Trial record 2 of 32 for:
Developmental Disabilities | ( Map: Minnesota, United States )
A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT01047306 |
Recruitment Status :
Completed
First Posted : January 12, 2010
Results First Posted : March 30, 2016
Last Update Posted : March 30, 2016
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Sponsor:
Shire
Information provided by (Responsible Party):
Shire
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Study Type | Observational |
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Study Design | Observational Model: Cohort; Time Perspective: Prospective |
Condition |
Sanfilippo Syndrome Type A |
Intervention |
Other: assessment |
Enrollment | 25 |
Participant Flow
Recruitment Details | |
Pre-assignment Details |
Arm/Group Title | Children < 6 Years Old | Children ≥ 6 Years Old |
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Children ≥1 to < 6 years of age with Sanfilippo Syndrome Type A (mucopolysaccharidosis type IIIA; MPS IIIA) who were untreated with any investigational products (drugs and/or devices). | Children ≥ 6 years of age with Sanfilippo Syndrome Type A (mucopolysaccharidosis type IIIA; MPS IIIA) who were untreated with any investigational products (drugs and/or devices). |
Period Title: Overall Study | ||
Started | 19 | 6 |
Discontinued From the Study | 3 | 0 |
Completed | 16 | 6 |
Not Completed | 3 | 0 |
Reason Not Completed | ||
Withdrawal by Subject | 2 | 0 |
Lost to Follow-up | 1 | 0 |
Baseline Characteristics
Arm/Group Title | Children < 6 Years Old | Children ≥ 6 Years Old | Total | |
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Children ≥1 to < 6 years of age with MPS IIIA who were untreated with any investigational products (drugs and/or devices). | Children ≥ 6 years of age with MPS IIIA who were untreated with any investigational products (drugs and/or devices). | Total of all reporting groups | |
Overall Number of Baseline Participants | 19 | 6 | 25 | |
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A maximum of 25 patients with MPS IIIA will be enrolled in the study to provide at least 12 patients who complete the study. This number was not based on any statistical consideration, but is expected to be adequate to describe the natural progression of Sanfilippo A syndrome.
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Age, Continuous
Mean (Standard Deviation) Unit of measure: Years |
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Number Analyzed | 19 participants | 6 participants | 25 participants | |
4.29 (1.996) | 12.44 (4.500) | 6.24 (4.452) | ||
Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 19 participants | 6 participants | 25 participants | |
Female |
6 31.6%
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3 50.0%
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9 36.0%
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Male |
13 68.4%
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3 50.0%
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16 64.0%
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Region of Enrollment
Measure Type: Number Unit of measure: Participants |
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United States | Number Analyzed | 19 participants | 6 participants | 25 participants |
19 | 6 | 25 |
Outcome Measures
Adverse Events
Limitations and Caveats
[Not Specified]
More Information
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts
the PI's rights to discuss or publish trial results after the trial is completed.
Shire's agreements with investigators vary. All agreements provide Shire the right to embargo communications regarding trial results prior to public release for a period ≤180 days from the time submitted to Shire for review. Shire does not prohibit publication, but can require the removal of confidential information (excluding trial results) and can request postponement of a single-center publication until after disclosure of the trial's multi-center publication.
Results Point of Contact
Name/Title: | Study Physician |
Organization: | Shire Development LLC |
Phone: | +1 866 842 5335 |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Shire |
ClinicalTrials.gov Identifier: | NCT01047306 |
Other Study ID Numbers: |
HGT-SAN-053 |
First Submitted: | January 11, 2010 |
First Posted: | January 12, 2010 |
Results First Submitted: | February 9, 2015 |
Results First Posted: | March 30, 2016 |
Last Update Posted: | March 30, 2016 |