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Growth Factor
Insulin-Like Growth Factor-1 (IGF-1) for Phelan-McDermid Syndrome
Phase 2
Waitlist Available
Led By Alexander Kolevzon, MD
Research Sponsored by Icahn School of Medicine at Mount Sinai
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline and week 12
Awards & highlights
Summary
The purpose of this study is to pilot the use of Insulin-Like Growth Factor-1 (IGF-1) treatment in 22q13 Deletion Syndrome (Phelan-McDermid Syndrome) caused by SHANK3 gene deficiency in order to evaluate safety, tolerability, and efficacy. IGF-1 is an injection under the skin that contains human IGF-1. IGF-1 is approved by the FDA under the brand name Increlex for the treatment of children with short stature due to primary IGF-1 deficiency. It is being used off-label in the current study and is not FDA approved, nor has it yet been studied in humans for the treatment of SHANK3 deficiency.
Eligible Conditions
- Deletion Syndrome
- Phelan-McDermid Syndrome
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline and week 12
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline and week 12
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Change in Aberrant Behavior Checklist - Social Withdrawal (ABC-SW) Subscale - Study 1
Change in Aberrant Behavior Checklist - Social Withdrawal (ABC-SW) Subscale Study 2
Secondary study objectives
Change in CGI-Improvement and Severity Scales; - Study 2
Change in Caregiver Strain Questionnaire
Change in Repetitive Behavior Scale - Study 2
+2 moreSide effects data
From 2016 Phase 2 trial • 19 Patients • NCT0152590158%
Sleep Disturbance
58%
Hypoglycemia
47%
URTI
47%
Mood changes/irritability
47%
Increased appetite
37%
Constipation
37%
Runny nose/congestion
37%
Fever
32%
Lethargy/decreased energy
21%
Decreased appetite
21%
Vomiting
16%
Gait changes/fell
16%
Rash
16%
Stomach virus
16%
Cough
11%
Increased energy
11%
Gagging
11%
Warmer body temperature
11%
Increased chewing/biting
11%
Increased urine frequency
11%
Facial swelling
11%
Diarrhea
5%
Increased aggression
5%
Erythema/swollen eyes
5%
Increased thirst
5%
Increased phlegm
5%
Teeth grinding
5%
Hand flapping
5%
Decreased visual acuity
5%
Conjunctivitis
5%
Breast buds developed
5%
Redness around perineum
5%
Seizure
5%
Increased reflux
5%
Increased bowel movements
5%
Hair loss
5%
Bruising at injection site
5%
Periobital/facial swelling
5%
Bloody nose
5%
Sedation
5%
Cooler body temperature/sweating
5%
Ear Infections
5%
Increased drooling
5%
Increased eye rolling
100%
80%
60%
40%
20%
0%
Study treatment Arm
Insulin-Like Growth Factor-1 (IGF-1)
Normal Saline
Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: Insulin-Like Growth Factor-1 (IGF-1)Experimental Treatment1 Intervention
Injection
Group II: Normal salinePlacebo Group1 Intervention
Injection
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Insulin-Like Growth Factor-1 (IGF-1)
2012
Completed Phase 2
~20
Find a Location
Who is running the clinical trial?
Icahn School of Medicine at Mount SinaiLead Sponsor
886 Previous Clinical Trials
535,024 Total Patients Enrolled
National Institute of Mental Health (NIMH)NIH
2,869 Previous Clinical Trials
2,777,517 Total Patients Enrolled
Alexander Kolevzon, MD4.34 ReviewsPrincipal Investigator - Icahn School of Medicine at Mount Sinai
Icahn School of Medicine at Mount Sinai
13 Previous Clinical Trials
488 Total Patients Enrolled
5Patient Review
Dr. Alexander Kolevzon is a rockstar. He headed a year long study for my son's chromosome deletion, as well as an annual study we have participated in. He is always helpful and kind. He has been there for us when we needed him.
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