STUDIES & CLINICAL TRIALS

The Chandler Project provides valuable information about clinical trials for informational purposes only. We strive to empower individuals with achondroplasia and other skeletal dysplasias with the knowledge to make informed decisions about their healthcare options. The Chandler Project does not sponsor, endorse, or is directly affiliated with any specific clinical trial mentioned or referenced on our platform. Please get in touch with the study coordinator if you have a question about a particular study or trial.

For the most up-to-date information on the following studies and trials, please refer to ClinicalTrials.gov
A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia
ID: NCT05353192
Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.
location: Shanghai, shanghai, china
extension study of inifgratinib in children with achondroplasia
ID: NCT05145010
Sponsor: QED Therapeutics, Inc.
A PARTICIPANT-MEDIATED OBSERVATIONAL VIRTUAL REGISTRY OF CHILDREN WITH ACHONDROPLASIA IN THE UNITED STATES
ID: NCT06168201
SPONSOR: BioMarin PHARMACEUTICAL
PROSPECTIVE CLINICAL ASSESSMENT STUDY IN CHILDREN WITH ACHONDROPLASIA
ID: NCT04035811
SPONSOR: QED THERAPEUTICS, INC.
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
ID: NCT06079398
SPONSOR: Ascendis Pharma Growth Disorders A/S
Study of Infigratinib in Children With Achondroplasia
ID: NCT04265651
SPONSOR: QED THERAPEUTICS, INC.
registry for patients with achondroplasia/hypochondroplasia (ompr-ACH/HY)
ID: NCT05328050
SPONSOR: Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico
location: Milan, Italy
Adaption and Testing of the Quality of Life in Short Stature Youth (QoLISSY) Questionnaire for Parents With Children From 0-4
ID: NCT05603936
SPONSOR: Universitätsklinikum Hamburg-Eppendorf
location: Hamburg, Germany
Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SAR442501 in Pediatric Participants With achondroplasia (upreach-2)
ID: NCT06067425
SPONSOR: Sanofi
location: Australia, China, Italy, Republic of Korea, Spain
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With achondroplasia
ID: NCT05246033
SPONSOR: Ascendis Pharma A/S
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With ACHONDROPLASIA (WITH RESULTS)
ID: NCT04085523
SPONSOR: ASCENDIS PHARMA A/S
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia (ApproACH)
ID: NCT05598320
SPONSOR: Ascendis Pharma Growth Disorders A/S
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With achondroplasia
ID: NCT04554940
sponsor: BioMarin Pharmaceutical
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With achondroplasia
ID: NCT03424018
sponsor: BioMarin Pharmaceutical
An Extension Study to Evaluate the Safety and efficacy of BMN 111 in Children With achondroplasia
ID: NCT03989947
sponsor: BioMarin Pharmaceutical
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With achondroplasia (ach) (ach)
ID: NCT02724228
sponsor: BioMarin Pharmaceutical
A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
ID: NCT03875534
Sponsor: Ascendis Pharma A/S
The Norwegian Adult Achondroplasia Study
ID: NCT03780153
SPONSOR: Sunnaas Rehabilitation Hospital
Lifetime Impact Study for Achondroplasia (LISA)
ID: NCT03872531
sponsor: Biomarin pharmaceutical
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
ID: NCT01603095
sponsor: BioMarin Pharmaceutical
Lifetime Impact of Achondroplasia Study in Europe-LIAISE
ID: NCT03449368
sponsor: BioMarin Pharmaceutical
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia *WITH RESULTS*
ID: NCT03197766
sponsor: BioMarin Pharmaceutical
A Study to Learn About Recifercept in Patients With Achondroplasia
ID: NCT05659719
sponsor: Pfizer
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia *WITH RESULTS*
ID: NCT02055157
sponsor: BioMarin Pharmaceutical
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
ID: NCT03583697
sponsor: BioMarin Pharmaceutical
A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
ID: NCT01590446
sponsor: BioMarin Pharmaceutical
C-Type Natriuretic Peptide and Achondroplasia
ID: NCT01541306
sponsor: Nemours Children’s Clinic
Issues Surrounding Prenatal Genetic Testing for Achondroplasia
ID: NCT00001536
sponsor: National Human Genome Research Institute (NHGRI)
A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With Somatropin
ID: NCT01435629
sponsor: Novo Nordisk A/S
Special Survey for Long Term Application
ID: NCT01516229
sponsor: Novo Nordisk A/S