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

JRCT ID: jRCT2031220338

Registered date:16/09/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Achondroplasia
• Date of first enrollment: 21/12/2023
• Target sample size: 12
• Study type: Interventional
• Intervention(s): Administration of RBM-007 at a dose of 0.3 or 0.6 mg/kg, every 1 to 4 weeks. The administration period is 1 to 5 years (including the administration period of the early phase II study) or the period until the height reaches the average height of healthy children, whichever is shorter.

Outcome(s)

Primary Outcome

(1) Comparison of height growth velocity in the observational study and height growth velocity after the early phase II study entry
(2) Change in serum ALP and NT-proCNP
(3) Change in body weight, head circumference and chest circumference
(4) Change in BMI
(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)
(6) Condition of the elbow and hip joints based on clinical findings
(7) Bone age
(8) Morphological shape of the femoral growth plate
(9) Tanner Staging Classification
(10) Adverse event

Key inclusion & exclusion criteria

• Age minimum: >= 5age old
• Age maximum: <= 14age old
• Gender: Both

Include 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

Exclude 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

Related Information

• Primary Sponsor: Nonaka Yosuke