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Summary

for people ages up to 16 years (full criteria)
at Oakland, California and other locations
study started
estimated completion:

Description

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.

Keywords

Achondroplasia dwarfism

Eligibility

You can join if…

Open to people ages up to 16 years

  • 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.

You CAN'T join if...

  • 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

Locations

  • Children's Hospital and Research Center Oakland
    Oakland, California, 94609, United States
  • Harbor-UCLA Medical Center
    Los Angeles, California, 90048, United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
BioMarin Pharmaceutical
ID
NCT01603095
Study Type
Observational
Last Updated
September 26, 2017