Achondroplasia clinical trials at University of California Health

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

  open to eligible people ages 3 months to 10 years

  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

• 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 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: July 14, 2021