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 : July 5, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Autism Spectrum Disorder||Drug: IGF-1 Drug: Placebo/saline||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
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.
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.
Placebo Comparator: Placebo
Other Name: Saline
- Aberrant Behavior Checklist - Social Withdrawal Subscale [ Time Frame: at 12 weeks ]Aberrant Behavior Checklist - Social Withdrawal Subscale
- Social [ Time Frame: at 12 weeks ]Social: Pervasive Developmental Disorder-Behavior Inventory (PDD-BI; Cohen 2003).
- Language [ Time Frame: at 12 weeks ]Language: Vineland Adaptive Behavior Communication Domain (Sparrow, 1984).
- 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
- Overall Functional Outcomes [ Time Frame: at 12 weeks ]Overall Functional Outcomes: CGI-Improvement and Severity Scales (Guy, 1976); Vineland Adaptive Behavior Composite
- Caregiver Strain [ Time Frame: at 12 weeks ]Caregiver Strain Index (CSI; Brannan et al., 1997).
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.
|Ages Eligible for Study:||5 Years to 12 Years (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- 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
- Closed epiphyses
- Active or suspected neoplasia
- Intracranial hypertension
- Hepatic insufficiency
- Renal insufficiency
- History of allergy to IGF-1
- Patients with comorbid conditions deemed too medically compromised to tolerate the risk of experimental treatment with IGF-1
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
|Contact: Lexie Massafirstname.lastname@example.org|
|United States, New York|
|Icahn School of Medicine at Mount Sinai||Recruiting|
|New York, New York, United States, 10029|
|Principal Investigator: Alexander Kolevzon, MD|
|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|
|Other Study ID Numbers:||
|First Posted:||October 28, 2013 Key Record Dates|
|Last Update Posted:||July 5, 2022|
|Last Verified:||June 2022|
Autism Spectrum Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Physiological Effects of Drugs