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ASD-specific Induced Pluripotent Stem Cells for Disease Modeling

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ClinicalTrials.gov Identifier: NCT02720939
Recruitment Status : Unknown
Verified March 2016 by National Taiwan University Hospital.
Recruitment status was:  Recruiting
First Posted : March 28, 2016
Last Update Posted : March 28, 2016
Sponsor:
Information provided by (Responsible Party):
National Taiwan University Hospital

Tracking Information
First Submitted Date March 22, 2016
First Posted Date March 28, 2016
Last Update Posted Date March 28, 2016
Study Start Date January 2016
Estimated Primary Completion Date December 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: March 25, 2016)
iPSC derivation and characterization [ Time Frame: 12 weeks ]
To generate induced-pluripotent stem cells (iPSCs) from peripheral blood mononuclear cells (PBMCs) isolated from whole blood of ASD probands and healthy controls
Original Primary Outcome Measures Same as current
Change History No Changes Posted
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title ASD-specific Induced Pluripotent Stem Cells for Disease Modeling
Official Title ASD-specific Induced Pluripotent Stem Cells for Disease Modeling
Brief Summary

Specific Aims:

  1. To generate induced-pluripotent stem cells (iPSCs) from peripheral blood mononuclear cells (PBMCs) isolated from whole blood of ASD probands and healthy controls.
  2. To derive neural progenitor cells (NPCs) and neurons from iPSCs of ASD probands and healthy controls and compare differences between patients and healthy controls to investigate the cellular phenotype of ASD and uncover the pathophysiology of the disease.
Detailed Description

Autism spectrum disorders (ASDs) is a common, lifelong impairing childhood-onset neurodevelopmental disorder with clinical and genetic heterogeneity. The etiology of ASD is complicated and remains largely unknown. Evidences indicate the high heritability (54-77%) in ASD, and researchers also found copy number variations (CNVs) play an important role in the etiology of ASD. However, due to the ethical issue, researchers haven't had an appropriate platform to investigate the copy number change effect to date. In the following study, researchers are going to derive ASD-specific induced pluripotent stem cells and neurons for disease modeling.

10 ASD patients and 10 healthy controls will be included in the present study and iPSCs and neurons will be generated from their PBMCs. Electrophysiological recording and analysis of neuronal morphology will be conducted to reveal the cellular phenotype of ASD after comparing differences between patients and healthy controls.

The study will provide a platform to reveal the etiology of genetic basis and molecular mechanism of ASD. The cellular phenotype of ASD will be revealed by comparing differences between patients and healthy controls. The findings from this work are expected to contribute to the basic and clinical research on ASD.

Study Type Observational
Study Design Observational Model: Case Control
Time Perspective: Cross-Sectional
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples With DNA
Description:
Peripheral blood will be collected in the INSEPACK Disposable Vacuum Venous Blood Specimen collection Tube (Green Tube with li-Heparin) and each tube will contain 5-10ml of blood from ASD probands and healthy controls.
Sampling Method Non-Probability Sample
Study Population The sample will consist of 10 ASD patients and 10 healthy controls.
Condition Autism Spectrum Disorder
Intervention Not Provided
Study Groups/Cohorts
  • ASD group
    ASD patients who met the diagnostic criteria of either autistic disorder or Asperger's disorder defined by the DSM-IV criteria
  • TD group
    Typically development controls without lifetime diagnosis with ASD or any psychiatric disorders
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Unknown status
Estimated Enrollment
 (submitted: March 25, 2016)
20
Original Estimated Enrollment Same as current
Study Completion Date Not Provided
Estimated Primary Completion Date December 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • ASD group: Subjects who met the diagnostic criteria of either autistic disorder or Asperger's disorder defined by the DSM-IV criteria.

Exclusion Criteria:

  • TD group: Subjects with any current or lifetime DSM-IV psychiatric disorders.
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Taiwan
Removed Location Countries  
 
Administrative Information
NCT Number NCT02720939
Other Study ID Numbers 201507086RINA
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party National Taiwan University Hospital
Study Sponsor National Taiwan University Hospital
Collaborators Not Provided
Investigators
Principal Investigator: Susan Shur-Fen Gau, MD, PhD National Taiwan University Hospital & College of Medicine
PRS Account National Taiwan University Hospital
Verification Date March 2016