Ignite Creation Date:
2024-05-05 @ 11:05 PM
Last Modification Date:
2024-10-26 @ 10:28 AM
Study NCT ID:
NCT01253317
Status:
COMPLETED
Last Update Posted:
2017-06-22
First Post:
2010-12-02
Brief Title:
Treatment of Rett Syndrome With rhIGF-1 Mecasermin rDNAInjection
Sponsor:
Boston Childrens Hospital
Organization:
Boston Childrens Hospital
Study Overview
Official Title:
Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation With IGF-1
Status:
COMPLETED
Status Verified Date:
2017-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 childs 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 childs behavior communication and speech In order to measure this the investigators will evaluate your child once during each month of treatment with neurodevelopmental assessments and a neurological exam Investigators will also ask you about her behavior and day-to-day functioning through a structured parental interview and questionnaires
4 We will examine your childs cortical function through use of electroencephalography EEG in conjunction with presentation of visual and auditory stimuli EEG is a non-invasive way of recording the electrical activity of your childs brain
5 Children with Rett Syndrome sometimes experience flushing in their cheeks or have exceptionally cold hands or feet andor abnormal perspiration The Qsensor is a non-invasive device worn on a fabric bracelet that continually measures your childs perspiration level and body temperature We would like to use the Qsensor to determine whether or not IGF-1 improves these 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 childs 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 childs behavior communication and speech In order to measure this the investigators will evaluate your child once during each month of treatment with neurodevelopmental assessments and a neurological exam Investigators will also ask you about her behavior and day-to-day functioning through a structured parental interview and questionnaires
4 We will examine your childs cortical function through use of electroencephalography EEG in conjunction with presentation of visual and auditory stimuli EEG is a non-invasive way of recording the electrical activity of your childs brain
5 Children with Rett Syndrome sometimes experience flushing in their cheeks or have exceptionally cold hands or feet andor abnormal perspiration The Qsensor is a non-invasive device worn on a fabric bracelet that continually measures your childs perspiration level and body temperature We would like to use the Qsensor to determine whether or not IGF-1 improves these symptoms
Detailed Description:
There are two treatment periods in the trial The multiple ascending dose MAD period is an intensive 4-week pharmacokinetic study which will require 3 inpatient stays and 4 half-day outpatient visits During in-patient sessions an IV line will be placed for frequent blood samples A lumbar puncture will be performed by a physician at the beginning and again at the end of the MAD The primary goal of the MAD is to determine the safety of IGF-1 therapy for girls with RTT As such the investigators will ask that you monitor your childs blood sugar levels using a glucometer At the end of the MAD you will have the option of enrolling your daughter in an additional 20 weeks of open-label IGF-1 treatment
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None