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Top 10 Achondroplasia Trials [2022 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.

Approximately 63 participants will be randomized to one of three doses to receive Recifercept either

Low Dose
Medium Dose
High Dose

Participants will will attend the clinic at baseline and at Day 1, 4, 8, 15, 29 & then Month 2, 3 6, 9 & 12. Assessments include safety, blood sampling, physical examination, vital signs, anthropometric body measurements & patient/caregiver quality of life questionnaires

Participants will received treatment with Recifercept for 12 months. All participants who complete the study and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to enroll into an open-label extension (OLE) study.

A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia

This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for at least 6 months. The study includes dose escalation with extended treatment, and dose expansion. The study also includes a PK Substudy to fully characterize the pharmacokinetics of infigratinib in children with ACH.

Study of Infigratinib in Children With Achondroplasia

This study is evaluating whether a drug called infigratinib can help children with Achondroplasia.

This is a Phase 2, open-label multi-center long-term extension study, with approximately 70 subjects, to evaluate the safety and efficacy of BMN111 in children with Achondroplasia until subjects reach near-adult final height. Eligible subjects will have completed 1 year of BMN111 or placebo treatment in the 111-206 study and once enrolled in the 111-208 extension study will receive a daily dose of BMN111 by subcutaneous injection according to their age as determined by 111-206.

An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia

This study is evaluating whether a drug called BMN111 can help children with Achondroplasia grow taller.

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

An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

This study is evaluating whether a drug called BMN 111 can help children with Achondroplasia.

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.

A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)

This study is evaluating whether a drug called BMN 111 is safe and effective for children with Achondroplasia.