Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Acho… (NCT03794609) | Clinical Trial Compass
TerminatedNot Applicable
Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.
Stopped: The study was terminated on 18th November 2022. The decision to terminate the trial was not based on any safety and/or efficacy concerns.
United States, Australia, Belgium315 participantsStarted 2018-06-15
Plain-language 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 characteristics, related symptoms, tests, \& treatments
Children's information will be collected in the registry for a maximum of 5 years.
Who can participate
Age range
0 Years – 15 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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 criteria
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change From Baseline in Standing Height
Timeframe: Baseline, 1 year, 2 years, 3 years, 4 years
2
Change From Baseline in Sitting Height
Timeframe: Baseline, 1 year, 2 years, 3 years, 4 years
3
Change From Baseline in Knee Height
Timeframe: Baseline, 1 year, 2 years, 3 years, 4 years
4
Change From Baseline in Head Circumference
Timeframe: Baseline, 1 year, 2 years, 3 years, 4 years
5
Change From Baseline in Arm Span
Timeframe: Baseline, 1 year, 2 years, 3 years, 4 years
6
Number of Participants With Achondroplasia-Related Treatments
Timeframe: Baseline to end of study visit (up to 55 months)
. 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