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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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16 December 2017 |
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Main ID: |
NCT01253317 |
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Date of registration:
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02/12/2010 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Treatment of Rett Syndrome With rhIGF-1 (Mecasermin [rDNA]Injection)
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Scientific title:
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Pharmacological Treatment of Rett Syndrome by Stimulation of Synaptic Maturation With IGF-1 |
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Date of first enrolment:
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December 2010 |
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Target sample size:
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12 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01253317 |
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Study type:
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Interventional |
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Study design:
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Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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Mustafa Sahin, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Boston Children’s Hospital |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- female
- with RTT (typical or variant) as defined using the internationally agreed 2010
RettSearch criteria.
- genetically defined mutation or deletion of the MECP2 gene.
- Girls will have the following prepubertal status: (1) Tanner stage 1 or 2 breast
development; (2) Tanner stage 1 or 2 pubic hair development; (3) and younger than 12
years by bone age.
- Chronological age must be 2 years or older
Exclusion Criteria:
- prior therapeutic use of IGF-1, growth hormone, Lupron® or sex steroids
- allergy to the trial product
- co-morbid or chronic illness beyond that known to be associated with Rett Syndrome:
diabetes mellitus, fatty acid oxidation disorder, chromosomal aneuploidy, syndromes
associated with high risk of malignancy, current or previous exposure to spinal
irradiation or history of malignancy.
- severe scoliosis (defined as a spinal curve of 70 degrees or more as measured on
clinical and radiological examination)
Age minimum:
2 Years
Age maximum:
12 Years
Gender:
Female
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Health Condition(s) or Problem(s) studied
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Rett Syndrome
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Intervention(s)
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Drug: rhIGF-1
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Primary Outcome(s)
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Pharmacokinetic (PK) Profile - Areas Under the Curve (AUCt)
[Time Frame: 60 minutes pre-dose and 0.5, 1.0, 1.5, 2.0, 3.0, 4.0, 8.0, and 12.0 hours post-dose on days 1, 8, 15 and 29.]
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Adverse Events
[Time Frame: biweekly during the MAD and every five weeks during the OLE]
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Secondary Outcome(s)
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Change From Pre-MAD Apnea Index at Post-OLE
[Time Frame: pre-MAD (baseline) to post-OLE (after 20 weeks of IGF-1 treatment)]
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Change in Social Avoidance Subscale Scores on the ADAMS From Pre-OLE to Post-OLE
[Time Frame: Pre-OLE (visit 1) and post-OLE (after 20 weeks of IGF-1 therapy)]
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Secondary ID(s)
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10-08-0403
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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