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10 Achondroplasia Trials

Power is an online platform that helps thousands of Achondroplasia patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

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).
Pivotal Trial
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 3
Age:3 - 17
Sex:All
110 Participants Needed
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 3
Age:6+
Sex:All
119 Participants Needed
This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a QED-sponsored interventional study, and potentially in additional subjects who are naïve to infigratinib treatment. Quality of Life assessments for this subject population will also be evaluated. Treatment-naïve subjects must have at least a 6-month period of growth assessment in study QBGJ398-001 (PROPEL) and will be enrolled in this OLE study only after a dose to be explored further is identified in Phase 2 Study QBGJ398-201 and subjects are not otherwise eligible to enroll in another QED-sponsored Phase 2 or Phase 3 ACH study.
No Placebo Group
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 2
Age:3 - 18
Sex:All
300 Participants Needed
This trial is testing BMN111, an injection, in children with Achondroplasia to help them grow taller. BMN111 has shown promise in promoting growth in children with achondroplasia.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:15+
Sex:All
73 Participants Needed
This is an open-label, phase 1/2 study evaluating the safety, tolerability, pharmacokinetic (what the body does to the drug), pharmacodynamic (what the drug does to the body), and antitumor activity of CGT4859 in adult participants with intrahepatic cholangiocarcinoma (iCCA) or other advanced solid tumors with FGFR2 and/or FGFR3 genetic alternations.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 1, 2
Age:18+
Sex:All
110 Participants Needed
This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.
No Placebo Group
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2
Age:7+
Sex:All
30 Participants Needed
This trial uses TransCon CNP to help children and adolescents with achondroplasia grow their bones properly. It is for those who have already been part of a previous trial. The treatment continues for an extended period.
No Placebo Group
Pivotal Trial
Trial Details
Trial Status:Enrolling By Invitation
Trial Phase:Phase 2, 3
Age:3 - 15
Sex:All
140 Participants Needed
Image of trial facility.

TYRA-300 for Achondroplasia

Hillsborough, North Carolina
The purpose of this study is to evaluate the safety, tolerability, and identify potentially effective dose(s) of TYRA-300 in children with achondroplasia with open growth plates.
No Placebo Group
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:3 - 10
Sex:All
92 Participants Needed
This trial tests if regular injections can help children with Achondroplasia grow taller by making their bones grow faster. Vosoritide has shown promising results in increasing growth in children with achondroplasia.
Pivotal Trial
Trial Details
Trial Status:Active Not Recruiting
Trial Phase:Phase 2, 3
Age:2 - 11
Sex:All
84 Participants Needed
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.
Trial Details
Trial Status:Recruiting
Trial Phase:Phase 2
Age:0 - 2
Sex:All
72 Participants Needed
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Frequently Asked Questions

How much do Achondroplasia clinical trials pay?

Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

How do Achondroplasia clinical trials work?

After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Achondroplasia trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Achondroplasia is 12 months.

How do I participate in a study as a "healthy volunteer"?

Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

What does the "phase" of a clinical trial mean?

The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

Do I need to be insured to participate in a Achondroplasia medical study ?

Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

What are the newest Achondroplasia clinical trials ?

Most recently, we added TYRA-300 for Achondroplasia, CGT4859 for Bile Duct Cancer and Navepegritide for Achondroplasia to the Power online platform.