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Top 10 Achondroplasia Trials [2024 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.

Pivotal Trial

This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of participation in the QED-sponsored study PROPEL (QBGJ398-001).

A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia

If you are sexually active, you must be willing to use a very effective form of birth control while taking the study drug and for 3 months after stopping the study drug.

I am between 3 and 18 years old, growing more than 1.5 cm a year, not fully through puberty, and meet the bone age requirement.

I am not pregnant or have tested negative for pregnancy.

My ACH diagnosis is confirmed by clinical assessment and genetic testing.

You must have been in the PROPEL study for at least 26 weeks before joining this study.

You and your parent(s) or guardian(s) are able to come to the study visits and follow the study instructions.

My condition matches the specific type and characteristics required.

You have given permission to take part in the study after understanding the details.

Infigratinib

Placebo Comparator

This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to < 2 years at the time of randomization.

A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia

My genetic test shows I have two copies of the ACH gene.

My caregiver is ready to follow the study rules and give me weekly shots.

My recent medical tests show I am fit for the trial.

I have been diagnosed with achondroplasia and genetic tests confirm it.

I am expected to have surgery, including for my neck or spinal cord, during the trial.

I have taken medication or products to influence my growth or body shape.

I need or will need long-term or frequent steroid treatment.

My infant is on daily Vitamin D, or I've started Vitamin D if my levels were low.

TransCon CNP

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

TransCon CNP for Achondroplasia

I have taken prescription or investigational drugs other than TransCon CNP.

I am not allergic to the trial drug or its components.

My guardian can give me weekly shots and follow the study rules.

I have achondroplasia and finished a trial with TransCon CNP.

I am not using effective birth control and am able to have children.

My guardian has signed the consent form, and I will too when I'm of age.

I am taking Vitamin D supplements due to low levels.

Achondroplasia Clinical Trials

Infigratinib +1 More for Achondroplasia

TransCon CNP for Achondroplasia

TransCon CNP for Achondroplasia