A long-term study of RBM-007 in children with achondroplasia

Phase 2

Recruiting

Conditions: Achondroplasia

Registration Number: JPRN-jRCT2031220338

Lead Sponsor: onaka Yosuke

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: All

Target Recruitment: 12

Inclusion Criteria

(1) Has completed the early phase II study, has completed the safety and efficacy evaluation (height growth velocity, etc.) by Investigator, and has been judged to have no safety issues
(2) Able to obtain written informed consent from surrogate decision maker (parents, relatives or legal representative) before study entry
Able to obtain written informed assent from the patient after explaining the contents of this study in the assent document (if possible)
(3) Able to perform all the tests and administration of investigational product described in the protocol, judged by Investigator

Exclusion Criteria

(1) Inadequate as a patient for this study based on examinations, observations, etc. at the end of the early phase II study
(2) Highly likely to be unable to complete this study due to poor compliance treated with RBM-007 or poor implementation of regular examinations and observation items, judged by the Investigator
(3) Diagnosed with a closed growth plate in the examination at the end of the early phase II study
(4) Has a positive pregnancy test at the end of the early phase II study (only female patients)
(5) Inadequate as a patient for this study for any other reason than above, considered by the Investigator

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
(1) Comparison of height growth velocity in the observational study and height growth velocity after the early phase II study entry<br>(2) Change in serum ALP and NT-proCNP<br>(3) Change in body weight, head circumference and chest circumference<br>(4) Change in BMI<br>(5) Change in body proportion ratios (ratio of upper arm length to forearm length, ratio of thigh length to lower leg length, ratio of arm span to height)<br>(6) Condition of the elbow and hip joints based on clinical findings<br>(7) Bone age<br>(8) Morphological shape of the femoral growth plate<br>(9) Tanner Staging Classification <br>(10) Adverse event

Secondary Outcome Measures