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Top 10 Achondroplasia Trials [2023 Studies] | Power

Achondroplasia Clinical Trials

Achondroplasia studies recruiting patients for novel treatments. Filter by phase, distance, and inclusion criteria to find your perfect clinical trial.

No Placebo-Only Group

TransCon CNP administered once-weekly in children and adolescents with achondroplasia who have completed a prior TransCon CNP clinical trial. Participants who complete a prior TransCon CNP trial and meet all eligibility criteria will be invited to continue into the long-term open label extension trial to receive 100 µg CNP/kg/week of TransCon CNP. Trial treatment will be completed when the participant reaches 16 years of age for females and 18 years of age for males and have femur and tibial epiphyseal closure. TransCon CNP treatment will continue if femur and tibial epiphyseal closure is not confirmed at the age of 16 years for females, and 18 years for males. Treatment with TransCon CNP will be completed once femur and tibial epiphyseal closure is confirmed by radiographic imaging. The trial duration is individual for each trial participant. Visits will occur every 12-14 weeks throughout the trial.

A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia

You are known or suspected to have a strong allergic reaction to the investigational product or its ingredients.

You have already taken part in a clinical trial for TransCon CNP if you have achondroplasia.

TransCon CNP

The purpose of this study is to establish the bioequivalence of a single SC dose of BMN 111 administered using a vial and syringe versus using an injector pen.

Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants

Non-smokers of any ethnic background, who have either never smoked or quit more than three months ago, are eligible to participate.

You must have no clinically significant abnormalities as per a medical assessment, which includes past medical history, physical examination and tests, cardiac monitoring, temperature check and evaluation of current medications.

All participants must be non-pregnant, non-lactating females and all other genders should use contraception.

This clinical study is open to both male and female participants.

You are between 18 and 55 years of age at the time you signed your informed consent.

You weigh between 60 and 110 kg, with a body mass index of 18.0 to 32.0 kg/m2.

Male and female participants must abide by local regulations regarding contraceptive methods when participating in clinical studies.

In order to participate, you must be able to provide a signed and informed consent that adheres to the requirements and restrictions specified in the Informed Consent Form (ICF).

You have participated in a clinical trial involving a new, experimental drug within the last 30 days or five times the amount of time it takes for the drug to leave your body (whichever is longer).

You have a skin condition or tattoos that could prevent you from receiving the study drug.

You have a significant medical history or symptoms of any health condition, as determined by the person in charge of the study.

You have a history of severe allergic reactions or intolerance to any medication, food or substance, unless the study doctor has approved it.

You are currently using medications that are not allowed in the study.

You cannot take any medication or products that might change how the study drug is processed by your body, like St. John's wort, for at least 30 days before starting the study unless the investigator says it is okay.

BMN 111

The purpose of this clinical trial is to evaluate efficacy and safety of once weekly SC doses of 100 µg CNP/kg compared to placebo on Annualized Growth Velocity after a 52-week randomized treatment period in children aged 2 to 11 years with genetically confirmed Achondroplasia. The double-blind, placebo-controlled treatment period is followed by an Open Label Extension (OLE) period of a 52-week duration.

Achondroplasia Clinical Trials

TransCon CNP 100 Mcgfor Achondroplasia

BMN 111 Injection With Injector Penfor Achondroplasia

TransCon CNPfor Achondroplasia