorder; IGF-1

Outcome Measures

Primary Outcomes (1)

• Aberrant Behavior Checklist - Social Withdrawal Subscale

  Aberrant Behavior Checklist - Social Withdrawal Subscale has16 items, Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). Total Subscale scores range from 0 to 48. Higher score indicates poorer health outcomes.

  Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8

Secondary Outcomes (9)

• Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)

  Week 4 of Phase 1

• Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)

  Week 12 of Phase 1

• Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)

  Week 4 of Phase 2

• Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)

  Week 8 of Phase 2

• Repetitive Behavior

  Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8

Study Arms (2)

IGF-1

EXPERIMENTAL

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

Placebo

PLACEBO COMPARATOR

Placebo

Drug: Placebo/saline

Interventions

IGF-1 DRUG

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.

Also known as: Increlex, mecasermin

IGF-1

Placebo/saline DRUG

Placebo

Also known as: Saline

Placebo

Eligibility Criteria

• Age: 5 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Icahn School of Medicine at Mount Sinai: lead
• Autism Science Foundation: collaborator

Study Sites (1)

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

Related Publications (2)

• Long J, Liao X, Han K, Niu M, Chen J, Wang X, Liu J, Zhang Y, Zhang H. Association of Thyroid Hormone and Insulin-Like Growth Factor-1 Levels With Autism Spectrum Disorders: A Systematic Review and Meta-Analysis. Autism Res. 2025 Jul;18(7):1497-1512. doi: 10.1002/aur.70052. Epub 2025 May 12.

  PMID: 40351246 DERIVED

• Iffland M, Livingstone N, Jorgensen M, Hazell P, Gillies D. Pharmacological intervention for irritability, aggression, and self-injury in autism spectrum disorder (ASD). Cochrane Database Syst Rev. 2023 Oct 9;10(10):CD011769. doi: 10.1002/14651858.CD011769.pub2.

  PMID: 37811711 DERIVED

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

Insulin-Like Growth Factor I; mecasermin; Sodium Chloride

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Somatomedins; Insulin-Like Peptides; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Blood Proteins; Proteins; Biological Factors; Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Results Point of Contact

• Title: Dr. Alexander Kolevzon
• Organization: Icahn School of Medicine at Mount Sinai

alexander.kolevzon@mssm.edu

(212) 659-8762

Study Officials

• Alexander Kolevzon, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Director, Seaver Autism Center for Research and Treatment

Study Record Dates

• First Submitted: October 22, 2013
• First Posted: October 28, 2013
• Study Start: May 1, 2014
• Primary Completion: September 13, 2017
• Study Completion: September 13, 2017
• Last Updated: February 8, 2024
• Results First Posted: February 8, 2024

Record last verified: 2024-01

Locations

US (1)