Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT01525901 · ClinicalTrials.gov registry record · Phase 2
Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)
A Phase 2 study, sponsored by Icahn School of Medicine at Mount Sinai.
- Completed
- Registry status
- Phase 2
- Development phase
- 19
- Enrollment target
NCT01525901: Completed Phase 2 study, sponsored by Icahn School of Medicine at Mount Sinai.
NCT01525901 is a Phase 2 study that has completed, run by Icahn School of Medicine at Mount Sinai. The registered enrollment target is 19 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (86% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01525901, a Phase 2 study, has completed, sponsored by Icahn School of Medicine at Mount Sinai.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 19 participants
- Enrollment target
Study 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.
Primary Outcome
16 items on ABC-SW subscale, Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). Total score from 0 to 48 with higher score indicating poorer health outcomes.
Interventions
- DRUG Normal saline
- DRUG Insulin-Like Growth Factor-1 (IGF-1)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 19 participants |
| Start Date | 2012-02 |
| Est. Completion | 2016-08-23 |
| Phase | Phase 2 |
What the finished NCT01525901 record still lists
NCT01525901 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 19 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (86% lower).
The record links to 0 conditions, and to 2 interventions - of which Normal saline is the first listed.
NCT01525901 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01525901 about?
NCT01525901 is a clinical study titled "Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)". 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...
What is the current status of trial NCT01525901?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 19 participants. The study started on 2012-02. Estimated completion is 2016-08-23.
What interventions are being tested in trial NCT01525901?
The interventions under investigation include: Normal saline (DRUG), Insulin-Like Growth Factor-1 (IGF-1) (DRUG).
Who is sponsoring clinical trial NCT01525901?
This trial is sponsored by Icahn School of Medicine at Mount Sinai, which has 708 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01525901's enrollment target sits among peer trials
19 1894th of 2000 higher than 103 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT03207464 · 19 participants · Phase 1
Molecular Imaging of NET Using [C-11]MRB-PET in MS
- NCT04318717 · 19 participants · Phase 2
Pembrolizumab and Hypofractionated Radiation Therapy for the Treatment of Mucosal Melanoma
- NCT05103904 · 19 participants · Phase 2
Lenvatinib for the Treatment of Recurrent Hepatocellular Carcinoma After Liver Transplant
- NCT05607407 · 19 participants · Phase 2
Methimazole in Patients With Progressive Glioblastoma
Similar registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia