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

Sponsor
BioMarin Pharmaceutical (Industry)
Overall Status
Completed
CT.gov ID
NCT01603095
Collaborator
(none)
363
Enrollment
20
Locations
106.1
Duration (Months)
18.2
Patients Per Site
0.2
Patients Per Site Per Month

Study Details

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 Intervention/Treatment Phase

    Study Design

    Study Type:
    Observational
    Actual Enrollment :
    363 participants
    Observational Model:
    Case-Only
    Time Perspective:
    Prospective
    Official Title:
    A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
    Study Start Date :
    Apr 1, 2012
    Actual Primary Completion Date :
    Feb 1, 2021
    Actual Study Completion Date :
    Feb 1, 2021

    Arms and Interventions

    Arm Intervention/Treatment
    Growth measurements

    Approximately 500 patients will be enrolled. Patients from birth to <= 17 years on the date of consent will be enrolled. Approximately equal numbers of boys and girls will be enrolled.

    Outcome Measures

    Primary Outcome Measures

    1. Collection of consistent growth measurements [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

    Criteria

    Ages Eligible for Study:
    N/A to 17 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    • 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 angiotensin-converting enzyme (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

    Locations

    Site City State Country Postal Code
    1 Harbor-UCLA Medical Center Los Angeles California United States 90048
    2 Children's Hospital and Research Center Oakland Oakland California United States 94609
    3 Nemours/Alfred I. duPont Hospital for Children Wilmington Delaware United States 19803
    4 Emory University Decatur Georgia United States 30033
    5 Ann and Robert H Lurie Children's Hospital of Chicago Chicago Illinois United States 60614
    6 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
    7 Vanderbilt University Nashville Tennessee United States 37232
    8 Baylor College of Medicine Houston Texas United States 77030
    9 Medical College of Wisconsin, Children's Hospital Milwaukee Wisconsin United States 53226
    10 Murdoch Children's Research Institute Parkville Victoria Australia 3052
    11 The Children's Hospital at Westmead Westmead Australia 2145
    12 Osaka University Hospital Osaka Japan
    13 Saitama Children's Medical Center Saitama Japan
    14 Tokushima University Hospital Tokushima Japan
    15 Institut Catala de Traumatologica I Medicina de l'Esport Barcelona Spain 08028
    16 Hospital Sant Joan de Deu Barcelona Barcelona Spain 08950
    17 Hospital Universitario Virgen de la Victoria Málaga Spain 29010
    18 Acibadem University School of Medicine Istanbul Turkey 34752
    19 Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital London United Kingdom SE1 9RT
    20 Sheffield Children's NHS Foundation Trust Sheffield United Kingdom S10 2TH

    Sponsors and Collaborators

    • BioMarin Pharmaceutical

    Investigators

    • Study Director: Medical Director, MD, BioMarin Pharmaceutical

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    BioMarin Pharmaceutical
    ClinicalTrials.gov Identifier:
    NCT01603095
    Other Study ID Numbers:
    • 111-901
    • 2017-000701-21
    First Posted:
    May 22, 2012
    Last Update Posted:
    Apr 13, 2021
    Last Verified:
    Apr 1, 2021
    Keywords provided by BioMarin Pharmaceutical
    Achondroplasia
    dwarfism
    Additional relevant MeSH terms:
    Achondroplasia

    Study Results

    No Results Posted as of Apr 13, 2021