Status:
COMPLETED
Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)
Lead Sponsor:
Icahn School of Medicine at Mount Sinai
Collaborating Sponsors:
National Institute of Mental Health (NIMH)
Conditions:
22q13 Deletion Syndrome
Phelan-McDermid Syndrome
Eligibility:
All Genders
5-12 years
Phase:
PHASE2
Brief 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 evalu...
Detailed Description
Overall, there will be 1-3 screening visits, a baseline visit where study drug will first be administered, and then 10 follow-up visits. Follow-up visits will occur at week 2, week 4, week 8, and week...
Eligibility Criteria
Inclusion
- 5 to 12 years old
- pathogenic deletions or mutations of the SHANK3 gene
- stable medication regimens for at least three months prior to enrollment
Exclusion
- closed epiphyses
- active or suspected neoplasia
- intracranial hypertension
- hepatic insufficiency
- renal insufficiency
- cardiomegaly / valvulopathy
- history of allergy to IGF-1 or any component of the formulation (mecasermin)
- history of extreme prematurity (\<1000 grams) with associated early neo-natal complications, e.g. intra-cerebral hemorrhage, prolonged hypoxia, prolonged hypoglycemia
- patients with comorbid conditions deemed too medically compromised to tolerate the risk of experimental treatment with IGF-1
Key Trial Info
Start Date :
February 1 2012
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 23 2016
Estimated Enrollment :
19 Patients enrolled
Trial Details
Trial ID
NCT01525901
Start Date
February 1 2012
End Date
August 23 2016
Last Update
May 12 2022
Active Locations (1)
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1
Seaver Austin Center, Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10029