Status:
TERMINATED
Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.
Lead Sponsor:
Pfizer
Conditions:
Achondroplasia
Eligibility:
All Genders
Up to 15 years
Brief Summary
This is a registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries. Information collected will include in anthropometric charac...
Eligibility Criteria
Inclusion
- Written informed consent is obtained from the children's parent(s) / legal guardian(s) before any study-related activity is carried out
- The child is able to provide written informed assent, where this is required according to national legislation, before any study related activity is carried out
- The child has been diagnosed as having achondroplasia documented by clinical diagnosis
- The child is between 0 years and 10 years of age, inclusive, on the date of consent / assent
- The investigator has considered the family and prospective participating child being able to comply with the study procedures
Exclusion
- The child has a diagnosis of hypochondroplasia or any short stature condition other than achondroplasia (eg, spondyloepiphyseal dysplasia congenital \[SEDC\], pseudoachondroplasia, trisomy 21)
- The child has any medical condition that may impact growth or where the treatment is known to impact growth, such as but not limited to hypothyroidism or hyperthyroidism, insulin-requiring diabetes mellitus, autoimmune inflammatory disease (including celiac disease, systemic lupus erythematosus \[SLE\], juvenile dermatomyositis, scleroderma, and others), autonomic neuropathy, or inflammatory bowel disease
- Treatment in the previous 12 months prior to consent and assent with growth hormone, insulin-like growth factor 1 (IGF-1), anabolic steroids, or any other drug expected to affect growth velocity
- Any surgery that affects the growth plate of the long bones that is planned, or has occurred in the past 18 months
- Participation in any interventional study (investigational product or device) for treatment of achondroplasia or short stature
- Has had bone-related surgery impacting assessment of anthropometric measurements or is expected to have it during the study period. Children with previous limb-lengthening surgery may enroll if surgery occurred at least 18 months prior to the date of consent/assent and healing is complete without sequelae as determined by the investigator
- Has any condition that in the view of the investigator places the child at high risk of poor compliance with the visit schedule or of not completing the study.
- Any concurrent disease or condition that in the view of the investigator would interfere with study participation
Key Trial Info
Start Date :
June 15 2018
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
January 13 2023
Estimated Enrollment :
315 Patients enrolled
Trial Details
Trial ID
NCT03794609
Start Date
June 15 2018
End Date
January 13 2023
Last Update
October 17 2024
Active Locations (57)
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1
Los Angeles Biomedical Research Institute At Harbour-UCLA Medical Centre
Los Angeles, California, United States, 90502
2
Los Angeles BioMedical Research Institute at Harbor-UCLA Medical Center
Torrance, California, United States, 90502
3
Alfred I. duPont Hospital for Children - Wilmington
Wilmington, Delaware, United States, 419803-3607
4
Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States, 419803