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A phase III study of JR-401 in patients with SHOX dificiency

Phase 3
Completed
Conditions
Short stature patients with SHOX deficiency
Registration Number
JPRN-jRCT2080223889
Lead Sponsor
JCR Pharmaceuticals Co., Ltd.
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
completed
Sex
All
Target Recruitment
32
Inclusion Criteria

1.The children have a diagnosis of SHOX deficiency by geneanalysis.
2.A girl patients who is clearly not a Turner syndrome base on chromosome testing.

Exclusion Criteria

Pediatric patients who have received continuous GH treatment.
Pediatric patients who have received any treatment that may have effect on the growth stimulation.
Pediatric patients with a disease that causes a short stature unrelated to SHOX deficiency.
Pediatric patients with diagnosis of Langer type middle limb bone dysplasia.

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
efficacy<br>confirmatory<br>Change in Height Standard Deviation Score (SDS) for chronogical Age
Secondary Outcome Measures
NameTimeMethod
efficacy<br>Height Standard Deviation Score (SDS) for chronogical Age
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