A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia This study is currently recruiting participants. Verified July 2013 by BioMarin Pharmaceutical Sponsor: BioMarin Pharmaceutical Information provided by (Responsible Party): BioMarin Pharmaceutical ClinicalTrials.gov Identifier: NCT01603095 First received: May 11, 2012 Last updated: July 23, 2013 Last verified: July 2013 History of Changes Full Text View Tabular ViewNo Study Results PostedDisclaimerHow to Read a Study Record Purpose Multicenter, multinational study to collect consistent baseline growth measurements on pediatric patients with Achondroplasia being considered for subsequent enrollment in Study 111-201. No study drug is administered. Condition Achondroplasia Study Type: Observational Study Design: Observational Model: Case-Only Time Perspective: Prospective Official Title: A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia Resource links provided by NLM: Genetics Home Reference related topics: achondroplasia metatropic dysplasia mucopolysaccharidosis type IV pseudoachondroplasia MedlinePlus related topics: Dwarfism U.S. FDA Resources Further study details as provided by BioMarin Pharmaceutical: Primary Outcome Measures: Collection of consistent growth measurements [ Time Frame: Assessed every 3 months for up to 60 months ] [ Designated as safety issue: No ] Patients will be screened and undergo a series of growth measurements on Day 1 and every 3 months thereafter. No study drug is administered. Estimated Enrollment: 200 Study Start Date: April 2012 Estimated Study Completion Date: November 2017 Estimated Primary Completion Date: May 2017 (Final data collection date for primary outcome measure) Groups/Cohorts Growth measurements Approximately 200 patients aged 0-13.5 will be enrolled. Approximately equal numbers of boys and girls will be enrolled. Eligibility Ages Eligible for Study: up to 13 Years Genders Eligible for Study: Both Accepts Healthy Volunteers: No Sampling Method: Probability Sample Study Population 200 boys and girls with achondroplasia aged 0-13.5 will be enrolled Criteria Inclusion Criteria: Parent(s) or guardian(s) willing and able to provide signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure. Also, willing and able to provide written assent (as needed) after the nature of the study has been explained and prior to performance of any research-related procedure. Aged 0 to 13.5 years, inclusive, at study entry Have ACH, documented by clinical diagnosis Are ambulatory and able to stand without assistance (not applicable for infants) Are willing and able to perform all study procedures as physically possible. Exclusion Criteria: Have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia) Have any of the following disorders: Hypothyroidism Insulin-requiring diabetes mellitus Autoimmune inflammatory disease Inflammatory bowel disease Autonomic neuropathy Have an unstable clinical condition likely to lead to intervention during the course of the study, including progressive cervical medullary compression Growth plates have fused Have a history of any of the following: Renal insufficiency Anemia Cardiac or vascular disease, including the following: Cardiac dysfunction (abnormal echocardiogram [ECHO] including left ventricle [LV] mass) at Screening Visit Hypertrophic cardiomyopathy Congenital heart disease Cerebrovascular disease, aortic insufficiency Clinically significant atrial or ventricular arrhythmias Current treatment with antihypertensive medications, ACE inhibitors, angiotensin II receptor blockers, diuretics, beta-blockers, calcium-channel blockers, cardiac glycosides, systemic anticholinergic agents, any medication that may impair or enhance compensatory tachycardia, drugs known to alter renal function that is expected to continue for the duration of the study Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or long-term treatment (> 3 months) at any time Have had regular long-term treatment (> 1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable) Concomitant medication that prolongs the QT/QTc interval within 14 days or 5 half-lives, whichever is longer, before the Screening visit Have used any other investigational product or investigational medical device for the treatment of ACH or short stature Have had bone-related surgery or expected to have bone-related surgery during the study period. Subjects with previous limb-lengthening surgery may enroll if surgery occurred at least 18 months prior to the study and healing is complete without sequelae. Have any condition that, in the view of the Investigator, places the patient at high risk of poor compliance with the visit schedule or of not completing the study. Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation Contacts and Locations Please refer to this study by its ClinicalTrials.gov identifier: NCT01603095 Contacts Contact: Tylea Fuhrmann tfuhrmann@bmrn.com Locations United States, California Cedars-Sinai Medical Center Recruiting Los Angeles, California, United States, 90048 Contact: Tara Funari, M.S. Tara.Funari@cshs.org Children's Hospital and Research Center Oakland Recruiting Oakland, California, United States, 94609 Contact: Jacqueline Madden JMadden@mail.cho.org United States, Illinois Ann and Robert H Lurie Children's Hospital of Chicago Recruiting Chicago, Illinois, United States, 60614 Contact: Victoria Roberts, MS vroberts@luriechildrens.org United States, Maryland Johns Hopkins McKusick- Institute of Genetic Medicine Recruiting Baltimore, Maryland, United States, 21287 Contact: Carrie Blout Cblout1@jhmi.edu United States, Tennessee Vanderbilt University Recruiting Nashville, Tennessee, United States, 37232 Contact: Amanda Bawcom amanda.d.bawcom@vanderbilt.edu United States, Texas Baylor College of Medicine Recruiting Houston, Texas, United States, 77030 Contact: Catherine Loffredo ccarter@bcm.edu Australia, Victoria Murdoch Children's Research Institute Recruiting Parkville, Victoria, Australia, 3052 Contact: Kate Pope kate.pope@mcri.edu.au France Institut Necker Recruiting Paris, France, 75015 Contact: Kim Hanh Le Quan Sang, MD kh.lequansang@nck.aphp.fr United Kingdom Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital Recruiting London, England, United Kingdom, SE1 9RT Contact: Melita Irving Melita.Irving@gstt.nhs.uk Sponsors and Collaborators BioMarin Pharmaceutical Investigators Study Director: Suyash Prasad, MD BioMarin Pharmaceutical More Information No publications provided Responsible Party: BioMarin Pharmaceutical ClinicalTrials.gov Identifier: NCT01603095 History of Changes Other Study ID Numbers: BMN 111-901 Study First Received: May 11, 2012 Last Updated: July 23, 2013 Health Authority: United States: Institutional Review Board Keywords provided by BioMarin Pharmaceutical: Achondroplasia dwarfism Additional relevant MeSH terms: Achondroplasia Dwarfism Bone Diseases, Developmental Bone Diseases Musculoskeletal Diseases Osteochondrodysplasias Genetic Diseases, Inborn ClinicalTrials.gov processed this record on November 22, 2013 TO TOP For Patients & Fami