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

Completed

Conditions studied: Achondroplasia

In brief

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.

Key facts

Study ID

NCT01603095

Run by

BioMarin Pharmaceutical

People needed

363

Starts

2012-04-01

Expected to finish

2021-02-01

Last updated by the study team

2021-04-13

Who can join

Age: any, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify 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 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.

Where it is running

• Harbor-UCLA Medical Center — Los Angeles, California, United States
• Children's Hospital and Research Center Oakland — Oakland, California, United States
• Nemours/Alfred I. duPont Hospital for Children — Wilmington, Delaware, United States
• Emory University — Decatur, Georgia, United States
• Ann and Robert H Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Vanderbilt University — Nashville, Tennessee, United States
• Baylor College of Medicine — Houston, Texas, United States
• Medical College of Wisconsin, Children's Hospital — Milwaukee, Wisconsin, United States
• Murdoch Children's Research Institute — Parkville, Victoria, Australia
• The Children's Hospital at Westmead — Westmead, Australia
• Osaka University Hospital — Osaka, Japan
• Saitama Children's Medical Center — Saitama, Japan
• Tokushima University Hospital — Tokushima, Japan
• Institut Catala de Traumatologica I Medicina de l'Esport — Barcelona, Spain
• Hospital Sant Joan de Deu Barcelona — Barcelona, Spain
• Hospital Universitario Virgen de la Victoria — Málaga, Spain
• Acibadem University School of Medicine — Istanbul, Turkey (Türkiye)
• Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital — London, United Kingdom
• Sheffield Children's NHS Foundation Trust — Sheffield, United Kingdom

Full record on ClinicalTrials.gov

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