Beyond Achondroplasia

Growing together with Clara

Update in multicenter study from BioMarin

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

Eligibility

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

Contacts
Contact: Trial Specialist  E-mail: clinicaltrials@bmrn.com
Please refer to this study by its ClinicalTrials.gov identifier: NCT01603095

Locations

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

jmadden@mail.cho.org

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

cassondra.brown@nemours.org

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

Elizabeth.d.smith@emory.edu

Contact: William Wilcox, MD

William.wilcox@emory.edu

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

aalade2@jhmi.edu

Contact: Kira Lurman, Study Coord

Kmarant1@jhmi.edu

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

umhsorthoenroll@health.missouri.edu

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

leeanna.melton@vanderbilt.edu

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

catherine.loffredo@bcm.edu

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

Parkville, Victoria, Australia, 3052

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

kh.lequansang@nck.aphp.fr

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

joanna.poulter@gstt.nhs.uk

Principal Investigator: Melita Irving, MD

6 Comments

  1. Inés como siempre mil gracias por toda tu información. Estos días han sido muy esperanzadores para nuestros bebes.

    Quería me aclararas Pf en que Trial se plantea el potencial da hacer pruebas en niños menores de 4 años.

    De igual forma quería preguntarte si podías ayudarnos a cuadrar la cita vía internet con la Dra Homeopata de Alpe.

    Un abrazo y mil gracias.

  2. Disculpa la pregunta no quedó clara :
    En qué fase se piensa hacer pruebas en niños menores de 4 años??
    Gracias.

    • Hola Alvaro,
      Empezara en el final del año el estudo multicentrico (fase 2 inicial) con niños menores de 5 años. Los centros clinicos estan listados

  3. Bom dia como posso inscrever o meu filho com 3 anos

  4. Why this website don’t have other languages?

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