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

This study is currently recruiting participants.
See Contacts and Locations
Verified July 2017 by BioMarin Pharmaceutical
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01603095
First received: May 11, 2012
Last updated: July 25, 2017
Last verified: July 2017
  Purpose
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
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:


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 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.


Estimated Enrollment: 250
Study Start Date: April 2012
Estimated Study Completion Date: February 2025
Estimated Primary Completion Date: October 2024 (Final data collection date for primary outcome measure)
Groups/Cohorts
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.

  Eligibility

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01603095

Contacts
Contact: Trial Specialist medinfo@bmrn.com

  Hide Study Locations
Locations
United States, California
Harbor-UCLA Medical Center Recruiting
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 Recruiting
Oakland, California, United States, 94609
Contact: Jacqueline Madden    510-428-3885 ext 5745    jmadden@mail.cho.org   
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Laurel Ware, BSN    720-777-5378    laurel.ware@childrenscolorado.org   
Principal Investigator: Gary Bellus         
United States, Delaware
Nemours/Alfred I. duPont Hospital for Children Recruiting
Wilmington, Delaware, United States, 19803
Contact: Cassie Brown    302-298-7930    cassondra.brown@nemours.org   
Contact: Michael Bober, MD         
United States, Georgia
Emory University Recruiting
Decatur, Georgia, United States, 30033
Contact: Elizabeth Smith       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 Recruiting
Chicago, Illinois, United States, 60614
Contact: Victoria Sanders    312-227-6120      
United States, Indiana
Indiana University, Riley Children's Hospital Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Susan Romie    317-278-6650    sromie@iu.edu   
Principal Investigator: David Weaver, MD         
United States, Maryland
Johns Hopkins McKusick- Institute of Genetic Medicine Recruiting
Baltimore, Maryland, United States, 21287
Contact: Adekemi Alade       aalade2@jhmi.edu   
Contact: Kira Lurman       Kmarant1@jhmi.edu   
United States, Missouri
University of Missouri Recruiting
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, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Racheal Powers    800-647-4805      
Principal Investigator: Howard Saal, MD         
United States, Tennessee
Vanderbilt University Recruiting
Nashville, Tennessee, United States, 37232
Contact: LeeAnna Melton, Study Coord    615-343-6761    leeanna.melton@vanderbilt.edu   
United States, Texas
Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
Contact: Catherine Loffredo       catherine.loffredo@bcm.edu   
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Tressa Mattioli-Lewis       tressa.mattiolilewis@seattlechildrens.org   
Contact: Klane White, MD       klane.white@seattlechildrens.org   
Principal Investigator: Klane White, MD         
United States, Wisconsin
Medical College of Wisconsin, Children's Hospital Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Paula Engelking    414-266-3289    pengelki@mcw.edu   
Contact: Donald Basel, MD       dbasel@mcw.edu   
Principal Investigator: Donald Basel, MD         
Australia, Victoria
Murdoch Children's Research Institute Enrolling by invitation
Parkville, Victoria, Australia, 3052
France
Institut Necker Active, not recruiting
Paris, France, 75015
Spain
Institut Catala de Traumatologica I Medicina de l'Esport Recruiting
Barcelona, Spain, 08028
Contact: Miriam Perez    +34 932 05 43 62 ext 21148    miriam.perez@icatme.com   
Contact: Ignacio Ginebreda    +34 619 21 35 77    ignacio.ginebreda@icatme.com   
Principal Investigator: Ignacio Ginebreda         
Hospital Sant Joan de Deu Barcelona Recruiting
Barcelona, Spain, 08950
Contact: Jose Maria de Bergua    +34 650 80 60 71      
Contact: Rosendo Ullot    +34 650 80 60 71    rullot@hsjdbcn.org   
Principal Investigator: Rosendo Ullot         
Hospital Universitario Virgen de la Victoria Recruiting
Málaga, Spain, 29010
Contact: Antonio Leiva    +34 646 56 87 89      
Contact: Felipe Luna    +34 607 85 81 22      
Principal Investigator: Felipe Luna         
Turkey
Acibadem University School of Medicine Recruiting
Istanbul, Turkey, 34752
Contact: Beyzanur Gonenc    +90 532 300 09 13    beyzanur.gonenc@acibadem.edu.tr   
Contact: Yesemin Alanay    +90 532 511 50 37    yesemin.alanay@acibadem.edu.tr   
Principal Investigator: Yesemin Alanay, MD         
Sub-Investigator: Selda Karaayvaz         
United Kingdom
Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital Recruiting
London, United Kingdom, SE1 9RT
Principal Investigator: Melita Irving, MD         
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 )
Study First Received: May 11, 2012
Last Updated: July 25, 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

ClinicalTrials.gov processed this record on July 26, 2017