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An early phase II study of RBM-007 in children with achondroplasia

An early phase II study of RBM-007 in children with achondroplasia

Nonaka Yosuke

RIBOMIC Inc.

3-16-13 Shirokanedai, Minato-ku, Tokyo 108-0071, Japan

y.nonaka@ribomic.com

Nonaka Yosuke

RIBOMIC Inc.

3-16-13 Shirokanedai, Minato-ku, Tokyo 108-0071, Japan

+81-3-3440-3303

y.nonaka@ribomic.com

Complete

Aug. 26, 2022

Interventional

non-randomized controlled trial

open(masking not used)

uncontrolled control

single assignment

treatment purpose

(1) 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 trial in the assent document (if possible)
(2) Has achondroplasia, diagnosed by genetic diagnosis
(3) Aged 5 to 14 years, inclusive, at study entry
(4) Able to lead a normal life without assistance
(5) Able to perform all the tests and administration of investigational product described in the protocol, judged by Investigator
(6) Participates in the observational study and has a height measurement record at the trial site at least 26 weeks (6 months) before study entry

(1) Has short stature condition other than achondroplasia
(2) Has congenital diseases other than achondroplasia
(3) Has history of treatment with growth hormone or VOXZOGO within 38 weeks before study entry
(4) Has history of limb-lengthening surgery
(5) With a closed growth plate
(6) Has any of the following disorders that require surgical treatment on MRI or CT scans, or has clinically significant neurological symptoms or obstructive sleep apnea syndrome caused by these disorders: foramen magnum stenosis, spinal stenosis, hydrocephalus, spinal cord and cauda equina compression, etc (However, patients H146who have undergone surgical treatment for these disorders at least 52 weeks (12 months) before study entry and have no clinical problems can be enrolled)
(7) Has a history of allergies against the following products containing RBM-007 components (e.g., nucleic acid, polyethylene glycol, mannitol): prescribed medicines, vaccines, over-the-counter medicines, cosmetics, foods, etc
(8) Has history of cardiac or vascular disease
(9) Shows clinically significant abnormalities in the clinical laboratory test result, which is related to hepatic function or renal function before study entry
(10) Had a fracture of the long bones or spine within 52 weeks (12 months) before study entry (except for fracture of digits or toes)
(11) Has history of hip surgery, severe hip dysplasia or serious hip injury
(12) Highly likely to be unable to comply with RBM-007 treatment or to complete the trial, judged by the Investigator
(13) Used any other IP or investigational medical device within 52 weeks (12 months) before study entry
(14) Inadequate as a patient for this trial for any other reason than above, considered by the Investigator

Both

Achondroplasia

Administration of RBM-007 at a dose of 0.3 or 0.6 mg/kg, every 1 to 4 weeks for 34 weeks

(1) Comparison of height growth velocity before and after administration (at least 26 weeks [6 months] each)
(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 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
(11) Blood concentration profile of RBM-007 and pharmacokinetic parameters

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