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

This study is currently recruiting participants.
See Contacts and Locations
Verified June 2017 by BioMarin Pharmaceutical
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01603095
First received: May 11, 2012
Last updated: June 13, 2017
Last verified: June 2017
  Purpose
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.

Condition
Achondroplasia

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia

Resource links provided by NLM:


Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • 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.


Estimated Enrollment: 250
Study Start Date: April 2012
Estimated Study Completion Date: February 2025
Estimated Primary Completion Date: October 2024 (Final data collection date for primary outcome measure)
Groups/Cohorts
Growth measurements
Approximately 250 patients will be enrolled. Patients from birth to < 17 years on the date of consent will be enrolled. 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. Approximately equal numbers of boys and girls will be enrolled.

  Eligibility

Ages Eligible for Study:   up to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Approximately 250 patients will be enrolled. Patients from birth to < 17 years, inclusive, on the date of consent will be enrolled. 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. Approximately equal numbers of boys and girls will be enrolled.
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. 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.

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, 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01603095

Contacts
Contact: Trial Specialist medinfo@bmrn.com

  Show 21 Study Locations
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
Study Director: Medical Director, MD BioMarin Pharmaceutical
  More Information

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT01603095     History of Changes
Other Study ID Numbers: 111-901
2017-000701-21 ( EudraCT Number )
Study First Received: May 11, 2012
Last Updated: June 13, 2017

Keywords provided by BioMarin Pharmaceutical:
Achondroplasia
dwarfism

Additional relevant MeSH terms:
Achondroplasia
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Osteochondrodysplasias
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on June 28, 2017