Achondroplasia clinical trials at UC Health

• Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.

  open to eligible people ages up to 10 years

  This is a registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries. Information collected will include in anthropometric characteristics, related symptoms, tests, & treatments Children's information will be collected in the registry for a maximum of 5 years.

  at UCLA

• Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

  open to eligible people ages 30 months to 10 years

  This is a long-term, multi-center, observational study in children 2.5 to 10 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications and treatments of study participants. No study medication will be administered.

  at UCSF

• A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

  Sorry, in progress, not accepting new patients

  Study 111-206 is a Phase 2 randomized, double-blind, placebo-controlled clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia.

  at UCLA UCSF

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

  Sorry, not currently recruiting here

  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.

  at UCLA

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

  Sorry, in progress, not accepting new patients

  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.

  at UCLA UCSF

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

  Sorry, accepting new patients by invitation only

  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.

  at UCLA UCSF

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

  Sorry, in progress, not accepting new patients

  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

  at UCLA UCSF

Last updated: May 11, 2021