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

April 8, 2021 updated by: BioMarin Pharmaceutical
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.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

363

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 17 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Probability Sample

Study Population

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

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Assessed every 3 months for up to 84 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BioMarin Pharmaceutical

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2012

Primary Completion (Actual)

February 1, 2021

Study Completion (Actual)

February 1, 2021

Study Registration Dates

First Submitted

May 11, 2012

First Submitted That Met QC Criteria

May 18, 2012

First Posted (Estimate)

May 22, 2012

Study Record Updates

Last Update Posted (Actual)

April 13, 2021

Last Update Submitted That Met QC Criteria

April 8, 2021

Last Verified

April 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 111-901
  • 2017-000701-21 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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