A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia (NCT03875534) | Clinical Trial Compass
CompletedNot Applicable
A Multi-center, Longitudinal, Observational Study of Children With Achondroplasia
United States, Australia, Austria260 participantsStarted 2019-06-19
Plain-language summary
This is a long-term, multi-center, longitudinal, observational study in children with achondroplasia (ACH). The aim is to study height velocity and comorbidities in children with ACH. This is a natural history study and no study medication will be administered.
Who can participate
Age range
0 Years – 8 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
. Legally authorized representative is willing and able to provide written, signed informed consent (with a written assent from the child when appropriate per local requirements)
. Willing and able to comply with study protocol per investigator judgement
. Clinical diagnosis of achondroplasia (confirmed by the investigator)
. Age between 0 to 8 years old at enrollment
. Able to stand without assistance (if the child is 24 months or older)
Exclusion criteria
. Have received chronic treatment (\> 3 months) of human growth hormone (hGH) or other medicinal products intended to affect stature or body proportionality at any time
. Have received any dose of medicinal products intended to affect stature or body proportionality within the previous 6 months of screening
. Have received any investigational medicinal product or device intended to affect stature or body proportionality at any time
. History or presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones
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
Annualized height velocity (centimeters/year) in children with achondroplasia
. History of any bone-related surgery that affects growth potential of long bones, such as orthopedic reconstructive surgery and osteotomy (foramen magnum decompression, and laminectomy with full recovery are allowed with minimum of 6 months of bone healing. Limb-lengthening with full recovery is allowed with a minimum of 12 months of bone healing.)
. Have forms of skeletal dysplasias other than achondroplasia or medical conditions that result in short stature or abnormal bone growth \[such as severe achondroplasia with developmental delay and acanthosis nigricans (SADDAN), hypochondroplasia, growth hormone deficiency, Turner syndrome, pseudoachondroplasia. uncontrolled hypothyroidism, uncontrolled diabetes mellitus, autoimmune disease requiring corticosteroid therapy, inflammatory bowel disease, and chronic renal insufficiency\]
. History or presence of malignant disease, other than basal cell epithelioma/carcinoma or completely resected squamous skin cancer with no recurrence for 12 months per medical records