Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.

Stopped early

Conditions studied: Achondroplasia

In brief

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 characteristics, related symptoms, tests, \& treatments Children's information will be collected in the registry for a maximum of 5 years.

Key facts

Study ID

NCT03794609

Run by

Pfizer

People needed

315

Starts

2018-06-15

Expected to finish

2023-01-13

Last updated by the study team

2024-10-17

Who can join

Age: any, up to 15. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Los Angeles BioMedical Research Institute at Harbor-UCLA Medical Center — Torrance, California, United States
• Alfred I. duPont Hospital for Children - Wilmington — Wilmington, Delaware, United States
• Alfred I. duPont Hospital for Children — Wilmington, Delaware, United States
• The John Hopkins — Baltimore, Maryland, United States
• The Johns Hopkins Hospital — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Boston Childrens Hospital — Boston, Massachusetts, United States
• Univesity of Missouri - Columbia — Columbia, Missouri, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Texas Children Hospital — Houston, Texas, United States
• Texas Children'S Hospital — Houston, Texas, United States
• University of Utah Health — Salt Lake City, Utah, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• Murdoch Childrens Research Institute — Parkville, Victoria, Australia
• Childrens Hospital Melbourne — Melbourne, Australia
• Antwerp University Hospital — Antwerp, Belgium
• Universitair Ziekenhuis Antwerpen (UZA) — Edegem, Belgium
• The Hospital for Sick Children — Toronto, Ontario, Canada
• CHU Sainte-Justine — Montreal, Quebec, Canada
• The Hospital for Sick Children — Toronto, Canada
• The First Affiliated Hospital, Sun Yat-sen University — Guangzhou, Guangdong, China
• Beijing Children's Hospital, Capital Medical University — Beijing, China
• Righospitalet — Copenhagen, Denmark
• Bispebjerg Hospital — Copenhagen, Denmark
• Los Angeles Biomedical Research Institute At Harbour-UCLA Medical Centre — Los Angeles, California, United States

Full record on ClinicalTrials.gov

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