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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Study Type | Interventional |
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Study Design | Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
Condition |
Rett Syndrome |
Intervention |
Drug: rhIGF-1 |
Enrollment | 12 |
Recruitment Details | |
Pre-assignment Details |
Arm/Group Title | Open-label IGF-1 Treatment |
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Twelve girls with MECP2 mutations participated in the 4-week multiple ascending dose (MAD) period of open-label treatment with IGF-1 (mecasermin). The MAD focused on obtaining PK data, determining cerebrospinal fluid (CSF) penetration, evaluating safety and tolerability, and estimating feasibility of automated cardiorespiratory measures as biomarkers. Mecasermin was escalated over a 4-week period, beginning with twice daily injections of 40 mcg/kg the first week, 80 mcg/kg the second week, and 120 mcg/kg during the third and fourth weeks. CSF samples were obtained prior to initiating treatment and after completing the fourth week. 10 of these subjects went on to complete the open label extension (OLE). The OLE was designed to obtain additional information on safety, tolerability, and cardiorespiratory measures after 20-weeks of treatment, as well as preliminary data on neurologic and behavioral parameters. During the 20-week OLE subjects were evaluated every 5 weeks. |
Period Title: Overall Study | |
Started | 12 |
Completed | 10 |
Not Completed | 2 |
Reason Not Completed | |
Adverse Event | 2 |
Arm/Group Title | Open-label IGF-1 Treatment | |
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Twelve girls with MECP2 mutations participated in the 4-week multiple ascending dose (MAD) period of open-label treatment with IGF-1 (mecasermin). The MAD focused on obtaining PK data, determining cerebrospinal fluid (CSF) penetration, evaluating safety and tolerability, and estimating feasibility of automated cardiorespiratory measures as biomarkers. Mecasermin was escalated over a 4-week period, beginning with twice daily injections of 40 mcg/kg the first week, 80 mcg/kg the second week, and 120 mcg/kg during the third and fourth weeks. CSF samples were obtained prior to initiating treatment and after completing the fourth week. 10 of these subjects went on to complete the open label extension (OLE). The OLE was designed to obtain additional information on safety, tolerability, and cardiorespiratory measures after 20-weeks of treatment, as well as preliminary data on neurologic and behavioral parameters. During the 20-week OLE subjects were evaluated every 5 weeks. |
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Overall Number of Baseline Participants | 12 | |
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Twelve girls with MECP2 mutations participated in the 4-week MAD period; ten of them continued and completed the subsequent 20-week OLE. Nine subjects met full diagnostic criteria for RTT, all of whom continued in the OLE.
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Age, Categorical
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 12 participants | |
<=18 years |
12 100.0%
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Between 18 and 65 years |
0 0.0%
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>=65 years |
0 0.0%
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Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 12 participants | |
Female |
12 100.0%
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Male |
0 0.0%
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Race (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 12 participants | |
American Indian or Alaska Native |
0 0.0%
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Asian |
2 16.7%
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Native Hawaiian or Other Pacific Islander |
0 0.0%
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Black or African American |
0 0.0%
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White |
10 83.3%
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More than one race |
0 0.0%
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Unknown or Not Reported |
0 0.0%
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Ethnicity (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 12 participants | |
Hispanic or Latino |
0 0.0%
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Not Hispanic or Latino |
12 100.0%
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Unknown or Not Reported |
0 0.0%
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Region of Enrollment
Measure Type: Number Unit of measure: Participants |
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United States | Number Analyzed | 12 participants |
12 |
Name/Title: | Walter E. Kaufmann, MD |
Organization: | Boston Children's Hospital |
Phone: | 617-355-5230 |
EMail: | rettresearch@childrens.harvard.edu |
Responsible Party: | Mustafa Sahin, Boston Children's Hospital |
ClinicalTrials.gov Identifier: | NCT01253317 |
Other Study ID Numbers: |
10-08-0403 |
First Submitted: | December 2, 2010 |
First Posted: | December 3, 2010 |
Results First Submitted: | May 13, 2014 |
Results First Posted: | June 22, 2017 |
Last Update Posted: | June 22, 2017 |