Achondroplasia clinical trials at University of California Health
7 in progress, 4 open to eligible people
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
Study of Infigratinib in Children With Achondroplasia
open to eligible people ages 3-11
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.
at 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, in progress, not accepting new patients
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
Our lead scientists for Achondroplasia research studies include Paul Harmatz, M.D..
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