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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01603095
Registration number
NCT01603095
Ethics application status
Date submitted
11/05/2012
Date registered
22/05/2012
Date last updated
13/04/2021
Titles & IDs
Public title
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
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Scientific title
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
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Secondary ID [1]
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2017-000701-21
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Secondary ID [2]
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111-901
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Achondroplasia
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Musculoskeletal
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Other muscular and skeletal disorders
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Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
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.
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Collection of consistent growth measurements
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Assessment method [1]
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Patients will be screened and undergo a series of growth measurements on Day 1 and every 3 months thereafter. No study drug is administered.
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Timepoint [1]
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Assessed every 3 months for up to 84 months
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Eligibility
Key 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.
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Minimum age
No limit
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Maximum age
17
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose
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Duration
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Selection
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Timing
Prospective
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/04/2012
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/02/2021
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Sample size
Target
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Accrual to date
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Final
363
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Murdoch Children's Research Institute - Parkville
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Recruitment hospital [2]
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The Children's Hospital at Westmead - Westmead
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Recruitment postcode(s) [1]
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3052 - Parkville
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Recruitment postcode(s) [2]
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2145 - Westmead
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Recruitment outside Australia
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United States of America
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California
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United States of America
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Delaware
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Georgia
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Illinois
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United States of America
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Ohio
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United States of America
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Tennessee
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United States of America
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Texas
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United States of America
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Wisconsin
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Japan
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Osaka
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Japan
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Saitama
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Japan
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Tokushima
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Spain
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Barcelona
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Spain
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State/province [13]
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Málaga
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Turkey
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Istanbul
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United Kingdom
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State/province [15]
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London
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Country [16]
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United Kingdom
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State/province [16]
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Sheffield
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
BioMarin Pharmaceutical
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01603095
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Medical Director, MD
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Address
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BioMarin Pharmaceutical
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Country
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Phone
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Email
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Contact person for public queries
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01603095
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