JPRN-jRCT1050210136
Not yet recruiting
未知
Research for the realization of criteria for on-demand provision of iPS cells for myotonic dystrophy type 1
Fujimoto Toshio0 sites8 target enrollmentDecember 18, 2021
Overview
- Phase
- 未知
- Intervention
- Not specified
- Conditions
- Myotonic dystrophy type 1
- Sponsor
- Fujimoto Toshio
- Enrollment
- 8
- Status
- Not yet recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Selection Criteria for Patients with Myotonic Dystrophy Type 1
- •(1\) Disease name: Myotonic dystrophy
- •(2\) Disease type: Myotonic dystrophy type 1
- •(3\) Patients with DMPK mutation
- •(4\) Age of onset: 40 years or younger
- •Diagnosis criteria of myotonic dystrophy type 1
- •Myotonic dystrophy type 1 is suspected in patients with characteristic muscle weakness, and is confirmed by DMPK gene analysis.
- •Selection criteria for healthy relatives of patients with myotonic dystrophy type 1
- •(1\) Blood relatives of patients with myotonic dystrophy type 1 within the third degree of consanguinity (biological parents, biological grandparents, biological siblings, biological siblings of biological parents)
- •(2\) Non\-symptomatic patients with myotonic dystrophy type 1
Exclusion Criteria
- •Exclusion Criteria for Patients with Myotonic Dystrophy Type 1
- •(1\) Age of onset: 41 years or older
- •(2\) Patients infected with HBV, HCV, HIV1, HIV2, or HTLV\-1
- •(3\) Other patients deemed inappropriate for this study by the investigators of the collaborating institutions.
- •Exclusion criteria for healthy relatives of patients with myotonic dystrophy type 1
- •(1\) Patients with myotonic dystrophy type 1
- •(2\) Patients infected with HBV, HCV, HIV1, HIV2, or HTLV\-1
- •(3\) Other healthy relatives of patients who are deemed inappropriate for this study by the investigators of the collaborating institutions.
Outcomes
Primary Outcomes
Not specified
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