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Metadata

Name
Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)
Repository
ClinicalTrials.gov
Identifier
clinicaltrials:NCT01253317
Description
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 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 child's 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 child's brain.

5. Children with Rett Syndrome sometimes experience "flushing" in their cheeks or have
exceptionally cold hands or feet and/or abnormal perspiration. The Qsensor® is a
non-invasive device worn on a fabric bracelet that continually measures your child's
perspiration level and body temperature. We would like to use the Qsensor® to determine
whether or not IGF-1 improves these symptoms.

.
Data or Study Types
clinical trial
Keywords
RTT, Rett Syndrome, IGF-1, MECP2, Mecasermin, Increlex, IGF1
Source Organization
Unknown
Access Conditions
available
Year
2010
Access Hyperlink
https://clinicaltrials.gov/ct2/show/NCT01253317

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