Meclozine for achondroplasia heading to clinical trial
Official scientific title of the study | Safety and pharmacokinetics of meclizine hydrochloride for achondroplasia children |
Title of the study (Brief title) | Safety and pharmacokinetics of meclizine |
Narrative objectives1 | Examination of safety as well as 24-hour pharmacokinetics and accumulation at 1 week after a single dose of meclizine. |
Basic objectives2 | Safety |
Primary outcomes |
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Key secondary outcomes | Simulation of pharmacokinetics in the repeated administration of meclizine for 14 days |
Age-lower limit |
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Age-upper limit |
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Gender | Male and Female | |||
Key inclusion criteria | achondroplasia children | |||
Key exclusion criteria | body weight of less than 11 kg | |||
Target sample size | 12 |
Meclozine for achondroplasia – Phase 1 clinical trial
The phase 1 will include 6 children that will take a single dose of Meclozine/meclizine (25mg/day). The team predicts to run a double dose study with the administration of 50mg/day (25mg twice per day) in other 6 children at the end of the year.
This is a critical study to evaluate the safety of Meclizine in young children.
Meclozine is already available in the market under several brand names, for the treatment of motion sickness, vertigo, nausea, and vomiting in adults and in children over 12 years old (5). But once there is no information of the use of meclozine in children under 12 years-old but is available adult PK data, now is required to know the PK for children. This is the reason why in this study, the phase 1 will include directly children and not healthy adults as like in another clinical trial, because the safety in adults is already known.

Example of a commercial brand of meclizine available in the market. Image credits: Phoenix Medical Services |
Repurposing drug for rare diseases
This is related to discovering new uses for approved drugs to provide the quickest possible transition from bench to bedside (6), or in other words, reducing the time from the early laboratory investigation until having a drug approved and available in the market for a specific disease.
Why repurposing drugs?
There is a poor success rate (16%) for drugs entering clinical trials, with a development timeline of 12 to 15 years and a jaw-dropping price tag approaching $1 billion, it makes economic sense for drug developers to explore better therapeutic fits for failed drugs. (7)
Meclozine for Achondroplasia – Phase 2
Sources
- Matsushita M et al., Meclozine Promotes Longitudinal Skeletal Growth in Transgenic Mice with Achondroplasia Carrying a Gain-of-Function Mutation in the FGFR3 Gene, Endocrinology, Volume 156, Issue 2, 1 February 2015, Pages 548–554
- Matsushita M et al., Meclozine has potential effects on short stature and foramen magnum stenosis in transgenic mice with achondroplasia, Bone Abstracts (2015) 4 OC13
- University hospital Medical Information Network (UMIN) Center, UMIN-CTR Clinical Trial. 2018, Infrastructure for Academic Activities.
- Lesson 1: Introduction to Pharmacokinetics and Pharmacodynamics, in Concepts in Clinical Pharmacokinetics. ASHP.
- Stewart, L.A.A.J., et al. Meclizine. Drugs A to Z 2017. [cited 2018 16-07].
- National Center for Advancing Translational Services. Repurposing Drugs. [cited 2018 16-07].
- Eureka Staff. Repurposing Drugs for Rare Diseases. Eye On The Science 2018 [cited 2018 16-07].