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Trial record 3 of 12 for:    achondroplasia

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01603095
Recruitment Status : Enrolling by invitation
First Posted : May 22, 2012
Last Update Posted : September 27, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
Multicenter, multinational study to collect consistent baseline growth measurements on pediatric patients with Achondroplasia being considered for subsequent enrollment in future studies sponsored by BioMarin. No study drug is administered.

Condition or disease
Achondroplasia

Study Design

Study Type : Observational
Estimated Enrollment : 250 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
Study Start Date : April 2012
Estimated Primary Completion Date : October 2024
Estimated Study Completion Date : February 2025


Groups and Cohorts

Group/Cohort
Growth measurements
Approximately 250 patients will be enrolled. Patients from birth to < 17 years on the date of consent will be enrolled. Patients aged 0 to < 4.5 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.


Outcome Measures

Primary Outcome Measures :
  1. Collection of consistent growth measurements [ Time Frame: Assessed every 3 months for up to 84 months ]
    Patients will be screened and undergo a series of growth measurements on Day 1 and every 3 months thereafter. No study drug is administered.


Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Approximately 250 patients will be enrolled. Patients from birth to < 17 years, inclusive, on the date of consent will be enrolled. Patients aged 0 to < 4.5 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.
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 < 17 years, inclusive, at study entry. Patients aged 0 to < 4.5 years on the date of consent may also be enrolled, the timing of which will be at the discretion of the sponsor.
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01603095


  Hide Study Locations
Locations
United States, California
Harbor-UCLA Medical Center
Los Angeles, California, United States, 90048
Children's Hospital and Research Center Oakland
Oakland, California, United States, 94609
United States, Delaware
Nemours/Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States, 19803
United States, Georgia
Emory University
Decatur, Georgia, United States, 30033
United States, Illinois
Ann and Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60614
United States, Indiana
Indiana University, Riley Children's Hospital
Indianapolis, Indiana, United States, 46202
United States, Maryland
Johns Hopkins McKusick- Institute of Genetic Medicine
Baltimore, Maryland, United States, 21287
United States, Missouri
University of Missouri
Columbia, Missouri, United States, 65201
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Tennessee
Vanderbilt University
Nashville, Tennessee, United States, 37232
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
United States, Wisconsin
Medical College of Wisconsin, Children's Hospital
Milwaukee, Wisconsin, United States, 53226
Australia, Victoria
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052
Australia
The Children's Hospital at Westmead
Westmead, Australia, 2145
France
Institut Necker
Paris, France, 75015
Germany
Otto-von-Guericke Universitaet, Universitaetskinderklinik
Magdeburg, Germany, 39120
Universitaetsklinikum Muenster, Klinik für Kinder- und Jugendmedizin
Muenster, Germany, 48129
Spain
Institut Catala de Traumatologica I Medicina de l'Esport
Barcelona, Spain, 08028
Hospital Sant Joan de Deu Barcelona
Barcelona, Spain, 08950
Hospital Universitario Virgen de la Victoria
Málaga, Spain, 29010
Turkey
Acibadem University School of Medicine
Istanbul, Turkey, 34752
United Kingdom
Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital
London, United Kingdom, SE1 9RT
Sheffield Children's NHS Foundation Trust
Sheffield, United Kingdom, S10 2TH
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
Study Director: Medical Director, MD BioMarin Pharmaceutical
More Information

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01603095     History of Changes
Other Study ID Numbers: 111-901
2017-000701-21 ( EudraCT Number )
First Posted: May 22, 2012    Key Record Dates
Last Update Posted: September 27, 2017
Last Verified: September 2017

Keywords provided by BioMarin Pharmaceutical:
Achondroplasia
dwarfism

Additional relevant MeSH terms:
Achondroplasia
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Osteochondrodysplasias
Genetic Diseases, Inborn