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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 : Completed
First Posted : May 22, 2012
Last Update Posted : April 13, 2021
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Tracking Information
First Submitted Date May 11, 2012
First Posted Date May 22, 2012
Last Update Posted Date April 13, 2021
Study Start Date April 2012
Actual Primary Completion Date February 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: June 13, 2017)
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.
Original Primary Outcome Measures
 (submitted: May 18, 2012)
Collection of consistent growth measurements [ Time Frame: Assessed every 3 months for up to 36 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.
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
Official Title A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
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.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.
Condition Achondroplasia
Intervention Not Provided
Study Groups/Cohorts 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.
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: April 8, 2021)
363
Original Estimated Enrollment
 (submitted: May 18, 2012)
48
Actual Study Completion Date February 2021
Actual Primary Completion Date February 2021   (Final data collection date for primary outcome measure)
Eligibility 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.
  • 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
Sex/Gender
Sexes Eligible for Study: All
Ages up to 17 Years   (Child)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries Australia,   Japan,   Spain,   Turkey,   United Kingdom,   United States
Removed Location Countries France,   Germany
 
Administrative Information
NCT Number NCT01603095
Other Study ID Numbers 111-901
2017-000701-21 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party BioMarin Pharmaceutical
Study Sponsor BioMarin Pharmaceutical
Collaborators Not Provided
Investigators
Study Director: Medical Director, MD BioMarin Pharmaceutical
PRS Account BioMarin Pharmaceutical
Verification Date April 2021