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

Achondroplasia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01603095
• Other study ID #: 111-901
• Secondary ID: 2017-000701-21
• Status: Completed
• Start date: April 2012
• Est. completion date: February 2021

Study information

• Verified date: April 2021
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 363
• Est. completion date: February 2021
• Est. primary completion date: February 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Achondroplasia

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

United States,  Australia,  Japan,  Spain,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Collection of consistent growth measurements	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