Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone)
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ClinicalTrials.gov Identifier: NCT02153723 |
Recruitment Status :
Completed
First Posted : June 3, 2014
Results First Posted : November 5, 2018
Last Update Posted : November 5, 2018
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Tracking Information | |||
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First Submitted Date ICMJE | May 26, 2014 | ||
First Posted Date ICMJE | June 3, 2014 | ||
Results First Submitted Date ICMJE | July 20, 2015 | ||
Results First Posted Date ICMJE | November 5, 2018 | ||
Last Update Posted Date | November 5, 2018 | ||
Study Start Date ICMJE | August 2013 | ||
Actual Primary Completion Date | August 2014 (Final data collection date for primary outcome measure) | ||
Current Primary Outcome Measures ICMJE |
Gait Velocity as Measured by GAITRite System [ Time Frame: Baseline and Final week of treatment (week 32) ] To perform quantitative gait assessments a computerized walkway (457 × 90.2 × 0.64cm) with embedded pressure sensors (GAIT Rite system) was used. Subjects walked on the walkway for two trials, while wearing comfortable footwear.
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Original Primary Outcome Measures ICMJE |
Gait speed [ Time Frame: 32 weeks ] To perform quantitative gait assessments a computerized walkway (457 × 90.2 × 0.64cm) with embedded pressure sensors (GAIT Rite system) will be used. Subjects will be asked to walk on the walkway for two trials wearing comfortable footwear.
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Change History | |||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
autonomic (respiratory) function [ Time Frame: 32 weeks ] Wake respirations using the NOX 3 . During one of the three days of monitoring, we will also monitor the same parameters with sleep monitoring equipment during the daytime at the polysomnography laboratory with additional oronasal airflow, EMG, EEG and video monitoring to confirm wakefulness during the period of study.
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Current Other Pre-specified Outcome Measures | Not Provided | ||
Original Other Pre-specified Outcome Measures |
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Descriptive Information | |||
Brief Title ICMJE | Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone) | ||
Official Title ICMJE | Pharmacological Treatment of Rett Syndrome With Glatiramer Acetate (Copaxone) | ||
Brief Summary | A phase 2 open label trial to test a potential drug treatment for Rett syndrome, the leading known genetic cause of severe neurological impairment in girls. The drug, Copaxone (generic name - Glatiramer acetate) is medication FDA approved for the treatment of multiple sclerosis. Copaxone's high safety profile has been documented in large cohorts of patients for more than 12 years. | ||
Detailed Description | Background/rationale for the study: In Rett syndrome brain cells aren't actually lost, instead poor maturation of connections between brain cells (synapses) prevents effective neurological functioning, and is the main morphological feature of the disease. The MeCP2 gene plays a major role in transcriptional regulation of other genes, one of which is the gene encoding brain-derived neurotrophic factor (BDNF). The disease progression and severity of symptoms is directly affected by the level of BDNF expression. An increase of BDNF levels (by genetic manipulations or pharmacological agents) leads to delayed onset of Rett syndrome-like symptoms in experimental models; rescued gait/mobility, improved quality of life and increased survival rates. Copaxone treatment by subcutaneous injection caused elevation of BDNF levels. Quantitative immunofluorescence assays showed about a twofold increase in neuronal expression of BDNF following Copaxone treatment. We expect that an increase in BDNF levels with Copaxone administration will stimulate communication between brain cells (synaptic maturation), which will lead to amelioration of symptoms (motor functions/gait, cognitive functions, breathing, encephalopathy and improve quality of life) for girls with Rett syndrome. |
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Study Type ICMJE | Interventional | ||
Study Phase ICMJE | Phase 2 | ||
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: Glatiramer Acetate
Other Name: Copaxone
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Study Arms ICMJE | Experimental: Copaxone
Dose escalation: Study drug will be administered once a week for 4 weeks, twice a week for 4 weeks and daily for 24 weeks. Drug is administered as a subcutaneous injection. Intervention: Drug: Glatiramer Acetate
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Publications * | Djukic A, Holtzer R, Shinnar S, Muzumdar H, Rose SA, Mowrey W, Galanopoulou AS, Shinnar R, Jankowski JJ, Feldman JF, Pillai S, Moshe SL. Pharmacologic Treatment of Rett Syndrome With Glatiramer Acetate. Pediatr Neurol. 2016 Aug;61:51-7. doi: 10.1016/j.pediatrneurol.2016.05.010. Epub 2016 May 27. | ||
* 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 |
10 | ||
Original Estimated Enrollment ICMJE |
20 | ||
Actual Study Completion Date ICMJE | January 2016 | ||
Actual Primary Completion Date | August 2014 (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 | 10 Years and older (Child, Adult, Older Adult) | ||
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 | NCT02153723 | ||
Other Study ID Numbers ICMJE | 13-05-117 | ||
Has Data Monitoring Committee | No | ||
U.S. FDA-regulated Product | Not Provided | ||
IPD Sharing Statement ICMJE |
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Current Responsible Party | Aleksandra Djukic, Montefiore Medical Center | ||
Original Responsible Party | Same as current | ||
Current Study Sponsor ICMJE | Montefiore Medical Center | ||
Original Study Sponsor ICMJE | Same as current | ||
Collaborators ICMJE | Rett Syndrome Research Trust | ||
Investigators ICMJE | Not Provided | ||
PRS Account | Montefiore Medical Center | ||
Verification Date | October 2018 | ||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |