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NCT01253317 · ClinicalTrials.gov registry record · Phase 1
Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)
A Phase 1 study of Rett Syndrome, sponsored by Boston Children's Hospital.
- Completed
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 1
- Study location
NCT01253317: Completed Phase 1 study of Rett Syndrome, sponsored by Boston Children's Hospital.
NCT01253317 is a Phase 1 study of Rett Syndrome that has completed, run by Boston Children's Hospital. The registered enrollment target is 12 participants, below the 525-participant average among 18 other Rett Syndrome trials with a reported enrollment target (98% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01253317, a Phase 1 study of Rett Syndrome, has completed, sponsored by Boston Children's Hospital.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 1
- Study location
Study Summary
The investigators are recruiting children for a research study using a medication known as IGF-1 (mecasermin or INCRELEX) to see if it improves the health of children with Rett syndrome (RTT). To participate in the study your child must be female, between the ages of 2 to 12 and have a genetic diagnosis (MECP2 deletion or mutation) of Rett Syndrome. As you may know, there is no treatment for this illness. Currently, the standard management of Rett syndrome is supportive, which means attempting to prevent complications and treatment of symptoms. This study involves testing an investigational drug, which means that even though IGF-1 is approved by the Food and Drug Administration (FDA) for use in children, it has not been used before to treat Rett syndrome specifically. Information from this research will help determine whether the drug should be approved by the FDA in the future for the treatment of Rett Syndrome. There are five major goals to this study: 1. As one of the features of Rett Syndrome is unstable vital signs, the investigators are trying to determine if IGF-1 has any effect on normalizing your child's pulse, blood pressure and breathing pattern. 2. The safety of IGF-1 in children with Rett syndrome. The study personnel will ask you to complete a medication diary and side effect reporting form on a regular basis. They will assist you in completing this by telephone interviews. Your child will undergo 2 lumbar punctures performed at the bedside in the clinical research facility. In addition, laboratory tests will be performed throughout the study to evaluate the safety of IGF-1. These will be blood tests similar to those provided in routine clinical care. Your child will undergo regular non-invasive comprehensive physical examinations including neurological and eye examination, tonsil evaluation, electrocardiograms (ECG), measurement of height, weight and head circumference. 3. IGF-1 may improve your child's behavior, communication and speech. In order
Conditions Studied
Interventions
- DRUG rhIGF-1
Study Locations (1)
Massachusetts
- Boston Children's Hospital - Boston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2010-12 |
| Est. Completion | 2013-01 |
| Phase | Phase 1 |
What the finished NCT01253317 record still lists
NCT01253317 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 525-participant average among 18 other Rett Syndrome trials with a reported enrollment target (98% lower).
The record links to 1 condition, with Rett Syndrome appearing as the primary indexed condition, and to 1 intervention - of which rhIGF-1 is the first listed.
NCT01253317 reports a single indexed study location in Massachusetts.
Frequently Asked Questions
What is clinical trial NCT01253317 about?
NCT01253317 is a clinical study titled "Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)". The investigators are recruiting children for a research study using a medication known as IGF-1 (mecasermin or INCRELEX) to see if it improves the health of children with Rett syndrome (RTT). To participate in the study your child must be female, between the ages of 2 to 12 and have a genetic diagn...
What is the current status of trial NCT01253317?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2010-12. Estimated completion is 2013-01.
What conditions does trial NCT01253317 study?
This clinical trial studies the following conditions: Rett Syndrome.
What interventions are being tested in trial NCT01253317?
The interventions under investigation include: rhIGF-1 (DRUG).
Who is sponsoring clinical trial NCT01253317?
This trial is sponsored by Boston Children's Hospital, which has 462 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01253317 being conducted?
This trial has 1 study location across Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Rett Syndrome
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT01253317's enrollment target sits among peer trials
12 17th of 18 higher than 2 of 18 other Rett Syndrome trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Rett Syndrome 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
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