Beyond Achondroplasia

Growing together with Clara

Update in multicenter study from BioMarin


A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia


Ages Eligible for Study: 2 Years to 13 Years
Genders Eligible for Study: Both
Accepts Healthy Volunteers: No
Sampling Method: Probability Sample

Study Population

  • Approximately 200 patients will be enrolled.
  • Patients aged 2 to 13.5 years, inclusive, on the date of consent will be enrolled. Patients aged 0 to < 2 years on the date of consent may also be enrolled, the timing of which will be at the discretion of the sponsor. Approximately equal numbers of boys and girls will be enrolled.

Please read here the inclusion and exclusion criteria

Contact: Trial Specialist  E-mail:
Please refer to this study by its identifier: NCT01603095


United States, California
Harbor-UCLA Medical Center
Los Angeles, California, United States, 90048
Contact: Nathalia Patritti Cressey, Study Coord    310-781-3682 ext Office
Contact    310-597-1960 ext Cell    – Principal Investigator: Patti Dickson, MD
Children’s Hospital and Research Center Oakland
Oakland, California, United States, 94609
Contact: Jacqueline Madden, Study Coord    510-428-3885 ext 5745

United States, Delaware
Nemours/Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States, 19803
Contact: Cassie Brown, Study Coord    302-298-7930

Contact: Michael Bober, MD
United States, Georgia
Emory University
Decatur, Georgia, United States, 30033
Contact: Elizabeth Smith, Study Coord

Contact: William Wilcox, MD

United States, Illinois
Ann and Robert H Lurie Children’s Hospital of Chicago
Chicago, Illinois, United States, 60614
Contact: Victoria Sanders, Study Coord   312-227-6120
United States, Maryland
Johns Hopkins McKusick- Institute of Genetic Medicine
Baltimore, Maryland, United States, 21287
Contact: Adekemi Alade, Study Coord

Contact: Kira Lurman, Study Coord

United States, Missouri
University of Missouri
Columbia, Missouri, United States, 65201
Contact: Vicki L Jones, Study Coord    573-882-7583

Contact: Daniel G Hoernschemeyer, MD    573-882-1351
Principal Investigator: Daniel G Hoernschemeyer, MD
United States, Tennessee
Vanderbilt University
Nashville, Tennessee, United States, 37232
Contact: LeeAnna Melton, Study Coord    615-343-6761

United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
Contact: Catherine Loffredo, Study Coord

Australia, Victoria
Murdoch Children’s Research Institute
Enrolling by invitation

Parkville, Victoria, Australia, 3052

Institut Necker
Paris, France, 75015
Contact: Kim-Hahn Le Quan Sang, MD    33 1 44 49 59 51

Principal Investigator: Valerie Cormier-Daire, MD
United Kingdom
Guy’s and St. Thomas NHS Foundation Trust Evelina Children’s Hospital
London, England, United Kingdom, SE1 9RT
Contact: Joanna Poulter, Study Coord    020 7188 7188 ext 52237

Principal Investigator: Melita Irving, MD


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