Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)
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ClinicalTrials.gov Identifier: NCT01253317 |
Recruitment Status :
Completed
First Posted : December 3, 2010
Results First Posted : June 22, 2017
Last Update Posted : June 22, 2017
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Tracking Information | ||||
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First Submitted Date ICMJE | December 2, 2010 | |||
First Posted Date ICMJE | December 3, 2010 | |||
Results First Submitted Date ICMJE | May 13, 2014 | |||
Results First Posted Date ICMJE | June 22, 2017 | |||
Last Update Posted Date | June 22, 2017 | |||
Study Start Date ICMJE | December 2010 | |||
Actual Primary Completion Date | September 2012 (Final data collection date for primary outcome measure) | |||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
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Change History | ||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | |||
Original Other Pre-specified Outcome Measures | Not Provided | |||
Descriptive Information | ||||
Brief Title ICMJE | Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection) | |||
Official Title ICMJE | Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation With IGF-1 | |||
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:
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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 child's 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 Type ICMJE | Interventional | |||
Study Phase ICMJE | Phase 1 | |||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Rett Syndrome | |||
Intervention ICMJE | Drug: rhIGF-1
1) Multiple ascending dose (MAD) period (4 weeks): Subjects will receive escalating twice-daily doses of IGF-1 over 4 weeks (40 µg/kg, 80 µg/kg, 120 µg/kg) and then continue treatment at 120 µg/kg BID for 20 weeks should they choose to enroll in the open-label extension period.
Other Name: Mecasermin (brand name Increlex)
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Study Arms ICMJE | Experimental: rhIGF-1
Subjects will receive escalating twice-daily doses of IGF-1 over 4 weeks (40 µg/kg, 80 µg/kg, 120 µg/kg) and then continue treatment at 120 µg/kg BID for 20 weeks should they choose to enroll in the OLE.
Intervention: Drug: rhIGF-1
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Publications * | Not Provided | |||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||
Recruitment Status ICMJE | Completed | |||
Actual Enrollment ICMJE |
12 | |||
Original Estimated Enrollment ICMJE |
40 | |||
Actual Study Completion Date ICMJE | January 2013 | |||
Actual Primary Completion Date | September 2012 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 2 Years to 12 Years (Child) | |||
Accepts Healthy Volunteers ICMJE | No | |||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries ICMJE | United States | |||
Removed Location Countries | ||||
Administrative Information | ||||
NCT Number ICMJE | NCT01253317 | |||
Other Study ID Numbers ICMJE | 10-08-0403 | |||
Has Data Monitoring Committee | Yes | |||
U.S. FDA-regulated Product | Not Provided | |||
IPD Sharing Statement ICMJE | Not Provided | |||
Current Responsible Party | Mustafa Sahin, Boston Children's Hospital | |||
Original Responsible Party | Omar, Khwaja, MD, PhD, Children's Hospital Boston | |||
Current Study Sponsor ICMJE | Boston Children's Hospital | |||
Original Study Sponsor ICMJE | Same as current | |||
Collaborators ICMJE |
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Investigators ICMJE |
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PRS Account | Boston Children's Hospital | |||
Verification Date | June 2017 | |||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |